Considering Global Development? Insights from Applications for FDA Breakthrough Therapy and EMA PRIME Designations-Reference-Cited by-同舟云学术

Considering Global Development? Insights from Applications for FDA Breakthrough Therapy and EMA PRIME Designations

Published:2022-10-28 Issue:2 Volume:57 Page:321-328
ISSN:2168-4790
Container-title:Therapeutic Innovation & Regulatory Science
language:en
Short-container-title:Ther Innov Regul Sci

Author:

Hanaizi Zahra,Kweder Sandra,Thor Shannon,Ribeiro Sonia,Marcal Anabela

Abstract

AbstractThe United States Food and Drug Administration and the European Medicines Agency (EMA) each have programs to expedite development of products identified as having potential to address unmet medical needs: the Breakthrough therapy (BT) and Regenerative Medicines Advanced Therapies designation programs in the US and the Priority Medicines (PRIME) scheme at EMA. We reviewed commonalities and differences in requests submitted and products designated through these programs, with the intent to explore ways to better support global development. During the period from PRIME’s launch in April 2016 to 31 December 2020, 151 requests were made to both BT and PRIME programs and the agencies reached concordant outcomes to grant or deny requests for almost two thirds of the cases (93/151, 62%), suggesting similar perspectives across international regulators on the potential of the products under study. Forty-two (42/151, 28%) products were granted both BT and PRIME, thus found by both Agencies to have the potential to address an unmet need for a serious condition, and thereby products for which efficient development would be highly desirable. Working toward better engagement on global development strategies is in the best interests of patients and public health. With this in mind, Agencies and sponsors should take advantage of existing collaborative opportunities, such as parallel scientific advice, and work to identify fresh approaches to support global development of products for unmet medical needs.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Public Health, Environmental and Occupational Health,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Link

https://link.springer.com/content/pdf/10.1007/s43441-022-00475-0.pdf

Reference9 articles.

1. US FDA breakthrough therapy information page. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy.

2. US FDA guidance document expedited programs for serious conditions: drugs and biologics, May 2014. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics.

3. Enhanced early dialogue to facilitate accelerated assessment of priority medicines (PRIME), EMA/CHMP/57760/2015, Rev. 1. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/enhanced-early-dialogue-facilitate-accelerated-assessment-priority-medicines-prime_en.pdf.

4. US FDA fast track, breakthrough therapy, accelerated approval, priority review information page. https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review.

5. US FDA regenerative medicine advanced therapy designation information page. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation.