US Congress called on to extend program for rare pediatric diseases
Voucher program set to expire Sept. 30 without congressional action
Since its launch 12 years ago, a program to incentivize drug development for rare diseases that affect children has led to the approval of dozens of new treatments, but without congressional action, the program is set to expire by the end of September.
The National Organization for Rare Disorders (NORD) recently released a report highlighting the success of the program, called the rare pediatric disease priority review voucher (RPD PRV) program. NORD has joined with nearly 200 other patient organizations calling on Congress to approve legislation, introduced by a bipartisan team of senators and congressional representatives, that would extend the program for another five years.
“This program gives hope to the estimated 15 million children living with a rare disease, of whom the vast majority are currently without FDA approved treatment options for their condition,” Pam Gavin, CEO of NORD, said in a press release. “Our research shows that the RPD PRV program is helping to address a substantial deficit in pediatric rare disease drug development. Yet, without Congressional action, this effective program will end September 30, 2024, and this is simply unacceptable.“
AADC deficiency one of thousands of rare diseases primarily affecting children
AADC deficiency is one of thousands of rare diseases that primarily affect children. Most of these disorders don’t have any approved treatments in the U.S. Getting new treatments developed and approved is a crucial need, but from an economic perspective, developing treatments for rare diseases faces notable obstacles.
To get a new therapy approved, companies need to invest a lot of money, not to mention time and other resources, into developing treatments and conducting clinical trials. Since rare diseases are by definition rare, relatively few patients would buy the treatments whose approvals are also not guaranteed. Therefore, there’s not much financial incentive for companies to invest in developing treatments for these diseases.
The RPD PRV program was launched in 2012 to address these obstacles by providing more incentives to companies developing treatments for rare pediatric illnesses. Under this program, a company that gets a treatment approved for a rare pediatric disease may be eligible to receive a priority review voucher. The company can either redeem the voucher for a future therapy to get it reviewed faster, or sell the voucher.
Since the launch of the RPD PRV program, 53 priority vouchers have been awarded to new therapies for 39 rare diseases, and nearly all of these diseases didn’t have any previously approved treatments, according to NORD’s new report. More than half of the vouchers have been issued in the last four years, which NORD says highlights how the program has helped accelerate the development of new treatments for conditions where there might not otherwise be incentives for companies to invest.
The report also notes these vouchers are not typically used to help accelerate review for so-called “blockbuster” drugs (medicines that are prescribed widely and rack up massive profits for companies). In fact, of the vouchers that have so far been redeemed, most are for medicines that are not among the top 100 medicines with the biggest fiscal impact on Medicare, the government-funded program that provides insurance to elderly Americans and younger people with disabilities.
For the children who still lack treatment options for their rare condition, letting this program lapse would be a devastating step backward.
Most companies that receive vouchers sell or transfer them
The report also notes companies that receive these vouchers rarely use them themselves. Instead, most of them are sold or transferred, often within the first few months after receipt. As such, NORD says these vouchers offer important incentives to companies that have taken the risky but important step of investing in new treatments for rare diseases.
The RPD PRV program has helped facilitate the development of new rare disease treatments, but most children with rare disorders still don’t have any approved treatment options in the U.S., so NORD is calling on Congress to quickly reauthorize the program.
“For the children who still lack treatment options for their rare condition, letting this program lapse would be a devastating step backward,” the report concludes. “Given the vast unmet medical needs that remain, Congress should act swiftly to reauthorize it to help resolve the treatment challenges faced by millions of children living with rare diseases.”
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