Value of Adaptive Trials and Surrogate Endpoints for Clinical Decision-Making in Rare Cancers.

ABSTRACT: Despite high-level endorsement, the number of adaptive Phase II/III trials in rare cancers needs to be improved, with better understanding of their value for clinical decisions in daily practice. This paper describes approaches to trial design in rare cancers, which has been supplemented by a search of ClinicalTrials.gov for adaptive trial designs in rare cancer. In addition, an online survey of 3,200 oncologists was conducted. Practicing physicians were questioned on the importance of different evidence levels, types of adaptive trial design, and categories of surrogate endpoints for clinical decision making. The results of the online survey revealed that evidence from Phase II/III trials with an adaptive design and relatively small sample size was considered high value in rare cancer by

SUBMITTER: Krendyukov A 

PROVIDER: S-EPMC7982798 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature