Adaptive Pathways: Generating Evidence of Value.
Adaptive pathways promise a “triple win”.
They enable early access for patients to the most promising innovative therapies, address concerns over affordability, cost-effectiveness and outcome uncertainty for payers, whilst meeting companies’ need to make R&D sustainable.
As such they are a critical tool in the market access toolbox, but their opportunities and challenges are not well understood.
Uncertainty remains on how to realise all the potential benefits, when adaptive pathways are superior to traditional market entry routes, and how to overcome real-world evidence generation challenges.
This Masterclass addresses these strategic issues, illuminating when adaptive pathways are optimal for innovative therapies, as well as bringing together multiple perspectives on these iterative pathways.
Participants will benefit from engaging with OHE’s global thought leaders, and from industry insights on successful examples of adaptive pathways applications, which have aligned regulatory and reimbursement evidentiary requirements to enable timely access to breakthrough therapies.
The Masterclass speakers will also reflect on the practical opportunities and potential barriers to the acceptability of real-world evidence, and will present optimal evidence generation plans for different treatment types.
Welcome and Introduction
Insights from the Experience in Europe and the UK
• Accelerated Pathways (AP) introduced by the EMA and MHRA
• What APs mean to development and access to medicines, including impact of early scientific advice
• Difference between compassionate use and APs
• Examples of early access in Europe
Regulatory and HTA experience in the US
• US experience
• Key challenges and future prospects
Panel Discussion 1
Bridging the gap between regulatory and reimbursement
• Elements driving uncertainty at an early stage of a treatment introduction, including surrogate endpoints
• Examples on how different types of uncertainty can be addressed, including conditional reimbursement schemes
Are APs always the answer?
Maximise AP use
• Application of value of information to inform optimal use of risk sharing schemes
• When companies should actively seek an AP, including scientific advice.
Panel Discussion 2
Summary and Reflections