Drug lag and associated factors of orphan drugs approved by the U.S. in China.

ABSTRACT:

Background

A pronounced disparity exists in the accessibility of orphan drugs between China and developed countries, such as the United States. Identifying and analyzing the critical determinants that contribute to this gap is essential for enhancing the availability of orphan drugs and promoting equitable access for patients.

Methods

We included all new orphan drugs approved by the US Food and Drug Administration (FDA) between 2013 and 2023 and collected their approval information in China and the United States. Major factors of interest included accelerated review pathway, locations where pivotal clinical trials were conducted, therapeutic category, and other factors affecting the delay in drug launch. They were analyzed using multinomial logistic regression, analysis of c

SUBMITTER: Mo Q 

PROVIDER: S-EPMC12425770 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature