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Photo of Shai Mulinari. Private photo.

Shai Mulinari

Associate Professor | Senior Lecturer

Photo of Shai Mulinari. Private photo.

Continued cancer drug approvals in Japan and Europe after market withdrawal in the United States : A comparative study of accelerated approvals

Author

  • Hayase Hakariya
  • Frank Moriarty
  • Akihiko Ozaki
  • Shai Mulinari
  • Hiroaki Saito
  • Tetsuya Tanimoto

Summary, in English

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of cancer drugs that received accelerated approval by the US Food and Drug Administration (FDA) but were subsequently withdrawn as of April 2023.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 Comparing regions, we found that for 52% (12/23) and 30% (7/23) of withdrawn accelerated approvals in the United States, sponsors had also sought marketing authorization from the European Medicines Agency (EMA) and Japan's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.

Department/s

  • Department of Sociology

Publishing year

2024-07-10

Language

English

Publication/Series

Clinical and Translational Science

Volume

17

Issue

7

Document type

Journal article

Publisher

Wiley-Blackwell

Topic

  • Sociology (excluding Social Work, Social Anthropology, Demography and Criminology)
  • Pharmacology and Toxicology

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

Status

Published

ISBN/ISSN/Other

  • ISSN: 1752-8062