Clinical Benefit and Regulatory Outcomes of Cancer Drugs Receiving Accelerated Approval.

ABSTRACT:

Importance

The US Food and Drug Administration's (FDA) accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered "reasonably likely" to predict clinical benefit. Postapproval clinical trials are then required to confirm whether these drugs offer clinical benefit.

Objective

To determine whether cancer drugs granted accelerated approval ultimately demonstrate clinical benefit and to evaluate the basis of conversion to regular approval.

Design, setting, and participants

In this cohort study, publicly available FDA data were used to identify cancer drugs granted accelerated approval from 2013 to 2023.

Main outcomes and measures

Demonstrated improvement in quality of life or o

SUBMITTER: Liu ITT 

PROVIDER: S-EPMC11000139 | biostudies-literature | 2024 May

REPOSITORIES: biostudies-literature