The Butterfly Effect: How Pediatric PRVs Shape Innovation and Access to Medicines

Executive Summary

The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program was extended on February 4, 2026, under the Mikaela Naylon Give Kids a Chance Act under the Consolidated Appropriations Act of 2026, now expiring on September 30, 2029.^1,2 The program had been reauthorized twice previously, in 2016 and 2020, both times with bipartisan support.³

The RPD PRV program was created to address a persistent market failure: the lack of economic incentive for companies to develop therapies for rare pediatric diseases. Rare diseases are defined as diseases or conditions that affect fewer than 200,000 people in the United States, although many have far smaller patient populations. There are an estimated 7,000-10,000 rare diseases affecting roughly 30 million Americans, with an estimated 50% of them impacting pediatrics and more than 95% of which have no approved treatments.^4,5,6 Despite this scale, small patient populations for individual rare diseases and high development costs historically discouraged investment.

RPD PRVs can be redeemed for faster review times or sold to other drug sponsors. Their transferability made them an important source of funding for biotech stakeholders that focus on rare disease development. To date, RPD PRVs have been awarded to 71 therapies, resulting in more than $4.4 billion of non-dilutive capital for their drug sponsors._⁷ The data and case studies in this paper demonstrate the importance of PRVs in driving innovation and access to medicine for pediatric rare disease patients.

Policymakers have historically relied on temporary reauthorizations rather than providing permanent statutory certainty for the program. Although the RPD PRV program was recently extended, the three and a half years extension is not enough for drug sponsors to plan their pipelines around. On average, drug development takes 10 years, making it impractical to initiate new programs when the period of legislative certainty spans only three to five years.^8,9

In addition, it takes 2-3 years of advocacy by rare disease, academic and biotech communities each time to get the RPD PRV program extended.¹⁰ Given the program’s short extension windows and the recurring need for legislative action, the RPD PRV program in its current form is no longer an ideal mechanism for incentivizing rare pediatric drug innovation. The independent analysis presented in this paper supports establishing permanency for the PRV program to provide certainty, encourage sustained investment, and ensure continued innovation in pediatric rare disease.

Research Methodology

This analysis draws on publicly available data from the Federal Register, relevant Securities and Exchange Commission (SEC) filings, prior reports from the U.S. Government Accountability Office (GAO) and the National Organization for Rare Disorders (NORD), peer-reviewed academic publications, news reporting, company press releases, and first-hand interviews with rare disease advocates and biotech executives.

From publicly available regulatory and financial records, RTW Institute compiled a comprehensive dataset of all PRVs awarded since inception in 2009, tracking each voucher’s award date, associated drug and sponsor, and any publicly available transaction information.

To complement the data analysis, the Institute team incorporated qualitative data to better contextualize the real-world impact of the RDP PRV program. Interviews were conducted with key stakeholders representing both industry and patient advocacy perspectives, including Terry Pirovolakis (CEO and founder of Elpida Therapeutics and rare disease parent), Amber Freed (CEO of SLC6A1 Connect and rare disease parent), Dr. Gaurav Shah (CEO of Rocket Pharmaceuticals^a), Ilan Ganot (CEO of Alesta Therapeutics and a rare disease parent), and Curran Simpson (CEO of RegenxBio). These interviews were used to capture experiential insights on how PRV-related uncertainty and incentives shape drug development strategy and patient access.

This analysis also faced several limitations, most notably the lack of fully transparent PRV sales data. The dataset includes all transactions publicly disclosed through company press releases and other official sources; however, several sale prices and transaction dates were not publicly reported. Access to these additional data points would allow for a more comprehensive and robust analysis.

Second, although PRV revenues and R&D spending appear positively correlated, it is difficult to quantify the extent to which voucher proceeds directly translate into increased investment in a company’s R&D pipeline. R&D expenditures are influenced by many factors, including broader revenue streams, operating costs, and strategic priorities. To address this limitation, RTW Institute selected two illustrative case studies that demonstrate a clear temporal association between PRV proceeds and subsequent increases in R&D spending.

Origins of Rare Pediatric Disease PRVs

The FDA's priority review voucher framework was first established in 2007 through the Food and Drug Administration Amendments Act, which created the Tropical Disease (TD) Priority Review Voucher program – Figure 1.^11,12 Tropical diseases include illnesses primarily affecting hot, humid subtropical regions, such as mosquito-borne diseases like malaria and dengue.¹³ In 2012, the framework was expanded under the FDA Safety and Innovation Act, which established the Rare Pediatric Disease (RPD) Priority Review Voucher program, designed to incentivize the development of treatments for rare pediatric conditions.¹⁴ Since then, rare pediatric vouchers have accounted for the majority of both PRVs awarded and PRVs sold, underscoring their prominent role in expediting approvals for valuable therapies and securing funding for biotech companies. The PRV program was further expanded in 2016 through the 21st Century Cures Act to include Material Threat Medical Countermeasures (MTMC).^15,16

How Rare Pediatric PRVs Create Value

A sponsor that receives FDA approval for a drug or biological product indicated for a rare pediatric disease may be awarded a Rare Pediatric Priority Review Voucher. This voucher has two important features: it can be redeemed for priority review or sold and transferred to another sponsor. When redeemed, the voucher reduces the FDA review timeline for a subsequent product application from the standard 10 months to approximately 6 months, measured from the 60-day filing date - Figure 2.^17,18

The value of RPD PRVs is closely tied to their transferability. Because expedited reviews can provide a first-to-market advantage and up to four additional months of commercial sales before competitors can enter the market, larger pharmaceutical companies have a strong incentive to purchase vouchers to accelerate the commercialization of products in their pipelines. The ability to transfer PRVs, in turn, improves the commercial viability of rare pediatric products that may serve small patient populations, making the vouchers especially valuable for smaller biotech firms seeking to sustain R&D and recoup development costs.

Since the first PRV was issued in 2009, the FDA has awarded 95 PRVs to drug sponsors, 71 of which were for rare pediatric disease products - Figure 3.⁷ These treatments span disease areas such as Duchenne muscular dystrophy, neuroblastoma, and sickle cell disease. Due to the rarity of these conditions, many did not have an FDA-approved therapy prior to the Rare Pediatric Disease PRV program’s implementation in 2012.¹⁹

Many drug sponsors who received PRVs chose to sell them to generate cash flow to support their R&D pipelines. As of March 2026, there have been 38 open-market Rare Pediatric Disease PRV sales, with a median transaction value of approximately $110 million – Figure 4.⁷

In January 2026, Jazz Pharmaceuticals sold its PRV for $200 million, marking the first time prices returned to the $200 million range since the historic $350 million sale by United Therapeutics in August 2015.^20,21

Collectively, RPD PRVs have generated more than $4.4 billion in direct, non-dilutive capital for sponsoring biotech companies.⁷ This infusion of non-dilutive capital from PRV sales creates opportunities for companies to sustain their pipelines and even pursue additional rare and nonrare disease programs, a dynamic this paper refers to as the “butterfly effect” on product innovation. When asked about the influence of PRVs, Dr. Gaurav Shah — Rocket Pharmaceuticals’ CEO, said:

“It was a huge influence, I don’t think the company would have started without the PRV program.”

On a similar note, Meg Dodge, LLM — Rocket’s head of external affairs, stated:

“The PRV system allows you to fund your other programs before you get revenue. What it is really meant to do is this infusion of capital that is non-dilutive. This infusion allows the machine to keep working”

Rocket Pharmaceuticals was formed in 2015 with an initial pipeline of three ex vivo lentiviral gene therapy programs targeting pediatric rare diseases, all eligible for PRVs. As the company achieved clinical success in its pediatric rare disease programs, it expanded its pipeline to include therapies for larger cardiac diseases affecting both children and adults.^22,23

Undoubtedly, rare disease drug development is a risky and burdensome venture, in many cases more so than conventional drug development. It shares the high failure rates, long timelines, and substantial costs, but often lacks the financial upside associated with larger patient populations. The following case studies illustrate how proceeds from PRV sales not only help sponsors sustain their development efforts but also generate broader spillover benefits.

United Therapeutics’ Sale of the Unituxin PRV and Rinvoq’s Expedited Approval

In August 2015, AbbVie purchased United Therapeutics’ rare pediatric PRV for $350 million.²¹ The voucher had been awarded following FDA approval of Unituxin, a therapy for children with high-risk neuroblastoma.²⁴ Neuroblastoma is an ultra-rare pediatric cancer with approximately 600 to 800 new cases diagnosed annually in the U.S., more than half of which are classified as high-risk.^25,26

Unituxin was the first FDA-approved drug specifically indicated for the treatment of children with high-risk neuroblastoma.²⁷ In a pivotal Children’s Oncology Group study involving nearly 1,200 children, dinutuximab (Unituxin) was added to the prior standard post-consolidation therapy, isotretinoin. Compared to isotretinoin alone, the addition of Unituxin increased two-year overall survival from 75% to 86% and event-free survival from 46% to 66%.²⁸

Yet, despite this efficacy, the commercial revenues generated by Unituxin remained modest. United Therapeutics reported approximately $85 million in Unituxin revenue in 2018, a fraction of the value realized from the PRV sale.²⁹ This contrast illustrates how the PRV program can create economic incentives for developing therapies that serve small patient populations where traditional market returns alone would be unlikely to justify the investment.

AbbVie’s $350 million purchase of the voucher provided substantial non-dilutive capital to United Therapeutics, which was subsequently reflected in the company’s research and development spending. While R&D project expenses remained relatively flat at ~$157 million in 2016, spending increased rapidly in subsequent years, rising to ~$256 million in 2017 and $370 million in 2018.^29,30,31 In its annual filings, United Therapeutics attributed this growth in R&D spending to expanded investment across its pipeline, including multiple cardiopulmonary programs and organ manufacturing initiatives. Many of these programs, like Unituxin, target rare or ultra-rare conditions, underscoring how PRV-generated capital can be redeployed to sustain and broaden innovation in areas with limited commercial markets.

This pattern is not unique to United Therapeutics. Across the Rare Pediatric Disease voucher program, voucher sale proceeds have repeatedly been used to support further rare disease innovation. As of March 31, 2026, there have been a total of 71 RPD PRVs awarded across ~50 rare diseases. Prior to the creation of the RPD PRV program, only four of those diseases had any FDA-approved treatments available.¹⁹ The 2019 GAO Report found that seven out of seven drug sponsors they contacted told them that the PRV programs were a factor in their decision-making for drug development.¹⁸

In February 2019, AbbVie redeemed a PRV (likely the one purchased from United Therapeutics)^b to secure expedited FDA review of Rinvoq, resulting in approval in less than six months.³² Rinvoq’s original indication was to treat adults with moderate to severe rheumatoid arthritis, a condition affecting more than 1.3 million Americans.³³ Since the drug was first marketed in 2019, Rinvoq has been prescribed to over 100,000 patients, highlighting its substantial reach and public health impact.³⁴

AbbVie redeemed another PRV (likely the one purchased from Eiger BioPharmaceuticals) in 2021 to obtain priority review for the supplemental NDA supporting Rinvoq’s ulcerative colitis indication, a condition that affects ~1.5 million individuals in the U.S.^35,36,37 The application was subsequently approved by the FDA in March 2022.³⁸

Undoubtedly, Rinvoq’s expedited review was valuable in granting patients earlier access to treatment. The Unituxin–Rinvoq case study shows that the rare pediatric PRV program not only advances innovation for ultra-rare pediatric diseases but also accelerates patient access to therapies for more common conditions.

The Case of the Progeria Research Foundation

In some cases, funding received by small biotech firms can also produce meaningful spillover benefits for their non-profit partners. Such spillover effects are illustrated by the partnership between the Progeria Research Foundation (PRF) and Eiger BioPharmaceuticals in the development and approval of Zokinvy.

Progeria is an ultra-rare, fatal pediatric genetic disorder affecting roughly 400 children worldwide.³⁹ On November 20, 2020, the FDA approved Zokinvy (lonafarnib) as the first treatment for Progeria and processing-deficient Progeroid Laminopathies.⁴⁰ This milestone was the result of a 2018 Collaboration and Supply Agreement between the Progeria Research Foundation (PRF) and Eiger Biopharmaceuticals in which PRF granted Eiger access to its research and clinical data to support the NDA filing. In return, Eiger managed the regulatory process, sponsored the NDA, conducted any additional studies required, and developed a pediatric formulation of the drug.⁴¹

Upon the drug’s approval in November 2020, Eiger received a PRV. The PRV was then sold to AbbVie for $95 million, closing in January 2021, with proceeds shared equally under the collaboration agreement. The PRF received approximately $47.5 million.⁴²

The funding from the PRV was transformative for PRF. The 2024 newsletter notes that the organization’s net assets totaled $47.4 million, largely from the PRV sale, and that “revenue from the gain on sale of the PRV represented 92% of the Organization’s total support and revenue”.^43,44 Roughly 50% of PRF’s total expenditures are dedicated to research programs, with an additional 20% allocated to research grants and clinical trials. Following Zokinvy’s approval, PRF has continued to advance Progeria research, launching its first Progerinin clinical trial and exploring gene therapy as a potential therapeutic option. As of the end of 2025, PRF has funded 85 grants and 69 principal investigators from 55 different institutions across 14 countries. PRF also holds international scientific meetings aimed at promoting collaboration between basic and clinical scientists.⁴⁵ This sustained reinvestment after initial drug approval underscores that PRVs do not just reward past success but rather drive continued innovation in the rare and ultra-rare disease space.

From a small-biotech perspective, Eiger increased its investment in research and development following the sale of the PRV. According to the company’s Security and Exchange Commission 10-k filings, R&D spending rose from $41.6 million in 2020 to $64.4 million in 2021, making it the company’s largest operating expense. Over the same period, Eiger reduced its net loss by 48%, from $65.1 million in 2020 to $33.9 million in 2021.⁴⁶ While Eiger had historically incurred operating losses since its inception, the company reported profitability for the three months ended March 31, 2021, attributable to proceeds from the PRV sale. Although Eiger ultimately ceased operations in 2024, Zokinvy’s approval and its subsequent acquisition by Sentnyl Therapeutics make it a compelling case study of how a PRV can catalyze value beyond the lifespan of a single sponsor.⁴⁷

As Amber Freed, rare disease advocate and CEO of SLC6A1 Connect, noted in an interview, PRF exemplifies how a PRV can create a “flywheel of research,” in which initial drug approval unlocks sustained funding, improved research infrastructure, and new therapeutic pathways.

PRV’s Social Bargain

Beyond its ability to improve commercial viability for biotech companies by generating non-dilutive capital, the Rare Pediatric Disease PRV program has another important feature: it is effectively budget neutral.

Because PRV transfers happen strictly within the biopharmaceutical ecosystem, the program effectively creates a market in which vouchers function as scarce, tradable commodities. While purchasing a voucher is costly, sponsors often view the expense as justified by the commercial advantage it provides. As a result, the PRV program contributes to funding socially valuable but commercially fragile rare pediatric disease research through private capital, accelerating development at no direct cost for U.S. taxpayers.

In fact, because sponsors redeeming a voucher must pay a substantial user fee to redeem the PRV, the program can be seen as generating incremental revenue for the FDA. According to the Federal Register, the priority review user fee for FY 2026 is ~$1.96 million.⁴⁸ This fee is intended to offset the additional resources required for expedited review. As noted in the Congressional Budget Office cost estimate of PRV reauthorization legislation, the presence of these user fees allows the Give Kids a Chance Act to have a net positive effect on federal revenues.⁴⁹

Advocating for Permanence

As a program with a measurable track record and a critical role in expediting drug approvals for some of the most vulnerable patients in the rare disease community, the recurring uncertainties surrounding the Rare Pediatric Disease Priority Review Voucher program are unwarranted.

Unfortunately, recurring uncertainty has become a theme for the program. Although the RPD PRV program is now extended through September 2029, the FDA’s authority to award new vouchers lapsed in December 2024, creating roughly a 14-month window during which no new pediatric PRVs could be issued.¹⁴ In addition, since the standard five-year extension was applied to the December 2024 sunset date, the effective period of certainty from February 2026 is only about three and a half years.

This brief extension is not sufficient for most drug sponsors. As Curran Simpson, RegenxBio’s CEO, states, drug development cycles can take up to 10 years, which is far longer than the current period of legislative certainty. This timeline is consistent with estimates reported in academic literature.^8,9

Given the status quo of recurring uncertainty every few years despite the program’s demonstrated record in incentivizing rare pediatric drug development, reauthorizing the pediatric PRV program on a temporary basis is no longer sufficient.

The totality of evidence supports that it would be prudent and cost-effective to advocate for the permanence of the Rare Pediatric Disease Priority Review Voucher Program. Making the PRV program permanent would bring much-needed certainty and funding to rare disease drug sponsors at no direct cost to taxpayers while expediting rare disease drug development.

In addition to the value added by PRVs elaborated above, the broader, downstream impact of PRVs, what Freed describes as the “butterfly effect,” elucidates how these incentives catalyze a feedforward mechanism across the biomedical ecosystem.

By sustaining research and development for small biotechs, PRVs enable more clinical trials, facilitate the identification of previously undiagnosed patients, and deepen the understanding of rare disease biology. Beyond this, recent scientific literature underscores that rare disease research plays a critical role in advancing understanding across the broader disease landscape. A 2025 review highlights how advances in genomics, machine–learning–based gene network analysis, and gene-editing technologies have revealed shared biological features between rare diseases and common diseases, highlighting PRVs’ wider impact.⁵⁰

That said, there are arguments for why making the pediatric PRV program permanent warrants caution. Because vouchers derive value from scarcity, a permanent program could make supply more predictable and potentially reduce what sponsors are willing to pay. However, biotech stakeholders and rare disease advocates interviewed by RTW Institute indicated they would prefer a certain, lower voucher value over the uncertainty created by recurring legislative lapses. As Ganot pointed out, PRVs represent the hope that there was a bigger, immediate carrot at the end of the rainbow, making it one of the only ways for developers to pursue a rational development plan. In other words, when incentives to develop rare disease therapies are scarce, the mere presence of a credible incentive can matter more than its dollar value.

Some are also concerned about the additional resource strain that priority review vouchers place on the FDA. The agency is already facing political and leadership uncertainty along with workforce attrition, which has contributed to fewer new orphan approvals across both CDER and CBER compared with previous years.^7,51 However, priority review user fees are intended to offset this burden by providing the agency with additional resources to support accelerated reviews. Under 21 USC §360ff(c)(5), voucher fees are deposited directly into the FDA’s appropriations account, and the agency notes that these fees help fund the staffing and resources needed for more efficient application review.^52,53

Conclusion

Recurring uncertainty around the RPD PRV program doesn’t just affect drug sponsors and policy makers; it directly burdens patients, families, and rare disease advocacy groups. Each legislative cycle demands substantial advocacy resources; in 2024, the National Organization for Rare Disorders (NORD) and the EveryLife Foundation brought together over 190 patient groups to urge Congress to maintain the program.⁵⁴

Many advocates take on this work amid already overwhelming personal challenges: one parent may already leave the workforce to care for a sick child, only to spend full-time fundraising and coordinating with scientists to advance a therapy. The economic burden of rare disease reached nearly $1 trillion in the U.S. in 2019, with more than $400 billion attributed to indirect costs and productivity loss.⁵⁵

Simpson highlighted the unique challenges faced by the rare disease community:

Rare disease is often the 7th issue on a list of 6… The patient community is counting on these incentives to move new therapies forward, and this is the group that get hurts the worst in times of uncertainty.

The RPD PRV program is a proven mechanism to advance rare disease drug innovation at no direct cost to U.S. taxpayers. To reach its full potential, however, it must be reinvented to reduce recurring legislative uncertainty and ease the ongoing advocacy burden placed on the rare disease community.

Appendix A. The Commissioner’s National Priority Voucher (CNPV)

In parallel with the Rare Pediatric Disease PRV program, the FDA has introduced a new and separate priority review mechanism: the Commissioner's National Priority Voucher (CNPV) Program. The CNPV program pledges to reduce the review window from the typical 10-12 months to just 1-2 months. Since the program’s pilot in October 2025, several sponsors have redeemed their CNPVs. Fo products were approved via the CNPV pathway. However, one product—Disc Medicine’s bitopertin—received a complete response letter (CRL) despite being granted CNPV status⁷.

While both initiatives aim to reduce review time for eligible drug candidates, the CNPV program differs structurally from the RPD PRV program. CNPVs are issued at the discretion of the FDA Commissioner to drug candidates tied to national priorities, whereas pediatric PRVs are mandated by law and awarded based on statutory eligibility criteria.^56,14 Due to its less clearly defined eligibility framework, several FDA staffers have voiced their concerns on how CNPV could potentially “become vehicle for political interference in drug review decisions.” Moreover, CNPVs cannot be transferred or sold, making them far less effective as a mechanism for generating non-dilutive capital for smaller biotech companies.⁵⁷

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