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Health Aff (Millwood). 2019 Feb;38(2):313-319. doi: 10.1377/hlthaff.2018.05330.

Impact Of The Priority Review Voucher Program On Drug Development For Rare Pediatric Diseases.

Author information

1
Thomas J. Hwang ( thwang1@partners.org ) is a researcher in the Program on Regulation, Therapeutics, and Law in the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, both in Boston, Massachusetts.
2
Florence T. Bourgeois is an associate professor of pediatrics at Harvard Medical School and director of the Pediatric Therapeutics and Regulatory Science Initiative in the Computational Health Informatics Program at Boston Children's Hospital.
3
Jessica M. Franklin is an assistant professor of medicine in the Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital and Harvard Medical School.
4
Aaron S. Kesselheim is an associate professor of medicine at Harvard Medical School and director of the Program on Regulation, Therapeutics, and Law in the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School.

Abstract

Only an estimated 5 percent of rare pediatric diseases have a treatment, although collectively they affect more than ten million children in the US. To stimulate drug development for rare pediatric diseases, Congress expanded the priority review voucher (PRV) program in 2012. A pediatric PRV, which can be sold to another manufacturer, requires the FDA to provide priority six-month review rather than the standard ten-month review to another drug of the company's choosing. We compared rare pediatric disease drugs eligible for a PRV and rare adult disease drugs (which are not eligible for a PRV). We found that compared to drugs for rare adult diseases, drugs for rare pediatric diseases progressed more quickly through all phases of clinical testing and were more likely to be first-in-class. The voucher program was not associated with a change in the rate of new pediatric drugs starting or completing clinical testing, but there was a significant increase in the rate of progress from Phase I to Phase II clinical trials after the program was implemented. New policies may be needed to expand the pipeline of therapies for rare pediatric diseases.

PMID:
30715972
DOI:
10.1377/hlthaff.2018.05330

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