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Priority Review Voucher

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May 2015: A priority review voucher sold for $245 million from Retrophin to Sanofi.

March 2015: The Global Health Investment Fund announced that it is providing $10 million to non-profit drug developer Medicines Development of Australia to complete registration of a drug for river blindness. If the drug is approved and wins a priority review voucher from the Food and Drug Administration, the Fund will share in some of the rewards from sale of the voucher. (In 2014, a voucher sold for $125 million.) The fund can then use some of the returns to re-invest in global health.

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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 or rare pediatric disease receives a voucher for priority review from the 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 ($2,562,000 in fiscal year 2015).

The PRV is transferable.

Eligibility

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

  1. Treat one of the following diseases:
    • Blinding trachoma
    • Buruli Ulcer
    • Cholera
    • Dengue
    • Dracunculiasis
    • Fascioliasis
    • Filoviruses (including Ebola) (added in 2014)
    • Human African trypanosomiasis
    • Leishmaniasis
    • Leprosy
    • Lymphatic filariasis
    • Malaria
    • Onchocerciasis
    • Schistosomiasis
    • Soil transmitted helminthiasis
    • Tuberculosis
    • Yaws
    • A rare pediatric disease (added in 2012)
  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

 YearDiseaseDrugCompanyCommentsStatus

1)2009MalariaCoartem (artemether/lumefantrine)NovartisThe first voucher awardedUsed by Novartis
2)2012TuberculosisSirturo (bedaquiline)Janssen (JNJ)The first TB drug approved by FDA in decades.Unused
3)2014Morquio A syndromeVimizim (elosulfase alfa)BioMarinThe first voucher for a rare pediatric disease.Sold for $67.5 million
4)2014LeishmaniasisImpavido (miltefosine)KnightFor patients with visceral, mucosal and cutaneous Leishmaniasis.Sold for $125 million
5)2015High-risk neuroblastomaUnituxin (dinutuximab)United TherapeuticsFor a type of cancer that most often occurs in young children.Unused
6)2015Rare bile acid synthesis disordersCholbamAsklepion PharmaceuticalsFDA press release.Sold for $245 million

Pipeline

BIO Ventures for Global Health (BVGH) reports on the pipelines for treatments for neglected diseases such as Ebola, malaria, river blindness, and tuberculosis. For example, there are several late-stage clinical trials for tuberculosis drugs and vaccines that could be eligible for vouchers. One of the companies developing a tuberculosis drug and pursuing a voucher is Sequella.

Another neglected disease 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.

In 2014, BioMarin sold its voucher for $67.5 million to Sanofi/Regeneron and Knight sold its voucher for $125 million to Gilead. In 2015, Retrophin sold a voucher for $245 to Sanofi.

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."
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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 on a trial basis.

In 2014, the U.S. President signed into lawAdding Ebola to the FDA Priority Review Voucher Program Act which makes (1) adds filoviruses (including Ebola) to the list of eligible diseases, (2) allows multiple transfers of the voucher, and (3) reduces the notification period for FDA from one year to 90 days.

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
Tropical Rare Pediatric
Legislative authority FDAAA (2007) (PDUFA IV) FDASIA (2012) (PDUFA V)
Voucher winner Number of vouchers to be issued Unlimited Pilot expires one year after third pediatric voucher awarded
Eligibility 17 neglected diseases (may be extended by Secretary of HHS) Rare, pediatric diseases (may ask FDA in advance for an indication of whether disease qualifies as a rare, pediatric disease)
Obligations for voucher winner None (other than public pressure) Winner must market drug within a year and report to FDA about use of drug within five years
Voucher user Notification 90 days prior to voucher redemption
Transferability Unlimited
Additional user fee $2,562,000 in fiscal year 2015
Voucher expiration date None

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