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Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019.
JAMA Netw Open. 2022 06 01; 5(6):e2216183.JN

Abstract

Importance

Ensuring patients have access to safe and efficacious medicines in a timely manner is an essential goal for regulatory agencies, one which has particular importance in oncology because of the substantial unmet need for new therapies. The 2 largest regulatory agencies, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have pivotal global roles, and their recommendations and approvals are frequently followed by other national regulators.

Objective

To compare market authorization dates for new oncology therapies approved in the US and Europe over the past decade and to examine and contrast the regulatory activities of the FDA and EMA in the approval of new cancer medicines.

Design, Setting, and Participants

This cross-sectional study reviewed the FDA and EMA regulatory databases to identify new oncology therapies approved in both the US and Europe from 2010 to 2019, and characterization of the timings of regulatory activities. Statistical analysis was performed from January to April 2022.

Main Outcomes and Measures

Regulatory approval date, review time, submission of market authorization application, accelerated approval or conditional marketing authorization status and proportion of approvals prior to peer-reviewed publication of pivotal trial results.

Results

In total, 89 new concomitant oncology therapies were approved in the US and Europe from 2010 to 2019. The FDA approved 85 oncology therapies (95%) before European authorization and 4 therapies (5%) after. The median (IQR) delay in market authorization for new oncology therapies in Europe was 241 (150-370) days compared with the US. The median (IQR) review time was 200 (155-277) days for the FDA and 426 (358-480) days for the EMA. Sixty-four new licensing applications (72%) were submitted to the FDA first, compared with 21 (23%) to the EMA. Thirty-five oncology therapies (39%) were approved by the FDA prior to pivotal study publication, whereas only 8 (9%) by the EMA.

Conclusion and Relevance

In this cross-sectional study, new oncology therapies were approved earlier in the US than Europe. The FDA received licensing applications sooner and had shorter review times. However, more therapies were approved prior to licensing study publication, leaving uncertainty for practitioners regarding clinical utility and safety of newly approved therapies.

Authors+Show Affiliations

Department of Surgery and Cancer, Imperial College London, London, United Kingdom.Division of Medical Oncology, Mayo Clinic, Rochester, Minnesota.Division of Cancer Care and Epidemiology, Departments of Oncology and Public Health Sciences, Queen's University Cancer Research Institute, Kingston, Canada.Brighton and Sussex University Hospitals NHS Trust, Brighton, United Kingdom.Department of Surgery and Cancer, Imperial College London, London, United Kingdom.Division of Hematology and Oncology, Vanderbilt University, Nashville, Tennessee.Division of Oncology, Department of Medicine, Stanford University School of Medicine, Palo Alto, California.

Pub Type(s)

Journal Article
Research Support, N.I.H., Extramural

Language

eng

PubMed ID

35687337

Citation

Lythgoe, Mark P., et al. "Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019." JAMA Network Open, vol. 5, no. 6, 2022, pp. e2216183.
Lythgoe MP, Desai A, Gyawali B, et al. Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019. JAMA Netw Open. 2022;5(6):e2216183.
Lythgoe, M. P., Desai, A., Gyawali, B., Savage, P., Krell, J., Warner, J. L., & Khaki, A. R. (2022). Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019. JAMA Network Open, 5(6), e2216183. https://doi.org/10.1001/jamanetworkopen.2022.16183
Lythgoe MP, et al. Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019. JAMA Netw Open. 2022 06 1;5(6):e2216183. PubMed PMID: 35687337.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019. AU - Lythgoe,Mark P, AU - Desai,Aakash, AU - Gyawali,Bishal, AU - Savage,Philip, AU - Krell,Jonathan, AU - Warner,Jeremy L, AU - Khaki,Ali Raza, Y1 - 2022/06/01/ PY - 2022/6/10/entrez PY - 2022/6/11/pubmed PY - 2022/6/15/medline SP - e2216183 EP - e2216183 JF - JAMA network open JO - JAMA Netw Open VL - 5 IS - 6 N2 - Importance: Ensuring patients have access to safe and efficacious medicines in a timely manner is an essential goal for regulatory agencies, one which has particular importance in oncology because of the substantial unmet need for new therapies. The 2 largest regulatory agencies, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have pivotal global roles, and their recommendations and approvals are frequently followed by other national regulators. Objective: To compare market authorization dates for new oncology therapies approved in the US and Europe over the past decade and to examine and contrast the regulatory activities of the FDA and EMA in the approval of new cancer medicines. Design, Setting, and Participants: This cross-sectional study reviewed the FDA and EMA regulatory databases to identify new oncology therapies approved in both the US and Europe from 2010 to 2019, and characterization of the timings of regulatory activities. Statistical analysis was performed from January to April 2022. Main Outcomes and Measures: Regulatory approval date, review time, submission of market authorization application, accelerated approval or conditional marketing authorization status and proportion of approvals prior to peer-reviewed publication of pivotal trial results. Results: In total, 89 new concomitant oncology therapies were approved in the US and Europe from 2010 to 2019. The FDA approved 85 oncology therapies (95%) before European authorization and 4 therapies (5%) after. The median (IQR) delay in market authorization for new oncology therapies in Europe was 241 (150-370) days compared with the US. The median (IQR) review time was 200 (155-277) days for the FDA and 426 (358-480) days for the EMA. Sixty-four new licensing applications (72%) were submitted to the FDA first, compared with 21 (23%) to the EMA. Thirty-five oncology therapies (39%) were approved by the FDA prior to pivotal study publication, whereas only 8 (9%) by the EMA. Conclusion and Relevance: In this cross-sectional study, new oncology therapies were approved earlier in the US than Europe. The FDA received licensing applications sooner and had shorter review times. However, more therapies were approved prior to licensing study publication, leaving uncertainty for practitioners regarding clinical utility and safety of newly approved therapies. SN - 2574-3805 UR - https://neuro.unboundmedicine.com/medline/citation/35687337/Cancer_Therapy_Approval_Timings_Review_Speed_and_Publication_of_Pivotal_Registration_Trials_in_the_US_and_Europe_2010_2019_ L2 - https://jamanetwork.com/journals/jamanetworkopen/fullarticle/10.1001/jamanetworkopen.2022.16183 DB - PRIME DP - Unbound Medicine ER -