Page 1 DIA 2017 PROPOSED SESSIONS · Page 1 DIA 2017 PROPOSED SESSIONS Note: Part 1 is a list of the titles of the proposed sessions within each primary interest area, and Part 2

Page 1DIA 2017 PROPOSED SESSIONS

Note: Part 1 is a list of the titles of the proposed sessions within each primary interest area, and Part 2 includes the individual session details and speaker requests.PART 1: TITLES OF PROPOSED SESSIONS IN PRIMARY INTEREST AREAS01: DATA/BIG DATA/eHEALTH01-01 EHRs/eSource and Emerging Data Streams01-02 Quantified Self Tracking01-03 Mobile Health: Participatory Research01-04 FDA Session: Electronic Submissions and Data01-05 FDA Session: Health IT at FDA01-06 Big Data: Combining Registries and Legacy Clinical Trial Databases01-07 Big Data: Genomics and Personalized Medicine01-08 Big Data: Current and Emerging Technologies 01-09 Big Data: Management and Analytics01-10 Big Data: Case Studies in Clinical Trials01-11 Big Data: Regulatory Information01-12 Data and Privacy Protection01-13 Risk-Based Monitoring: Convergence of Technology, Processes, and People01-14 Data Quality01-15 Evolving Data Standards and Regulations

02: DISRUPTIVE INNOVATION02-01 New Site Paradigm: Just in Time Sites02-02 How 3D Printing May Disrupt Pharmaceutical Manufacturing and Personalized Medicine02-03 Next Generation Patient Recruitment02-04 New Frontiers in Pharmaceutical Manufacturing02-05 Online Patient Networking02-06 Next Generation Predictive Analytics02-07 New Trial Design Approaches: Including New Endpoints02-08 Microbiome02-09 Leveraging Wearables and Sensors in Clinical Trials02-10 Artificial Intelligence/Machine Learning02-11 Are We Ready for Telemedicine-Powered Clinical Trials?02-12 Gene Editing/CRISPR

03: MEDICAL AFFAIRS AND SCIENTIFIC COMMUNICATION03-01 Handling Patient Requests for Medical Information03-02 Creating Adaptive Contents for a Multichannel World03-03 Medical Information and Digital Channels03-04 Quality Oversight for Medical Affairs03-05 Off-Label Communications to Health Care Providers and Consumers 03-06 Digital Innovations for Medical Information Contact Centers03-07 EMA Policy 70: What Have We Learned?03-08 Best Practice for Advisory Committee Meeting Preparation03-09 Writing of Layperson Summaries of Clinical Trial Results According to the EU: Regulation and Its Impact on Global Pharmaceutical Activities03-10 Driving International Awareness and Use of Regulatory Writing Guidelines: Japanese Case Study of the Clarity and Openness in Reporting (CORE) Reference Guidelines03-11 Submission Dossiers: Communications Excellence to Drive Speed and Quality03-12 Effective Collaboration Between Sponsors and Medical Writing Vendors03-13 How to Build a Patient-Centric Model Within Medical Affairs03-14 Building the Medical Strategy and Tactics03-15 FDA Marketing Enforcement Update03-16 The New Business Reality for the Medical Science Liaison Community03-17 Globalization of Medical Affairs Departments

04: PATIENT ENGAGEMENT04-01 How Do You Build a Collaborative Community? What Advice Would You Give?04-02 Patient Engagement: 4 W's and an H04-03 Partnering with Patients: Virtual and Traditional Communities - When for What04-04 Data Sharing: 2017 and Beyond

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04-05 Not Your Grandma's Patient Advocacy Group Anymore04-06 Does Diversity Matter? 04-07 Will Twitter (or Baidu) Change What's In Your Medicine Cabinet? 04-08 If You See Something (About a Clinical Trial), Do You Say Something (to Your Doctor?)04-09 We Can't Forget the Children in This Process04-10 How Is Digital Patient Engagement Impacting Trial Participation?04-11 Sharing the Burden04-12 No More Swag Items for Retention, So Now What Do We Do? 04-13 Adoption and Implementation of Digital Platforms to Further Patient Interactions04-14 Challenges of Patient Reported Data via Wearable Devices04-15 For Better or Worse, Health Data Going Public Can Change Your Life04-16 Defining the Science of Patient Input to Enhance Drug Development and Approval

05: REGULATORY05-01 Expanded Access and Compassionate Use05-02 Regulatory Frameworks Amidst Disruptive Innovation05-03 Recent Trends in Asian Regulations for Drug Development05-04 Minding the Gaps: Using Totality of Evidence to Support Regulatory Approval05-05 FDA Intercenter Collaboration: Oncology Center of Excellence 05-06 What’s New in Devices: European Medical Device Regulations and MDUFA IV05-07 Regulatory Challenges with Next Generation Sequencing05-08 Lessons Learned from the Sarepta Exondys 51 Approval05-09 Evolution of Novel Registration Endpoints as Diseases Become Chronic05-10 How to Work through Divergent Regulatory Advice in Global Development Programs05-11 PDUFA at 25: A Historical View of the Prescription Drug User Fee Agreement and a Look to the Future - PDUFA VI05-12 The Reauthorization of BsUFA and its Impact on Both the Regulator and Industry05-13 GDUFA II and Regulatory Science Initiatives: Challenges in Developing Complex Generics05-14 Is Getting the Right Evidence During Development to Support Both Registration and Reimbursement an Achievable Endpoint?05-15 The Evolving Model of Using Artificial Intelligence to Deliver Actionable Regulatory Intelligence05-16 Challenges in Developing a Global Strategy for Postapproval CMC Changes for Combination Products05-17 International Collaboration in Times of Public Health Crisis 05-18 Global Compliance Scorecard on Supply Chain Issues

06: SAFETY AND PHARMACOVIGILANCE06-01 Safety Evaluation: Transforming Information into Evidence06-02 Postmarketing Safety Studies: Approaches and Regulatory Insights06-03 Counting on Safety06-04 Integrated Life Cycle Approaches to Benefit-Risk Assessment, Communication, and Evaluation06-05 Justifying Pharmacovigilance Legislation Based on its Impact06-06 Designing and Implementing a Robust Pharmacovigilance System for Vaccines06-07 Automation and Innovation in Clinical Trial Safety Assessment06-08 A New Age for Physician and Patient Labeling: Recommendations for Innovative Change in How We Communicate06-09 The Brave New World: The Ongoing Globalization of Pharmacovigilance: 06-10 Exploring the Evolution of Signal Detection 06-11 Changing Environments within Pharmacovigilance06-12 New Considerations in Benefit-Risk Management06-13 Pharmacovigilance 2.0: Redesigning for the Future

07: SPECIAL POPULATIONS07-01 Integrating the Patient's Voice Across the Development Program of Rare Diseases07-02 Engagement, Education, Networks, Media and Societies in Rare Diseases: The MUST Haves07-03 Innovative Designs and Statistical Approaches for Small Trials (Rare Disease or Pediatric Indications) 07-04 Recruiting Rare Disease Patients: Objects in the Mirror May Be Closer Than They Appear 07-05 Pregnancy and Lactation Labeling Rule: Unique Challenges to Meet Requirements07-06 Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes07-07 Collaborative Efforts to Accelerate Rare Disease Research and Development07-08 CNS Impairment Guidelines and the Impact on Relevant Topics Pertaining to the Brain

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07-09 Is the Future Bright for Treatment of Neurodegenerative Diseases?07-10 Exploration of Metabolic Diseases: Is the Liver the New Heart?07-11 Issues in Early-Phase Oncology Trials07-12 Increasing Participation in Clinical Trials Among Under-represented Populations07-13 Looking to the Future for EU Pediatric Investigation Plans (PIPs)

08: STRATEGIC PLANNING, EXECUTION, PARTNERSHIPS08-01 So You Say You're an Extrovert: Being an Effective Leader When All You Really Want Is a Stage08-02 Effective Collaborations: Navigating the Grey Space08-03 Post-Trial Access: Ensuring Patient Access Across the Development Spectrum08-04 Strategic Planning Across Research Organizations 08-05 The Art of Negotiation: Preparing Yourself and Your Team for Their Seat at the Table 08-06 Effective Life Cycle Management08-07 Flexing Your Project Management Muscles Outside of Traditional Roles08-08 The Impact of Industry Consortia on Creating Collaborative Solutions to Drug Development Challenges: What Does the Future Hold?08-09 Keys to Effective Product Launch Preparation08-10 Effective Portfolio Management and How to Ensure You Get the Value Out of the Decisions Made08-11 Accelerating Drug Development Through Innovation08-12 Crisis Management in the Face of a Global Health Pandemic08-13 Leading in the Midst of Ambiguity

09: TRANSLATIONAL SCIENCE: PRECLINICAL, CLINICAL, AND PRODUCT DEVELOPMENT09-01 The Use of Wearable Devices in Clinical Trials, or My iPhone Just Took My Blood Pressure!? 09-02 Use of Novel Trial Designs to Accelerate Decision Making: To 'P' No More09-03 What Question Are You Trying to Answer? The Use of Estimands, Potential Revisions to ICH E-9 and the Impact to Trial Design and Analysis09-04 Novel Technological Approaches to Identify Subjects and Augment Recruitment09-05 Prior Elicitation: Estimating the Unknown09-06 Master Protocols: New Approaches to Enrolling Hard-to-Find Subjects and Expediting Drug Development09-07 Audits and Agency Inspections of Risk-based Monitored Studies: The Results Are In…09-08 Trial Master File: MHRA/EMA Inspections and Sponsor Audit Learnings09-09 Evolving Clinical Trial Guidance and Regulations: Am I Ready?09-10 Of Course My Data Has Integrity … And I Can Prove It09-11 Patient-Relevant, Fit-For-Purpose Endpoints: Is this Achievable?09-12 Patient-Reported Outcomes (PROs): Hot Topics09-13 Experimental Studies: New Approaches to Study Designs to Facilitate Early Phase Decision-Making09-14 Novel Techniques for Improving Clinical Trial Subject Retention09-15 Optimizing Early Clinical Strategies to Support Breakthrough Therapy Designation09-16 Calling All Angels to the Valley of Death09-17 Fusion Study Designs: Balancing Cost and Risk 09-18 Exploration of PK/PD in NDA Enabling or Early Proof of Concept Studies 09-19 Safety Issues in First-in-Human Studies

10: VALUE AND ACCESS10-01 Breaking Down the Wall for FDA to Leverage Real World Data10-02 Breaking Down the Wall for Global Regulators to Leverage Real World Data10-03 The Increasing Role for Big Data for Late Phase Drug and Postapproval Purposes10-04 The Intersection of FDA Labeling and Value-Based Conversations with Payers10-05 Drug Mortgages: Benefit-Risk and Pricing Convergence10-06 Outcomes Standardization: An Imperative to Value Assessments10-07 Comparative Efficacy Trials: The Role of FDA and Other Regulators10-08 Mind the Efficacy-Effectiveness Gap: Will the New European Regulations on Clinical Trials Revolutionize Health Technology Assessment10-09 Real World Data to Real World Evidence for Assessing Efficacy and Effectiveness10-10 Drug Pricing: Access Versus Innovation10-11 Payer Evaluation in Today's Value Framework: Impact on Patient Care

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PART 2: DIA 2017 PROPOSED SESSIONS DETAILS01: DATA/BIGDATA/eHEALTH

Working Title Brief Overview Overall Learning Objective(s)Potential Speaker Perspective #1

Potential Speaker Perspective #2

Potential Speaker Perspective #3 Keywords

01-01 EHRs/eSource and Emerging Data Streams

This session will discuss the convergence of clinical trials with clinical practice – bringing all the data together to use to identify new questions and answers.

Discuss the potential and complexities associated with EHR to EDC data transfer.

01-02 Quantified Self Tracking

This session will discuss how research is done using tools remotely to minimize patient visits – much broader accessibility to clinical research as well as additional types of information being generated. Wearables, apps

Discuss the support structure necessary for combining traditional clinical research with emerging technologies.

01-03 Mobile Health: Participatory Research

*Social media, crowd-sourced research, personal health records, commercial genomics

Discuss the use of mobile health and social media to increase Participatory Health Research (PHR) and other innovative approaches to data collection, analysis, and use.

01-04 FDA Session: Electronic Submissions and Data

Study Data Standards are required in clinical and nonclinical studies that start after December 17, 2016. Technical rejection criteria will added to the existing eCTD validation criteria to enforce the deadlines. This session will provide an update on these topics and others focused on eCTD and standardized study data.

Discuss the status of electronic submissions, standardized data requirements, and data validation at FDA.

Regulatory speaker

01-05 FDA Session: Health IT at FDA

This session will focus on the status of health informatics at the FDA. Topics will include data standards for health care and clinical research, as well as use of EDC and EHRs in clinical research.

Discuss the status of health informatics, eSource and EHRs at the FDA.

Regulatory speaker

01-06 Big Data: Combining Registries and Legacy Clinical Trial Databases

*Rare data, post marketing, leveraging RWE, data sharing, open access, identifying cohorts for clinical trials, interoperability, differing standards, submission considerations

Identify the potential of combining various types of studies and requirements to make it a success.

01-07 Big Data: Genomics and Personalized Medicine

Using big data to identify correlations and determinants of disease or to predict response to therapy

Describe challenges and opportunities of using big data to achieve more precise modeling of complex diseases.

01-08 Big Data: Current and Emerging Technologies

*scalable and expandable big data infrastructure; cloud, outsourcing, remote monitoring

Discuss how big data and new technology can be used to enhance patient experience, manage outcomes, and enhance research.

01-09 Big Data: Management and Analytics

Includes predictive analytics, what do we do with it, cleaning it Identify how to use big data analytics to develop new insights from patient data.

01-10 Big Data: Case Studies in Clinical Trials

*Actual current actual use of big data in trials Discuss real-life examples and lessons learned from big data application.

01-11 Big Data: Regulatory Information

*Leveraging information assets to identify correlations and relationships; effective management of regulatory information

Discuss effective management of regulatory information.

01-12 Data and Privacy Protection

*HIPAA, PHI, PII, cyber security Discuss data and privacy protection.


01-13 Risk-Based Monitoring: Convergence of Technology, Processes, and People

Real-life challenges and Lessons Learned. Impact on traditional roles, new opportunities, cross-functional collaboration, identifying data trends and issues

Define key challenges in RBM implementation; Understand how processes and roles need to change in the organization to support the RBM; Demonstrate how results from analytics can be turned into actionable items to mitigate risks.

01-14 Data Quality This session will discuss data quality in the light of risk-based methodologies: data fit for purpose, appropriate risk treatment, quality by design.

Discuss if and how the approach to data quality has evolved with the adoption of risk-based methodologies.

01-15 Evolving Data Standards and Regulations

This session will describe the current state and evolution of data standards.

Describe the current state and evolution of data standards, understand the direction of changes and what they are trying to achieve.

02: DISRUPTIVE INNOVATION




Keywords

02-01 New Site Paradigm: Just in Time Sites

Can use of just in time sites enable trials of the future? What are ways in which we can diversify and expand the types of sites used for clinical trials (e.g., physician office) and how can we rapidly help inexperienced sites get up and running? "Just in time sites" are they the wave of the future? Consider identifying potential clusters of patients and then opening sites to come to them. Discover the elements to consider in this new paradigm

Evaluate new structure needs for sites of the future; Identify new support that will need to be provided to the sites.

clinical trialist site monitor

02-02 How 3D Printing May Disrupt Pharmaceutical Manufacturing and Personalized Medicine

3D printing of pharmaceutical treatments has the potential to revolutionize the ability to tailor treatment to individual need in a manner not possible in current processes. This session will explore emerging 3D printing approaches in pharmaceutical manufacture and particularly in the provision of personalized medicine where dosage can be finely tuned to individual needs and current disease status. This session will explore the current landscape and consider the promise and potential of this approach, how close we are to achieving it, and practical considerations such as cost, quality, privacy and implications on the supply chain.

Identify the potential for 3D printing to revolutionize the delivery of health care to patients

pharmaceutical drug supply / vendor

Speaker to discuss the impact on clinical trials

3D printing

02-03 Next Generation Patient Recruitment

Patient recruitment remains a major cause of delay in today’s clinical trials, despite many patients indicating a willingness to participate in trials should opportunities arise. In this session we explore experience with approaches to reach patients outside the clinic pool. We will explore why leveraging pharmacy has been unsuccessful to date, explore experience of leveraging primary care EHR tools to provide direct patient referrals, using artificial intelligence solutions to gain deeper and more focused insights from medical records data and the use of social listening and social media to present opportunities to potential participants. Security and privacy concerns will be considered.

Describe how the use of technology can help identify potential study patients; Gain a greater understanding the value and limitations machine learning can assist regarding identifying potential study patients.

FARSITE or EHR4CR projects

Artificial intelligence perspective

Social listening and social media approaches

Identifying trial patients


02-04 New Frontiers in Pharmaceutical Manufacturing

This session explores novel and disruptive approaches to pharmaceutical formulation development and manufacturing. Translational pharmaceutics is the emerging practice of integrating formulation development, real-time manufacturing and clinical testing within early phase drug development. We explore the utility and potential of this approach to provide optimal formulations to take forward into later phases of clinical testing. We also explore the potential of 3D printing and how this may be integrated to provide personalized medicine within clinical trials and on the market. We consider cost, utility and benefits of these approaches, and implications on the regulatory requirements.

Describe how new techniques for formulation and product manufacturing will modernize the delivery of products to patients.

3D printing perspective Regulatory perspective continuous approaches, decentralized vs centralized, 3D, on-demand biotechnology

02-05 Online Patient Networking

This session explores the use of online patient communities and networks in clinical research and development. We will explore the use of social listening to gain insights into patient conditions, issues, and concerns and how this can be useful in designing clinical trials and engagement programs. We will also explore the development of online communities and the involvement of pharma – what is appropriate involvement for pharma companies to ensure communities and twitter chats continue to be valuable and popular with participants. We will also explore the kind of information these communities can reveal about unmet needs and current treatments.

Identify the potential and limitations of tapping into online patient communities to develop insights for product design and testing.

Social listening, pharmaceutical perspective

Twitter chats bioethicist Social listening

02-06 Next Generation Predictive Analytics

How can sophisticated predictive analytic tools transform product development and supply? Can we go beyond standard analytic techniques? Clinical trial optimization and enrollment? Can we forecast clinical trial outcomes? How do we anticipate problems before they arise?

Recognize an awareness of capabilities for predictive analytics and how success can drive efficiency in product development.

pharmaceutical statisticians/quantitative sciences or supply chain management

regulator consortium in this space or academia

Predictive Analysis

02-07 New Trial Design Approaches: Including New Endpoints

Can better endpoint structures be developed that capture disease status and medical product performance, yet still allow interpretability? Can we employ trial designs that are not ‘one size fits all’ and generate better data more efficiently?

Discuss how the clinical trial enterprise may be enhanced with novel designs.

pharmaceutical perspective with a clinical success story

pharmaceutical perspective with a statistical success story

regulator perspective Endpoints

02-08 Microbiome Emerging research points to the significant role of microbiome in contributing to disease and how promoting maintenance of a “healthy microbiome” across a wide variety of disease states can maintain health, prevent, intercept, and treat disease states. How do we handle the complexity of the microbiome in conducting clinical research and monitoring? Does addressing the microbiome impact how we view disease and clinical practice? Do we need new regulatory and medical approaches to manage?

Describe the complexity of the microbiome in both health and disease management; Identify how addressing the microbiome can fundamentally change our approach to medicine.

pharmaceutical speaker regulator clinician Microbiome

02-09 Leveraging Wearables and Sensors in Clinical Trials

Due to the increased miniaturization of sensors and circuitry the health and wellness industry is seeing rapid proliferation in the number and diversity of wearables and sensors to provide personal wellness applications. This provides opportunity to collect greater and richer data during clinical trials and post-marketing studies, and also to develop beyond the pill strategies to support launching or marketing products. This session explores the impact of wearables and sensors on clinical trial conduct and participation. We also consider how to ensure the validity and acceptability of health outcomes that are derived from wearables data, especially when using to provide endpoints that may support labelling claims. Finally, we consider how pharma is levering wearables and sensors to deliver beyond the pill strategies to support patients and physicians in the effective use of their treatments.

Describe the impact of wearables and sensors on clinical trial conduct and participation; Identify how pharma is leveraging wearables; Discuss the importance of validity and acceptability of the data from wearables on labeling claims.

Wearables expert from pharma

Outcomes validation expert

Pharma beyond the pill Wearables


02-10 Artificial Intelligence/Machine Learning

What are the ways in which artificial intelligence (AI) can impact clinical decision making and how we design and run clinical trials? How effective or robust is AI in these areas? What parts of clinical research and care will AI take over? What are the barriers to full deployment of AI in clinical care and research? What are the privacy and policy implications of learning software that continuously builds on patient experience to better deliver to the next patient?

Discuss how artificial intelligence can impact product development, research, and clinical care; Identify potential benefits and barriers to use of artificial intelligence.

pharma bioethicist patient perspective or privacy advocate

02-11 Are We Ready for Telemedicine-Powered Clinical Trials?

Telemedicine enables remote monitoring and interaction with patients to enhance care provision and treatment decisions. These tools used in routine care have enormous potential in providing ways to enable better monitoring of trial participants, and facilitate participation and engagement with less reliance on clinic visits. This session explores whether we are adequately primed to effectively utilize telemedicine, and where challenges exist what needs to be changed about the system or telemedicine approach to enable better usage? We will also consider how best telemedicine is used, or not, in clinical trials with reference to some practical examples. Are we driving to a world in which clinical care is not based on an in-person interaction with a health care provider?

Learn about telemedicine, including the drivers for and barriers to its use in clinical trials and care.

speaker representing video interactions with patients

Technology provider pharmaceutical perspective

Telemedicine

02-12 Gene Editing / CRISPR

New techniques that enhance precision and efficiency in gene splicing and editing could revolutionize disease prevention and cure. Are we on the cusp of being able to treat all genetic diseases? Can we rid ourselves of disease-bearing mosquitos? What are the implications for this technology in our approach to product development and patient care? What could the cost to society be, and are they worth it?

Discuss the potential for gene editing technology to revolutionize disease management; Hear perspectives on how the technology can be used.

speaker engaged in CRISPR

bioethicist academia

03: MEDICAL AFFAIRS AND SCIENTIFIC COMMUNICATION




Keywords

03-01 Handling Patient Requests for Medical Information

This session will share ideas for how to better engage patients who contact us for medical information. The session will explore novel approaches or new methods for engaging patients. This includes conversations with patients in the contact center setting and creating and fulfilling patient questions with lay language answers.

Discuss ways in which to better meet patient needs for medical information.

Traditional medical information pharmaceutical representative

Medical information Contact Center Representative

Patient advocate or Traditional medical information pharmaceutical representative

patients, consumers, medical information, lay language

03-02 Creating Adaptive Contents for a Multichannel World

This session will discuss the importance of creating structurally rich, discoverable, adaptable content to meet the high demands of our multichannel world. Includes a discussion on component authoring and how to transition from traditional content creation and the importance of developing a content strategy for content reuse.

Describe an approach to transforming medical information content for multiple channels.




content strategy, adaptive content, digital channels, medical information

03-03 Medical Information and Digital Channels

This session will explore customer usage and preferences (health care provider and consumer), what does good look like in this space (including pharmaceutical medical information websites), what is the "payoff" for pharma's investment in this area (i.e.. where should resources be focused.)

Identify what good looks like in the medical information digital space.


medical information, digital channels, customer preferences, innovation

03-04 Quality Oversight for Medical Affairs

Describe the global and local regulations as it pertains to medical affairs and how to establish and oversee/monitor a quality framework for medical affairs.

Identify and review an example quality framework for medical affairs.

quality, oversight, audit, compliance, medical affairs

03-05 Off-Label Communications to Health Care Providers and Consumers

Preparing for new FDA policies on off-label communication to health care providers and consumers in light of FDA, court, and other developments.

Discuss the newest developments at FDA and industry recommendations for changes in FDA off-label policies.

Industry representative Consumer/patient representative

off-label, consumer, patient, health care provider, regulation, marketing, enforcement


03-06 Digital Innovations for Medical Information Contact Centers

A review of cutting edge technologies and channels for medical information contact centers. Approaches to adoption and implementation, challenges, and successes.

Gain insight into cutting edge tools and technologies in the contact center space.

digital channel, innovation, medical information, contact center, call center

03-07 EMA Policy 70: What Have We Learned?

By June 2017, the Final Rule should be available and we will know more about Policy 70 implementation issues and what is happening to EMA and the EU Portal for the Clinical Trials. Regulation because of Brexit. There will be a huge amount of information to discuss.

Share experience with and the impact to pharmaceutical companies, medical writers, regulators and patient safety.

EMA representative redaction, clinical trial disclosure, EMA Policy 70

03-08 Best Practice for Advisory Committee Meeting Preparation

Often pharmaceutical sponsors are requested by the FDA to participate in an Advisory Committee meeting during the review of a NDA/sNDA. Understanding the best practices for preparation of an advisory committee meeting is crucial (e.g., slide deck, briefing book, review of FDA's Advisory committee brief ) is critical for success.

Discuss how to generate a quality briefing book; Recognize considerations for preparing for the Advisory Committee meeting such as expectation and FDA information needs.

Pharmaceutical representative with a medical writing focus

FDA representative Advisory Committee panel voting member

advisory committee, briefing books, regulatory writing

03-09 Writing of Layperson Summaries of Clinical Trial Results According to the EU: Regulation and Its Impact on Global Pharmaceutical Activities

For the first time there is regulation in a key geographical area that mandates that trial results are presented in a lay-friendly way. This session will discuss how this is a unique opportunity to re-define the relationship between sponsors, investigators, study participants, and patient organizations. Although limited to the EU, most sponsors will make lay summaries available globally.

Describe the challenges of actual writing of lay summaries and the implementation of a process for individual sponsors.

EU regulatory perspective

clinical trial disclosure, layperson summary

03-10 Driving International Awareness and Use of Regulatory Writing Guidelines: Japanese Case Study of the Clarity and Openness in Reporting (CORE) Reference Guidelines

The Clarity and Openness in Reporting: E3-based (CORE) guidelines were developed to help companies prepare disclosure-ready clinical study reports mandated by the EMA in a timely and cost-effective manner, and without risking inadvertent disclosure of sensitive information. Using the CORE guidelines as an example, this session will examine how to develop guidelines that can be used to enhance transparency in the real world, across the world.

Identify how to prepare disclosure-ready clinical study reports.

A regulatory writer from Japan who will share results from original research on the awareness and use of the CORE guidelines in Japan.

clinical trial disclosure, clinical study reports, CORE

03-11 Submission Dossiers: Communications Excellence to Drive Speed and Quality

Pharmaceutical companies are driving the 'time to submission' paradigm in today's market pressured by submitting dossiers to multiple markets in the same time period, as quickly as possible following last database lock of pivotal studies. In turn cross-functional teams are looking for strategies/approaches to realize a quicker yet just as effective submissions. This session will explore optimal authoring approaches to plan, execute, and deliver marketing applications to time and quality. Case studies will provide cross-disciplinary views to highlight successful approaches and best communications practice.

Define communications strategy linked to draft label; Discuss authoring strategies to maximize time to submission and approval from LSLV.

Pharmaceutical representative

Regulatory Writer Regulatory writer or Project manager

CTD, marketing applications, regulatory writing, communication strategy

03-12 Effective Collaboration Between Sponsors and Medical Writing Vendors

With pharmaceutical companies outsourcing more medical/regulatory writing activities, the need for medical writing service providers is growing. The ultimate goal of outsourcing should be to build a productive, long-term relationship with a core team of writers who are an integral part of a clinical team. This session is of relevance to both medical writing departments and outsourcing departments in order to discuss and find ways how sponsors can be more effective at bringing vendor medical writers’ particular skill sets to bear and help clinical teams prepare their regulatory documentation more efficiently.

Discuss using case studies to highlight what success looks likes and the benefits of a strategic partnership; Describe the role of the vendor medical writer and project manager in partnership with a pharmaceutical sponsor.

Medical writer or pharmaceutical representative

Project manager strategic partnerships, outsourcing, medical writing, project management

03-13 How to Build a Patient-Centric Model Within Medical Affairs

This session will discuss the role of medical affairs in patient involvement, and best practices for incorporating patient-centric initiatives into the medical affairs unit.

Discuss appropriate methods for medical affairs units to engage patients.

Medical information/communication representative

Medical Director Patient education liaison

patients, medical strategy


03-14 Building the Medical Strategy and Tactics

This session will focus on how to develop a comprehensive medical plan for your medical affairs unit. Speakers will discuss key considerations, such as effective collaboration and value add to clinical development, R&D, and commercialization groups.

Discuss key considerations and strategies for medical manager/directors when developing medical plans.

Medical Director Commercialization or Marketing representative

medical strategy

03-15 FDA Marketing Enforcement Update

FDA marketing officials from the CDER Office of Prescription Drug Promotion and other related centers will outline the latest major enforcement actions and regulatory guidance and explain major priorities in enforcement actions and area of enforcement concern. Participants can expect a review of the structure and priorities of the marketing enforcement centers, including review of warning letters and current guidances.

Describe the latest enforcement priorities and warning letters from FDA OPDP and other enforcement centers that focus on marketing matters.

FDA representatives marketing, promotion, warning letters, advertising, DTC

03-16 The New Business Reality for the Medical Science Liaison Community

The development of Affordable Care Organizations (ACO) and the new Integrated Delivery Networks (IDN) / Integrated Delivery Systems (IDS) have created a new business reality for the medical science liaison (MSLs) community. While ACOs generally focus on specific medical touch-points, such as lowering A1C for diabetics, IDNs and IDSs are focused on more general improvement of healthy outcomes. This session would focus on the business challenges MSLs face from these new customers and how best to adjust.

Discuss business challenges MSLs face from Affordable Care Organizations (ACO) and the new Integrated Delivery Networks (IDN) / Integrated Delivery Systems (IDS); Describe methods on how best to adjust MSL practices.

Representative from an ACO

Representative from an IDN

MSL, strategy, health care

03-17 Globalization of Medical Affairs Departments

Traditionally, medical affairs departments have focused most on domestic operations but more and more companies are broadening their reach to international operations, especially in the EU, Pacific Rim, and emerging markets. These new markets vary considerably from the US and from country to country, requiring new knowledge and skill development. The session will share perspectives from medical affairs professionals across the globe who will discuss the necessary skills and best practices for working globally and cross functionally.

Discuss the necessary skills and best practices for medical affairs professionals to work globally and cross functionally.

Panel with individuals from each region across medical affairs: medical director, medical information

global, MSL, medical communication, medical director/ manager, phase 3, phase 4

04: PATIENT ENGAGEMENT




Keywords

04-01 How Do You Build a Collaborative Community? What Advice Would You Give?

In this panel discussion, we want to tell the story of multiple stakeholders involved, the effort in organizing everyone and getting them on the same page, and the challenges and lessons learned. Panel members should include patient(s) and how they got involved, FDA, other stakeholders.

Describe the challenges and lessons learned in building a collaborative community.

Advocacy organization Sponsor (i.e., pharmaceutical)

Regulator agency or organization that influences policy

Patient voice

04-02 Patient Engagement: 4 W's and an H

Patient engagement is a hot topic across the industry and has potential touchpoints throughout the life cycle of a product or portfolio. Because it's a new idea in product development, we want to hear from those who've used patient engagement techniques while planning and executing development programs, as well as during and post approval. We are seeking clear examples on why, what, when, who, and how these engagement tactics were used and the value they added. Ideally, this session will include an update from the TUFTS/ DIA working group on Patient Engagement and provide some benchmarking data about what's happening across industry. When these tactics were used, what changed in the planning and execution of programs? And what surprises arose, both in the process of engagement and in the outcomes?

Provide data on patient engagement practices across industry; Prepare a 'map' of potential points of engagement across product life cycle; Discuss case studies about patient engagement tactics and what was learned, applied based on the outcomes.

Industry: Possibly Clinical Operations and Clinical Science or Commercial representation.

Academic and/or Regulatory

Patient or patient advocate

Patient engagement


04-03 Partnering with Patients: Virtual and Traditional Communities - When for What

Communities - we're all part of them as humans. But how do you engage with or create communities for patient engagement? And which are most effective to achieve your business and patient-centric objectives? How does one work through the process of selecting which community to work with, or to build one from the ground up? Is it more effective to work with virtual or traditional communities? Ideally, this would be crafted as a workshop with facilitators helping participants think through these questions. For example, define a disease area and create high level objectives for the community engagement (e.g., input to development, compliance / adherence, patient recruitment, product experience feedback). This will be a guided workshop - it might take a community to execute it - so please invite those who've been involved to participate.

Test decision making and logistics planning to use virtual / traditional communities; Explore how to determine which traditional communities are best fit for program objectives; Explore the benefits and challenges of working with both types of communities.

Industry: Clarify questions to be addressed

Digital/vendor: Who's got a platform to use (e.g., iAdvise or My Breast Cancer Team)?

Patient/patient group: Representative who has been on a patient panel or has provided input through a community

Patient community

04-04 Data Sharing: 2017 and Beyond

Society is increasingly expecting data transparency in many aspects of life, including clinical data. The European Medicines Agency (EMA) has set a timeframe for lay summaries of trials to be published, while FDA has expressed concerns about inappropriate promotional practices if data are shared with the trial patients prior to FDA review/approval. No clear guidelines or processes established on how to share results back to the trial patients themselves. At the same time, some ethics review boards are expecting individual data results back to the patient, which is a whole different level of data specificity.

Discuss the legislations changes that are expected and the impact if there are differences between regulatory agencies; Discuss the mechanisms or barriers for dissemination of individual research results and/or clinical trial lay summaries.

TransCelerate representative / Sponsor

Representative(s) from EU Ethics Board or "Be the Partner"

Representative(s) from EUPATI / CISCRP

Clinical trial data

04-05 Not Your Grandma's Patient Advocacy Group Anymore

This session will share end-to-end stories of how advocates have gained influence with researchers, policy makers and regulators to drive the approvals of new therapies. The panelists will tell the story from the point of view of the advocates who organized resources differently to increase the volume and speed of research in their disease, and addressed gaps in the environment to speed review and approvals. Panelists from regulatory agencies and HTAs will discuss different models practices they are using to acquire patient input into the decision-making process. Speakers should be able to articulate both the internal and external barriers for patients and agencies and they should outline solutions. Panelists will offer practical advice on how to build a coalition of patients, researchers, advocates and regulators to bring promising new therapies to patients. We are looking for 1-2 Patient Advocacy representatives who can represent and end-to-end story, plus a researcher from Industry, and a representative from a Health Authority, US and/or ex-US).

Discuss how to work with patient advocacy to develop, assess and approve new medicines.

Patient advocates Health authority representative (US or non-US)

Researcher (Industry or HCP)

Clinical trial data

04-06 Does Diversity Matter?

The theme of diversity in health care has been discussed for years, but industry and health authorities have yet to show sustainable increases in the participation of under-represented populations in research, prescriber behavior and patient uptake. How do we define diversity - age, gender, race, all of the above or something else? What are the elements of a "diverse patient population"? In an environment increasingly driven by biomarkers, does demographic diversity even matter? This session will address these questions and more in the form of a debate, sharing medical, social and cultural frameworks to explore the concept of diversity and the implications of efforts to address it in health care. Aspects discussed will include operational efforts, such as clinical trial operations, labeling, prescriber and consumer behavior, as well as geographic and community-based approaches. The panelists will speculate on what efforts may be most effective to achieve diversity in drug development and clinical practice, and what these successes might ultimately mean for patients. We are looking for speakers from a Health Authority, Patient Advocate, Researcher or Sponsor representative (such as Epidemiologist, Statistician, Recruitment Specialist, and/or Patient Engagement role), Site Community Outreach coordinator, Payer.

Recognize multiple ways to consider diversity in medicines development, labeling, approval and clinical practice.

Panelists to represent:(1) Health Authority representative(2) Patient advocacy(3) Sponsor representative (i.e., epidemiologist, statistician, recruitment specialist, patient engagement role)(4) Site community outreach coordinator(5) Payer

Diversity


04-07 Will Twitter (or Baidu) Change What's In Your Medicine Cabinet?

From online patient communities and global forums to blogs and individual accounts, people are using social media to learn about options, ask questions from peers and experts alike, explain their treatment decisions and share their experiences. At the same time, those involved in healthcare activities, such as recruiting clinical trials, marketing branded therapeutics and developing regulations, are increasingly relying on the conversations and behaviors they observe in social media to inform strategies and tactics. The panel will discuss how this dual use of social media for healthcare is likely to evolve over time, influencing drug development activities and treatment decisions. We will explore the concepts of content creation and observation as well as discuss concerns that present challenges, such as privacy, inequities in access around the world and other challenges. We are looking for speakers who can represent patients (advocacy, blogger), industry (clinical trial design, content creation, clinical trial and marketing communications), legal, regulator, social media platform provider. Preference will be given to those speakers who can suggest interactive formats as well as include a global perspective, especially Asia.

Participants will learn how to use multiple layers of social media to support conversations and shared decision making related to global drug development activities and treatment choices.

Patient/blogger Industry Regulator Social media

04-08 If You See Something (About a Clinical Trial), Do You Say Something (to Your Doctor?)

Patient enrollment and participation in clinical trials is a necessary part of bringing safe and effective medicines to the patients who need them, yet it presents a continuing challenge in drug development. Efforts to increase patient and provider participation in research often start with clinical trial awareness and education initiatives. This session features the latest generation of clinical trial awareness efforts (for example, the crowdsourcing Ideathon organized by INC Research and CISCRP), to campaigns led by patient advocacy groups as well as the industry coalition TransCelerate. The session will explore how these efforts are designed to bring new participants into the system, both patients and providers. Speakers will discuss measurable design elements of campaigns, such as calls to action that can help us trace awareness to enrollment. We are looking for speakers who can represent patients (advocates, individuals), providers (investigators, referring physicians), industry (individual sponsors or CROs or consortia such as TransCelerate). Preference will be given to speakers who can lead in a workshop format.

Describe elements of content creation, media planning and execution, as well as how to develop a metrics pathway and reports illuminating awareness to enrollment.

Patient / Patient advocate

Provider (investigators, referring physicians)

Industry (individual sponsor, CRO, or consortia such as TransCelerate)

Patient enrollment

04-09 We Can't Forget the Children in This Process

For this panel, we are seeking speakers to add to the discussion on how patient groups are incorporated into the process. We can't forget the children in this process. The trend of the regulators is that it will become mandatory to involve a Young Person’s Advisory Group (YPAG) in the process of approving the design of clinical studies and being integrated in all related developmental processes. Regulators are promoting the development in Europe of a European Network to centralize the collaboration between YPAGs, to ensure common curricula to train their members and to work with the same procedures and methodologies. Ideally, this panel will include: a representative from one of the 18 worldwide groups inside the umbrella of International Children's Advisory Network (ICAN) that has specific outcomes and best practices regarding active participation of young patients in the different stages of the clinical trial development process; as well as an Industry representative with experience involving a YPAG to discuss how to increase the input and influence of children and their families/ caregivers into the development of clinical trials. The panel will illustrate their internal challenges and solutions involved in interacting with a YPAG. Also open to including a Regulatory voice on how these guidelines are incorporated.

Demonstrate how to increase the input and influence of children and their families/caregivers into the development of clinical research.

Representative(s) from: (1) a Young Person's Advisory Group (YPAG)(2) International Children's Advisory Network (ICAN)

Industry representative with experience with a YPAG

Regulatory agency or regulatory affairs

Pediatrics; Patient Engagement;


04-10 How Is Digital Patient Engagement Impacting Trial Participation?

Seeking patients to participate in clinical research is still challenging, despite the advent of new digital engagement tactics. Where are the choke points in patient participation? How is digital engagement of patients addressing these chokepoints? How do we measure the outcomes of these engagements, i.e., address the return on engagement? Which qualitative and quantitative metrics are useful to assess impact?

Discuss data on choke points in patient engagement throughout the study planning and execution life cycle and get examples of how to intervene digitally; Define alternative metrics and understand how to utilize this information.

Industry / Vendor Patient community or other group that has been involved in seeking patient input

Digital patient engagement

04-11 Sharing the Burden Many in industry are interested in identifying new and innovative technologies to engage with patients throughout and after a clinical trial. The industry is enamored with multi-media eConsent platforms, but do they really help speed clinical trials to completion and reduce regulatory burden? When patients receive patient diaries, digital data capture tools, or even digital patient stipends does the learning curve and maintenance of use really benefit the patient? When sites have to log into multiple platforms to obtain study related information, it might require a new password for each platform. A more connected world enabled by digital technology can potentially help open up conversations in the area of clinical research, but it can also create more burden for investigator sites and patients when it is not streamlined or integrated. This forum will explore how these innovations are being implemented without adding burden to the sites and patient users. Speakers will discuss how they are innovative but also reducing the added burden onto the site or patient. We are looking for speakers who can represent sites and patient experience, as well as, sponsors, CROs, and vendors who have implemented solutions to these concerns.

Recognize how to anticipate site and patient burden due to the increased trend of introducing new technology processes; Describe ways to address site and patient burden.

Investigator Site Sponsor/CRO Patient Advocacy and Vendor

Digital technology, site burden, patient burden

04-12 No More Swag Items for Retention, So Now What Do We Do?

Over the years, it is harder to provide sites and patients with items to help retain their interest in a long-term or even short-term clinical trial. Now that the landscape has changed, how can sponsors create a solid retention strategy? Sponsors, clinical sites, and patient advocacy organizations will explore current best practices to retain a patient in a clinical trial. One group will explore measuring long-term treatment acceptance to help understand the factors leading patients with chronic diseases to accept their treatment. While adherence assesses behavior toward treatment, acceptance explains and possibly predicts behavior towards treatment. The ACCEPTance by the Patients of their Treatment (ACCEPT) questionnaire, which provides a comprehensive appraisal of acceptance of long-term medication, with six scores measuring acceptance of treatment-specific attributes, and one score measuring general treatment acceptance. ACCEPT was used on the Carenity platform, a European online patient community in which patients with a chronic disease can share their experience. These findings may help when developing a long-term retention strategy. Other representatives (e.g., Pharma/CRO/Investigator Site/Vendor/Patient) will provide a case study on how they retained patients in a long-term study. They will discuss their challenges and solutions.

Discuss the holistic view in the patient retention space; Describe a multi-strategy approach to encourage long-term retention.

Patient advocacy organization (e.g., ACCEPT representative - EU)

Industry representatives:(1) Sponsor (Pharma/CRO)(2) Vendor (implemented plan)

Investigator Site Patient retention, Patient adherence


04-13 Adoption and Implementation of Digital Platforms to Further Patient Interactions

Patients (also prospective study subjects) are using the internet to facilitate unprecedented levels of analysis, engagement, and communication around an explosion of online information exchange. This content ranges from social media posts to review of policy documents to conversation threads on research platforms. Health care product developers, professionals, researchers, and practitioners are leveraging these opportunities for non-traditional engagement to further engage with patients via platforms of interchange in the context of a clinical trial. This session would share experiences of real-world- platforms including how the patient perspective was incorporated in the design of the solution and how patients will benefit from their adoption.

Describe the characteristics of a platform that will improve the patient experience.

Patient advocate Sponsor/CRO Site/Institution Digital platform

04-14 Challenges of Patient Reported Data via Wearable Devices

Many recent efforts to have focused on directly engaging patients with improved communication or introducing flexibility in how they participate in a clinical trial with wearables. The introduction of patient‘s direct access to technology in the form of wearables has changed the patient experience. With access to accurate and timely insights on key patient-related indicators, Biopharma professionals can react quickly to and resolve issues before they more broadly impact the patient or the trial. This proactive approach to issue management is anticipated to improve the patient experience and potentially overcome other patient-related challenges such as adherence and retention. Wearables for patients reporting data digitally offers the potential for introducing new, innovative ideas for accomplishing both patient engagement but there are challenges to be managed. With planning and mitigation strategies in place, the patient wearables can lead to enhanced patient engagement. Speakers will share real-life experiences from patient, practitioner or vendor perspective. A Sponsor/CRO Data Manager may speak to the transition to wearables and the effect on data quality.

Identify challenges to the adoption of wearable technology.

Patient/patient advocate

Clinical monitor managing site

Data manager who receives wearable output

Wearables

04-15 For Better or Worse, Health Data Going Public Can Change Your Life

Patients have many tools available to them that can support and assure their privacy. Global and regional privacy regulations, such as HIPAA, offer protections for patients. These regulations have been both a blessing and a curse, depending on the role one serves. Many patients and some entire patient populations have tossed aside any personal requirement for privacy. Many positive opportunities are available for sharing information and this public-sharing may offer a treasure trove of data to support and enhance clinical development as well as medical treatment. From a research perspective, trying to find ways to leverage healthcare data for their purposes, many use (perhaps overuse?) HIPAA as an excuse for not innovating. In this panel discussion, we’ll offer insights on practical ways for development teams to assess how HIPAA rules and patient privacy guidelines impact both clinical trial operations and the daily lives of patients. Patient representatives will share insights to how full public disclosure may not entirely be the best choice in their daily experiences. Researchers will share best practices to balance the need for privacy with the progressing research opportunities.

Recognize the impact to patients of their sharing their personal history; Explain the impact of privacy rule enforcement.

Privacy advocate Patient/Patient Advocate

medical/ research practitioner

Privacy, clinical trial data

04-16 Defining the Science of Patient Input to Enhance Drug Development and Approval

Presenters will discuss how patient input and real world evidence can be used to meet the "adequate and well-controlled studies" standard. The panel will discuss the meaning of the science of patient input, and provide examples of successfully using patient input (e.g., in making trial endpoint decisions; weighing benefit/risk; modifying study protocols based on patient input, etc.) within the regulatory context to enhance drug development and approval by incorporating the patient voice. Gaps and challenges with suggested solutions will be discussed, and differences among US and non-US regulatory agencies may be considered.

Define the science of patient input; Describe the challenges and solutions to successfully incorporating the patient voice into drug development.

Policy expert to provide an overview of the landscape, policy changes, and policy implications impacting the science of patient input, may identify similarities and differences between US and non-US policy

Industry Representative to provide case study or examples of how patients have been successfully incorporated into the drug development program and decision-making

Regulatory (US and/or non-US) to discuss ongoing regulatory changes to facilitate improvements in the science of patient input, sharing examples of challenges and successes from the regulatory perspective

The Science of Patient Input


05: REGULATORY




Keywords

05-01 Expanded Access and Compassionate Use

Early access to medicines is increasingly sought by patients, physicians and legislators. Regulators and industry must work together to optimize patient access ethically and in a medically appropriate way while remaining focused on registration efforts. How to find the right balance? What are some case examples?

Discuss case studies of patient need and access approaches prior to regulatory approval.

Patient view Industry view Regulator or legislator view

expanded access, compassionate use, patients, unmet need

05-02 Regulatory Frameworks Amidst Disruptive Innovation

Regulators and sponsors must adapt today’s structures and expectations to effectively evaluate new technologies (such as gene therapy) and new treatment modalities (such as combination products or microbiome modification). In addition, the voice of the patient is being integrated into development and registration planning. This session will explore how regulatory frameworks are changing amidst such disruptive innovation.

Explore new regulatory paradigms needed when disruptive innovation approaches emerge.

New technology example: sponsor and regulator considerations

New treatment approach example: sponsor and regulator considerations

regulatory, innovation, technology, strategy

05-03 Recent Trends in Asian Regulations for Drug Development

Speakers from East Asian countries (Japan, China, Korea, and/or Taiwan) will discuss hot regulatory topics in each country. Discussions may cover multiregional clinical trials covering Asian countries, as well as drug development regulations and harmonization efforts in this geographic area

Discuss how Asia is an emerging region for worldwide drug development, especially with its large and aging population and the intense level of activity with respect to modernization and harmonization of regulations.

China hot topics Japan hot topics 3rd country hot topics Asia, Japan, China, Korea, Taiwan, harmonization

05-04 Minding the Gaps: Using Totality of Evidence to Support Regulatory Approval

Regulatory approval dossiers often lack full evidence in some areas. Sponsors and regulators can sometimes accommodate data gaps by relying on a totality of evidence evaluation. Accelerated or conditional approval regulations are examples where evidentiary flexibility enables earlier access to medicines. This session will explore cases in which regulatory approval was enabled through a totality of evidence presentation.

Describe using totality of evidence to support regulatory approval.

US accelerated approval example

EU conditional approval example

Etiplersen example accelerated pathways; strategy

05-05 FDA Intercenter Collaboration: Oncology Center of Excellence

Collaboration between FDA Centers is critical for product development and public health. The Oncology Center of Excellence is the first step in a possible broader reorganization of FDA.

Explore the latest progress resulting from recent FDA efforts.

FDA representatives FDA, CDER, CBER, CDRH, collaboration, Oncology Center of Excellence

05-06 What’s New in Devices: European Medical Device Regulations and MDUFA IV

With the new medical device and IVD regulations in the EU, plus the renewal of MDUFA in the USA, the year 2017 is a pivotal and disruptive period for medical device and diagnostic regulations. This session explores notable developments in global device regulation

Summarize recent global device regulatory changes; Describe potential approaches for development and lifecycle management strategies to ensure future compliance.

Regulator from EU or USA

Notified body representative

Medical device or diagnostic industry representative (regulatory, compliance

medical devices, MDUFA IV, IVD, Europe, US

05-07 Regulatory Challenges with Next Generation Sequencing

The high interest and rapid uptake of next-generation sequencing in the clinic speaks to the promise of this technology as a valuable adjunct for optimizing therapeutic treatment, yet much of this promise remains untapped. The challenge associated with establishing a regulatory framework stems from the lack of available standards to help establish the safety and effectiveness of this technology, and from the lack of high quality, public databases with data curated from validated methods for use in clinical decision-making

Explore recent efforts by regulatory bodies, academia, and regulated industry to curate data from this technology and use it for regulatory decision making.

Regulators involved in curating next-gen sequencing technology and data

Academic contributors advancing the technology in practical ways for the broader community

Regulated industry (diagnostic or drug)

next generation sequencing, technology


05-08 Lessons Learned from the Sarepta Exondys 51 Approval

FDA’s approval amidst controversy of Sarepta’s Duchenne muscular dystrophy drug Exondys 51 (eteplirsen) was unprecedented in terms of the level of flexibility used to support accelerated approval in a rare disease. This session explores the unique conditions surrounding the drug’s patient-focused drug development program and its approval.

Describe circumstances surrounding the development and ultimate approval of eteplirsen; Identify how to apply lessons learned to other drug development efforts.

FDA speaker who can talk about the drug’s approval

Patient perspective Industry perspective orphan drug, patient advocacy, muscular dystrophy, accelerated approval, dispute resolution

05-09 Evolution of Novel Registration Endpoints as Diseases Become Chronic

Recent advances in medicine have rendered once-acute diseases into chronic burdens, but serious unmet medical needs remain for these patients. Addressing these unmet medical needs with novel therapies requires new and flexible endpoints acceptable to global regulators. This session tackles possible approaches to address the endpoint gap using the immuno-oncology development paradigm as a case study.

Explore endpoint gaps for long-term diseases; Evaluate alternative endpoint approaches using immuno-oncology as a case example.

Health authority perspective

Industry perspective Clinical/Academic perspective

novel endpoints, registration criteria, chronic diseases

05-10 How to Work through Divergent Regulatory Advice in Global Development Programs

Although there has been a strong focus on harmonizing guidance and requirements across countries and regions, sponsors continue to receive burdensome divergent advice with respect to their global development programs. This session will discuss the status of several harmonization initiatives, review the opportunities and challenges, and examine selected case studies.

Discuss the complexity of regulatory convergence; Identify the progress being made through case studies.

convergence, divergence, harmonization

05-11 PDUFA at 25: A Historical View of the Prescription Drug User Fee Agreement and a Look to the Future - PDUFA VI

Development and approval timelines for drugs and therapeutic biologic products affect us all. This will be the first reauthorization occurring in parallel with a new Congress and a new administration. This session will focus on how PDUFA has affected both the regulator and regulated industry and what we can expect in the sixth iteration of the agreement.

Discuss a historical review of PDUFA from expediting approvals to expediting development; Identify what's new in PDUFA VI and the impact on sponsor and regulator.

FDA Speaker Trade Association Speaker

Regulatory Policy Industry Speaker

user fee, PDUFA, reauthorization, real world evidence, patient-focused drug development, biomarkers

05-12 The Reauthorization of BsUFA and its Impact on Both the Regulator and Industry

FDA and Industry have taken lessons learned from the first four years of the biosimilar user fee agreement (BsUFA) and applied them to this first reauthorization of the program. This session will focus on changes to the program and their affect on both industry and the agency.

Discuss what's new in BsUFA II and the impact on sponsor and regulator.

FDA Speaker Trade Association Speaker

Regulatory Policy Industry Speaker

user fee, BsUFA, biosimilars, new review program,

05-13 GDUFA II and Regulatory Science Initiatives: Challenges in Developing Complex Generics

GDUFA II creates a new program to support development of complex generic products including development meetings, pre-filing meetings and mid-cycle communications. This session will look at the details of the draft commitment letter and the future environment for complex generics.

Discuss what’s new in GDUFA II and the impact on sponsor and regulator

Regulatory Speaker Trade association representative

GDUFA,generics, complex generics, combination products

05-14 Is Getting the Right Evidence During Development to Support Both Registration and Reimbursement an Achievable Endpoint?

The current dynamics of bringing new medicines to market are being influenced by conflicts between the agendas of regulators, HTA and payers. The two processes of licensing and reimbursement are introducing an additional uncertainty into drug development decisions as market approval does not necessarily mean that the product will be reimbursed. The aim of this topic is to discuss the direction of change within the current landscape towards alignment and as more synchronized decision making between regulatory and HTA agencies, how companies are managing uncertainty by determining where the key differences are, and the implications of this to the changing development and approval models seeking increased flexibility in regulatory and access pathways.

Discuss the direction of change within the current landscape towards alignment and as more synchronized decision making between regulatory and HTA agencies; Identify how companies are managing uncertainty by determining the key differences ; Describe the implications of this to the changing development and approval models seeking increased flexibility in regulatory and access pathways.

HTA, reimbursement


05-15 The Evolving Model of Using Artificial Intelligence to Deliver Actionable Regulatory Intelligence

The breadth and scope of a regulatory intelligence function is often dependent on the knowledge of its employees, the tools they can access or the consultants they employ. What if we were able to employ machine learning and cognitive computing to help deliver actionable intelligence? This session will look at evolving models for Artificial Intelligence and the challenges in developing and validating these systems

Explore artificial intelligence concepts, tools, and case studies in the development of actionable regulatory intelligence.

Vendor speaker regulatory intelligence, artificial intelligence, regulatory strategy, precedent

05-16 Challenges in Developing a Global Strategy for Post approval CMC Changes for Combination Products

Drug/device combinations and biologic/device products continue to grow in complexity and offerings. One of the most complex issues for regulatory professionals is how to manage and submit post-market changes for a combination product. This session will explore the global regulatory challenges of lifecycle considerations for drug/device combination products.

Discuss the challenges in developing a global strategy for post approval changes for combination products.

CMC, strategy, challenges, drug, device, combination product

05-17 International Collaboration in Times of Public Health Crisis

International collaboration amongst regulators is important from the industry aspiration that it should suffice to develop one global dossier. In the wake of the Ebola crisis, and again during the current Zika event, regulators have and are demonstrating a willingness to find new and pragmatic ways of working together with industry with the ultimate goal of providing health care solutions which are in demand by society. Are there lessons learned that can be shared and discussed? Can the envelope be pushed even further?

Recognize using an interactive exchange of points of view on the current state of cooperation amongst regulators during Public Health Emergencies, the challenges faced by the stakeholders, and lessons learned in addressing them.

Ebola, Zika, tropical disease, collaboration

05-18 Global Compliance Scorecard on Supply Chain Issues

Creating and delivering safe and effective products that reliably meet customers' needs around the world is central to the health care industry . This session will look at how industry is making improvements to prevent diversion and counterfeit products from entering the supply chain and to discuss lessons learned from design, implementation, and serialization strategies.

Describe issues surrounding regulatory compliance within a global supply chain.

Quality, supply chain, track and trace

06: SAFETY AND PHARMACOVIGILANCE




Keywords

06-01 Safety Evaluation: Transforming Information into Evidence

Safety evaluation is a continuous process taking place throughout the life cycle of a medical product. Safety concerns have often been the primary driver in stopping or delaying development for many drug candidates. The lack of necessary safety data can compromise the regulatory acceptance of a program or a decision on a drug reimbursement. What are the different sources of safety data? How can one gather the necessary safety information without adding too much to drug development cost and without delaying the process of taking the effective medicine to a patient? In this session, presenters will review different sources of safety data and present medical considerations and statistical strategies of looking at pre-marketing stage of drug development, post-marketing safety surveillance, and other sources of real world data, and discuss how to use the totality of information to generate evidence for regulators, payers, physicians, and patients.

Identify different sources of safety data; Discuss how to use the totality of information to generate evidence.

Industry perspective: real world data to generate evidence and supplement clinical trials data

FDA perspective: post-marketing safety surveillance

Payer perspective safety surveillance, real world data, totality of information

06-02 Postmarketing Safety Studies: Approaches and Regulatory Insights

We are seeing an increase globally in the number of postmarketing safety studies requested by regulatory agencies. This session will discuss approaches to planning and conducting observational postmarketing studies, regulatory perspectives on such studies and a case example.

Discuss approaches to postmarketing safety studies and the ways in which they can have impact and provide value.

Speaker to discuss an overview of kinds of studies and reasons why might do one or the other.

Regulatory perspectives on the value and utility of postmarketing safety studies.

Interesting case study. For example, something that solved a difficult problem or had an interesting impact.

Postmarketing safety studies, PASS


06-03 Counting on Safety The regulatory landscape for safety monitoring is evolving, particularly in emerging regulation/guidance from the US FDA and EMA. This maturing regulatory landscape calls for a systematic and proactive process, multi-disciplinary collaboration, quantitative framework/methods/tools, built on a solid safety data architecture. Results from the efforts of the ASA Biopharm Safety Monitoring Working Group (2015-16), Clinical Trial Transformation Initiative (CTTI), and the proposed cross-function and cross-region joint ASA-DIA Safety Monitoring Task Force beginning in 2017 will have numerous topics, case studies and speakers of interest on this emerging topic.

Discuss the latest regulatory landscape for safety monitoring.

Industry / pharma rep(s) statistical perspective

US regulatory representative involved with the IND Reporting Rule and 2015 draft guidance

Medical judgment that comes before, during, and after quantification (this rep may also be from the ASA-DIA Safety Monitoring Task Force)

06-04 Integrated Life Cycle Approaches to Benefit -Risk Assessment, Communication, and Evaluation

Effective benefit-risk (B-R) communication is important in ensuring that key stakeholders understand how to use medicines appropriately. It requires the creation of processes to deliver communications that are relevant to their audiences, robust dissemination mechanisms, and evaluations to show they promote behaviors optimizing B-R. This session will address these areas.

Identify the importance of effective benefit-risk communication.

Speaker to discuss the use of B-R assessments throughout the product lifecycle and the importance of including patients’ perspectives in both the process and communications.

Speaker to demonstrate the advantages offered through the use of digital B-R communications in terms of availability, tailored content, links to relevant information, and ease of maintenance.

Speaker to provide insights into implementing postauthorization safety studies (PASS) for risk minimization material effectiveness evaluations across Europe

Benefit-risk

06-05 Justifying Pharmacovigilance Legislation Based on its Impact

A trend is emerging to consider that pharmacovigilance regulations need to be justified in terms of their impact on patient safety, rather than simply being reactions to past concerns. If this idea catches on, it has huge implications for industry and regulators. This session will explore the implications of justifying pharmacovigilance regulations based on their impact on patient safety.

Describe the implications of justifying pharmacovigilance regulations based on their impact on patient safety.

Industry / pharma rep(s)

US regulatory rep Patient advocate rep(s)

06-06 Designing and Implementing a Robust Pharmacovigilance System for Vaccines

This session will provide insights into the distinct challenges in vaccine pharmacovigilance. Vaccine postmarketing surveillance is critical to mitigating risks, aiding in a response to vaccine safety concerns, and building public trust. The session will present the common need around the globe to manage public concerns about events related to vaccine safety. It will also discuss the differences in safety reporting requirements in vaccines in the US versus EU and the specific points to be taken into account in safety management and signal evaluation of vaccines both from the US and EU perspective.

Discuss differences in safety reporting requirements in vaccines in the US versus EU and the specific points to be taken into account in safety management and signal evaluation of vaccines.

US perspective EU perspective An interesting case study.

06-07 Automation and Innovation in Clinical Trial Safety Assessment

Many aspects of monitoring and assessing safety data are resource intensive and becoming more so. For example, changes in laws such as the U.S. IND safety reporting rule have resulted in ever-growing demands on sponsors. The current model of adding more staff and using the same process and tools is not sustainable. In this session we will look to innovation and technology for practical solutions to support clinical trial safety monitoring and reporting needs.

Learn some practical ways to use innovative tools to support clinical trial safety monitoring and reporting.

Any novel ideas for innovating and automating safety-related monitoring and/or reporting.

Infrastructure-related topic related to automation and innovation

Automation of patient narratives

06-08 A New Age for Physician and Patient Labeling: Recommendations for Innovative Change in How We Communicate

Recently there has been interest from many groups to change the way labeling is written. This session will explore recommendations and recent research on this topic.

Discuss recent recommendations and research on communication of product information via the product label.

Industry / pharma rep(s)

Regulatory rep Patient advocate rep(s), or researchers who have investigated what methods are effective in communicating with patients


06-09 The Brave New World: The Ongoing Globalization of Pharmacovigilance:

Increasing pharmacovigilance requirements and guidelines by global regulatory agencies is causing the pharmaceutical industry to globalize and harmonize across pharmacovigilance organizations. Practical strategies for breaking down silos, sharing best practices and expected outcomes will be presented. Topics to include: process harmonization, optimal management of local safety offices, optimal governance models, and successful change management.

Discussions will explore the challenges, benefits, best practices, and lessons learned in globally harmonizing one's pharmacovigilance organization.

Industry/pharma rep from emerging/mid-size pharma currently engaged in globalization, with a focus on the challenges and key considerations

Lessons learned from a industry/pharma speaker who have completed globalization

Industry/pharma representative to discuss global governance along with local affiliate and partner oversight

06-10 Exploring the Evolution of Signal Detection

Since the initial release of GVP Module IX - Signal Management, pharmacovigilance organizations have gained a considerable amount of experience. The EMA has released Module 9 R2, which incorporates the key learnings to date. This session will explore notable changes, advances in signal management, and how organizations are adapting to meet the new guidelines.

Describe the updates to Module 9 and how they impact one's pharmacovigilance organization.

Industry / Pharma rep(s) engaged in signal detection

Regulatory representative providing perspective on the new guidelines

Industry / pharma representative to discuss the impact of new guideline on processes and organization

06-11 Changing Environments within Pharmacovigilance

Updates in pharmacovigilance and safety in various markets with changing regulatory environments. Speakers will include: FDA, EMA, as well as other key regions such as Japan, China, and emerging markets like Africa.

Discuss changes in regional and global regulations and how they impact one's pharmacovigilance organization.

FDA regulatory representative to overview recent updates

MHRA/EMA regulatory representative to overview recent updates

Industry/pharma representative to overview recent updates from Japan, China, Africa, Latin America

06-12 New Considerations in Benefit-Risk Management

Benefit-Risk Management continues to evolve as noted changes have been seen with ICH's M4E (R2) -Guideline on Enhancing the Format and Structure of Benefit-Risk Information along with the proposed changes to the EMA's Module V - Risk Management Systems and the FDA's draft Guidance on FDA's Application of Statutory Factors in Determining When a REMS is Necessary. This session will focus on proposed changes and the impact on MAHs. Best practices and change management efforts will be discussed.

Describe the updates and proposed revisions will impact one's pharmacovigilance organization.

Speaker from ICH Expert Working Group responsible for M4E (R2)

Regulatory representative providing perspective on the new guidelines

Industry / pharma representative to discuss impact of new guideline on processes and organization

06-13 Pharmacovigilance 2.0: Redesigning for the Future

Pharmacovigilance is a highly regulated environment with defined requirements for compliance and quality. The rapidly evolving world of artificial intelligence and machine learning has been applied to many industries with much success. How can these technological advances be applied to pharmacovigilance? Do they have a place in pharmacovigilance? Can they truly deliver the 3 C's: control, consistency, and cost savings? Real world examples will presented where these innovations are already in use, the difficulties encountered, and how organizations are harnessing the art of the possible.

Explore available automation and artificial intelligence technologies; Discuss where these innovations are being applied; Describe tools to assess organizational readiness for this major redesign.

Industry/pharma /CRO representative to discuss real world examples and application of artificial intelligence and machine learning

07: SPECIAL POPULATIONS




Keywords

07-01 Integrating the Patient's Voice Across the Development Program of Rare Diseases

Patients living with a rare disease have a direct stake in the end-benefit, and as a result should have their perspective included in the process of developing drugs. Inclusion of the patient's knowledge and experience, the Patient’s Voice, throughout the investigational continuum can help address the many drug development challenges, yield better clinically meaningful outcomes and get therapies to patient's faster.

Describe how the patient’s perspective profoundly affects all dimensions of drug development.

Patient advocacy Group

Pharmaceutical or CRO representative:(1) US(2) EU

FDA, COA staff


07-02 Engagement, Education, Networks, Media and Societies in Rare Diseases: The MUST Haves

Patient networks, patient engagement and education is critical to patients' involvement in clinical trials. This plays out through social media and patient networks to disseminate information about opportunities more rapidly through a much broader audience. Due to the limited number of potential subjects , the contribution of multiple medical groups to a given trial necessitates the development of physician/patient networks as an integral part of the recruitment process. Societies are instrumental in disseminating , demystifying and educating patients about clinical trial opportunities which can assist in recruiting, retention and conducting these trials.

Describe and discuss communication pathways and approaches between key stakeholders in the development of rare disease therapies

Patient advocacy Group - Non-profit


Pharmaceutical representative or CRO

07-03 Innovative Designs and Statistical Approaches for Small Trials (Rare Disease or Pediatric Indications)

With the advance of the computer technology, developing personalized medicine is not an unachievable dream in the 21st century, although it remains a challenging task to efficiently conduct clinical trials with a limited number of patients. It is important to develop new methods by utilizing all available information and resources without lowering the current standard for assessing drug safety and efficacy.

Discuss how implementation of innovative designs and newly developed statistical approaches for small clinical trials such as rare disease or pediatric populations will aid the drug development process and draw all relevant stakeholders’ attention and interests.

FDA Representative Pharmaceutical Representative

Industry Representatives:(1) Pharma (2) CRO

07-04 Recruiting Rare Disease Patients: Objects in the Mirror May Be Closer Than They Appear

Many rare disease clinical trials fail due to enrolment challenges caused by the scarcity of the rare disease patient population and the corresponding logistical challenge of identifying sites within the reasonable vicinity of the patients. But what if it was possible to identify where the rare disease patients were, and even help confirm the diagnosis by enabling earlier detection? What if it was possible to map the investigators closest to the patients to help increase likelihood of recruitment AND retention, bringing in the need for patient centricity in the design of rare disease protocols? Alongside the therapeutic innovations revolutionizing the future treatment of rare diseases are two broader, parallel developments: 1) the expansion of real-world data (RWD) and 2) innovation in the analytical methods that can be used to interrogate the data. When brought together and supported by clinical insight, these allow for the development of screening algorithms. This in turn presents a major opportunity to help detect new, undiagnosed patients and subsequently map them to potential investigators, for a more sophisticated Physician to Patient heat-mapping methodology, referral plan or even telemedicine strategy.

Identify new tools leveraging real-world data and innovation in advanced analytics including the growing field of machine learning technology, and how the advancements in this field are creating opportunities for dramatic improvements in identifying hard-to-find patients.

Pharmaceutical Representative

CRO Advocacy Group

07-05 Pregnancy and Lactation Labeling Rule: Unique Challenges to Meet Requirements

In December 2014, the FDA published “The Content and Format of Labeling for Human Prescription Drug and Biological Products; Requirements for Pregnancy and Lactation Labeling,” referred to as the “Pregnancy and Lactation Labeling Rule” (PLLR). Drug manufacturers are required to provide specific details pertaining to its use in pregnancy, lactation, and reproductive risks associated with the drug's use. The rule allows for statements in labeling to indicate that no data exists, however, companies are encouraged to generate data for both pregnancy and lactation, which raise very interesting questions for drug manufacturers and the patient populations that they treat. This session will examine many of these questions. What are the obligations for manufacturers to generate data on the safety of their products for use during pregnancy or lactation? Are current methods of data generation or capture, such as registries, effective, and are there alternatives? In the case of lactation, how do you recruit patients for lactation studies? Should breastfeeding be encouraged as much as possible, even for mothers who need to take drugs that might be passed to the infant via breast milk? Is the obligation to generate data and encourage breastfeeding greater for products that are known to be used more by women?

Discuss and learn about both the public health benefits of breastfeeding, the ethical challenges of pharmaceutical use during lactation, and the challenges of generating sufficient data to comply with the requirements of the Pregnancy and Lactation Labeling Rule

FDA representative(s) Pharmaceutical representative

CRO


07-06 Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes

As most rare diseases have no approved therapies, these patient populations are frequently desperate for any kind of treatment. As a result, they request earlier and more extensive access to investigational drugs before many of the safety and dosing questions have been addressed. This could result in erroneously high adverse event profiles or subjective benefits that skew the development program. How do we better define how to broaden access while simultaneously maintaining rigid control of the data?

Discuss the importance of collaboration between key stakeholders in the for profit and non-profit sectors in the development of Early Access therapeutic approaches for rare disease.

FDA representative(s) Non-profit Industry Pharmaceutical representative or CRO

07-07 Collaborative Efforts to Accelerate Rare Disease Research and Development

This session will provide rare disease case studies where regulatory agencies, industry and advocacy groups collaborated together to accelerate and advance clinical research .

Describe case studies where regulatory agencies, advocacy groups and industry can work together to advance clinical research.

FDA representative(s) Pharmaceutical Representative

Pharmaceutical Representative or CRO with EU Perspective

07-08 CNS Impairment Guidelines and the Impact on Relevant Topics Pertaining to the Brain

This session will review the general evaluation of cognitive function for new drugs being tested and improvements in function. Areas of interest may include concussion management as a topic to assess scales used or suicidality assessments for new drug assessments.

Identify new tools to look at brain function in drug research from a safety and efficacy perspective.

Specialty vendors for screening evaluations


Regulator

07-09 Is the Future Bright for Treatment of Neurodegenerative Diseases?

Despite decades of research, we continue to search for meaningful treatment in neurodegenerative diseases such as Alzheimer's and Parkinson's. This session will focus on the impact of patient associations on the progress of treatment. A case study could be explored.

Discuss where we are today and what is the future in treatment of diseases such as Alzheimer's or Parkinson's; Describe the impact the associations have had in improving treatment.

Foundation representative(s)

Patient advocacy Pharma

07-10 Exploration of Metabolic Diseases: Is the Liver the New Heart?

With the increasing prevalence of metabolic disease, this session will explore the rising incidence of metabolic disease such as diabetes as well as emerging areas of metabolic diseases such as NAFLD/NASH. With new imaging technique enhancements, we observe previously undetected liver scarring progressing to fibrosis and onto cirrhosis. With no NAFLD/NASH-specific treatment to date, the FDA has provided recommendations in this indication.

Discuss why liver fibrosis is becoming a concern in the pharmaceutical industry and is an area needed for therapy.

Academic site PI Industry:(1) Pharma(2) CRO

FDA

07-11 Issues in Early-Phase Oncology Trials

Subjects in early-phase oncology trials represent a special population, particularly in the age of precision medicine with many of the new therapies targeted to specific mutations or being developed for rare types of cancer. This session will provide background on early-phase oncology trials, review some of the study designs that are often used, and discuss issues with recruitment and retention unique to these trials.

Discuss the complex issues in early-phase oncology trials


Pharmaceutical Representative ex-US

CRO

07-12 Increasing Participation in Clinical Trials Among Under-represented Populations

Increasing diversity in clinical trial participation has been and remains a goal of those involved in developing medicines, including pharmaceutical industry organizations, investigative sites and regulators. Several high profile campaigns have failed to move the needle, while smaller scale initiatives are showing promise. This session would mention regulatory guidance, but would focus more on the "how" of gaining clinical trial participants from diverse backgrounds, including age, gender, race/ethnicity and geography. Sessions under this topic may include initiatives based on ethnic/racial diversity, age, gender, geography and other characteristics of special populations. Beyond regulatory mandates, key elements of successful efforts include focusing on the 1:1 conversation between patients and physicians, resources related to improving access to care (financial, geographic, insurance), referral arrangements between practicing physicians and investigators, establishing broad community-based networks around research institutions. Additional aspects to cover include inclusive clinical trial design and data generation with diverse populations.

Discuss strategies to engage and retain patients in under-represented populations; Describe challenges of including under-represented patients in novel clinical trial designs and data generation.

Patient Advocacy representative(s)


Academic representative


07-13 Looking to the Future for EU Pediatric Investigation Plans (PIPs)

EU pediatric investigation plans (PIPs) have become a routine part of the global drug development process. Small and medium-sized US companies that develop new and efficacious drugs are less prepared for the enthusiastic pediatric requirements from the European Medicines Agency. As a consequence, once they approach EU registration, they give in more to EMA's PIP requirements than would be necessary. Even large companies are unable to avoid commitment to questionable studies in children. This has become a serious obstacle in the development of efficacious new drugs, and industry needs better arguments to push back requirements that are expensive and do not improve child health. In 2017 there will be new report on the EU pediatric legislation to the EU parliament. While the EMA and EU Commission will report on its successes (more PIPs, more clinical trials in children), there are concerns being voiced about having even stronger pediatric obligations from the pharmaceutical industry. This session will focus on the need for better PIP analysis, analysis of lessons learned, and reflections on how to modify the EUPL, while offering a chance for a substantial improvement in industry’s relationship with clinicians and parents. It will also examine how we can prepare the groundwork for countering new proposals to impose stronger pediatric obligations in the EU and US.

Discuss the need for better PIP analysis and analysis of lessons learned.


US Regulatory Affairs (Pediatric)

EU Regulatory Affairs (Pediatric)

08: STRATEGIC PLANNING, EXECUTION, PARTNERSHIPS




Keywords

08-01 So You Say You're an Extrovert: Being an Effective Leader When All You Really Want Is a Stage

Some might argue that extroverts make natural leaders given their love of people and engagement. But not all extroverts are effective leaders. Their need for interaction and attention can sometimes hinder their ability to allow space for others and hear the positions of their peers and direct reports. In this forum, we'll hear from individuals who have worked at overcoming being extroverted as well as introverts who have effectively collaborated with extroverts and learned how to engage others to create a collaborative, equitable environment in which a variety of personalities can thrive.

Describe the pros and cons of being an extroverted leader; Identify effective strategies for collaborating with both extroverts and introverts.

Individual in leadership position who is an extrovert

Individual in a leadership position who is an introvert

Individual in leadership position who is an extrovert

leadership, influence

08-02 Effective Collaborations: Navigating the Grey Space

R&D partnership models are a common arrangement that can be found in small and large companies alike. While legal agreements provide the guardrails for those engagements, the success of a partnership can hinge on how well the alliance managers and relevant team members manage the grey spaces not explicitly covered in the pre-arranged contract. In this forum, we will talk through some areas that often fall into this grey space such as data ownership, firewalls, and governance committees.

Identify areas that may not be commonly covered in collaboration agreements and learn how to effectively negotiate those spaces while maximizing the health of the collaboration.

Legal/contracting expertise in M&A

Alliance Manager Speaker with experience forming a joint governance committee for an alliance

collaboration, alliance management, contracts

08-03 Post-Trial Access: Ensuring Patient Access Across the Development Spectrum

Clinical trials are an important way many patients gain access to life saving treatment they might not otherwise be able to obtain. Once the trial ends for an individual patient, what happens during the time treatment stops and the investigational agent becomes approved? Sponsors of clinical trials for life threatening diseases need to plan proactively for the management of this transition and engage with downstream stakeholders to ensure continued access to beneficial treatments.

Define what is meant by post-trial access; understand the bioethical principles behind providing post-trial access; describe the planning that should be undertaken in advance of initiating a trial for a life threatening disease.

Speaker with policy or guideline development experience in this area (e.g., Harvard MRCT Working Group on Post-Trial Responsibilities)

Bioethicist with an expertise in pharmaceutical R&D or this particular topic

Clinical / Medical leader that has dealt with this issue

access, bioethics

08-04 Strategic Planning Across Research Organizations

Leveraging innovative technologies, cutting edge research, and unique capabilities are the hallmarks of robust drug discovery and development. Large academic centers, hospital research facilities, government organizations and for-profit pharmaceutical companies approach these activities differently. Understanding these perspectives can lead to more effective collaborations.

Inform audience about how different industries conduct clinical research; Identify key differences that can be leveraged in executing effective collaborations; Exposure and learnings from variety (type) of R&D organizations.

Speaker from an Academic Research Organization

Speaker from government research organization (e.g., NIH) or hospital

Pharma industry representative

Academic Research; Collaboration; Operations


08-05 The Art of Negotiation: Preparing Yourself and Your Team for Their Seat at the Table

The ability to facilitate rapid decision making is a key skill for a project manager; however, sometimes speed can come at the expense of one of your stakeholder groups. How can you improve your influence and negotiation skills in order to help those critical conversations be more well rounded and focused on collective goals rather than personal agendas?

Identify tools and tips for effective negotiations.

Project manager or Project Leader with experience in negotiating/influencing

Project Manager or Project Leader with experience in negotiating/influencing

Project Manager or Project Leader with experience in negotiating/influencing

Leadership; Negotiation, teams, decision making

08-06 Effective Life Cycle Management

During development of a novel pharmaceutical candidate, the focus is often on near term and immediate success as costs and attrition need to balance across a portfolio of candidates. However, subsequent to regulatory approval, commercial asset teams are obliged to address several important life cycle expectations. How do organizations manage this space and allocate resources to support? What work/planning can or should be completed pre-approval that would set the program up for success? This forum will give an overview of typical life cycle management activities and walk through some case studies that highlight different approaches (e.g., serial planning, parallel planning).

Discuss activities and resources needed to effectively manage product life cycle.

Commercial /New Product planner who can help give perspective on value, tradeoffs, market influences

Clinical or Project leader who has experience in commercializing a late stage asset

Portfolio manager Life Cycle, Resource planning

08-07 Flexing Your Project Management Muscles Outside of Traditional Roles

Typically the expectations for a Project Manager on a drug development team are very clear and defined. However, there are opportunities to leverage project management (PM) skills and experience in areas of organizations that may not have a formal PM function or might call the role something different. In this forum, hear from different project managers about their experience in flexing their PM muscles in different ways.

Describe the various roles and responsibilities of project managers either in non-drug development projects or in junior roles.

Project Planner/ Coordinator/ Associate/ Junior Project Manager role across companies

Non-R&D project managers

Project Manager who has experience in a drug development role but who is now in a different one

Project Management Roles; Project Manager Development

08-08 The Impact of Industry Consortia on Creating Collaborative Solutions to Drug Development Challenges: What Does the Future Hold?

It takes $2.6 billion in capitalized costs to develop a new drug. Only one in 10 new drug applications is approved, and cycle times are not faster compared to the mid-90s. Many companies continue to spend time and energy on addressing challenges that do not bring competitive differentiation. The Avoca Quality Consortium along with other industry consortia such as TransCelerate, CTTI and others have enabled sponsors, CROs, and specialty providers to leapfrog solution development through common, collaboration platforms. In this forum, we will bring together leaders of consortia to talk about how the availability of consortia has helped organizations shave time and cost from drug development.

Discuss how consortia can provide benefits in drug development.

Speaker who is a senior from TransCelerate (e.g., sits on the board)

Speaker who is a senior from CTTI

Speaker who is a senior from a relevant CRO

consortium, collaboration, disruptive innovation

08-09 Keys to Effective Product Launch Preparation

Given the small number of products that often make it successfully through development, not many people have the chance to launch a product. This session will describe a framework for effective launch preparation and how different functions should begin preparing and planning for a successful launch.

Describe a launch development model; Discuss examples of global launch successes and failures.

Speaker who can describe the launch framework

Speaker who could describe a case study for a successful launch

Speaker who could describe a case study for an unsuccessful launch

launch preparation, global

08-10 Effective Portfolio Management and How to Ensure You Get the Value Out of the Decisions Made

Making timely portfolio decisions is critical to ensure organizations achieve the value of the exercise. This session will discuss how those decisions can be made and communicated effectively. Speakers will also address how to ensure timely closeout once a decision to discontinue or deprioritize has been made.

Discuss basic concepts of portfolio management decision making; Identify best practices for ensuring timely close-out of a project.

Portfolio manager to provide insight into how portfolio prioritization decisions are made and communicated

Project Manager with experience in winding down or closing out projects

Portfolio Management, decision making, prioritization

08-11 Accelerating Drug Development Through Innovation

There has been a lot of discussion recently around accelerating drug development via innovative trial design. This can lead to shortened timelines and bringing products to patients more quickly. There are a variety of stakeholders involved in the successful execution of these innovative plans. In this workshop, participants will be given a case study in which they have to create a solution leveraging innovative ideas that will have been highlighted by subject matter experts.

Describe the application of innovative drug development methodologies.

Speaker with experience in executing innovative clinical trials (e.g., adaptive design) or other non-traditional approaches to generating clinical evidence

Innovation, drug development, adaptive design


08-12 Crisis Management in the Face of a Global Health Pandemic

Crises can never be fully anticipated. There is a need to activate a response plan with little preparation. This requires a blend of skill and organization so that resources and individuals can be activated quickly to address the crisis. Learn about a crisis management framework and how this can be applied to the management of a public health crisis.

Describe a crisis management framework and how it can be applied to health care.

Crisis management specialist - preferably with health care background, but may be multidisciplinary

Public health worker who has done planning for global health pandemic

Researcher who was involved in response to a global public health crisis (e.g., Ebola, Zika)

crisis management, global public health,

08-13 Leading in the Midst of Ambiguity

While projects are often well-defined, project leaders can often find themselves in situations where a project or initiative does not have many clear guardrails or defined outcomes. Being effective in this type of situation requires the confidence and ability to take prudent risks and navigate teams through uncertainty. Participants in this workshop will work through case studies and practice skills needed to be successful in these situations.

Practice leadership skills that can be applied to the effective management of ambiguous or uncertain situations.

Project leader with experience leading strategic initiatives

Project leader with experience in leading managing complex drug development program

leadership, career development

09: TRANSLATIONAL SCIENCE: PRECLINICAL, CLINICAL, AND DRUG DEVELOPMENT




Keywords

09-01 The Use of Wearable Devices in Clinical Trials, or My iPhone Just Took My Blood Pressure!?

This session will cover aspects of design, implementation, and data analysis related to wearable devices. Case studies are strongly encouraged.

Discuss regulatory framework, development and validation of instruments/devices, and analysis issues; Describe examples of use in clinical trials.

US Regulatory Affairs Professional

Content development - experienced content developer

Implementation - experienced with implementation of Clinical trials that include wearables

Wearables

09-02 Use of Novel Trial Designs to Accelerate Decision Making: To 'P' No More

This session will provide case studies where Bayesian statistical approaches and adaptive designs have facilitated go/no-go decisions in small proof of concept studies.

Discuss Bayesian framework for decision-making; Describe early phase scenarios for adaptive trial designs.

Statistician Clinical lead Trial design, adaptive design

09-03 What Question Are You Trying to Answer? The Use of Estimands, Potential Revisions to ICH E-9 and the Impact to Trial Design and Analysis

This session will cover the proposed revision to ICH E9 that introduces the use of estimands and their use in answering clinical trial questions, particularly in the case of missing data. Examples of interactions with regulators and the impact on protocols and analysis plans will be discussed.

Identify the definitions and intended use of estimands.

Regulatory Perspective:(1) FDA representative(2) European regulatory(3) Someone currently working in Regulatory Affairs

Statistician Statistics, ICH, estimand

09-04 Novel Technological Approaches to Identify Subjects and Augment Recruitment

This session will look at emerging sources of information such as electronic medical records and associated technology to find subjects. It will also examine metrics of case studies that demonstrate their ability to improve recruitment metrics and strategy.

Describe the changing environment of data availability; Explain metrics from case studies; Identify data sources.

Big Data vendor/expert Clinical recruitment specialist

Patient recruitment

09-05 Prior Elicitation: Estimating the Unknown

Clinical trial designs are often based on assumptions on effects (e.g., efficacy, safety) that are not robust. One approach to augment these assumptions is the use of prior elicitation to characterize the uncertainty of such estimates. These estimates can be used to determine probability of success, mitigate risk, and improve trial designs.

Describe a Prior Elicitation approach; Discuss the type of information generated; Identify uses for elicited estimates.

Clinical Project/Program manager

Statistician Elicitation, Bayesian design

09-06 Master Protocols: New Approaches to Enrolling Hard-to-Find Subjects and Expediting Drug Development

Master ("umbrella") protocols that have multiple treatment options are being used to optimize enrollment and expedite drug development based on biomarkers/specific disease traits. This session will discuss this approach and the benefits and challenges of these multi-company collaborations.

Describe the Umbrella concept; Discuss key considerations.

Clinical Operations Master protocol, Umbrella protocol, protocol design

09-07 Audits and Agency Inspections of Risk-based Monitored Studies: The Results Are In…

Sponsors, investigator sites, regulators and CRO organizations are invited to share the results of audits and inspections in which risk-based monitoring studies have been assessed.

Discuss the effectiveness of the implementation of the risk-based monitoring approach as judged by auditors and inspectors.

Regulators (US and non-US)

Sponsor Auditor Investigators, CROs with risk-based monitored studies that have been audited or inspected

Risk-based Monitoring


09-08 Trial Master File: MHRA/EMA Inspections and Sponsor Audit Learnings

Sponsors and MHRA or EMA GCP inspectors are invited to share their experiences and learnings from the MHRA or EMA inspection of the TMF.

Discuss the activities performed pre-audit or pre-inspection, the reactions of the auditor or MHRA or EMA inspector during the inspection as a result of those efforts, as well as plans for future improvements.

MHRA or EMA inspector

Clinical Operations Auditor Trial Master File

09-09 Evolving Clinical Trial Guidance and Regulations: Am I Ready?

Presenters are invited to share 1) an overview of the ICH E6 addendum and EU Clinical Trial Regulation 536/2014; and 2) a practical demonstration of how to perform an impact assessment to existing processes and procedures.

Describe the changes and assess the impact to existing procedures and processes.

Quality Clinical Operations Good Clinical Practice

09-10 Of Course My Data Has Integrity … And I Can Prove It

Presenters will share 1) an overview of the data integrity expectations for good clinical practice (GCP) (using MHRA draft guidance as foundation); 2) case studies (with different data sources and media) of how the described expectations have been executed in the GCP environment; and 3) differences between data quality and data integrity.

Distinguish between data quality and data integrity; Describe attributes and application of data integrity expectations in GCP environment.

Quality Data Management Data Integrity

09-11 Patient-Relevant, Fit-For-Purpose Endpoints: Is this Achievable?

It is not easy to develop novel patient-relevant clinical outcome assessments (COAs) and endpoints for drug approval, leading some to question if it is even achievable to do so given the practical demands of drug development. This session will cover regulatory and industry perspectives on successfully developing such COAs and endpoints that are fit for purpose in drug approval. Regulators will share examples of the tools and partnerships they are developing in order to facilitate use of novel assessments and endpoints that incorporate the patient voice. An industry representative will provide insight into decision-making within drug development teams regarding the development of novel endpoints, as well as discuss practical and logistical challenges that can be overcome. Best practices for engaging with the FDA (and EMA), and even patients, in order to increase the likelihood of regulatory acceptance of proposed novel endpoints will be covered.

Describe the challenges and solutions, including available tools, meeting types, and partnerships, to successfully develop and implement patient-relevant, fit-for-purpose endpoints in clinical trials.

US Regulatory (FDA COA Staff) to share perspectives on tools and collaborations with FDA that can assist in developing patient-relevant, fit-for-purpose endpoints (e.g., update on the compendium, Critical Path Innovation Meetings examples, qualification partnerships, best practices for sponsors to interact during IND process, etc.)

Industry (HEOR) representative to share perspective on convincing clinical teams of the value of "taking the risk" to incorporate new patient-relevant endpoints; share success stories as well as practical and logistical challenges overcome

COA developer to share perspective on best practices for developing novel outcome assessments to use as key endpoints in clinical trials, and provide practical suggestions for engaging with FDA and EMA regarding novel patient-relevant endpoints.

Clinical Outcome Assessments

09-12 Patient-Reported Outcomes (PROs): Hot Topics

Patient-reported outcomes (PROs)are not new. The FDA released a final guidance on this topic in 2009, and there have been many presentations and discussions on the evidence and measurement principles used to guide development of fit-for-purpose PRO assessments. This session will move the conversation from basic PRO development to discuss the latest hot topics regarding PROs in drug development. Hot topics might include (but are not limited to): FDA's flexibility in implementing principles in the guidance; methods to evaluate meaningful change on PRO assessments; strategies for incorporating PRO endpoints into multiplicity-adjusted endpoint hierarchy with traditional endpoints; novel methods to validate new PRO tools).

Summarize practical insights and methods that can be used to facilitate the development and use of PROs in clinical trials.

FDA representative Industry/PRO Expert Patient-Reported Outcomes

09-13 Experimental Studies: New Approaches to Study Designs to Facilitate Early Phase Decision-Making

This session will follow the life of a biomarker from the nonclinical arena to its use in clinical trials. The characteristics of a useful biomarker will be discussed. Examples of protocol designs and statistical analysis approaches that use mechanism of action at the molecular level to inform go/no-go decisions will be discussed.

Describe the characteristics of a useful biomarker; Illustrate examples of protocol designs and statistical analysis approaches at the molecular level.

Nonclinical scientist Clinical lead Statistician Biomarkers, Experimental Medicines

09-14 Novel Techniques for Improving Clinical Trial Subject Retention

Speakers are invited to share innovative techniques and approaches to reduce the number of subjects lost to follow up in a clinical trial and discuss the statistical impact of reducing loss to follow up on the validity of clinical trial results.

Describe approaches and techniques for decreasing the number of subjects lost-to-follow-up to reduce the impact on study validity.

Statistician Clinical operations Patient retention


09-15 Optimizing Early Clinical Strategies to Support Breakthrough Therapy Designation

Preliminary clinical evidence showing substantial improvement over existing therapies on at least one clinically significant outcome is required to qualify for breakthrough therapy designation. Presenters are invited to share insights for defining “acceptable” therapeutic thresholds.

Describe the key considerations and challenges in the design of early clinical trials to provide sufficient evidence of early benefit; Discuss successful case examples of novel therapies that have never before received FDA approval.

FDA representative Industry representative(s)

Breakthrough therapy

09-16 Calling All Angels to the Valley of Death

The Valley of Death refers to the obstacles that are keeping innovative medical research discoveries from becoming new therapies or even making it to clinical trials. Funding is necessary to bridge the gap. This session will provide insight into strategies used by translational research scientists and funding agencies to expedite the transformation of basic research into break through clinical applications.

Describe strategies used to expedite the transformation of basic research into breakthrough clinical applications.

Scientist or Physician Scientist to present case study

Representative from funding agency (e.g., government, nonprofit, foundation, venture capital, investor)

Breakthrough therapy

09-17 Fusion Study Designs: Balancing Cost and Risk

The need for rapid development timelines has encouraged rapid dose escalation, adaptive designs, inclusion of multiple biomarkers in clinical research and in some cases abbreviated investigator brochures. How does this fusion development conflict with signal dosing, extensive animal data and more leisurely timelines? here are additional challenges around cost and speed to be considered. How do we balance these conflicting interests and what are the trade offs?

Describe the impact of adaptive or fusion study designs; Explain how and if they save time and money.



CRO

09-18 Exploration of PK/PD in NDA Enabling or Early Proof of Concept Studies

The session explores sophisticated PK/PD modelling in NDA enabling studies such as in hepatic and renal studies conducted for labelling requirements or provides an exploration of PK/PD for early proof of concept (POC) studies which may help to progress development more quickly. Specific therapeutic areas of PK/PD models in patients may be explored. Examples of a topic may include antiviral drugs revealing a drop in viral load associated with exposure to the drug.

Discuss how sophisticated studies help in early clinical research to determine if you have a winning drug; Describe how PK/PD in patients give you better information for POC decision making.

Pharmaceutical / Regulatory agency perspectives

Modelling group, Academic group

CRO (preparing sophisticated analysis)

09-19 Safety Issues in First-in-Human Studies

The session deals with overcoming or assessing safety concerns and issues early in development and exploring some of the safety issues that you can address in early clinical trials with a focus on biomarker. It showcases where preclinical and clinical meet demonstrating a variety of organ markers. Examples of different organ systems impacted or assessed may include use of markers in renal toxicity, testicular safety or cardiac assessments as examples.

Identify methods that are useful to address early safety issues in FIH studies which is originally seen in toxicity data.

Clinical site Pharmaceutical representative

CRO

10: VALUE AND ACCESS




Keywords

10-01 Breaking Down the Wall for FDA to Leverage Real World Data

This forum will explore the use of real world evidence/big data in drug and device development, regulatory decision making under PDUFA VI and MDUFA IV, and transactional data sources.

Discuss FDA's plans for implementing the RWE provisions once PDUFA VI begins in October 2017; Describe potential methodologies for gathering and analyzing RWE; Explain perspectives from those who have conducted RWE/pragmatic clinical trial pilots or demonstrations; Discuss prospects for use of RWE to help inform efficacy not only in the postmarket space but also premarket for original NDAs.

FDA (CDER and CDRH) representative(s), preference to someone from device side re: guidance on RWE.

Industry representative-pharma

Industry perspective-device

Real world data


10-02 Breaking Down the Wall for Global Regulators to Leverage Real World Data

This forum will present global perspectives on use of real world evidence for drug and device regulatory decision making including postmarket surveillance, fast track or acceleration programs, conditional approval programs.

Describe a global view of access and use of data.

Regulatory agency representative (US)

Regulatory agency representatives (non-US)

industry perspective (drug-primary and device-secondary)

Real world data

10-03 The Increasing Role for Big Data for Late Phase Drug and Postapproval Purposes

This session will clarify the growth of big data assets both in the private and public sector, the tools that can be used to evaluate drug real world safety and effectiveness, and the increasingly favorable view of big data by regulators as a more comprehensive and less expensive approach to postapproval monitoring for safety and effectiveness.

Explain use of data postapproval. FDA or policy representative (Sentinel), representing view of FDA Commissioner and others on use of RWEALSORepresentative from organization such as PCORnet on what has and is being done

Industry examples of developing big data capabilities to prepare for these changes in regulatory view point

Payer perspective on the role of big data in considering reimbursement

Big Data

10-04 The Intersection of FDA Labeling and Value-Based Conversations with Payers

This session will review the drivers for value-based contracting including risk taking by providers and payers, and the barriers. These barriers include FDA labeling decisions, communication of unapproved uses, Medicaid best price and anti-kickback concerns.

Describe the regulatory and legal considerations surrounding value-based contracting conversations with payers.

Payer representative involved in outcomes based contracts

Industry representative with experience in outcomes based contracts

Regulatory perspectives: (1) OIG representative or legal to discuss regulatory changes or modifications needed to allow these programs to go forward; (2) FDA representative to discuss how label could be modified to mitigate using broader outcomes based metrics in contracting.

Labeling

10-05 Drug Mortgages: Benefit-Risk and Pricing Convergence

In the US and EU, today's innovations and health advances are changing cost parameters. Conversations about paying higher costs now for a cure versus paying lower costs over years for treatment are no longer isolated to the patient and health care provider. Can easing human suffering bear the price, and how is the payer environment changing?

Discuss whether or not human suffering can bear the price; Describe how the payer environment is changing.

Patient advocate representative

Payer representativeALSOIndustry representative for a high cost therapy that cures (e.g. Bluebird bio)

Health Economist working on the mechanics of how it might work

Pricing

10-06 Outcomes Standardization: An Imperative to Value Assessments

Data from both premarket and postmarketing clinical trials and formal registries of medical products can be used to assess safety and effectiveness across the medical product life cycle. Sources outside of clinical trials, such as EHRs, can provide evidence of continued effectiveness when used in larger populations in the postmarketing setting. However, the lack of consistent outcome assessments is a potential barrier to evaluating safety and efficacy across the continuum of the product life cycle.

Discuss the gaps and challenges in the current environment resulting from a lack of outcomes standardization.

Regulatory perspectives: (1) AHRQ/NLM representative regarding Outcomes Measures Framework efforts(2) CDC representative

FDA representative on standardization efforts for FDA clinical endpoints and how pre and post-approval might harmonize

HIT industry representative on what it would take to standardize EMR systems technically and politically

Outcomes

10-07 Comparative Efficacy Trials: The Role of FDA and Other Regulators

In this session, we will think through the designs and endpoints for comparative efficacy and safety. This work is typically performed by NIH and PCORI in the US but has been proposed to CDER and CBER.

Review key areas for assessment for clinical trials; Describe what roles can and should the FDA and other regulators play.

FDA representative Regulator view point from EU or Canada

Single payer representative

Endpoints


10-08 Mind the Efficacy-Effectiveness Gap: Will the New European Regulations on Clinical Trials Revolutionize Health Technology Assessment

The new European regulations introduce the lower-intervention clinical trial, with streamlined safety monitoring requirements versus conventional clinical trials. This study design can contribute to real-world evidence development and has the potential to close the efficacy-effectiveness gap that undermines HTA and clinical decision making.

Discuss if the new regulations drive sufficient research to impact HTA in Europe.

EMA representative to discuss the new regulations

HTA representative to discuss impact of these new regulations on HTA in Europe

Industry representative to discuss impact on relative to the prior regulations and the potential utility of streamlining with HTA needs.

HTA, efficacy

10-09 Real World Data to Real World Evidence for Assessing Efficacy and Effectiveness

In the US and EU, there are opportunities and challenges for new medicines development, regulatory review, and health technology. The efficacy and effectiveness gap is challenging the value in decision making and the generalizability of findings from the traditional gold standard randomized controlled trials (RCT) to use of medicines outside of the studied population. Many decisions that need to be made by HTA agencies must rely on the RCT-generated evidence in their analysis and deliberations with respect to the expected effectiveness of new medicines.

Evaluate whether RWD can enable decision making by complementing evidence from RCTs using data from other sources, including observational studies, EHRs, claims databases, or other nonstandard information sources.

Industry representative HTA perspective (i.e., NICE representative)

Regulatory:(1) FDA perspective(2) EMA perspective

Real world data

10-10 Drug Pricing: Access Versus Innovation

This panel will review the balance between expediting generics to market for pricing balance based on competition and the need for the innovation of new drugs (e.g., EpiPen pricing and competitors).

Recognize the need for continued innovation with the need to expedite approvals for generics and biosimilars.

Industry representatives:(1) Biosimilars(2) Patented drugs

Policy think tank view on the problem of innovation versus competition, OR non pharma view; other industries have faced similar pricing v. innovation, & pharma has looked to other industries on how to do better.

Payer representative on the selection of generics and biosimilars versus brand drugs and how to maintain innovation

Drug pricing

10-11 Payer Evaluation in Today's Value Framework: Impact on Patient Care

Some of the value frameworks include ASCO's oncology value framework, ICER, NCCN Evidence Blocks, ACC/AHA evidence+value guidelines, Premera Blue Cross value-based formulary, and more. What are the implications of the proliferation of value-based frameworks? How can we guide better usage? Do they work as intended? What are the risks of value frameworks to patients, providers, and society?

Describe how payers evaluate the data/results from various value frameworks and how this is impacting patient care and medical management.

Representative(s) from value framework:(1) ISPOR(2) Organization such as Greenpark or ICER

Industry representative Payer representative Value Framework, Payer

Documents

Page 1 DIA 2017 PROPOSED SESSIONS · Page 1 DIA 2017 PROPOSED SESSIONS Note: Part 1 is a list of the titles of the proposed sessions within each primary interest area, and Part 2