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Surrogate Endpoints Used in NICE Technology Appraisals for Oncology and Non-Oncology Indications, 2022–23

Amy Heptinstall and Lizzie Adkins

Poster Session: 1. 13 November 2023. Poster Session Time: 10:30-13:30. Discussion Period: 12:30-13:30.

Abstract

OBJECTIVES: Surrogate endpoints may be used to predict patient-relevant clinical outcomes when it would be unethical or impractical to conduct a trial to assess the final patient-relevant outcome (e.g., requiring unattainably long follow-up, or when the clinical benefit of improving the surrogate endpoint is well established). The use of surrogate endpoints in health technology assessment (HTA) is increasing, particularly in oncology. While this can allow faster access to new treatments, it increases uncertainty for decision-makers and payers. Consequently, the National Institute for Health and Care Excellence (NICE) is working with international organisations to develop guidance on their use in cost-effectiveness analysis.

We aimed to understand the frequency and type of surrogate primary efficacy endpoints currently being used in NICE decision-making.

METHODS: HTAs published on the NICE website from 01.06.2022–31.05.2023 were reviewed. The use of surrogate endpoints as primary efficacy outcomes, therapeutic area, and recommendation decision were recorded.

RESULTS: Of the 76 HTAs reviewed, 68% included at least one surrogate primary efficacy endpoint. Six (8%) were not recommended by NICE, of which five used surrogates. The most common surrogates were progression-free survival (31%) and measures of response rate (31%). For HTAs in oncology indications, surrogates were used in 82% (32/39), mainly progression-free survival (50% [16/32]), response rate (28% [9/32]), and other survival outcomes such as disease- or event-free survival (25% [8/32]) (with two randomised controlled trials having dual-primary endpoints). For non-oncology indications, surrogates were used in 54% (20/37), mainly response rate (35% [7/20]).

CONCLUSIONS: Surrogate primary efficacy endpoints are frequently used and widely accepted by NICE when making a recommendation in both oncology and non-oncology indications. The introduction of clear guidance on their use in HTA will help to ensure a consistent approach, reduce uncertainty in decision-making, and allow fairer access to new treatments.

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