By Gabrielle Wanneh / November 22, 2022
This article was published by Inside Health Policy.
Rare disease advocacy groups hope the new Congress will prioritize investment in the development of rare disease treatments by passing bills that restore and reinforce seven-year exclusivity for drugs being developed for specific rare conditions, require regular reporting to improve the approval process of orphan drug designation and ensure patient input is counted throughout the development process.
With Republicans set to control the House and Democrats to maintain control of the Senate, and new committee leaders yet to be formally announced, it’s too early to tell how legislation aimed at accelerating the development of rare disease treatment will be handled, sources say, despite bipartisan support for some measures.
Amanda Malakoff, executive director of the Rare Disease Company Coalition, is hopeful that bills like the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act and the Helping Experts Accelerate Rare Treatments (HEART) Act have the bipartisan support needed to be pass in one or both chambers next year.
The BENEFIT Act would make sure patient experiences, patient-focused drug development and other related data are considered as part of FDA’s risk-benefit assessment, while the HEART Act would require FDA to consult external experts and stakeholders while also providing reports on how FDA is handling applications for a drug to be designated for the treatment of a rare disease.
“There’s just a lack of expertise in general in [the area of] rare disease, so we really advocate for the FDA making sure they’re bringing people with the most timely and relevant knowledge into the process,” Malakoff told Inside Health Policy.
“Anything that can be done to streamline the process at FDA will help give that confidence back to investors on rare disease and biotech, and those two bills are just examples of steps Congress can take to help make that happen,” Malakoff added.
Another bipartisan bill, Cameron’s Law, would increase the tax credit for clinical testing expenses under the Orphan Drug Act from 25% back to the original 50%, years after the Tax Cut and Jobs Act reduced the credit in 2017. Malakoff said restoring the tax credit to its original parameters would make it more economically feasible for drug companies to develop rare disease treatments and could also send a positive message to investors.
Malakoff noted that provisions from both the BENEFIT Act and HEART Act had been included in Congress’ user fee reauthorization bill at different points in time before it passed in September. It’s unclear whether measures from either bill will make it into the upcoming year-end legislation.
Heidi Ross, vice president of Policy and Regulatory Affairs at the National Organization for Rare Disorders, said that reinforcing the seven-year exclusivity period for rare diseases and conditions under the Orphan Drug Act, and specifying that this period prohibits the approval of other drugs for the same use or indication, is another issue that may be able to garner bipartisan support. The Retaining Access and Restoring Exclusivity (RARE) Act, co-sponsored by Sens. Tammy Baldwin (D-WI) and Bill Cassidy (R-LA), would do just that.
Cassidy is expected to be the ranking Republican on the Senate health committee.
The bill would reverse the September 2021 Catalyst v. Becerra decision that upended FDA’s longstanding narrow interpretation of how and under what circumstances the exclusivity period should be granted.
“Our concern is that you will end up in a world where if you are awarding exclusivity to just cystic fibrosis, then you’re not going to have companies have the proper incentives to study those more challenging patient populations. The biggest population might be genetic mutation XYZ, but the patients that have genetic mutation ABCD also deserve to know that a therapy is safe and effective for them,” Ross said.
Similar language that codifies the FDA’s prior interpretation of how to award orphan drug exclusivity was included in the version of the Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act and the Food and Drug Amendments of 2022 passed by the House passed in June. The provision ultimately did not make it into the final clean user fee package that Congress passed in September, but many of the FDA reforms that were left out of the final package could still be included in year-end legislation.
If not included in Congress’ year-end legislation, Ross said it’s also possible the RARE Act alone could garner bipartisan support next Congress should Cassidy become the ranking Republican of the Senate health committee. — Gabrielle Wanneh (firstname.lastname@example.org)