Cargando…

Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study

OBJECTIVE: We aimed to provide insight into differences in drug review decisions made by the US Food and Drug Administration’s (FDA) accelerated approval (AA) pathway and the European Medicines Agency’s (EMA) conditional marketing authorisation (CMA) pathway, and to add to the current knowledge base...

Descripción completa

Detalles Bibliográficos
Autores principales: Xie, Jinping, Li, Jinlian, Liu, Yue, Wang, Haoyang, Wang, Yifei, Yang, Yifan, Chen, Yi, Jiang, Rong, Shao, Rong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10255285/
https://www.ncbi.nlm.nih.gov/pubmed/37286329
http://dx.doi.org/10.1136/bmjopen-2022-069132
_version_ 1785056834271313920
author Xie, Jinping
Li, Jinlian
Liu, Yue
Wang, Haoyang
Wang, Yifei
Yang, Yifan
Chen, Yi
Jiang, Rong
Shao, Rong
author_facet Xie, Jinping
Li, Jinlian
Liu, Yue
Wang, Haoyang
Wang, Yifei
Yang, Yifan
Chen, Yi
Jiang, Rong
Shao, Rong
author_sort Xie, Jinping
collection PubMed
description OBJECTIVE: We aimed to provide insight into differences in drug review decisions made by the US Food and Drug Administration’s (FDA) accelerated approval (AA) pathway and the European Medicines Agency’s (EMA) conditional marketing authorisation (CMA) pathway, and to add to the current knowledge base of drug approval processes. DESIGN, SETTING, PARTICIPANTS: This cross-sectional study thoroughly examines novel oncology drugs with dual approval through FDA AA and EMA CMA between 2006 and 2021. Statistical analysis was performed from June to July 2022. PRIMARY AND SECONDARY OUTCOME MEASURES: The study examined the regulatory differences between regions for dually approved novel oncology drugs, including approval decisions, pivotal efficacy clinical trials, speed of review and postmarketing obligations. RESULTS: During this time period, there was a difference in the use of the FDA AA and the EMA CMA (FDA: EMA: 41.2%: 70.0%, p<0.05). Of the 25 drugs approved by both the FDA AA and the EMA CMA, 22 (88.0%) of the regulatory decisions were based on the same pivotal clinical trials. But there were more differences in the requirements for postmarketing obligations, with the EMA’s postmarketing obligations focusing on the efficacy and safety of the drug (EMA: FDA: 63.0%: 27.0%, p<0.05) and the FDA’s postmarketing obligations focusing more on the efficacy (FDA: EMA: 73.0%: 23.9%, p<0.05). In addition, both the USA and EU had some postmarketing obligations completed beyond the schedule (30.4% and 19.2% in the USA and EU, respectively), with the longest delays lasting 3.7 years (0.2–3.7 years) and 3.3 years (0.04–3.3 years) in the USA and EU, respectively. CONCLUSIONS: The FDA and EMA have different orientations and benefit–risk balance considerations in the use of AA or CMA. It is also the case that the shortcomings in the design and implementation of postmarketing studies have made it a challenge to obtain the evidence needed to confirm a drug’s benefits.
format Online
Article
Text
id pubmed-10255285
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher BMJ Publishing Group
record_format MEDLINE/PubMed
spelling pubmed-102552852023-06-10 Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study Xie, Jinping Li, Jinlian Liu, Yue Wang, Haoyang Wang, Yifei Yang, Yifan Chen, Yi Jiang, Rong Shao, Rong BMJ Open Health Policy OBJECTIVE: We aimed to provide insight into differences in drug review decisions made by the US Food and Drug Administration’s (FDA) accelerated approval (AA) pathway and the European Medicines Agency’s (EMA) conditional marketing authorisation (CMA) pathway, and to add to the current knowledge base of drug approval processes. DESIGN, SETTING, PARTICIPANTS: This cross-sectional study thoroughly examines novel oncology drugs with dual approval through FDA AA and EMA CMA between 2006 and 2021. Statistical analysis was performed from June to July 2022. PRIMARY AND SECONDARY OUTCOME MEASURES: The study examined the regulatory differences between regions for dually approved novel oncology drugs, including approval decisions, pivotal efficacy clinical trials, speed of review and postmarketing obligations. RESULTS: During this time period, there was a difference in the use of the FDA AA and the EMA CMA (FDA: EMA: 41.2%: 70.0%, p<0.05). Of the 25 drugs approved by both the FDA AA and the EMA CMA, 22 (88.0%) of the regulatory decisions were based on the same pivotal clinical trials. But there were more differences in the requirements for postmarketing obligations, with the EMA’s postmarketing obligations focusing on the efficacy and safety of the drug (EMA: FDA: 63.0%: 27.0%, p<0.05) and the FDA’s postmarketing obligations focusing more on the efficacy (FDA: EMA: 73.0%: 23.9%, p<0.05). In addition, both the USA and EU had some postmarketing obligations completed beyond the schedule (30.4% and 19.2% in the USA and EU, respectively), with the longest delays lasting 3.7 years (0.2–3.7 years) and 3.3 years (0.04–3.3 years) in the USA and EU, respectively. CONCLUSIONS: The FDA and EMA have different orientations and benefit–risk balance considerations in the use of AA or CMA. It is also the case that the shortcomings in the design and implementation of postmarketing studies have made it a challenge to obtain the evidence needed to confirm a drug’s benefits. BMJ Publishing Group 2023-06-07 /pmc/articles/PMC10255285/ /pubmed/37286329 http://dx.doi.org/10.1136/bmjopen-2022-069132 Text en © Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. https://creativecommons.org/licenses/by-nc/4.0/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/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Health Policy
Xie, Jinping
Li, Jinlian
Liu, Yue
Wang, Haoyang
Wang, Yifei
Yang, Yifan
Chen, Yi
Jiang, Rong
Shao, Rong
Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title_full Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title_fullStr Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title_full_unstemmed Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title_short Comparison of novel oncology drugs that received dual approval from the US accelerated approval and EU conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
title_sort comparison of novel oncology drugs that received dual approval from the us accelerated approval and eu conditional marketing authorisation pathways, 2006–2021: a cross-sectional study
topic Health Policy
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10255285/
https://www.ncbi.nlm.nih.gov/pubmed/37286329
http://dx.doi.org/10.1136/bmjopen-2022-069132
work_keys_str_mv AT xiejinping comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT lijinlian comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT liuyue comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT wanghaoyang comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT wangyifei comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT yangyifan comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT chenyi comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT jiangrong comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy
AT shaorong comparisonofnoveloncologydrugsthatreceiveddualapprovalfromtheusacceleratedapprovalandeuconditionalmarketingauthorisationpathways20062021acrosssectionalstudy