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Experience with the Priority Review Voucher Program for Drug Development

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Experience with the Priority Review Voucher Program for Drug Development Opinion VIEWPOINT Experience With the Priority Review Voucher Program for Drug Development Aaron S. Kesselheim, In 2007, Congress authorized a program intended to but was found to cure visceral leishmaniasis in the late MD, JD, MPH promote development of new treatments for ne- 1990s. Paladin Laboratories acquired rights to the drug Program on Regulation, glected tropical diseases, conditions that disproportion- in 2008 for $8.5 million5 and submitted an application to Therapeutics, and Law ately affect poor people in developing countries. Ne- the FDA for miltefosine in 2013 based on trials it had not (PORTAL), Division of Pharmacoepidemiology glected tropical diseases lack treatments for many conducted dating back to 1999. The drug was approved andPharmacoeconomics, reasons, including attracting little interest from multi- in2014.ThevoucherwassoldtoGileadScienceslaterthat Harvard Medical School, national pharmaceutical manufacturers, which prefer- year for $125 million and remains unused. Boston, Massachusetts. entially invest in developing products that offer the pos- Three pediatric disease PRVs have been awarded sibility for more profitable returns. since 2012. The first was issued in February 2014 to Bio- Lara R. Maggs, MBioethics To help overcome such barriers, 2007 federal leg- Marin after FDA approval of elosulfate alfa for the treat- Program on Regulation, islation offering priority review vouchers (PRVs) to com- mentofMorquioAsyndrome,aninheritedmetabolicdis- Therapeutics, and Law panies that sponsored drugs newly approved by the US ease affecting approximately 800 children in the US. The (PORTAL), Division of Pharmacoepidemiology Food and Drug Administration (FDA) to treat qualifying pivotal Phase III trial for the drug was launched in Feb- andPharmacoeconomics, neglected tropical diseases such as tuberculosis, ma- ruary 2011, 17 months prior to the initiation of the pro- Harvard Medical School, laria, schistosomiasis, and yaws. Once granted, the gram. This voucher was sold to Regeneron and Sanofi for Boston, Massachusetts. vouchers could be transferred or sold, or redeemed at $67.5 million and used to accelerate the review of ali- the FDA to accelerate the regulatory review of a differ- rocumab, a PCSK9-inhibitor approved in July 2015 for Ameet Sarpatwari, JD, PhD ent product (eTable in the Supplement) from the stan- patients with familial hypercholesterolemia. Program on Regulation, dard 10-month period to the priority 6-month period in- In March 2015, 2 additional rare pediatric disease Therapeutics, and Law tended to be reserved for drugs that appear to represent vouchers were awarded. The first was granted to (PORTAL), Division of therapeutic advances.1 Earlier access to the US market Asklepion Pharmaceuticals, the sponsor of cholic acid, Pharmacoepidemiology andPharmacoeconomics, leads to longer market exclusivity periods and greater approved for the treatment of bile acid synthesis disor- Harvard Medical School, revenues. In 2012, PRVs were also made available to ders. The efficacy and safety of cholic acid was demon- Boston, Massachusetts. sponsors of FDA-approved drugs treating a rare pediat- strated in an investigator-initiated trial begun in 1994. ric diseases, with a plan to reexamine the effects of the After approval, the rights to the drug and voucher were voucher program in this clinical context after 3 rare pe- obtained by Retrophin, which subsequently sold the diatric disease vouchers were granted.2 voucher to Sanofi for $245 million in May 2015. Supplemental ThroughAugust2015,theFDAhasissued6vouchers The most recent voucher was granted to United content at jama.com (Table). The first tropical disease voucher was granted in Therapeutics for approval for dinutuximab, a drug for 2009 to Novartis for the antimalarial artemether- combination use with other treatments for pediatric pa- lumefantrine,eventhoughatthetimeofFDAapproval,this tients with high-risk neuroblastoma. This drug was de- drug had been approved in more than 80 countries.3 veloped by the National Cancer Institute, which synthe- Artemether-lumefantrinequalifiedbecausethestatutere- sized it, conducted its initial preclinical studies, and then quired only that the drug was not yet registered in the manufactured it through the Biopharmaceutical Devel- United States. Toearn the voucher, Novartis submitted to opment Program to perform pivotal phase 3 testing be- the FDA 8 of the 20 studies it had sponsored from 1993 to tween 2001 and 2009. In August 2015, United Thera- 2007 to support approval of the drug abroad. peutics sold its voucher to AbbVie for $350 million. The next tropical disease voucher was for bedaqui- Based on this experience, some preliminary conclu- line, indicated for multidrug resistant tuberculosis. The sions can be drawn about the PRV program. First, little re- surrogate measure of efficacy agreed on by the FDA was liable evidence that the program’s primary intention of the rapidity of conversion of patients’ sputum samples spurring novel drug development has been met exists, at from growth to no growth of the tuberculosis bacillus in least for tropical diseases, for which PRVs have now been Corresponding culture. Although preapproval studies demonstrated an available for 7 years. Artemether-lumefantrine was regis- Author: Aaron S. improvement in this measure among bedaquiline- tered worldwide prior to FDA approval. Miltefosine was Kesselheim, MD, JD, MPH, Division of treated patients, those studies also showed a signifi- registered outside the United States a decade before its Pharmacoepidemiology cantly higher mortality (mostly from tuberculosis) for pa- FDAreview.ThesponsorofitsFDAapplicationearnedthe andPharmacoeconomics, tients treated with bedaquiline than those who were not voucher despite not being involved in its development. Department of 4 Medicine, Brigham and (10 of 79 vs 2 of 81). Despite these concerns, the FDA ap- Artemether-lumefantrine, miltefosine, dinuximab, and Women’s Hospital, proved bedaquiline in 2012 for use “when an effective cholic acid were also based on significant public sector in- Harvard Medical School, treatment regimen cannot otherwise be provided.” vestment. One way to potentially prevent such windfalls 1620 Tremont St, In 2014, Knight Therapeutics received a voucher for wouldbetoredesignthevouchersystemsothatdrugcom- Ste 3030, Boston, MA 02120 (akesselheim miltefosine,atreatmentforleishmaniasis.Miltefosinewas panies would have to show some level of investment in a @partners.org). originally developed as an anticancer agent in the 1980s new drug’s development before earning the reward. jama.com (Reprinted) JAMA Published online September 28, 2015 E1 Copyright 2015 American Medical Association. All rights reserved. Downloaded From: http://jama.jamanetwork.com/ by Patty Fegan on 10/01/2015 Opinion Viewpoint Table. Awarded Priority Review Vouchers, September 2008 to August 2015a Drug Year Sponsor Disease Sold Used Neglected tropical diseases Artemether- 2009 Novartis Malaria No; used internally Canakunimab for gouty lumefantrine arthritis in 2011 (indication rejected) Bedaquiline 2012 Johnson & Multidrug-resistant Johnson tuberculosis Miltefosine 2014 Knight Leishmaniasis $125 million Therapeutics to Gilead Sciences, 2014 Rare pediatric diseases Elosulfate alfa 2014 BioMarin Morquio A syndrome $67.5 millionto Alirocumab for high Regeneron and Sanofi, cholesterol (approved 2014 July 2015) Cholic acid 2015 Asklepiona Bile acid synthesis $245 million disorders involving to Sanofi, 2015 single-enzyme defects and peroxisomal a Rights to approved drug and disorders voucher subsequently purchased by Dinutuximab 2015 United High-risk neuroblastoma $350 million Retrophin for approximately $40 Therapeutics to Abbvie, 2015 million. The voucher may have had limited influence on drug develop- Whether the voucher is successful at improving drug innova- ment so far because its value only accrues after drug approval. The de- tion, it clearly does not ensure affordable access to the products velopmentprocesscanbeuncertainandmaytake6to10yearsormore either in the United States (elosulfase costs $380 000 per year7) from the time of original molecule identification. The voucher may be or overseas. As a result, making use or sale of the voucher condi- optimallyusefulforcaseslikeartemether-lumefantrineormiltefosine— tional upon demonstrating equitable marketing of the drug has drugs that are known to be effective but have not been taken through been suggested.8 For example, differential pricing across coun- the final step to FDA approval for reasons that could include the rela- tries is commonly used to make treatments available in resource- tively minor costs of drug registration. Experience with the program, poor settings, having successfully reduced the cost of antiretrovi- though, suggests that few effective and safe drugs for tropical dis- ral human immunodeficiency virus and AIDS in developing eases are undeveloped and could be taken through any final steps but countries by up to 90% of the profit-making price charged in for the additional late-stage incentive that the vouchers provide. high-income countries. The voucher program could be further The value of the voucher has increased from $67.5 million to amended to make its issuance dependent on successful demon- $350 million. The prospect of a payment of as much as $350 mil- stration of such a plan. lion a decade or so in the future may nevertheless be insufficient for Several more promising approaches exist to promote discov- large pharmaceutical
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