Health Economic Evaluations Alongside Adaptive Platform Trials: A Systematic Review

Adaptive platform trials (APTs) present an unprecedented opportunity to enhance clinical trial efficiency and accelerate decision making. Limited evidence exists on how health economic evaluation studies are planned or conducted alongside APTs for translation and resource allocation. This review aims to identify and synthesize published evidence on the planning and conduct of economic evaluations (EEs) alongside the flexible APT design.

Eligible trials were identified from electronic bibliographic databases, clinical trial registries, and other sources - MEDLINE, Embase, Cochrane Trials, EconLit, CINAHL, Web of Science, Scopus, ClinicalTrials.gov, ANZCTR, the World Health Organization's ICTRP, reviews, and trial alliances up to November 2024. APTs with EE analysis planned or conducted, and accessible core protocols, were included. Information was extracted from protocols and health economic analysis plans. EE methods were narratively synthesized, with strengths and limitations appraised.

Seventeen APTs mentioned plans to embed EEs, among which 15 (14%; n = 104) had EE results published or core protocols accessible. The EEs were primarily designed as secondary objectives or outcomes of APTs, with methods centrally planned for individual domains within the platform. Efficient use of long-term data collection methods was observed. None planned to conduct EE during interim analysis. Value of information analysis was conducted in one and planned within another APT to inform value of future research.

Almost 1 in 5 APTs embedded EE plans. APTs utilize health economics methods to achieve outcomes beyond cost-effectiveness results, such as research resource prioritization and data collection. Platform-level, centrally planned EE methods were observed, indicating a focus on methodological efficiency.