Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers

ExplainersExplainersRegulatory NewsRegulatory News | 25 February 2020 |  By 

Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers (PRVs) which allow its recipient to expedite the review of any one of its new drug products. What are these vouchers, why is FDA issuing them and what benefit might they have for society? Find out in our latest Regulatory Explainer on the Priority Review Voucher system.

Last updated on 25 February 2020 to include:

  • Vifor Pharma said on 17 February that it purchased a PRV, and said on 25 February the PRV was purchased from Sarepta Therapeutics. The company said in an SEC filing that the PRV will be used to speed the review of vadadustat, which is a treatment for anemia due to chronic kidney disease. Vifor purchase the PRV for $111 million, according to Sarepta.
  • According to a new report from GAO on PRVs, Gilead was the unknown purchaser of Ultragenyx's second PRV for about $80 million. Gilead said last month that it has used the voucher to speed the review of filgotinib, an investigational, oral, selective JAK1 inhibitor for the treatment of adults who are living with moderate-to-severe rheumatoid arthritis (RA). 
  • Novartis confirmed to Focus that it will use two of its PRVs to speed the approvals of ofatumumab to treat multiple sclerosis and Cosentyx (secukinumab) for axial spondyloarthritis. Novartis said: "FDA decisions for both are expected in the first half of 2020."
  • Merck won a PRV for the approval of its Ebola vaccine.
  • Bavarian Nordic sold its PRV for $95 million.
  • Sarepta Pharmaceuticals received a PRV for the accelerated approval of Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. This is Sarepta's second PRV (the first was sold to Gilead).
  • How Jazz Pharmaceuticals plans to use the PRV it purchased from Spark Therapeutics.


I Don't Know Much About Drug Development - What are we Talking About Here?

FDA's priority review voucher (PRV) programs are about drug development, and specifically the time and cost of drug development and the conditions a new drug is intended to treat.

Developing a new drug is a costly and time-intensive affair. The cost of developing a new drug can range from the tens of millions to billions of dollars—and that assumes the development program is successful. Only about one in 10 drug products which enter phase I testing are ever approved in the US. For some hard-to-treat indications, like conditions affecting the central nervous system, success rates can be even lower.

The time it takes to obtain that approval can also be a major factor. Companies often take years, sometimes decades, to develop a drug before sending it to regulatory officials for review. Once FDA begins its review, it takes more than a year on average for regulators to approve a drug.

Drug Development Failure and Success

Why Does the Time and Cost of Drug Development Matter?

Assume, for a minute, that you're a pharmaceutical or biopharmaceutical company hoping to develop a drug. The time and cost it takes to develop a drug means you're going to need funding, either from private investors or public shareholders. In return for their initial investment, investors usually want a return on their investment. Most companies generate this return by selling their approved products, either directly to consumers or to payers like health insurers and government healthcare systems.

In other words, if a company is looking to make the most money it can, it's unlikely to be able to do so by developing products for rare or neglected diseases.

In addition, developing drugs for rare and neglected diseases can sometimes be disproportionately difficult. Most prominent diseases in western countries benefit from research conducted by government entities like the US National Institutes of Health (NIH) or public health foundations. Their research helps expedite drug development by better helping companies to understand the pathology of the diseases they hope to develop treatments or cures for. Companies hoping to develop treatments for rare or neglected diseases often have to conduct more, if not all, of this early-stage research.

Are There Ways to Incentivize Drug Development to Benefit Specific Diseases?

Since the 1984 passage of the Orphan Drug Act, the US has begun to recognize that incentives can help spur development of new drugs for historically underserved conditions. Under the Orphan Drug Act, companies are eligible for several extra years of marketing exclusivity, during which time FDA is not permitted to approve a generic, for getting a drug for a "rare" disease approved. This is meant to give companies an added incentive to produce drugs intended for rare diseases, as it allows the company extra time to recoup its development costs and likely turn a profit as well.

To date, these incentives have proven wildly popular. Prior to the passage of the Orphan Drug Act, few medicines were approved to treat rare conditions—defined as affecting fewer than 200,000 persons in the US. Just three "orphan" drugs were approved in 1984, for example. In 2014, 49 orphan drug products were approved.

Approved Orphan Products

Not all drugs are reviewed by FDA in the same way. Most drugs are reviewed by FDA under "standard" review times, meaning FDA has 10 months to review each product before it is supposed to render a decision.

However, for certain drugs, FDA accelerates its regulatory review in the hopes of getting products to market more quickly.

Drugs intended for use in "serious conditions," or which "demonstrate the potential to be a significant improvement in safety or effectiveness," are permitted to be reviewed under FDA's Priority Review Designation pathway. As explained in an FDA guidance document, the pathway allows FDA to review a drug in just six months instead of the standard 10.

For companies and patients alike, FDA's priority review process can be extremely beneficial. For patients with serious conditions, the expedited review means they have access to a potentially life-saving or -changing treatment. For companies, it means they can market their product more quickly and begin recouping their often considerable development costs.

What Does all This Have to do with a Priority Review Voucher?

FDA's priority review vouchers (there are three types) are incentives meant to spur the development of new treatments for diseases that would otherwise not attract development interest from companies due to the cost of development and the lack of market opportunities.

To do this, companies are given a special voucher which allows them to have any one of their drugs reviewed under FDA's priority review system.

Priority Voucher System Figure

That Sounds Interesting. What is the History of the PRV Programs?

The priority review voucher system was first proposed in a March 2006 paper published in the journal Health Affairs, and written by Duke University's David Ridley, Henry Grabowski and Jeffery Moe.

In their paper, "Developing Drugs for Developing Countries," they proposed the creation of a priority voucher system specifically targeted at neglected tropical diseases, which the authors argued lacked sufficient development incentives to produce.

This "prize," as they called it, could be used in one of two ways: Either it could be redeemed by its recipient, or it could be sold to another company, which might want to have its own drug reviewed in a six-month timeframe.

The initiative later caught the attention of Sen. Sam Brownback (R-KS), who worked to introduce the first voucher system into a bill known as the Food and Drug Administration Revitalization Act, which was eventually renamed and passed as the Food and Drug Administration Amendments Act (FDAAA) of 2007.

What did the FDAAA do for the Priority Voucher System?

Section 1102 of FDAAA, "Priority Review to Encourage Treatments for Tropical Diseases," created the Neglected Tropical Disease Priority Review Voucher system.

As explained in the law and subsequent guidance documents, any new drug intended to treat the following list of tropical diseases is eligible to receive a transferrable voucher for priority review:

Eligible Diseases Under the Tropical Disease Priority Review Voucher System
Malaria Blinding trachoma
Buruli Ulcer Cholera
Dengue/Dengue haemorrhagic fever Dracunculiasis (guinea-worm disease)
Fascioliasis Human African trypanosomiasis
Leishmaniasis Leprosy
Lymphatic filariasis Onchocerciasis
Schistosomiasis Soil transmitted helminthiasis
Yaws Tuberculosis
Added by Congress
Cueva virus Ebola virus
Marburg virus Zika virus
Added by FDA Order
Chagas Neurocysticercosis
Chikungunya Virus Disease Lassa Fever
Rabies Cryptococcal Meningitis

FDAAA also gave FDA the regulatory authority to add "any other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations."

In December 2014, legislators added several new viruses to the list of diseases eligible for a voucher including Cuevavirus, five strains of Ebolavirus and two strains of Marburgvirus (more on that below). In August 2015, FDA exercised its authority to add to the list of eligible neglected tropical diseases for the first time, adding, Chagas disease and neurocysticercosis to the list.

Are There any Limitations on How Tropical Disease Priority Review Vouchers Can be Used?

Yes, though subsequent legislation eliminated some of those limitations.

For example, FDAAA originally required a drug sponsor to alert FDA at least 365 days before it planned to apply a voucher to the submission of a new product. That limited the usefulness of the voucher for many companies, as it slowed down the drug development process. Some companies do not know if they plan to file a new drug application until they receive the results of late-stage (Phase III) clinical testing.

Another limitation contained in FDAAA was the number of times a voucher could be sold: only once. While a 2008 guidance by FDA allowed companies to buy and sell options on vouchers, once a company purchased a voucher, it could not be resold—a major risk for the purchaser. For example, if a company's sole drug product failed before it could use the voucher, it would have been effectively useless.

A third limitation (if you can call it that) is the cost to redeem the voucher. Under FDAAA, FDA is permitted to charge a company redeeming a voucher the added cost of conducting a priority review in addition to the normal new drug user fee.

Since the priority voucher user fee was first established in FY 2011, the cost to use it has ranged as high as $5.28 million (2012) and as low as $2.32 million (2014).

Tropical Disease Priority Review Voucher User Fee
Fiscal Year Voucher Fee
FY 2011 $4,582,000
FY 2012 $5,280,000
FY 2013 $3,559,000
FY 2014 $2,325,000
FY 2015 $2,562,000
FY 2016 $2,727,000
FY 2017 $2,706,000
FY 2018 $2,830,579
FY 2019 $2,457,140
FY 2020 $2,167,116

What Other Types of Vouchers Exist?

So far, two other types of voucher exists: rare pediatric disease priority review voucher and medical countermeasure priority review vouchers.

The rare pediatric voucher was created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), and specifically targets the need for additional therapies for rare pediatric subsets of other diseases.

Section 908 of FDASIA defines a "rare pediatric disease" as one which "primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children and adolescents," and is a rare disease according to federal statute (200,000 persons in the US or fewer).

The rare pediatric voucher system is closely modeled off the tropical disease voucher system, with several exceptions. Perhaps the most notable difference at the time of the pediatric voucher's initial passing was its relative ease of use. Unlike the tropical voucher, which required FDA to be notified 365 days prior to its use, the rare pediatric voucher could be used just 90 days prior to its use.

Another key difference: The rare pediatric voucher could be transferred (i.e. sold) an unlimited number of times, unlike the tropical disease voucher which could only be sold once.

However, as of December 2014, both of these differences have been erased under the Adding Ebola to the FDA Priority Review Voucher Program Act, which eliminated the disparity between the two vouchers. Tropical vouchers may now be redeemed in just 90 days, and may be resold an unlimited number of times.

In 2016, the 21st Century Cures Act was signed into law creating a priority review voucher program for medical countermeasures.

Rare Pediatric Disease Priority Review Voucher User Fee
Fiscal Year Voucher Fee
FY 2014 $2,325,000
FY 2015 $2,562,000
FY 2016 $2,727,000
FY 2017 $2,706,000
FY 2018  $2,830,579
FY 2019 $2,457,140
FY 2020 $2,167,116
Medical Countermeasure Priority Review Voucher User Fee
Fiscal Year Voucher Fee
FY 2018 $2,830,579
FY 2019 $2,457,140
FY 2020 $2,167,116

How Many Vouchers Have Been Awarded so Far?

Priority Review Vouchers Awarded to Date
Date Voucher Awarded Voucher Type Company Voucher Awarded to:
2009 Tropical Disease Novartis
2012 Tropical Disease Janssen
2014 Rare Pediatric Disease BioMarin
2014 Tropical Disease Knight Therapeutics
2015 Rare Pediatric Disease United Therapeutics
2015 Rare Pediatric Disease Asklepion Pharmaceuticals
2015 Rare Pediatric Disease Wellstat Therapeutics
2015 Rare Pediatric Disease Alexion Pharmaceuticals
2015 Rare Pediatric Disease Alexion Pharmaceuticals
2016  Tropical Disease  PaxVax Bermuda
2016 Rare Pediatric Disease Sarepta Therapeutics 
2016 Rare Pediatric Disease  Ionis Pharmaceuticals 
2017  Rare Pediatric Disease  Marathon Pharmaceuticals
2017 Rare Pediatric Disease  BioMarin
2017  Tropical Disease  Chemo Research, S.L. 
2017 Rare Pediatric Disease  Novartis 
2017  Rare Pediatric Disease  Ultragenyx Pharmaceutical 
2017  Rare Pediatric Disease  Spark Therapeutics 
2018 Rare Pediatric Disease Ultragenyx
2018 Tropical Disease Medicines Development
2018 Rare Pediatric Disease GW Pharma
2018 Material Threat Medical Countermeasure SIGA Technologies
2018 Tropical Disease GlaxoSmithKline
2018 Rare Pediatric Disease Leadiant Bioscience Inc
2018 Rare Pediatric Disease Sobi and Novimmune
2019 Tropical Disease Novartis
2019 Rare Pediatric Disease Vertex
2019 Rare Pediatric Disease Alexion
2019 Tropical Disease Sanofi
2019 Rare Pediatric Disease Novartis
2019 Tropical Disease Global Alliance for TB Drug Development
2019 Material Threat Medical Countermeasure Bavarian Nordic
2019 Rare Pediatric Disease Vertex Pharmaceuticals
2019 Rare Pediatric Disease Sarepta Pharmaceuticals
2019 Tropical Disease Merck

Have any Vouchers Been Used or Sold so Far?

Just as interesting as who obtained the vouchers is what has happened to those vouchers.

Status of Existing Priority Review Vouchers
Company Voucher Type Status of Voucher
Novartis Tropical Disease Unsuccessfully used by Novartis to accelerate the review of its Biologics Licensing Application (BLA) for Ilaris (canakinumab).
Janssen Tropical Disease Successfully used to accelerate the approval of Tremfya (guselkumab) to treat plaque psoriasis.
BioMarin Rare Pediatric Disease Sold to Sanofi and Regeneron for $67 million. Used successfully to speed the approval of Praluent.
Knight Tropical Disease Sold to Gilead Sciences for $125 million. Gilead announced it had used the voucher in support of its NDA filing for its HIV drug Odefsey. FDA approved the drug in six months on 1 March 2016.
United Therapeutics Rare Pediatric Disease Sold to AbbVie for $350 million in August 2015. AbbVie has decided to use it to speed the review for upadacitinib for the treatment of adult patients with moderate to severe rheumatoid arthritis. Upadacitinib was approved by FDA on 8/16/19.
Asklepion Pharma Rare Pediatric Disease Transfered to Retrophin under an existing agreement. Sold to Sanofi for $245 million in May 2015. In February 2016 Sanofi redeemed the voucher to support its NDA for a new type 2 diabetes drug.
Wellstat Therapeutics  Rare Pediatric Disease  Transferred to AstraZeneca under an existing agreement. Unused by AstraZeneca.
PaxVax Bermuda Tropical Disease  Unused. Likely sold to Gilead for ~$200 million in 2016. Used to speed FDA's review of an sNDA for Descovy (emtricitabine 200 mg and tenofovir alafenamide 25 mg tablets) for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 infection among individuals who are HIV-negative and at risk for HIV.
Alexion Pharmaceuticals Rare Pediatric Disease Used to speed the review of ALXN1210, which is a treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and which won approval as Ultomiris (ravulizumab-cwvz) in December 2018.
Alexion Pharmaceuticals  Rare Pediatric Disease Unused
Sarepta Therapeutics  Rare Pediatric Disease Sold to Gilead for $125 million in February 2017. Used by Gilead to speed FDA's review of its new HIV treatment, which was approved in February 2018.
Ionis Pharmaceuticals Rare Pediatric Disease  Unused.
Marathon Pharmaceuticals  Rare Pediatric Disease  Unused.
BioMarin  Rare Pediatric Disease  Sold to an undisclosed party for $125 million in November 2017
Chemo Research SL  Tropical Disease  Unused. 
Novartis  Rare Pediatric Disease  Used to speed FDA's review of Novartis' BLA for brolucizumab (RTH258) for the treatment of wet age-related macular degeneration (AMD), also known as neovascular AMD. FDA approved the biologic in October 2019.
Ultragenyx  Rare Pediatric Disease  Sold to Novartis for $130 million in December 2017. Used to speed the review of siponimod for the treatment of secondary progressive multiple sclerosis (SPMS) in adults, which won FDA approval in March 2019.
Spark Therapeutics  Rare Pediatric Disease  Sold in April 2018 for $110 million to Jazz Therapeutics. Jazz said on 5 November 2019 that it intends to use the PRV to speed the review of its NDA for a treatment of cataplexy and excessive daytime sleepiness (EDS) in adults with narcolepsy.
Ultragenyx Rare Pediatric Disease Sold to an undisclosed party for $80.6 million. According to the GAO, the purchaser was Gilead. Gilead said in December 2019 that it has used a voucher to speed the review of filgotinib, an investigational, oral, selective JAK1 inhibitor for the treatment of adults who are living with moderate-to-severe rheumatoid arthritis (RA). 
Medicines Development Tropical Disease Sold to Novo Nordisk for an undisclosed sum. Novo said it used the PRV to speed the review of oral semaglutide, which was filed in March 2019. FDA approved the drug in September 2019.
GW Pharma Rare Pediatric Disease Sold to Biohaven Pharmaceutical Holding Company for $105 million on 18 March 2019. Biohaven said it will use the PRV to speed its potential migraine drug Rimegepant Zydis.
SIGA Technologies Material Threat Medical Countermeasure Sold to Eli Lilly for $80 million on 1 November 2018.
GlaxoSmithKline Tropical Disease ViiV Healthcare used the PRV for a New Drug Application for a single-tablet, two-drug regimen of dolutegravir and lamivudine for the treatment of HIV-1 infection. FDA approval on 8 April 2019.
Leadiant Bioscience Inc Rare Pediatric Disease Unused
Sobi and Novimmune Rare Pediatric Disease Purchased by AstraZeneca for $95 million.
Novartis Tropical Disease Used to speed the review of ofatumumab to treat multiple sclerosis, Novartis said.
Vertex Rare Pediatric Disease Unused
Alexion Rare Pediatric Disease Unused
Sanofi Tropical Disease Unused
Novartis Rare Pediatric Disease Used to speed the review of Cosentyx (secukinumab) for axial spondyloarthritis, Novartis said.
Global Alliance for TB Drug Development  Tropical Disease Unused
Bavarian Nordic Material Threat
Medical Countermeasure
The company sold its PRV for $95 million in December 2019 to an undisclosed buyer.
Vertex Pharmaceuticals Rare Pediatric Disease Unused
Sarepta Pharmaceuticals Rare Pediatric Disease Purchased by Vifor Pharma for about $100 million to speed the review of Akebia Therapeutics' vadadustat, which is intended to be a treatment for anemia due to chronic kidney disease.
Merck Tropical Disease Unused

Perhaps the most notable aspect of the vouchers so far is the price they have been able to command. BioMarin's voucher, the first ever to be sold, was purchased for $67 million. Several months later, Gilead Sciences purchased Knight Therapeutics' voucher for $125 million. In May 2015, Sanofi purchased yet another voucher for a record-setting $245 million, and in August 2015, AbbVie shattered that record by paying $350 million for a voucher originally awarded to United Therapeutics. However, the selling price for PRVs has continued to slide, with the prices hovering around $100 million.

In June 2017, GlaxoSmithKline (GSK) and ViiV announced they used a priority review voucher bought by GSK for $130 million from an undisclosed party to speed the review of ViiV's two-drug HIV maintenance therapy, Juluca, which won approval in 2017.

In its Q3 2017 earnings call, Teva Pharmaceuticals disclosed that it used a priority review voucher bought for $150 million from an unnamed party to get a priority review for its investigational migraine biologic fremanezumab. This rare pediatric PRV was used successfully.

In early 2019, Doctors without Borders took issue with Novartis winning its third priority review voucher: "This is the second time Novartis has gotten a PRV for an old medicine," MSF said. Meanwhile, changes to the tropical disease PRV in FDARA should have prevented Novartis from winning the PRV, but FDA says those changes "did not apply to Novartis’s application."

Are There any Risks to Using a Priority Review Voucher?

There are several risks inherent to using a PRV.

The first and most obvious is that FDA is under no obligation to approve a product using a voucher. Both FDASIA and FDAAA stipulate that FDA only needs to come to a decision within six months. As Novartis proved in the first-ever use of a priority review voucher, FDA will not necessarily approve a product just because its sponsor used a voucher.

Priority review will likely accelerate the approval of a good drug to market, but it will not save a bad drug from being rejected. And for companies that are unsuccessful in their use of a priority review voucher, the cost of failure can be especially high due to the cost to procure and use the voucher.

New Drugs Vaccines Rate of PRVs

Another little-known limitation is that FDA claims it isn't actually required to review all drugs submitted using a voucher in six months. As explained in FDA's tropical disease voucher guidance, sponsors are not "guaranteed a six-month review" of their new drug product.

"We believe the intent of this section is that drugs for which priority review vouchers are used should be treated as if they were any other priority review drug. Therefore, these applications would be placed in the priority review group. The Agency has committed to a goal of completing 90 percent of priority reviews within 6 months."

The agency's rare pediatric disease voucher guidance contains similar language:

"Although FDA's goal is to take action on the application within 6 months after the 60 day filing period for an application involving a new molecular entity or within 6 months after the date of receipt of an application not involving a new molecular entity, this timeframe is not guaranteed. Note that "take action" in this context means that FDA aims to complete its review of the filed application and issue an approval or complete response letter within this timeframe; it does not mean that the application will be approved within this timeframe."

Have There Been any Changes to the Priority Review Voucher Program?

In 2014, US legislators made a major change to the tropical disease priority review voucher program with the intent of providing additional incentives to fight the Ebola virus and several other related viruses.

The changes were spurred in part by an op-ed by David Ridley, one of the fathers of the original voucher plan, in which he proposed adding "other infectious diseases" to the list of diseases eligible to receive a voucher. While FDA was already permitted to add additional diseases to its list through the regulatory process, legislators said they feared that process would take too long and would not be completed until the Ebola outbreak had potentially ended.

On 16 December 2014, President Barack Obama signed into law the Adding Ebola to the FDA Priority Review Voucher Program Act. The law made several subtle but important changes to the neglected tropical disease voucher program.

First, the law permitted the tropical vouchers to be used just 90 days after a company notified FDA of its intent to file a new drug. Previously, companies were required to notify FDA 365 days in advance.

Second, the law allows tropical vouchers to be resold an unlimited number of times. FDA had previously interpreted the statute to allow only one sale of the voucher.

Third, the law adds " filovirus"—a category of diseases which include five strains of Ebolavirus, two strains of Marburgvirus and the lone strain of Cuevavirus, known as Lloviu virus.

Genus name Virus name
Cuevavirus Lloviu virus
Ebolavirus Bundibugyo virus
Reston virus
Sudan virus
Taï Forest virus
Ebola virus
Marburgvirus Marburg virus
Ravn virus

Fourth, the law permits FDA to add any new viruses to the list of tropical diseases eligible for a voucher "by order" instead of "by regulation." This change will permit FDA to make changes much more quickly, and to avoid the lengthy notice-and-comment provisions of federal regulation.

Are There any Criticisms of the Priority Review Voucher Program?

There have been several major criticisms levied against the priority review voucher program.

First, there have been some questions about whether the voucher system is actually incentivizing development, or is rather just acting as a giveaway to companies which would have developed the new products anyways. In a January 2015 Public Library of Science article, authors Bernard Pécoul and Manica Balasegaram looked at the tropical disease voucher obtained by Knight Therapeutics, and found that other companies and entities had done much of the development work to get the company's tropical disease drug approved.

The group Medicines san Frontiers has levied similar claims against the voucher program, and argued that companies should be required to show they conducted the research necessary to obtain approval for the drug.

Other experts, including the Drugs for Neglected Disease Initiative's (DNDi) Rachel Cohen, have noted that the voucher systems actually do not require a company to sell a drug. In other cases, companies might seek regulatory approval for a tropical disease drug which is marketed outside the US in the hopes of obtaining a voucher. This should be banned, one of the founders of the voucher program, David Ridley, has said.

The voucher program also doesn't require the drugs provided through the program to be affordable. David Ridley has proposed requiring companies to report on the affordability of drugs that receive a voucher from FDA, and called on companies to voluntarily ensure that the drugs are affordable to those who need them.

Then there's a more fundamental question: Would these drugs have been developed were it not for the possibility of obtaining a voucher? While defenders of the voucher program have said the existence of the vouchers provides a powerful incentive for companies to consider developing new drugs, there's little evidence to show the drugs wouldn't have been developed anyway.

Unfortunately, it's not yet possible to determine whether FDA's priority review voucher programs are having an effect. As we stated at the outset of this article, it often takes a decade to develop a new drug, and any new development catalyzed by the creation of the voucher system may not be evident for several more years. One study recently published in the Public Library of Science estimated that FDA will issue between five and six new vouchers between 2016 and 2018.

Another criticism, levied by Aaron Kesselheim in the New England Journal of Medicine in 2008, is that while the voucher program is intended to be used only for new drug ingredients, it ignores the potential utility of new innovations.

"As a result, an effective novel antimalarial drug that degrades in the heat and must be taken six times a day would earn its sponsor a voucher, but no voucher would be granted for a follow-on formulation that might be more useful in resource-poor settings," he wrote.

Kesselheim also noted a broader concern, raised by other experts as well, that the priority review process is improper to use for drugs lacking an urgent need. "The voucher program will allow drugs for which there is little or no clinical urgency to be subject to accelerated deadlines and may lead to approval of products without adequate consideration by the FDA," he wrote.

Do Any Other Countries Have a Voucher System in Place?

No, although a September 2010 article in The Lancet—again authored by David Ridley—proposed the creation of a European voucher system which would accelerate the review of medicines as well as pricing and reimbursement decisions. The proposal has not yet been adopted.

Are There Other Diseases FDA Might Add to its List of Eligible Neglected Tropical Diseases?

The World Health Organization (WHO) recently called on governments to "increase investment to tackle neglected tropical diseases."

In August 2015, FDA exercised its ability to add to the list for the first time, adding Chagas disease and neurocysticercosis to the list of eligible neglected tropical diseases.

Many of the diseases on its list are already contained on FDA's list of neglected tropical diseases, but several are not:

Which Diseases Aren't on FDA's List of Neglected Tropical Diseases?
Rabies Echinococcosis
Taeniasis Foodborne trematodiases

And with the inclusion of the Ebolavirus, Chagas and other diseases to the list, it is possible Congress and FDA may soon eye other diseases for inclusion on the list as well, including the Middle East respiratory syndrome coronavirus (MERS-CoV), Chikungunya, the avian flu virus and other diseases thought to present a security risk to the US.

Are There any Potential Changes Coming to the Priority Review Voucher System?

The Rare Pediatric Disease PRV program was re-authorized by Congress in the 21st Century Cures legislation, extending the program through 2020, and also creating a new PRV program for medical countermeasures. 

Where Can I Learn More About Priority Review Vouchers?

You should read FDA's two related guidance documents:

Guidance for Industry: Tropical Disease Priority Review Vouchers

Guidance for Industry: Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry


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