FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis.

ABSTRACT:

Objective

To analyze the US Food and Drug Administration (FDA) approval, trials, unmet needs, benefit, and pricing of ultra-rare (<6600 affected US citizens), rare (6600-200 000 citizens), and common (>200 000 citizens) orphan cancer drug indications and non-orphan cancer drug indications.

Design

Cross sectional analysis.

Setting

Data from Drugs@FDA, FDA labels, Global Burden of Disease study, and Medicare and Medicaid.

Population

170 FDA approved drugs across 455 cancer indications between 2000 and 2022.

Main outcome measures

Comparison of non-orphan and ultra-rare, rare, and common orphan indications regarding regulatory approval, trials, epidemiology, and price. Hazard ratios for overall survival and progression-free survival were meta-analyzed.

Re

SUBMITTER: Michaeli T 

PROVIDER: S-EPMC10167557 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature