Use of alternative and confirmatory data in support of rare disease drug development.

Abstract

Recently, the use of alternative and confirmatory data in support of rare disease drug development has received much attention (NASEM 2024). This article attempts to provide an overview regarding the limitations and major challenges of the use of ACD that are commonly encountered in rare disease drug (including biologics) product development. In addition, some innovative approaches using ACD under a novel two-stage hybrid adaptive trial design are proposed to assist the sponsors in rare disease drug development are proposed. Under the proposed hybrid adaptive trial design, statistical considerations regarding the implementation of ACD in support of the demonstration of the safety and efficacy in rare disease drug development are discussed.