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Breakthrough therapy designation

Breakthrough therapy designation is a United States Food and Drug Administration designation that expedites drug development, created by Congress under Section 902 of the Food and Drug Administration Safety and Innovation Act of 9 July 2012[1]. It was created for drugs that may be significantly better treatments for serious diseases or conditions[2].

A drug can be given the designation if it is intended, alone or in combination with other drugs, to treat a serious or life-threatening disease or condition[3]. The preliminary clinical evidence must also indicate that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development[3].

Requests are submitted as an amendment to the IND application, usually before the end of Phase II meeting[4]. They are reviewed by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER)[5]. CDER receives approximately 100 requests per year, and historically about one third were approved[5].

Drugs that have been granted the designation are given priority review, and the FDA works with the sponsor to expedite the approval process[4]. This can include rolling reviews, smaller clinical trials and alternative trial designs[4]. The designation is not intended to imply that a drug is actually a breakthrough or that there is high-quality evidence of efficacy for a particular condition[6]. If the designation is no longer supported by subsequent data, the FDA may rescind it[7].

Why it matters to investors

The FDA guidance states that not all products designated as breakthrough therapies will ultimately be shown to have the substantial improvement over available therapies that the preliminary clinical evidence suggested at the time of designation[7]. Critics note that designations rest on preliminary evidence, including surrogate markers such as laboratory measurements, which often do not reflect meaningful clinical benefit[8].

Related: Priority review, Fast track designation, Accelerated approval, Investigational New Drug (IND)

Sources

  1. Darrow, JJ; Avorn, J; Kesselheim, AS (27 March 2014). "New FDA breakthrough-drug category—implications for patients" . The New England Journal of Medicine . 370 (13): 1252– 8. doi : 10.1056/nejmhle1311493 . PMID 24670173 . doi.org; Yandell, Kate (1 January 2016). "Picking Up the Pace" . The Scientist . the-scientist.com (via Wikipedia: Breakthrough therapy)
  2. "Fact Sheet: Breakthrough Therapies" . Food and Drug Administration . Archived from the original on 2017-01-18 . Retrieved 2019-12-16 . web.archive.org (via Wikipedia: Food and Drug Administration Safety and Innovation Act)
  3. "Fact Sheet: Breakthrough Therapies" . United States Food and Drug Administration. 10 December 2014. Archived from the original on 9 October 2017 . Retrieved 9 October 2017 . A breakthrough therapy is a drug: • intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and • preliminary clinical evidence indicates that the drug may dem web.archive.org; Food and Drug Administration Safety and Innovation Act (FDASIA) , Silver Spring, MD: Food and Drug Administration , 29 January 2015, archived from the original on 18 July 2015 , retrieved 20 July 2015 web.archive.org (via Wikipedia: Breakthrough therapy)
  4. Research, Center for Drug Evaluation and (10 April 2019). "Expedited Programs for Serious Conditions––Drugs and Biologics" . U.S. Food and Drug Administration . Archived from the original on 14 June 2019. web.archive.org (via Wikipedia: Breakthrough therapy)
  5. Research, Center for Drug Evaluation and (5 October 2021). "IND Activity Reports - Breakthrough Therapy Designation Requests" . www.fda.gov . Archived from the original on 30 March 2014. web.archive.org (via Wikipedia: Breakthrough therapy)
  6. Schwitzer G (13 May 2016). "CBS proclaims 'cancer breakthrough' – doesn't explain what FDA means by that term" . Health News Review . Archived from the original on 8 December 2020 . Retrieved 9 October 2017 . web.archive.org; Kepplinger EE (February 2015). "FDA's Expedited Approval Mechanisms for New Drug Products" . Biotechnol Law Rep . 34 (1): 15– 37. doi : 10.1089/blr.2015.9999 . PMC 4326266 . PMID 25713472 . The fourth expedited approval mechanism—the Breakthrough Therapy designation—applies to a new drug product if it "is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-thre ncbi.nlm.nih.gov (via Wikipedia: Breakthrough therapy)
  7. "Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics" (PDF) . FDA. May 2014. p. 10. [ dead link ] fda.gov (via Wikipedia: Breakthrough therapy)
  8. Schwitzer G (13 May 2016). "CBS proclaims 'cancer breakthrough' – doesn't explain what FDA means by that term" . Health News Review . Archived from the original on 8 December 2020 . Retrieved 9 October 2017 . web.archive.org (via Wikipedia: Breakthrough therapy)

Checked 8 October 2026. How we check sources