Uptake of orphan drugs in the WHO essential medicines lists.

ABSTRACT:

Objective

We evaluated the uptake of medicines licensed as orphan drugs by the United States Food and Drug Administration (FDA) or European Medicines Agency (EMA) into the WHO Model list of essential medicines and the WHO Model list of essential medicines for children from 1977 to 2021.

Methods

We collated and analysed data on drug characteristics, reasons for adding or rejecting medicines, and time between regulatory approval and inclusion in the lists. We compared trends in listing orphan drugs before and after revisions to the inclusion criteria of the essential medicines lists in 2001, as well as differences in trends for listing orphan and non-orphan drugs, respectively.

Findings

The proportion of orphan drugs in the essential medicines lists increa

SUBMITTER: Costa E 

PROVIDER: S-EPMC10753278 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature