Health agencies recommend best practices for using surrogate endpoints
A consortium of health technology assessment (HTA) agencies has released a white paper that aims to help pharmaceutical manufacturers provide better data on using surrogate endpoints to demonstrate drug effectiveness and to help HTAs make better decisions on long-term efficacy outcomes.The white paper was produced by the UK National Institute for Health and Care Excellence (NICE), and other members of the working group, including Canada’s Drug Agency (CDA-AMC), the Institute for Clinical and Economic Review (ICER) in the US, the Australian Department of Health and Aged Care, the National Health Care Institute (ZIN) in the Netherlands, the Institute for Technology Assessment in Health (IETS) in Colombia, and Rubix Health in the US.
“There is some published methodological guidance on using surrogate endpoints to inform decision-making,” the paper said. “But the level of detail varies, and there is limited guidance on using surrogate endpoints for economic modeling, and therefore, this paper aims to address some of these gaps.
A surrogate endpoint is a biomarker that can substitute for a clinical endpoint and reasonably predict a drug’s benefits in situations when measuring the clinical outcome is difficult. For example, lowered blood pressure could be a surrogate endpoint for stroke reduction or prevention from an antihypertensive medication.
Canada’s Drug Agency stated that when surrogate endpoints are used in drug submissions for reimbursement review, its committees may lack sufficient evidence to approve reimbursement.
The committees “need to understand the relationship between short-term effects and long-term outcomes in order to make accurate predictions about the long-term health effects of new drugs.”
The white paper assessed four key areas: the regulatory standards for validating surrogate endpoints, the published statistical methods used for this validation, the existing criteria set by health technology assessment (HTA) bodies for validating surrogate endpoints, and how these surrogate endpoints have been addressed in previous HTA evaluations.
After addressing these questions, the authors listed recommendations for considering surrogate endpoints. These recommendations aim to enhance current best practices for model conceptualization:
- Efforts should be made to align the HTA definitions of surrogate endpoints with widely accepted evidence-based medicines applications like those proposed by the Grading of Reoemdains, Assessments, Development, and Evaluation (GRADE) Working Group.
- Technology developers should engage with regulators and HTA bodies at an early stage when developing evidence-generation plans to ensure that the selected outcomes align with the expectations of these bodies.
- Pharmaceutical manufacturers should use clear, non-technical language in their submissions when reporting the evidence of a surrogate relationship and its use in an economic model.
- The model should also be designed to incorporate new information on the surrogacy relationship as it becomes available.
NICE report, Announcement
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