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THE POWER OF REAL-WORLD INFORMATION
TCD OUTCOMES RESEARCH © 2016 | 1
THE POWER OF REAL-WORLD INFORMATION
TCD OUTCOMES RESEARCH © 2016 | 2
OBJECTIVES
It is NOT to create OR specialists ….
Use a product/industry life-cycle perspective ….
Using OR smartly ....
THE POWER OF REAL-WORLD INFORMATION
TCD OUTCOMES RESEARCH © 2016 | 3
• Framing Outcomes Research (OR)
• Techniques
• Case studies
• Application
CONTENTS:
THE POWER OF REAL-WORLD INFORMATION
TCD OUTCOMES RESEARCH © 2016 | 4
THE POWER OF REAL-WORLD INFORMATION
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1. FRAMING OUTCOMES RESEARCH
In your view:
• Why are you here today?
• What is OR?
• Where, if at all, should OR reside in
your organization?
FRAMING OR
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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• New questions from regulators, geographies, insurers, payers, patients, providers
• Disruptive new technologies: New pressures on price (NDOH Pricing Committee)
• New pressures on illustrating value
• Internal and external validity
MARKET ACCESS ANDREIMBURSEMENT CHALLENGES
FRAMING OR
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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EVOLUTION OF EVIDENCE
Dynamic Solutions to Dynamic QuestionsTM
Source: http://www.quintiles.com/~/media/library/presentations/the-emerging-role-of-real-world-studies-and-registries.pdf
FRAMING OR
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 8TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
DRUG DEVELOPMENT CYCLE
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 9TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
COMMERCIALIZATION CYCLE
High-value vs. non-high-value
assets
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 10TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS™
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 11TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
THEREFORE …
• What is OR? • What makes it different?
• How can OR contribute towards policy?
?
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 12TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
• Outcomes Research “seeks to understand the end results of particular health care practices and interventions” on individual patients and populations
• Often includes:
» Clinical impact
» Economic impact
» Financial impact
» Funding impact
» Market impact
» Public health impact
Clinical impact
Economic impact
Financial impact
Funding impact
Market impact
Public health impact
WHAT IS OR??
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 13TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
1. The Question It AsksA hallmark of outcomes research is the breadth of issues it addresses. Outcomes research touches all aspects
of health care delivery, from the clinical encounter itself to questions of the organization, financing and
regulation of the health care system. Each of these factors plays a role in the outcome of care, or the ultimate
health status of the patient. Understanding how they interact requires collaboration among a broad range
of health services researchers, such as physicians and nurses, economists, sociologists, political scientists,
operations researchers, biostatisticians and epidemiologists.
2. The Setting It StudiesOutcomes research evaluates the results of the health care process in the real-life world of the doctor’s office,
hospital, health clinic and even the home. This contrasts with traditional randomized controlled studies,
funded mainly through the National Institutes of Health, which test the success of treatments in controlled
environments. These are called efficacy studies. Research in real-life settings is called effectiveness research.
WHAT MAKES IT DIFFERENT?
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 14TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
3. The Health Status Measures It UsesTraditionally, studies have measured health status, or health outcomes, in terms of physiological measurements
– through laboratory test results, complication rates (e.g. infections) or death. These measures alone do not
adequately capture health status. A patient’s functional status, well-being, and satisfaction with care must
compliment the traditional measures.
4. The Methods It UsesConstraints of money and the time it takes to conduct large, long term clinical trials is a major challenge of
outcomes research. One strategy of overcoming this barrier is to use existing computerized databases, such as
those available from Medicare, Medicaid, private insurance companies, and major employers. Another strategy
is to use simple questionnaires that patients can use to evaluate their own health and health care. Outcomes
research also uses meta-analysis, a technique to summarize comparable findings from multiple studies.
These approaches complement the more standard research strategies involving the collection and analysis of
information from medical records and other clinical and management data.
WHAT MAKES IT DIFFERENT?
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 15TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM
FRAMING OR
CONTRIBUTION TO POLICY
• Global policy trends …
• Country policy
• Funder policy
• Clinical policy
Bridge the gap between clinical and commercial functions, throughout the product life-cycle. By infusing insights from the marketplace in the form of real-world data into the development process, companies can better align their development efforts with unmet patient needs and accelerate time to market with the right evidence package. In the post-marketing realm, collecting more data on real patients in a real-world setting is becoming the new mandate.
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES
2. OUTCOMES RESEARCH TECHNIQUES
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FRAMING OR TECHNIQUES
TECHNIQUESOur armamentarium:
• Systematic review and Meta-analysis
• Health economics
• RWD analysis
• Observational studies
• Comparative effectiveness trials
• Patient reported outcomes
• Phase IIIB and IV studies
THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES
TECHNIQUES: SCIENTIFIC METHODOLOGY
Just because it is OR, does not mean we do not need scientific rigor …
The research question: Why are red cherries black?
Hypothesis: Because they’re green.
• Protocol development, PI and GCP
• Regulatory
• Ethics
• Project management
• Site management, CRF, data collection
• Data management
• Biostatistics
• Medical writing: Clinical study Report
• Value messages related to different stakeholders
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FRAMING OR TECHNIQUES
TECHNIQUES: RWD ANALYSISExamples:
• Retrospective data review
» Medical schemes claims data
» Expanded access programs
• Retrospective chart review
Types of questions answered:• Total cost of illness
• Patient flow modeling
• Adherence
• Data inputs for PE modeling
• Epidemiology
• Medicine initiation and switching rates
• Competitor analysis
• Local patient outcomes
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FRAMING OR TECHNIQUES
TECHNIQUES: RWD ANALYSIS
HOW?:• Research question
• Primary and secondary objectives
• Data specification
• SAP
• Data collection and analysis
• Results
• Impact and value messages
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FRAMING OR TECHNIQUES
TECHNIQUES: OBSERVATIONAL STUDIES
Involves the direct observation of phenomena in their natural setting
Examples:
• Registries
• Epidemiological studies
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FRAMING OR TECHNIQUES
TECHNIQUES: OBSERVATIONAL STUDIES
Registries:
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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FRAMING OR
A PATIENT REGISTRY is an organized system that uses observational study methods to collect uniform
data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease,
condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy Purposes.
A REGISTRY DATABASE is a file (or files) derived from the registry.
FOCUSED on the outcomes, effectiveness, comparative clinical effectiveness, and appropriateness of
pharmaceuticals, devices, and health care services.
DEFINITIONS
TECHNIQUES
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 25TCD OUTCOMES RESEARCH © 2016
FRAMING OR
PROCESS
Creating registries:
• Planning
• Design
• Data elements
• Use
• Data sources
Source:
https://effectivehealthcare.ahrq.gov/ehc/products/420/1897/
registries-guide-3rd-edition-vol-1-140430.pdf
TECHNIQUES
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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FRAMING OR
PROCESS
The key points to consider in designing a registry include:
• formulating a research question;
• choosing a study design;
• translating questions of clinical interest into measurable exposures and outcomes;
• choosing patients for study, including deciding whether a comparison group is needed;
• determining where data can be found; and
• deciding how many patients need to be studied and for how long.
TECHNIQUES
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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FRAMING OR
PROCESS
TECHNIQUES
Ethical considerations:
• Ethics, data ownership, privacy
• Patient informed consent
• Data protections (FDA CRF Part 11 compliance)
http://www.fda.gov/RegulatoryInformation/Guidances/ucm125067.htm
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 28TCD OUTCOMES RESEARCH © 2016
FRAMING OR
PROCESS
TECHNIQUES
Operating registries:
• Recruiting and retaining patients
• Data collection and QA
• AE’s recording and reporting
• Data analysis
• Medical writing
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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FRAMING OR TECHNIQUES
SCIENTIFIC RIGOR
https://www.graceprinciples.org/doc/GRACE-Checklist-031114-v5.pdf
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FRAMING OR TECHNIQUES
Static Registries to Dynamic eRegistriesTM
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FRAMING OR TECHNIQUES
Static vs. Dynamic Registries
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FRAMING OR TECHNIQUES
• Paper based CRF
• Transcription into e-CRF
• Significant monitoring
• Data management cumbersome
• Takes time: high touch, low tech
• Reporting is laborious
Static Registries
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FRAMING OR TECHNIQUES
E-REGISTRY:
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FRAMING OR TECHNIQUES
Computational Model + E-Registry = Dynamic e-Registry
DYNAMIC e-REGISTRIES
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FRAMING OR TECHNIQUES
DYNAMIC e-REGISTRIES
• Automated descriptive analyses: Simple descriptive statistics
• Automated predictive analyses: Predict heterogeneity of treatment effect; predict primary/secondary outcomes
• Automated prescriptive analyses: Machine learning - Prescribe what patient characteristics will provide best outcomes
• Dashboards: Create bespoke dashboards as required by stakeholders
• Automated exposure to stakeholders: All technology platforms
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FRAMING OR TECHNIQUES
TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)
“While the implementation of CER has
implications across global healthcare
systems, the US focus on CER points to the
growing influence of payers and their desire
to control spending.”1
1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0
THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES
TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)
“In an era marked by increasing prominence
of CER evidence, successful pharmaceutical,
biotechnology and medical device manufacturers
will need to develop products with unambiguous
evidence of economic as well as clinical value
relative to alternatives.”1
1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0
DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
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FRAMING OR
“CER is defined as research comparing interventions in the real world settings”1
1. http://www.med.upenn.edu/sleepctr/documents/FederalCoordinatingCoucilforCER_2009.pdf
CER DEFINITION
TECHNIQUES
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FRAMING OR TECHNIQUES
• Non-inferiority trials • Superiority trials • Observational studies
CER EXAMPLES
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FRAMING OR TECHNIQUES
TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)
“PRO is a health outcome directly reported by the patient
who experienced it.”
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FRAMING OR TECHNIQUES
TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)
Examples:
• Symptoms (impairment)
• Functioning (disability)
• General health perceptions
• Quality of life
• Rating health care
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THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES CASE STUDIES
3. CASE STUDIES
Question:
• Can you think of a specific clinical/competitive issue that might be addressed by using OR techniques?
• Would you like to use your specific problem as a case study for this session?
THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES CASE STUDIES
SETTING THE CASE STUDY
CONTEXT:
• As product manager, you are informed that, in 2018, casestudymab, your company’s MAB in the treatment of prostate cancer, will be launched in SA
• You are told it will cost approximately US$40,000 per treatment
• There are at least 3 competitors in this setting in SA
• Their comparative cost is about US$25,000 per treatment
Using the knowledge gained in this session, how will you proceed?
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FRAMING OR TECHNIQUES CASE STUDIES
FRAMING THE RESPONSE
Activity Activity plan
Pre-launch activities
Launch activities
Post-registration activities
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THE POWER OF REAL-WORLD INFORMATION
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FRAMING OR TECHNIQUES CASE STUDIES APPLICATION
4. APPLICATION
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FRAMING OR TECHNIQUES CASE STUDIES APPLICATION
4. APPLICATION
CONTEXT:
• Clinical affairs
• Medical affairs
• Regulatory affairs
• Market access
• Commercial
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FRAMING OR TECHNIQUES CASE STUDIES APPLICATION
4. APPLICATION
THE BIG OR OBJECTIVE:
• It is part of your competitive strategy …
• It is about building a comprehensive portfolio of real world evidence …
• That can be used by all departments …
• To reach commercial objectives of market access and reimbursement ...
• Using S.M.A.R.T. Goals.
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“In times of change the learners shall inherit the earth … while the learned will find
themselves perfectly equipped to manage a world that no longer exist” – Eric Hoffer
www.tcd-global.com
Dynamic Solutions to Dynamic QuestionsTM