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Progesterone for Prevention of Preterm Birth

Shortcomings and Unintended Consequences of the Orphan Drug Act

Gee, Rebekah E., MD, MPH; Kuy, SreyRam, MD, MHS; Karas, Laura O., MD, MPH

doi: 10.1097/AOG.0000000000002308
Contents: Current Commentary

Preterm birth is a problem of major public health significance that continues to plague our country despite the existence of a therapy, 17α-hydroxyprogesterone caproate, with known efficacy in reducing the risk of spontaneous preterm birth among high-risk women. Over the past several years, the Louisiana Department of Health has undertaken a robust, multifaceted initiative to improve access to 17α-hydroxyprogesterone caproate, which resulted in a 3.5-fold increase in the percentage of eligible high-risk pregnant women in the Medicaid program who received the therapy between 2013 and 2016. Yet despite Louisiana's progress, the vast majority of the eligible population still fails to receive 17α-hydroxyprogesterone caproate. In this Current Commentary, we argue that the high price of progesterone since U.S. Food and Drug Administration approval has unnecessarily complicated access, and our nation has potentially suffered nearly 60,000 avoidable premature births as a consequence. We present the history of the orphan drug approval and manufacturer-imposed price increase for injectable progesterone, the interplay between the drug's high price and the persistence of racial and ethnic disparities in preterm birth, which are particularly germane in Louisiana, and Louisiana's broad-reaching efforts to improve progesterone coverage. The story of 17α-hydroxyprogesterone caproate highlights the durable barriers that high prices place in the way of access and helps illuminate the shortcomings and unintended consequences of the Orphan Drug Act. This case, however, is not an outlier; it is the far-too-common product of monopoly pricing in the U.S. pharmaceutical market, inadvertently bolstered by existing law, at the expense of affordability and patient access.

Since the orphan drug approval of 17α-hydroxyprogesterone caproate for prevention of preterm birth in 2011, the drug's high price has impeded access and exacerbated health care disparities.

Louisiana Department of Health, Baton Rouge, Louisiana; the Department of Health Policy and Management, School of Public Health, and the Department of Obstetrics and Gynecology, School of Medicine, Louisiana State University, New Orleans, Louisiana; the Department of Surgery, Louisiana State University School of Medicine, New Orleans, Louisiana; and the Department of Health Policy and Management, the Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.

Corresponding author: Rebekah E. Gee, MD, MPH, Louisiana Department of Health, 628 N. Fourth Street, P.O. Box 629, Baton Rouge, Louisiana 70821-0629; email:

Financial Disclosure The authors did not report any potential conflicts of interest.

Each author has indicated that he or she has met the journal's requirements for authorship.

© 2017 by The American College of Obstetricians and Gynecologists. Published by Wolters Kluwer Health, Inc. All rights reserved.