How Priority Review Vouchers Can Save Lives

An important US government program is on the chopping block September 30

When developing therapies in a capitalist society, rare diseases often get sidelined in favor of more common (and therefore more profitable) ailments. To incentivize the development of treatments for rare or orphan diseases, many governments have developed programs to bring money or attention to neglected patients.

One such program was created in 2007: the US Priority Review Voucher Program. The purpose: to offer vouchers for expedited FDA review to organizations who were researching treatments for rare pediatric or tropical diseases, as well as anything related to public health emergencies. The PRV can, in theory, make a drug available up to four months earlier. The program is set to expire on September 30, 2024, and must be reauthorized in order to continue. However, some politicians are concerned since the program did not go exactly as intended.

A provision of the program was that rare disease researchers could sell their vouchers. This has proved to be as popular as keeping the voucher for yourself: of the 64 vouchers awarded since 2009, 37 have been sold and 19 of those have been redeemed. Those 19 have gone to treatments for more common diseases such as arthritis, diabetes, and HIV that would not have been eligible for a PRV.

Some politicians, including US Senator Bernie Sanders of Vermont, are worried that this trend is merely lining the pockets of larger pharma companies. After all, if they buy a voucher for US$100 million that gets their drug to market four months faster, they could make much more than $100 million back on that drug in those four months. And the purpose of the voucher was to make rare disease treatments available faster, not to promote drugs for diseases that already have available treatments.

However, that $100 million, while a drop in the bucket to a large company, is the difference between a rare drug continuing to market or stalling out due to lack of funds.

"The PRV voucher serves as a lifeline for rare diseases affecting fewer than 2,000 patients globally," said Terry Pirovolakis, CEO and founder of Elpida Therapeutics, a biotech looking for a treatment for SPG50, an ultra-rare neurodegenerative disease that his son Michael has. "In the current biotech economic crisis, the elimination of the PRV voucher could doom rare disease research, deterring companies from pursuing such programs. The ecosystem for rare diseases hinges on the existence of the PRV voucher; it's not ideal, but it's our only option."

According to a report by the US Government Accountability Office (GAO), vouchers have been sold from between $67 million and $350 million. The report did not find quantitative evidence that the vouchers affected drug development. However, all the drug sponsors that GAO spoke with confirmed that PRVs were a factor in their drug development decisions.

Considering that the average time for a drug to be developed is 10-15 years, it is not surprising that a program that has really only been active since 2009 hasn't had a huge impact - yet.

"PRV vouchers are to the rare disease community what grants are to clean energy initiatives-while the latter aims to reduce CO2 emissions, PRV vouchers are crucial for saving the lives of our children," said Pirovolakis.

This is a crucial point that politicians should consider when they decide whether to reauthorize the program at the end of September, says Pirovolakis. Drug development, like climate change mitigation, is not something that can be accurately measured in a decade. Whether or not the program "works" in terms of getting rare drugs developed faster is not yet something that can be proven.

However, the impact of the sale of vouchers to companies with more resources has brought millions of dollars in research funding to rare drug developers. In essence, it has helped a little to shift the burden of rare disease research funding from government grants and donors to big companies.

Ultimately, the fate of the PRV program lies with our elected representatives. If you believe the program should continue, or if you have any other thoughts on the program, now is the time to reach out to your representative or congressperson.

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