Enhancing Health Technology Assessment Methods: Navigating Surrogate Endpoints and Associated Statistical Standards

PURPOSE:

The workshop will introduce the rationale, objectives and preliminary recommendations from the ISPOR Statistical Evaluation of Surrogate Endpoints for HTA Decision Making Good Practices Task Force. The preliminary recommendations will be linked to the recent joint recommendations issued from an international HTA collaboration. The audience will gain a deeper understanding of statistical methods for clinical and economic value demonstration when surrogate endpoints are employed for licensing purposes.

DESCRIPTION:

Those conducting HTA around the globe often have to make access and reimbursement decisions based on surrogate endpoints that have been accepted by regulators. Despite many HTA agencies publishing guidelines on surrogate endpoints, few HTA submissions explicitly present evidence supporting the translation of the treatment effect on early/surrogate endpoints to the treatment effect on late/target outcomes for clinical value and economic value demonstration.

Dr. Ciani will introduce the session (6 min). Ms. Kusel will discuss how the taskforce recommendations link to the work published by a collaboration between UK’s NICE, US’s ICER, Canada’s CDA-AMC, the Netherland’s ZIN and Australia’s PBAC on science and methods development for surrogacy (10 minutes).

Dr. Bujkiewicz will discuss statistical approaches available for surrogate endpoint evaluation, including considerations for predictions, associated uncertainty and emerging novel tools to tackle less optimal data scenarios to support decision making (12 minutes). Using two existing technical appraisals, Dr. Ouwens will discuss how surrogacy analyses can be used for health economic modeling of relative treatment effects on target outcomes or to support implicit and explicit assumptions for modelled relationships (12 minutes). Dr. Ciani (20 minutes) will open the interactive polling and Q&A session on the task force’s recommendations.

This informative workshop is designed for researchers, payers, regulators, and technology assessors, as well as for those working in the pharmaceutical industry or those interested in HTA and market access of health technologies.