By Mark A. Tobolowsky —
The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Designed to incentivize the development of drugs for pediatric rare diseases where such development may not otherwise have occurred, vouchers may be granted for drugs for serious or life-threatening rare diseases where the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Once granted, the vouchers may be used by the recipient or sold to another company for use with an application that may not otherwise qualify for priority review. A very important aspect of the program is that it costs FDA, and the government, no additional dollars, though we do not wish to minimize the additional resources a priority review demands over the standard review that such an application might otherwise receive. The value to industry for receiving a speedier review depends on the specific circumstances of the application, but it is clear that they are valued highly. The dollar prices of the vouchers themselves have fluctuated, recent sales have been in the neighborhood of $100 million.
Over 50 rare pediatric disease priority review vouchers have been awarded to date. We have heard over and over again how the potential for a priority review voucher has allowed small companies with limited resources to invest in the development of drugs for rare pediatric diseases. Although the program has not been without its critics (including as documented in two GAO reports from 2016 and 2020), there is ample evidence that the program has been successful at accomplishing its goals, which has become more evident with time. A recent analysis from NORD showed that among the 39 rare pediatric diseases for which vouchers were awarded, only three had any FDA-approved products on the market before the program’s enactment. Even FDA officials, who have historically been hesitant, at best, to support the program (this opposition was documented in the GAO reports), have recently issued statements of support. This also appears to be the administration’s position at this time, as it was included in the government’s “anomalies” list for purposes of inclusion in a continuing resolution.
The program, however, has always had a scheduled sunset date. Notably, it was reauthorized in 2016 (until 2020) and in 2020 (until 2024). We watched as the days ticked away as we crept closer to the first scheduled sunset date in the amended law, September 30, 2024. After this date, without action by Congress, FDA’s authority to grant rare pediatric disease designations would expire, and no vouchers could be granted unless previously granted such a designation. No rare pediatric disease vouchers could be awarded even to designated applications after September 30, 2026.
We heard and felt the anxiety from many of our clients about the uncertainty this caused. It is extremely difficult, if not impossible, to do the long-term planning and substantial fundraising required for drug development without knowing whether such an important and valuable incentive will be there. After all, this is what incentives are for. While the dates have been written in the law, the previous extensions provided a reasonable hope that the program would continue.
Nearly at the last minute, Congress acted to kick the can down the road just a little bit. On September 26, as part of a continuing resolution to keep the government funded, the President signed an extension into law that allows rare pediatric disease designations to be granted until December 20.
That brings us to today, where we are halfway through October without a longer-term fix. After coming so close to the brink, we were relieved to see the last-second short-term extension. However, this is only a brief reprieve, leaving substantial uncertainty for stakeholders as to what might happen in the coming months. Adding to the difficulty is that Congress is not scheduled back in session until mid-November, following Election Day.
However, there has been movement. A few days before the continuing resolution, on September 23, 2024, the House of Representatives passed H.R. 3433, the Give Kids a Chance Act of 2024. It was shortly thereafter introduced in the Senate and referred to the Committee on Health Education, Labor, and Pensions, where it remains. This bill would extend the designation deadline until September 30, 2029, a five-year extension (and would require yet another GAO report).
While we were heartened to see this movement, finally, we remain in an uncertain position, watching as the days tick away until December this time. With so much support from stakeholders, we are hopeful that Congress will recognize the immense value of this program, primarily for pediatric rare disease patients, the ultimate beneficiaries of the program, and authorize an extension. Hopefully, this can be accomplished without coming quite so close to the brink this time.
