Stakeholder Engagement Strategies
Development of engagement approaches, multi-stakeholder dialogue. Seek dialogue with payers and regulators as early as possible in the development of new therapies in line with recent EMA guidance facilitating dialogue between sponsors and payers on investigational product information.
Topics of discussion should include:
- ways to include or create meaningful patient-centered outcomes or validated surrogate outcomes in developmental trials
- options for making the registration studies as robust as possible, especially if RCTs will not be performed
- options for partnership on post-approval studies to reduce residual uncertainty about the safety and effectiveness of new therapies (particularly important in the context of possible adaptive licensing)
- methods for determining patient eligibility that can be feasibly translated into coverage criteria
- criteria for designation of potential centers of excellence for the delivery of the therapy
- size of the potential patient population
- the role of a therapy envisioned within the care pathway
- the elements of value that patients, clinicians, manufacturers, and payers can agree to should be considered in assessing value and judging what price fairly reflects the added value of the therapy
