PURPOSE:

The workshop will highlight practical issues around the use of surrogate endpoints in HTA decision-making, with particular focus on data limitations at regulatory and HTA stage of drug development. It aims to discuss with the audience emerging statistical methodologies for efficient utilisation of diverse evidence in this context.

DESCRIPTION:

Surrogate endpoints play an increasingly vital role in evaluation of new health technologies in accelerated regulatory approvals and HTA recommendations. Appropriately validated surrogate endpoints, as good predictors of clinical benefit, should be utilized to reduce decision uncertainty. However, traditional approaches to surrogate endpoint evaluation, using meta-analysis of data from RCTs, become challenging; particularly in areas of precision medicine and rare diseases where such data are increasingly limited. HTA organizations, including NICE and EUnetHTA, advise against generalization of surrogate relationships to other treatments or populations. In this workshop, we will explore emerging Bayesian meta-analytic models for efficient use of diverse sources of data, including from different treatment classes, indications and study designs, when evaluating surrogate relationships whilst avoiding their direct generalization.

Dr Bujkiewicz will introduce the session (6 minutes). Dr Papanikos will discuss hierarchical models for information-sharing across treatment classes with a case study in advanced colorectal cancer (12 minutes). Dr Singh will illustrate flexible methods for sharing information on surrogate relationships across multiple indications in a case study of bevacizumab (12 minutes). Dr Nikolaidis will discuss adaptive information-sharing methods for efficient utilisation of real-world evidence with a case study in acute myeloid leukaemia (12 minutes). Dr Bujkiewicz will moderate multi-stakeholder discussion on expected opportunities and challenges of introduced methodologies, with interactive polling questions and open discussion (18 minutes).

The workshop is relevant to analysts, methodologists and decision-makers, from all public and private sectors. The multi-stakeholder panelists are experienced methodologists and include authors of technical methods guidance for HTA submissions.