Home Media Research Seminar Teaching Vita Voucher

Priority Review Voucher

Video

Overview

The priority review voucher (PRV) was based on a 2006 paper (Ridley et al. 2006) and became law in 2007. Under the law, a developer of a treatment for a neglected disease receives a PRV for priority review from the Food and Drug Administration (FDA) to be used with a product of its choice or sold to another developer.

Priority review means that the FDA aims to render a decision in 6 months. In contrast, the FDA aims to complete a standard review in about 10 months, and it often takes even longer. The median difference in recent years was about 7 months (Grabowski et al. 2009).

The PRV is intended to reduce two types of inefficiency. First, the PRV speeds approval of potential blockbuster therapies in the US, getting US patients access to these treatments more quickly. Second, the PRV motivates more treatments for neglected diseases.

By moving one drug to faster review, there is the potential to slow other drugs. To provide FDA with more resources and mitigate this cost, the PRV holder must pay the FDA an additional user fee ($2,325,000 in fiscal year 2014) and provide the FDA with a one-year notice before redemption.

The PRV is transferable. The law reads as follows: "The sponsor of a tropical disease product that receives a priority review PRV under this section may transfer (including by sale) the entitlement to such voucher." FDA attorneys interpreted this language as permitting only one transfer, but a pending Senate bill would allow unlimited transfer.

Regulation

The PRV regulation appears in 21 U.S.C. 360N - Priority review to encourage treatments for tropical diseases.

FDA published draft "Guidance for Industry: Tropical Disease Priority Review Vouchers" in 2008.

Eligibility

To be eligible for a PRV, the drug or vaccine must satisfy at least four criteria.

  1. Treat one of the following sixteen diseases:
    • Blinding trachoma
    • Buruli Ulcer
    • Cholera
    • Dengue
    • Dracunculiasis
    • Fascioliasis
    • Human African trypanosomiasis
    • Leishmaniasis
    • Leprosy
    • Lymphatic filariasis
    • Malaria
    • Onchocerciasis
    • Schistosomiasis
    • Soil transmitted helminthiasis
    • Tuberculosis
    • Yaws
  2. Be a new drug application (NDA).
  3. Be a new molecular entity (NME) or new chemical entity (NCE). It must contain no active ingredient (including any ester or salt of the active ingredient) that has been approved in any other application.
  4. Offer major advances in treatment, or provide treatment where no adequate therapy exists, thus earning priority review on its own merit. (In other words, to win a bonus priority review, the treatment must first get its own priority review).
See "Extension to Rare Pediatric" below for eligibility for a pediatric vouchers.

Recipients

 YearDiseaseDrugCompanyComments

1)2009MalariaCoartem (artemether/lumefantrine)NovartisThe first voucher awarded
2)2012TuberculosisSirturo (bedaquiline)Janssen (Johnson & Johnson)The first TB drug approved by FDA in decades. It is for drug-resistant TB.
3)2014Morquio A syndromeVimizim (elosulfase alfa)BioMarin PharmaceuticalThe first voucher for a rare pediatric disease.
4)2014LeishmaniasisImpavido (miltefosine)Knight TherapeuticsFor patients with visceral, mucosal and cutaneous Leishmaniasis.

Pipeline

BIO Ventures for Global Health (BVGH) reports on the pipelines for treatments for more than 20 neglected diseases. For example in 2011, there were several late-stage clinical trials for tuberculosis drugs and vaccines that could likely be eligible for vouchers. One of the companies developing a tuberculosis drug and pursuing a voucher is Sequella.

Another disease in the BVGH data set is dengue. One of the firms engaged in early research on treatments for dengue is NanoViricides which has a technology for treating infectious diseases. Because of the voucher, the team at NanoViricides is applying the technology not just to infectious diseases that affect people in relatively rich countries, such as influenza, but also to infectious diseases that affect people in developing countries, such as dengue.

Value

The price of the voucher will depend on supply and demand. On the demand side, the PRV's value derives from three factors: shifting sales earlier, longer effective patent life due to earlier entry, and competitive benefits from earlier entry relative to competitors. Top-selling treatments can yield billions in sales each year, so being approved months earlier can be worth hundreds of millions of dollars to the PRV holder (Ridley et al. 2006; Grabowski et al. 2009). The value of the voucher will depend on the drug's therapeutic class (Noor 2009).

About half of the blockbuster drugs in the 1990s received a standard review, and thus could have benefited from a PRV. There are typically about three blockbusters per year with standard review.

On the supply side, researchers at BioVentures for Global Health estimate that over the next decade, 3.5 vouchers could be awarded per year. They forecast that the next voucher could be awarded as early as 2013, possibly for a drug to treat river blindness. They forecast that the distribution across years will not be smooth with most of the vouchers awarded in the latter half of the decade.

Quotations

"But some of the highest-leverage work that government can do is to set policy and disburse funds in ways that create market incentives for business activity that improves the lives of the poor. Under a law signed by President Bush last year, any drug company that develops a new treatment for a neglected disease like malaria or TB can get priority review from the Food and Drug Administration for another product they've made. If you develop a new drug for malaria, your profitable cholesterol-lowering drug could go on the market a year earlier. This priority review could be worth hundreds of millions of dollars."
-
Bill Gates speaking at the World Economic Forum in Davos in 2008 (transcript, video, video excerpt).

History

David Ridley, Henry Grabowski, and Jeff Moe proposed the voucher. David presented the idea at several conferences, including the June 2004 meeting of the Drug Information Association, the January 2005 meeting of the American Economic Association, and the July 2005 meeting of the International Health Economics Association. In 2006, the voucher proposal was the lead article in Health Affairs. After Health Affairs published the paper, David was invited to present the idea at the National Press Club on March 7, 2006. After the press conference, Laura Blinkhorn (a reporter for Congressional Quarterly) told David that Senator Brownback (R-KS) would be interested. David and Jeff met with Senator Brownback and his staff (including Melanie Benning). Senator Brown (D-OH) and others joined Senator Brownback in sponsoring the bill. In a later issue of Health Affairs, Senator Brownback wrote, "After reading their proposal in Health Affairs, I met with Ridley and colleagues to discuss the idea further, and I subsequently drafted an amendment... Indeed, their idea is the heart of my Elimination of Neglected Diseases (END) amendment" (Brownback 2007).

Extension to Europe

Writing in The Lancet, David Ridley and Alfonso Calles Sanchez proposed extending the voucher to the European Union (Ridley and Calles Sanchez 2010). The proposed EU voucher would provide priority regulatory review through the European Medicines Agency, as well as accelerated pricing and reimbursement decisions by EU member states.

Extension to Patents

Inspired by the priority review voucher, in 2012 the United States Patent and Trademark Office (USPTO) launched a pilot program called "Patents for Humanity" to encourage businesses to apply their patented technology to addressing humanitarian challenges. Winners receive a voucher with which they may (1) move a patent re-examination proceeding to the front of the queue; (2) move a patent appeal case in front of the Board of Patent Appeals and Interferences to the front of the queue; or (3) accelerate the examination of a patent with the goal of a final decision on the application within twelve months. Application categories include medical technology, food & nutrition, clean technology, and information technology. In 2013, the USPTO awarded ten acceleration certificates.

Extension to Rare Pediatric Diseases

In 2012, the U.S. President signed into law the FDA Safety and Innovation Act which includes Section 908 the "Rare Pediatric Disease Priority Review Voucher Incentive Program". The act extends the voucher program to rare pediatric diseases, but only on a trial basis. One year after the third pediatric voucher is awarded, no other pediatric vouchers may be awarded.

The pediatric voucher program includes several changes that had been sought for the neglected-disease voucher program, but apply only to pediatric vouchers. First, the pediatric treatment developer can ask the FDA in advance for an indication of whether the disease qualifies as a rare, pediatric disease. Second, the pediatric voucher can be transferred an unlimited number of times, whereas the neglected-disease voucher can only be transferred once. Third, the pediatric voucher user needs to notify FDA 90 days prior to using the voucher, rather than 1 year for the neglected-disease voucher. Fourth, the pediatric voucher winner risks having the voucher revoked if the treatment is not marketed within a year. Fifth, the pediatric voucher winner must report to FDA about use of the pediatric treatment within five years of approval.

To be eligible for a pediatric PRV, the drug or biological product must

  • be novel
    • contain no active ingredient that has been previously approved by FDA
    • qualify for priority review (in addition to the bonus priority review)
  • treat a rare pediatric disease
    • rely on clinical data from studies examining a pediatric population and dosages of the drug intended for that population
    • not seek approval for an adult indication in the original rare pediatric disease product application

Limitations

Bibliography

News Coverage

More Information