PRE-CONFERENCE WORKSHOPS MONDAY 22ND OCTOBER 2018

09:00 Welcome Coffee and Registration

09:30 Navigating the Regulatory Landscape Across the EU & US In this bespoke and highly interactive workshop, you will engage with the workshop leader and hear case to case specific best practice examples for EMAPs launches across Europe and the US. You will have the opportunity to submit your specific challenges surrounding regulatory policy for your specific programme ahead of the session and will be encouraged to share your experience with your peers. Solving for regulatory compliance you will get the opportunity to take down the best working strategy to resolve any of your standing regulatory roadblocks. Attendees will further discuss with the workshop leader current global regulatory constraints and the need for harmonization in terminology in order to establish the unified global policy.

What you will gain from this workshop: Discuss unique cases that have navigated successfully through the robust regulatory landscape and establish best practice Receive tailored solutions for your own programme challenges from pre – launch country specific regulations to policy changes on

current running programmes Incorporate an effective compliance strategy for your specific needs

Workshop Leader:Allison Morgan, Managing Director, Metis ClinicalBIO: Allison Morgan has over 20 years of industry experience and has worked for multi-national pharmaceutical companies, small start-up biotech’s and contract research organizations, both within Europe and the US. Allison has previously held a variety of senior management positions, including Vice President of Clinical R&D, where she designed

successful strategies for accelerated programme launch, which involved negotiation with both EMA and FDA. Within this framework Allison has generated bespoke regulatory agency best practice guidelines, introduced new concepts and developed proof of concepts, which have withstood the rigours of due diligence and scientific challenge and contributed significantly to a successful IPO which raised $90 million.

12:00 Networking Lunch Break

14:30 Devising a Successful Company-Wide Roll Out Strategy for an Early Access Programme that Benefits All Stakeholders

The second workshop in our series will focus on formulating the best in-house strategy for full company programme roll-out. We will discuss the challenges of big and small pharmaceutical companies and learn how to devise internal procedures for operational excellence. Regardless of whether you are focusing on pre-launch or programme efficiency, you will learn the best practice approaches of end-to-end programme management. The workshop will look at practical approaches in integrating the patient needs payer capabilities and managing expectations. From launch to funding, clinician collaboration and registration the workshop will aim to give you the most efficient and successful programme starategy that fits with your company model and capabilities. You will be able to submit and will be encouraged to share your current challenges

What you will gain from this workshop: Take home unique programme models in order to apply the best practice into your company stategy Incorporate the patient payer and clinician perspectives. Learn how to approach and benefit all stakeholders of your programme Take the opportunity to interact with peers and solve the challenges around programme launch and operational excellence

Workshop Leader:Tom Watson, Executive Vice President, Early Access Programs, BionicalBIO: Tom is an experienced professional who has spent 15+ years within the pharmaceutical industry fulfilling a range of leadership roles including Director of Global Business Development and Head of Marketing for multinational pharma companies. His current focus is on developing a best strategy for running Early Access Programs as the Vice President of

Bionical. He further consults on a wide range of global programme models for the Early and Pre- Approval Access landscape finding bespoke solutions for the challenges of companies launching and working in this space.

17:00 End of Workshop and Networking

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CONFERENCE DAY ONETUESDAY 23RD OCTOBER 201808:30 Morning Registration and Coffee

09:00 PharmaIQ Welcome

09:05 Chairman Opening Remarks

09:10 Keynote Address: The Need for Collaboration and Harmonisation Across the Board Drive regulatory policy harmonisation globally by clearly defining programme terminology Discuss the best practice approach to engage and solicit input from patients and caregivers – a manufacturer’s challenge Discover the need for effective communication with the HCPs in order to establish best practice for data capture

David Farber, Partner - FDA and Life Sciences, King & Spalding

09:50 Creating a Strategy for a Successful Early and Managed Access Launch Define programme implementation criteria and drive engagement across different functions Discuss case studies of successful programme launch and understand difference in approaches Understand the key challenges in internal communication and best practice in company-wide roll-out

Samantha Nunes Radimerski, PhD, Medical Governance Lead, Managed Access Programs, Chief Medical Office, Novartis Pharma AG

10:30 Morning Coffee Break

11:00 Case Study: An Inside Look at Managing an Early Access Programme Define best approach for cross-functional collaboration pre-launch from a rare disease perspective Explore patient inclusion, HCPs engagement and managing expectations Examine criteria for a successful launch and followed programme operational management CROs perspective

Venkata Sravan Jaggumantri, Clinical Scientist, Rare Diseases & Scientific Affairs, PRA Health Sciences

11:40 Interactive Round Table DiscussionsAfter a morning of presentations and debates, use this opportunity to confer with your peers on the key strategies in launching and optimising your access programme. Choose one round table and after 30 minutes rotate round to another topic area of discussion.

12:40 Networking Lunch Break

12:50 myTomorrows Lunch and Learn – By Invitation Only

Enjoy an exclusive networking lunch hosted by ‘myTomorrows’ where they will be presenting case studies on successfully integrating Real-World Evidence into Early Access Programmes.

13:40 Examining EU Regulatory Fast Track Schemes Analyse the EU fast track schemes and their differences, with reference to prime; adaptive pathways and accelerated assessment Discuss the criteria for MAH under exceptional circumstances and conditional MAH Examine the challenges and benefits of trying to utilise a fast track scheme Pedro Franco, Director Europe, Global Regulatory & Scientific Policy (GRASP), Merck

KEYNOTE ADDRESS

CASE STUDY

CASE STUDY

Strategies for a successful

programme launchHartwig Gajek,

Managing Director, Hesyra-Medical

GmbH

Regulatory differences between

the US & EUAllison Morgan,

Managing Director, Metis Clinical

Approaches to reimbursement

Dimitri F. Dimitriou, Chief Executive Officer, ImmuPharma plc

“My knowledge has increased, which I can take back to my organisation”

Business Development, MultiPharma

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14:20 Interactive Session: Best Strategies for Funding Debate the challenges of small vs big pharmaceutical companies in developing a successful EMAPs strategy Define criteria and mitigate risks for small and specialty pharma manufacturers Discuss the possibility of non-competitive collaboration in best practice and terminology harmonisation

Dimitri F. Dimitriou, Chief Executive Officer, ImmuPharma plc

15:00 Afternoon Coffee Break

15:40 Early Regulatory Strategic Insights Create successful global regulatory strategies for early access programmes Analyse the differences in national requirements Discover how your peers are complying on a global scale

Hartwig Gajek, Managing Director, Hesyra-Medical GmbH

16:20 Panel Discussion: Regulatory Landscape and Government Understanding of Terminology within Programmes Define country understanding on terminology and influence on policy roll-out Discuss country intelligence outside of USA and EMEA Analyse strategies for policy harmonisation

Cristian Brocchieri, Medical Director, Alexion Pharma Pedro Franco, Director Europe, Global Regulatory & Scientific Policy (GRASP), Merck David Farber, Partner: FDA and Life Sciences, King and Spalding

17:00 Chairman’s Closing Summary of Day One

17:15 Networking Drinks Reception

CONFERENCE DAY ONE CONTINUEDTUESDAY 23RD OCTOBER 2018

INTERACTIVE SESSION

“Good event, good networking opportunities and relationship

building”

Regulatory Affairs Manager, Idis

“A valuable event providing lots of very useful information and

relevant contacts”

GMP/GDP Systems Manager, Intercept Pharmaceuticals

INTERACTIVE SESSION

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CONFERENCE DAY TWOWEDNESDAY 24TH OCTOBER 201808:30 Morning Registration and Coffee

09:00 Chairman’s Opening Remarks

09:10 International Keynote: The US Patient Perspective on Expanded Access Incorporate the patient advocacy perspective to develop a review process and formalised procedure on expanded access Recognise actionable steps to optimising patient access Discuss working with patient groups to help demystify pre-approval access and create community-specific resourcesAndra Stratton, President & Co-Founder, Professional Patient Advocate & Rare Leader, Lipodystrophy United

09:50 Stakeholder Relationship Management Discuss how to optimise relationships with caregivers, clinicians and healthcare providers Explore ways to work effectively with patient advocacy groups Get to grips with patient centricity and understand how it can benefit pharma manufacturersAnne Bruns, Rare Disease Advocate and Board Member, Utah Rare, Rare & Undiagnosed Network RUN

10:30 Morning Coffee Break

11:00 Local Perspectives: The EU Patient Perspective on Expanded Access Learn best practice on collaboration between manufacturer and patient from a EU perspective Optimise your working relationships with key European stakeholders such as policy makers, NGOs, and the media Understand the decision making process behind European policies Thomas Smith, Rare Disease Advocate, Parliamentarian and Committee Member, European Health Parliament

11:40 Panel Discussion: Effective Collaboration Between Manufacturers and Patient Advocates Soliciting input - the rare disease patient advocates perspective on the process of accessing treatment in EMAPs Understand the tools/recourses and practices to best promote awareness/education with patients Discover the key challenges faced by patients seeking early access and suggested ways for medicines

manufacturers to help overcoming theseAnne Bruns, Rare Disease Advocate and Board Member, Utah Rare, Rare & Undiagnosed Network RUN Andra Stratton, President & Co-Founder, Professional Patient Advocate & Rare Leader, Lipodystrophy United Shannon Burkoth, Rare Disease & Narcolepsy Advocate, Program Mentor, Rare Advocacy Movement (RAM)Simon Stones, Trustee and Patient Leader, Fibromyalgia Action UKNadia Bodkin, Rare Disease Patient Advocacy Professional, Senior Executive Vice President, Maxify Life

12:40 Networking Lunch Break

13:40 RWE Acquisition – Collaboration and Investment from Healthcare Providers Formulate standards for RWD collected on patient health status and/or the delivery of healthcare Develop the expertise needed to design and implement RWE studies Engage in pre-competitive collaborations to define best practicesRamona Reichenbach, Global Medical Governance Lead – Global Governance Office, Novartis Pharma AG

14:20 Adding Value by Utilising RWD from Managed Access Programmes in Conjunction with Clinical Trial Data Create greater precision and clarity as to the safety and efficacy profile of new products Improve the amount and quality of information available to patients and physicians, leading to more informed decision-making

about new products Demonstrate benefit: examination of real-world use of products may accelerate the rate at which products prove their value to

patients, providers and payers, supporting reimbursement with real-world evidence Tom Watson, Executive Vice President, Early Access Programs, Bionical

15:00 Afternoon Coffee Break

15:30 Ethical Crossroads: Expanded Access, Patient Advocacy and the #SaveJosh Social Media Campaign Perspectives and learning experiences from the CEO at the center of the 2014 social media campaign for pre-approval access to

Chimerix’s investigational antiviral drug Discussion of the ethical dilemmas surrounding expanded access Application of these lessons to future EAPs, and advice for companies looking to be well prepared Implications from US Right to Try LegislationKenneth Moch, President & CEO, Cognition Therapeutics CEO, Chimerix (2010-2014)

16:10 Panel Discussion: Future Steps and Calls to Action 2019 Discuss the requirements for policy harmonisation globally and terminology education across Define strategy for implementing data capture protocols and making use of RWD by also taking into account the Patient and Clinician input Discuss the most efficient path to manufacturer and patient collaborationRamona Reichenbach, Global Medical Governance Lead – Global Governance Office, Novartis Pharma AG

16:50 Chairman’s Closing Remarks

INTERNATIONAL KEYNOTE

PANEL DISCUSSION

PANEL DISCUSSION

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