Priority review is a designation granted by the U.S. Food and Drug Administration (FDA) to certain drug applications that, if approved, would provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Under this designation, the FDA aims to take action on an application within six months, compared to ten months under standard review.
Priority review does not change the standards for approval or the amount of evidence required to demonstrate a drug's safety and effectiveness. Instead, it allows the FDA to allocate additional resources to the evaluation of promising therapies. The designation is one of several expedited programs used by the FDA to facilitate the development and review of drugs that address unmet medical needs.
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability.
Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a fee (approximately $2.8 million) to use the voucher.
When seeking approval for a drug, manufacturers can apply to the FDA for priority review. This is granted when a drug is intended to treat a serious condition and would "provide a significant improvement in safety or effectiveness" over currently available treatments. A priority review voucher can be used when a drug does not fit these requirements, but the company wishes to expedite the review process.
In 2007, Title XI of the Food and Drug Administration Amendments Act of 2007 created the priority review voucher program for neglected tropical diseases. This was extended in 2012 by the Food and Drug Administration Safety and Innovation Act to include rare pediatric diseases. The act built upon the tropical disease system and made amendments including a shorter notification to the FDA before exercising a voucher, a designation system so that early in the drug development cycle sponsors may use the possibility of earning a voucher in their valuation of their company, a requirement of a marketing plan and reporting of marketing, and indefinite transferability of the voucher. In 2016, medical countermeasures were added to the program.
Priority review Prior to approval, each drug marketed in the United States must go through a detailed FDA review process. In 1992, under the Prescription Drug User Fee Act (PDUFA), FDA agreed to specific goals for improving the drug review time and created a two-tiered system of review times – standard review and priority review.
A priority review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. The 2002 amendments to PDUFA set a goal that a standard review of a new drug application be accomplished within a ten-month time frame. The FDA goal for completing a priority review is six months. Priority review status can apply both to drugs that are used to treat serious diseases and to drugs for less serious illnesses.
The distinction between priority and standard review times is that additional FDA attention and resources will be directed to drugs that have the potential to provide significant advances in treatment. Such advances can be demonstrated by, for example: evidence of increased effectiveness in treatment, prevention, or diagnosis of disease; elimination or substantial reduction of a treatment-limiting drug reaction; documented enhancement of patient willingness or ability to take the drug according to the required schedule and dose; or evidence of safety and effectiveness in a new subpopulation, such as children.
A request for Priority Review must be made by the drug company. It does not affect the length of the clinical trial period. FDA determines within 45 days of the drug company's request whether a priority or standard review designation will be assigned. Designation of a drug as "priority" does not alter the scientific/medical standard for approval or the quality of evidence necessary. Safety requirements for a priority review are equal to that of a standard review.
The amendment can be found on page 150 of the Food and Drug Administration Amendments Act of 2007.
Priority review voucher program
The statute authorizes the FDA to award a priority review voucher to the sponsor (manufacturer) of a newly approved drug or biologic that targets a neglected tropical disease or a rare pediatric disease. The provision applies to New Drug Applications (NDAs), Biological License Applications (BLAs) and 505(b)(2) applications. The voucher, which is transferable and can be sold, entitles the bearer to a priority review for another product.
Under current Prescription Drug User Fee Act targets, the FDA aims to complete and act upon reviews of priority drugs within six months instead of the standard ten-month review period. Actual FDA review timelines, however, can be longer than the target PDUFA review periods, particularly for new products that haven't previously been approved for any indications. Economists at Duke University, who published on this concept in 2006, estimated that priority review can cut the FDA review process from an average of 18 months down to six months, shortening by as much as a full year the time it takes for the company's drug to reach the market.
An intangible benefit of the voucher is the value created for a company if the faster review provides them "first mover advantage," allowing the voucher holder's product to be introduced ahead of a similar, competing product. By taking advantage of existing market forces, patients in the developing world can have faster access to lifesaving products that may not otherwise be developed. And sponsors of neglected disease drugs can be rewarded for their innovations
Sponsors must inform the FDA of their intention to use a priority review voucher 90 days before submission. Before adding Ebola to the FDA Priority Review Voucher Program Act in 2014, this requirement was 365 days, which was a hindrance to the process of speedy review, as companies do not typically determine when drugs will be submitted until the results of safety studies are available.
Companies may also sell vouchers to other drug companies. Thus far, priority review vouchers have sold for $50–350 million.
Cost Companies that use the voucher will be required to pay a supplemental priority review user fee to ensure that the FDA can recoup the costs incurred by the agency for the faster review, in addition to the fee for standard review of drugs. The additional user fee also aims to ensure that the new program will not slow the progress of other products awaiting FDA review. The cost has decreased dramatically from over $5 million in 2012. For the fiscal year 2018, this fee is $2.8 million.
Uses of the priority review program , fourteen priority review vouchers have been awarded, four for tropical diseases, and ten for rare pediatric diseases. The first priority review voucher was awarded in 2009 to Novartis for its approval of Coartem. The next voucher was not awarded until 2012. +Vouchers have been awarded for the following:ProgramDrugIndicationCompanyYearTropical diseaseCoartemMalariaNovartisTropical diseaseSirturoMulti-drug-resistant tuberculosisJanssen PharmaceuticaRare pediatric diseaseVimizimMorquio syndromeBioMarin PharmaceuticalTropical diseaseImpavidoLeishmaniasisKnight TherapeuticsRare pediatricUnituxinNeuroblastomaUnited TherapeuticsRare pediatricCholbamZellweger syndromeAsklepion PharmaceuticalsRare pediatricXuridenOrotic aciduriaWellstat TherapeuticsRare pediatricStrensiqHypophosphatasiaAlexion PharmaceuticalsRare pediatricKanumaLysosomal acid lipase deficiencyAlexionTropical diseaseVaxchoraCholera preventionPaxVaxRare pediatricExondys 51Duchenne muscular dystrophySarepta TherapeuticsRare pediatricSpinrazaSpinal muscular atrophyIonis PharmaceuticalsRare pediatricEmflazaDuchenne muscular dystrophyMarathon PharmaceuticalsRare pediatricBrineuraBatten diseaseBioMarin PharmaceuticalTropical diseasebenznidazoleChagas diseaseChemoResearchRare pediatricKymriahB-ALLNovartisRare pediatricMepseviiMucopolysaccharidosis type VIIUltragenyxRare pediatricLuxturnaRPE65-mutated retinal dystrophySpark TherapeuticsRare pediatricCrysvitaX-linked hypophosphatemiaUltragenyx 2020Tropical diseaseMoxidectinOnchocerciasisMedicines Development for Global HealthRare pediatricEpidiolexLennox–Gastaut syndromeGW PharmaceuticalsMaterial threat countermeasureTpoxxSmallpoxSIGA TechnologiesTropical diseaseKrintafelMalariaGlaxoSmithKlineRare pediatricRevcoviAdenosine deaminase-SCIDLeadiant BiosciencesRare pediatricGamifantHaemophagocytic lymphohistiocytosisNovimmuneTropical diseaseEgatenFascioliasisNovartisRare pediatricSymdekoF508del cystic fibrosisVertex PharmaceuticalsTropical diseaseDengvaxiaDengueSanofiRare pediatricZolgensmaSpinal muscular atrophyAveXisTropical diseasePretomanidTuberculosisTB AllianceMaterial threat countermeasureJynneosSmallpoxBavarian NordicRare pediatricTrikaftaCystic fibrosisVertex PharmaceuticalsTropical diseaseErveboEbolaMerck & Co. Capmatinib MET exon 14 skipping mutated non-small cell lung cancer Novartis KTE-X19 Mantle cell lymphoma Kite Pharma Selpercatinib RET fusion-positive non-small cell lung cancer Eli Lilly elantamab mafodotin Multiple myeloma GlaxoSmithKline Cedazuridine and decitabine Chronic myelomonocytic leukemia Astex Pharmaceuticals Rare pediatric Vyondys 53 Duchenne muscular dystrophy Sarepta Therapeutics 2020 Rare pediatric Oxlumo Primary hyperoxaluria type 1 Alnylam Pharmaceuticals 2020 Rare pediatric Rethymic Congenital athymia Enzyvant Therapeutics 2021
Extensions Extension to rare pediatric diseases
In 2012, President Obama signed into law the FDA Safety and Innovation Act which includes Section 908, the "Rare Pediatric Disease Priority Review Voucher Incentive Program". Section 529 extends the voucher program to rare pediatric diseases, but only on a trial basis. After the third voucher is awarded, the Comptroller General of the United States is to conduct a study on the effectiveness of the pediatric priority review voucher program.
The pediatric voucher program includes changes to the voucher program. First, the pediatric treatment developer can ask the FDA in advance for an indication of whether the disease qualifies as a rare, pediatric disease.
The awardee must market the drug within 365 days of approval, or the voucher may be revoked. Within five years of approval, the manufacturer must submit a report containing information on the estimated population in the United States suffering from the rare pediatric disease, the estimated demand in the United States for such rare pediatric disease product, and the actual amount of such rare pediatric disease product distributed in the United States.
The Advancing Hope Act of 2016 reauthorized the program until December 31, 2016 and instructed the GAO to compile a report on the effectiveness of the program.
In February 2026, the FDA announced that the priority review voucher program for rare pediatric diseases would end in September 2029.
Extension to ebola virus