PURPOSE: To introduce the concept of surrogacy and outline the levels of evidence and analytical techniques used to evaluate the surrogate relationship. To describe the current status of using surrogate endpoints in regulatory and health technology assessment (HTA) submissions and outline the international HTA agencies’ methodological guidance this area, providing an update on the changes currently being made to NICE methods guide relating to their use in submissions.

DESCRIPTION: Using surrogate endpoints has become common in trials; especially those informing regulatory decisions. This means that many drugs, particularly those for cancer and rare diseases, are increasingly being approved based on an evidence-base that lacks direct evidence on their actual impact on final outcomes that are important to patients, clinicians and HTA agencies – including overall survival and quality of life.

In this workshop, Sylwia Bujkiewicz will present the methodologist perspective, introducing the concept of surrogacy, outlining the levels of evidence used to validate the surrogate endpoints and the latest analytic techniques proposed by NICE Decision Support Unit (DSU) for their evaluation (12 minutes). Huseyin Naci will outline the regulatory context, describing the rationale for and extent of using surrogate endpoints in regulatory approvals, the limitations of the evidence used to support the surrogacy relationship in recent approvals and the societal impacts of the current regulatory context (12 minutes). Oriana Ciani will describe the HTA context, providing an overview of the use of surrogate endpoints in HTA submissions, and how the HTA context differs from the regulatory one, discussing the impact of their use on decision-making (12 minutes). Dalia Dawoud will present the proposed update of NICE methods in relation to the use of surrogate endpoints, comparing it with other international HTA agencies’ requirements (12 minutes). The workshop is relevant to attendees from various backgrounds including academics, regulators, HTA agencies and pharmaceutical companies.