Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU.

ABSTRACT:

Objective

To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).

Design and setting

Identification of all cancer drugs (initial or supplementary indication) with orphan status approved by the FDA between 2008-2017 based on publicly accessible reports. The European public assessment reports (EPAR) was searched to determine whether these FDA-approved drugs were also approved by the EMA.

Main outcome measures

Extraction of active ingredient, trade name, approval date and approved indication from two FDA data sources (Orphan Drug Product Designation Database, Drugs@FDA) and comparison with the same data from EPAR.

Results

The FDA approved 135 cancer dr

SUBMITTER: Vokinger KN 

PROVIDER: S-EPMC6797305 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature