ISSUE:

A multistakeholder discussion of the new EUnetHTA 2022 requirements on endpoints used for relative effectiveness assessment (clinical, composite, and surrogate endpoints; safety, HRQoL and utility measures)

OVERVIEW:

Clinical and patient relevant outcomes assessments are crucial in the development of new products. Novel and surrogate endpoints are more frequently used, but there is a risk that they will be disregarded in HTA, which may hinder patient access. The EUnetHTA guidelines on endpoints will be open for stakeholder review in October 2022 (EUnetHTA endpoint guidance). We believe that this guideline will inform an important discussion for ISPOR members involved in evidence submission for HTA submissions across Europe.

Dr. Rudell will moderate the session and the audience questions following the presentations. M. Schlichting (Merck KGaA, Darmstadt, Germany) will provide an overview of clinical outcome assessment (COA), in the context of HTA assessments, eg, issues related to clinical relevance, surrogate endpoints, composite endpoints, and PRO endpoints (10 minutes). Dr. Wieseler (IQWIG) will summarize the outcomes relevant for HTA (10 min). Dr. Ryll (Melanoma Patient Network Europe) will discuss the patient/carer perspective on clinical and surrogate endpoints (10 minutes). We believe ISPOR members would like the opportunity to directly discuss this topic with experts as well as to review whether it would address the patient needs regarding access to medication.