Adaptive Pathways for Transformative Medicinal Products. A New Paradigm with the Enhanced Application of Real-World Evidence? ISPOR 20 th Annual European Congress, Glasgow, Scotland Issue Panel 21, Wednesday, 8 th November 2017, 10:00-11:00 Biomedical Innovation Has Science Overtaken the System? Empowered patients Precision Medicine Need for Speed Adaptive Trials Adaptive Pathways (PRIME/CMA) Outcome and Value Uncertainties 1
The Panel Three Diverse Perspectives The Patients Voice Nicola Bedlington Secretary General, European Patients Forum and Co- Founder, The Patient Access Partnership PACT, Brussels, Belgium Health Technology Assessment Perspectives Ad Schuurman MA, Senior Medical Officer, EMA, London, UK, Head of the International Department, National Health Care Institute (ZIN), The Netherlands The Biopharma Viewpoint Rob Thwaites MA, MCom, Senior Director, Takeda, London, UK Nicola Bedlington The Patients Voice Adaptive Pathways (MAPPs) to foster access to beneficial treatment for the right patient groups at the earliest appropriate time in the product life span in a sustainable fashion (Adapt Smart) Opportunity to address bottlenecks evident in standard pathways 2
The Patients Voice Focus on : high unmet medical need Attention to : safety profile of products and patients trade-off preferences Attention to : capacity to collect of quality and useful data reducing uncertainties Key: Involvement all relevant stakeholders engaging early dialogue The Patients Voice Trade-off benefit/risk is key to patients Not static or linear. Expectations and perceptions of benefits and risks change over time (of illness) and according to age, role in society, culture. Analysis of benefits and risks may result in highly diverse perspectives 3
Priorities Communication/Information Education of patients enrolled in the process Key role of patient organisations as coaches and information providers Clear legal framework to address issues such as disinvestment Consideration of ethical aspects Early involvement of all stakeholders Ad Schuurman Payers Want Control on volume (indication, start-stop, dose) Control on data (real life, transparent) Agreement registry, definitions data, when assessed, consequences assessments, NL and EMA experience: dynamics 3-2-1 line therapy, near/off label, changing start/stop, doses, combination therapy, transaction costs Prevent decline in quality of evidence 4
Payers Want Control on costs (adaptive reimbursement, mutual acceptable prices) Initial prices, future price (per country?), how to be paid (confidential?) Restrict MAPPs to special cases Patients who cannot wait clinical development & benefit/risk evaluation: deteriorate irreversibly or die. Or urgent public health protection. Major improvement expected. Realistic exit strategy Agreement, patients/doctors aware Iterative development: Approval in stages Confirming benefit/risk balance of a product Gathering more evidence through time Early dialogue with multiple stakeholders The use of real world evidence to supplement clinical trial data Rob Thwaites Adaptive Pathways 5
GetReal and Evidence of Effectiveness Aim To develop a common understanding amongst health care decision makers and pharma R&D of the acceptability and usefulness of innovative development programmes which use RWE to estimate the effectiveness of new medicines HTAs Other stakeholders Regulators Alternative Evidence Development Pharma R&D Patients Clinicians GetReal: How can Pharma Take on Board? Enhance evidence plans and clinical development plans with learnings from GetReal Introduce a systematic approach for effectiveness planning Incorporate into project teams activities Support new ideas with internal and external expertise Engage earlier in scientific advice processes 6
Panel Debate The Role of the Patient Community Are patients willing to accept greater risks in return for early access to novel drugs? Which challenges to the equitable and timely access of transformative medicines remain across Europe? What is the patients role in HTA and P&R decisionmaking and how can this enhance timely access? Panel Debate Ensuring Value in Greater Uncertainty How do HTA bodies and payers deal with the challenges of fewer data & increased uncertainty? How can the stakeholders collaborate with HTA to achieve timely access to innovative treatments? What is the state of play for value-based MEAs in Europe and what are potential ways forward? 7
Panel Debate Advancing Evidence Generation with RWE RCTs and RWE polar opposites or complimentary? Can we accept some bias to get closer to the truth? Can regulatory and HTA requirements be aligned in early parallel EMA/HTA scientific consultations? Which areas of RWE require further development to enhance early access HTA/P&R decision-making? Questions & Answers Contributions from the Audience Please use the microphones State your name and affiliation Name the panellist(s) that you are addressing Thank you for your contributions to the debate 8
Thank You for Your Participation Slides and Debate Minutes will be available via ISPOR and LinkedIn Acknowledgements: Glasgow images by Shutterstock, icons by Freepic.com 9