A Framework for the Use and Likelihood of Regulatory Acceptance of Single-Arm Trials.

ABSTRACT:

Background

Single-arm clinical trials (SAT) are common in drug and biologic submissions for rare or life-threatening conditions, especially when no therapeutic options exist. External control arms (ECAs) improve interpretation of SATs but pose methodological and regulatory challenges.

Objective

Through narrative reviews and expert input, we developed a framework for considerations that might influence regulatory use and likelihood of regulatory acceptance of an SAT, identifying non-oncology first indication approvals as an area of interest. We systematically analyzed FDA and EMA approvals using SATs as pivotal evidence. The framework guided outcome abstraction on regulatory responses.

Methods

We examined all non-oncology FDA and EMA drug and biologic approvals for fir

SUBMITTER: Subramaniam D 

PROVIDER: S-EPMC11530569 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature