The drug development process under an Adaptive Pathway (AP) approach may entail a stepped development of evidence substantiating the value of a new treatment. Incremental development of value substantiation implies accumulating evidence over time, while requiring critical value assessment, pricing and reimbursement decisions at milestones.
Therefore, decision-making needs to account for a higher level of uncertainty, especially at the initial milestones. Uncertainty may result from more limited data regarding efficacy, effectiveness and safety, as well as HRQoL and health economic values.
The degree and nature of uncertainty have to be addressed for decisions on pricing and reimbursement strategies for manufacturers in an AP framework. How have HTA valued early data in pricing and reimbursement in the past and what are the perspectives?
To assess what the critical elements to successful health technology assessments (HTAs) for products in an AP framework, Evidera developed a number of case studies of products undergoing conditional market authorization in Europe and accelerated approval in the U.S. and reviewed the HTA strategies as observable based on publicly available documents in selected EU markets. It is critical to carry learnings forward from past assessments using early data and elucidate the individual market perceptions towards adaptive pathways – example Germany.
Who Should Attend
Senior members of the clinical development, health economics and outcomes research, global market access, and regulatory teams and anyone who is involved in the decision about early market access, development of HTA submissions and global market access.
Key Learning Objectives
- Understand some of the challenges arising from HTA submissions based on early data and how this may affect adaptive pathway for emerging products, in terms of evidence generation
- Understand some of the practices followed to date by manufacturers of products with conditional market authorization (CMA) or accelerated approval
- Provide a perspective from the German market
- Understand the elements that proved successful in the process and that may be critical for future successful HTA in these countries for products with early access
Susanne Michel, MD, European Practice Leader, Payer Strategy; Yvonne Beatrice Boehler, MD, MBA, Prof. TH Koln University; Agnes Benedict, MA, MSc, Sr. Research Scientist, M&S and Exec. Dir. Center of Excellence – HE; Noemi Muszbek, MA, MSc, Sr. Research Scientist, M&S