Presentation (CTI)

OBJECTIVES: Health policymakers in many jurisdictions face a persistent challenge: deciding whether to adopt new medical treatments based on clinical trial evidence generated in other countries. This decision entails a trade-off between providing early access to potentially beneficial therapies and postponing adoption until locally relevant evidence becomes available. While deferring adoption is a common regulatory strategy, it may not always be optimal, as delays can result in significant welfare losses for patients. This study develops a theoretical model to evaluate the social welfare implications of alternative policy strategies for adopting treatments supported by external clinical evidence.
METHODS: Using a Bayesian value-of-information framework with a power prior, we develop a theoretical model to evaluate expected social welfare under four policy strategies: (1) delayed adoption pending a local confirmatory trial, (2) conditional adoption with a local trial requirement, (3) immediate adoption without a trial, and (4) immediate rejection. We apply the model to an illustrative case study of sintilimab, a PD-1 inhibitor developed and trialed exclusively in China, for potential use in treating non-small cell lung cancer in the United States.
RESULTS: The optimal policy varies based on uncertainty in transportability, trial duration, strategic delays in result disclosure, and drug pricing. When uncertainty is moderate and trial delays are manageable, conditional adoption with a local trial requirement yields the highest expected net benefits. High uncertainty favors delayed adoption, while pricing flexibility—such as lower launch prices—can shift the balance toward immediate adoption.
CONCLUSIONS: There is no one-size-fits-all approach to adopting treatments based on external evidence. Policymakers should tailor regulatory strategies to the specific evidentiary context, considering uncertainty, trial timelines, potential strategic delays, and pricing.