Fragility of randomized trials supporting cancer drug approvals stratified by approval pathway and review designations.

ABSTRACT:

Background

It has been suggested that the results from fragile trials are less likely to translate into benefit in routine clinical practice.

Methods

We searched the Food and Drug Administration (FDA) archives to identify drug approvals for solid organ malignancies between 2010 and 2019. We calculated the Fragility Index (FI) supporting each approval, using methods to account for time-to-event. We compared FI and trial and approval characteristics using Mann-Whitney U and Kruskal-Wallis test. Using logistic regression, we examined study characteristics associated with withdrawal of consent or lost to follow-up (WCLFU) exceeding the calculated FI.

Results

The median FI among 125 included studies was 23 (range 1-322). The FI was ≤10 in 35 studies (28%), 11-20 in 21 (17%

SUBMITTER: Wilson BE 

PROVIDER: S-EPMC8366090 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature