Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2018 Jul 17;320(3):301-303.
doi: 10.1001/jama.2018.7619.

Clinical Trial Evidence Supporting FDA Approval of Drugs Granted Breakthrough Therapy Designation

Jeremy Puthumana  1 , Joshua D Wallach  2 , Joseph S Ross  3
Affiliations

Clinical Trial Evidence Supporting FDA Approval of Drugs Granted Breakthrough Therapy Designation

Jeremy Puthumana et al. JAMA. .

Abstract

This study used the Drugs@FDA database to review FDA-approved “breakthrough” therapies from 2012 to 2017 (characterizing the pivotal clinical trials that serve as the basis of FDA approval), and premarket development and review times to assess the strength of evidence supporting breakthrough approvals.

PubMed Disclaimer

Conflict of interest statement

Conflict of Interest Disclosures: All authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Wallach reported receiving grant funding from Laura and John Arnold Foundation. Dr Ross reported receiving grant funding from Laura and John Arnold Foundation, Johnson & Johnson, Medtronic, the US Food and Drug Administration, Blue Cross Blue Shield Association, Centers for Medicare & Medicaid Services, Agency for Healthcare Research and Quality, and from the National Heart, Lung, and Blood Institute of the National Institutes of Health. No other disclosures were reported.

Comment in

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. US Food and Drug Administration Guidance for industry: expedited programs for serious conditions—drugs and biologics. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformat.... Accessed March 14, 2018.
    1. Kesselheim AS, Woloshin S, Eddings W, Franklin JM, Ross KM, Schwartz LM. Physicians’ knowledge about FDA approval standards and perceptions of the “breakthrough therapy” designation. JAMA. 2016;315(14):1516-1518. doi:10.1001/jama.2015.16984 - DOI - PubMed
    1. Krishnamurti T, Woloshin S, Schwartz LM, Fischhoff B. A randomized trial testing US Food and Drug Administration “breakthrough” language. JAMA Intern Med. 2015;175(11):1856-1858. doi:10.1001/jamainternmed.2015.5355 - DOI - PubMed
    1. Downing NS, Aminawung JA, Shah ND, Krumholz HM, Ross JS. Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005-2012. JAMA. 2014;311(4):368-377. doi:10.1001/jama.2013.282034 - DOI - PMC - PubMed
    1. Wallach JD, Egilman AC, Dhruva SS, et al. . Postmarket studies required by the US Food and Drug Administration for new drugs and biologics approved between 2009 and 2012: cross sectional analysis. BMJ. 2018;361:k2031. - PMC - PubMed