Article ; Online: Accelerated Approval of 17α-Hydroxyprogesterone Caproate: A Cautionary Tale.
2020 Volume 135, Issue 5, Page(s) 1207–1213
Abstract: Before 2011, 17α-hydroxyprogesterone caproate (17P) was used to prevent recurrent preterm birth in women with singleton pregnancies and was compounded at a low cost (∼$15 per injection). In 2011, the U.S. Food and Drug Administration (FDA) approved a ... ...
| Abstract | Before 2011, 17α-hydroxyprogesterone caproate (17P) was used to prevent recurrent preterm birth in women with singleton pregnancies and was compounded at a low cost (∼$15 per injection). In 2011, the U.S. Food and Drug Administration (FDA) approved a commercial version of 17P (trade name "Makena") through their Accelerated Approval Program, and the price of 17P subsequently increased by nearly 100-fold. This approval was largely based on a methodologically limited, placebo-controlled trial, which found that although 17P significantly reduced preterm births, the placebo group had significantly more participants with a history of preterm birth, potentially confounding the results. The FDA required a confirmatory trial for continued approval that demonstrated clinical benefit. Eight years after accelerated approval, the confirmatory trial, PROLONG (Progestin's Role in Optimizing Neonatal Gestation), found no evidence of an effect of Makena for reducing recurrent preterm birth or perinatal mortality. Trial completion triggered an automatic review of Makena by an advisory committee, which voted 9-7 to recommend revoking approval of Makena for preterm birth. Although the FDA created the Accelerated Approval Program to introduce therapies for serious conditions that lacked treatment options, Makena is an example of the limitations of this program. We encourage the FDA to re-evaluate their program and consider improvements, such as shorter timeframes to complete confirmatory trials, potentially revoking approval if the studies are not completed within a predefined timeframe, and to hold manufacturers responsible, in part, for the costs of therapy if they cannot prove a clinical benefit. |
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| MeSH term(s) | 17 alpha-Hydroxyprogesterone Caproate/therapeutic use ; Clinical Trials as Topic/legislation & jurisprudence ; Clinical Trials as Topic/methods ; Drug Approval/methods ; Female ; Humans ; Infant, Newborn ; Perinatal Mortality ; Pregnancy ; Premature Birth/prevention & control ; Treatment Outcome ; United States ; United States Food and Drug Administration |
| Chemical Substances | 17 alpha-Hydroxyprogesterone Caproate (276F2O42F5) |
| Language | English |
| Publishing date | 2020-04-10 |
| Publishing country | United States |
| Document type | Journal Article |
| ZDB-ID | 207330-4 |
| ISSN | 1873-233X ; 0029-7844 |
| ISSN (online) | 1873-233X |
| ISSN | 0029-7844 |
| DOI | 10.1097/AOG.0000000000003787 |
| Database | MEDical Literature Analysis and Retrieval System OnLINE |
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