Cargando…

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

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 approve...

Descripción completa

Detalles Bibliográficos
Autores principales: Vokinger, Kerstin Noëlle, Kesselheim, Aaron S
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6797305/
https://www.ncbi.nlm.nih.gov/pubmed/31601584
http://dx.doi.org/10.1136/bmjopen-2018-028634
_version_ 1783459793772478464
author Vokinger, Kerstin Noëlle
Kesselheim, Aaron S
author_facet Vokinger, Kerstin Noëlle
Kesselheim, Aaron S
author_sort Vokinger, Kerstin Noëlle
collection PubMed
description 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 drugs with orphan indications that met our inclusion criteria, of which 101 (75%) were also approved by the EMA. 80/101 (79%) were first approved in the USA. Only 41/101 (41%) also received orphan designation by the EMA. 33/101 (33%) were approved for biomarker-based indications in the USA, however, only nine approved cancer drug indications by the EMA were biomarker-derived drugs. 78% (47/60) of approved cancer drugs that were only approved in the USA with orphan status were indicated for solid tumours, 22% (13/60) had indications for non-solid tumours. By contrast, out of those approved cancer drugs that received orphan designation by both agencies, 20% (8/41) were indicated for solid, and 80% (33/41) for non-solid tumours. CONCLUSIONS: Orphan designation was intended to encourage drug development for rare conditions. This study shows that the FDA approves more cancer drugs with such designations compared with the EMA, especially for subgroups of more prevalent cancers. One reason for the difference could be that the European Union requires demonstration of significant benefit for drugs that target the same indication as a drug already on the market to earn the orphan designation.
format Online
Article
Text
id pubmed-6797305
institution National Center for Biotechnology Information
language English
publishDate 2019
publisher BMJ Publishing Group
record_format MEDLINE/PubMed
spelling pubmed-67973052019-10-31 Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU Vokinger, Kerstin Noëlle Kesselheim, Aaron S BMJ Open Health Policy 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 drugs with orphan indications that met our inclusion criteria, of which 101 (75%) were also approved by the EMA. 80/101 (79%) were first approved in the USA. Only 41/101 (41%) also received orphan designation by the EMA. 33/101 (33%) were approved for biomarker-based indications in the USA, however, only nine approved cancer drug indications by the EMA were biomarker-derived drugs. 78% (47/60) of approved cancer drugs that were only approved in the USA with orphan status were indicated for solid tumours, 22% (13/60) had indications for non-solid tumours. By contrast, out of those approved cancer drugs that received orphan designation by both agencies, 20% (8/41) were indicated for solid, and 80% (33/41) for non-solid tumours. CONCLUSIONS: Orphan designation was intended to encourage drug development for rare conditions. This study shows that the FDA approves more cancer drugs with such designations compared with the EMA, especially for subgroups of more prevalent cancers. One reason for the difference could be that the European Union requires demonstration of significant benefit for drugs that target the same indication as a drug already on the market to earn the orphan designation. BMJ Publishing Group 2019-10-10 /pmc/articles/PMC6797305/ /pubmed/31601584 http://dx.doi.org/10.1136/bmjopen-2018-028634 Text en © Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.
spellingShingle Health Policy
Vokinger, Kerstin Noëlle
Kesselheim, Aaron S
Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title_full Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title_fullStr Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title_full_unstemmed Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title_short Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
title_sort application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the usa and eu
topic Health Policy
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6797305/
https://www.ncbi.nlm.nih.gov/pubmed/31601584
http://dx.doi.org/10.1136/bmjopen-2018-028634
work_keys_str_mv AT vokingerkerstinnoelle applicationoforphandrugdesignationtocancertreatments20082017acomprehensiveandcomparativeanalysisoftheusaandeu
AT kesselheimaarons applicationoforphandrugdesignationtocancertreatments20082017acomprehensiveandcomparativeanalysisoftheusaandeu