Continued cancer drug approvals in Japan and Europe after market withdrawal in the United States : A comparative study of accelerated approvals
(2024) In Clinical and Translational Science 17(7). p.13879-13879- Abstract
Regulatory authorities must balance ensuring evidence of efficacy and safety of new drugs. Various regulatory pathways, such as the accelerated approval program in the United States (US), allow authorities to quickly approve drugs for severely ill patients by granting market authorization based on surrogate end points and pending confirmatory trials. In this cross-sectional study, we considered 23 indications of cancer drugs that received accelerated approval by the US Food and Drug Administration (FDA) but were subsequently withdrawn as of April 2023. Our investigation extended to assessing the regulatory status of these accelerated approvals in the European Union (EU) and Japan, examining relevant regulatory documents and identifying... (More)
Regulatory authorities must balance ensuring evidence of efficacy and safety of new drugs. Various regulatory pathways, such as the accelerated approval program in the United States (US), allow authorities to quickly approve drugs for severely ill patients by granting market authorization based on surrogate end points and pending confirmatory trials. In this cross-sectional study, we considered 23 indications of cancer drugs that received accelerated approval by the US Food and Drug Administration (FDA) but were subsequently withdrawn as of April 2023. Our investigation extended to assessing the regulatory status of these accelerated approvals in the European Union (EU) and Japan, examining relevant regulatory documents and identifying factors contributing to the withdrawal in the United States. Comparing regions, we found that for 52% (12/23) and 30% (7/23) of withdrawn accelerated approvals in the United States, sponsors had also sought marketing authorization from the European Medicines Agency (EMA) and Japan's Pharmaceuticals and Medical Devices Agency (PMDA), respectively. As of the April 30, 2023 study cutoff date, 83% (10/12) of drug-indication pairs remained approved by the EMA, while the PMDA retained 100% (7/7). For these indications, the time from FDA withdrawal until the study cutoff date ranged from 0.23 years to 11.45 years for EMA approvals (median: 1.28 years) and 1.10 years to 11.45 years for PMDA approvals (median: 3.22 years). These findings highlight substantial regulatory discrepancies concerning cancer drugs with unconfirmed benefits. Addressing these discrepancies may involve requiring pharmaceutical companies to confirm clinical benefits using more robust end points and fostering international harmonization in regulators' assessment.
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- author
- Hakariya, Hayase ; Moriarty, Frank ; Ozaki, Akihiko ; Mulinari, Shai LU ; Saito, Hiroaki and Tanimoto, Tetsuya
- organization
- publishing date
- 2024-07-10
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- Drug Approval/legislation & jurisprudence, Japan, United States, Humans, Antineoplastic Agents/therapeutic use, United States Food and Drug Administration, Cross-Sectional Studies, Europe, European Union, Neoplasms/drug therapy
- in
- Clinical and Translational Science
- volume
- 17
- issue
- 7
- pages
- 13879 - 13879
- publisher
- Wiley-Blackwell
- external identifiers
-
- scopus:85198114898
- pmid:38987923
- ISSN
- 1752-8062
- DOI
- 10.1111/cts.13879
- language
- English
- LU publication?
- yes
- additional info
- © 2024 The Author(s). Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.
- id
- 040f267b-fa9a-43f1-9f8d-853d29cdb040
- date added to LUP
- 2024-07-17 11:40:38
- date last changed
- 2024-08-29 07:08:23
@article{040f267b-fa9a-43f1-9f8d-853d29cdb040,
abstract = {{<p>Regulatory authorities must balance ensuring evidence of efficacy and safety of new drugs. Various regulatory pathways, such as the accelerated approval program in the United States (US), allow authorities to quickly approve drugs for severely ill patients by granting market authorization based on surrogate end points and pending confirmatory trials. In this cross-sectional study, we considered 23 indications of cancer drugs that received accelerated approval by the US Food and Drug Administration (FDA) but were subsequently withdrawn as of April 2023. Our investigation extended to assessing the regulatory status of these accelerated approvals in the European Union (EU) and Japan, examining relevant regulatory documents and identifying factors contributing to the withdrawal in the United States. Comparing regions, we found that for 52% (12/23) and 30% (7/23) of withdrawn accelerated approvals in the United States, sponsors had also sought marketing authorization from the European Medicines Agency (EMA) and Japan's Pharmaceuticals and Medical Devices Agency (PMDA), respectively. As of the April 30, 2023 study cutoff date, 83% (10/12) of drug-indication pairs remained approved by the EMA, while the PMDA retained 100% (7/7). For these indications, the time from FDA withdrawal until the study cutoff date ranged from 0.23 years to 11.45 years for EMA approvals (median: 1.28 years) and 1.10 years to 11.45 years for PMDA approvals (median: 3.22 years). These findings highlight substantial regulatory discrepancies concerning cancer drugs with unconfirmed benefits. Addressing these discrepancies may involve requiring pharmaceutical companies to confirm clinical benefits using more robust end points and fostering international harmonization in regulators' assessment.</p>}},
author = {{Hakariya, Hayase and Moriarty, Frank and Ozaki, Akihiko and Mulinari, Shai and Saito, Hiroaki and Tanimoto, Tetsuya}},
issn = {{1752-8062}},
keywords = {{Drug Approval/legislation & jurisprudence; Japan; United States; Humans; Antineoplastic Agents/therapeutic use; United States Food and Drug Administration; Cross-Sectional Studies; Europe; European Union; Neoplasms/drug therapy}},
language = {{eng}},
month = {{07}},
number = {{7}},
pages = {{13879--13879}},
publisher = {{Wiley-Blackwell}},
series = {{Clinical and Translational Science}},
title = {{Continued cancer drug approvals in Japan and Europe after market withdrawal in the United States : A comparative study of accelerated approvals}},
url = {{http://dx.doi.org/10.1111/cts.13879}},
doi = {{10.1111/cts.13879}},
volume = {{17}},
year = {{2024}},
}