Josie Cooper
Congress needs to act to continue to spur new treatments for sick children with few options.
The Creating Hope Reauthorization Act would extend an existing program (1) that incentivizes the development of treatments for rare pediatric diseases by offering a voucher for expedited FDA review. (2) Companies can then buy and sell these vouchers to capture a return on investment that rare drugs seldom deliver.
There have been 49 vouchers issued under the program, generated by the approval of treatments for 40 diseases impacting children, including a rare muscular dystrophy mutation (3), cystic fibrosis (4), and haemophagocytic lymphohistiocytosis. (5) No approved drugs (6) were available for most of these conditions before the incentives program existed.
Research and Rare Diseases in Children
A complex cost-benefit equation applies to treatments for rare diseases.
Researchers and investors should prioritize technologies that can improve the well-being of children by addressing their unique diseases and challenges. The small number of patients affected by rare conditions makes it difficult to recover research and development costs, even if the treatment is wildly successful.
The difficulty of conducting clinical trials for children, the effects of delayed diagnoses, and a 15-year average for drug approval further complicate matters.
Fully 95% of rare diseases have no FDA-approved treatment. These medications serve a small patient population and are difficult to develop, test, and make available. The challenges are magnified for disease treatments for children, often discouraging traditional investment.
Patients and Providers Urge Congress to Reauthorize Vouchers
More than 600 patients, caregivers, providers, and advocates converged on the U.S. Capitol to tell their stories and plea for more public investment in policy solutions to healthcare challenges, just days after the Creating Hope Reauthorization Act was introduced. (7)
The bipartisan bill is sponsored by U.S. Representatives Gus Bilirakis (R-FL), Anna Eshoo (D-CA), Michael McCaul (R-TX), Lori Trahan (D-MA), Michael Burgess (R-TX) and Nanette Barragán (D-CA.). In remarks announcing (8) their sponsorship, several representatives referred to personal acquaintances or constituents whose children live with rare diseases.
The voucher program, formally known as the Rare Pediatric Disease Priority Review Voucher Program, must be renewed before its September 2024 expiration (9), or programs currently developing treatments for America’s most vulnerable may be shuttered or delayed.
References:
- https://go2.bio.org/NDkwLUVIWi05OTkAAAGRUWwN3BiLtufOa6ucbOcA0-gwJvbiJwhqe4N_86ZNMyVuMy1OYT- 2RbUmFISoHTw0oBQW7Fc=?_gl=1*1fc1arn*_gcl_au*NDUwNjAyMDczLjE3MDk1NjY5Mzg.&_ga=2.168779745.171284454.1709566938-1724185872.1709566938
- https://www.raps.org/news-and-articles/news-articles/2017/12/regulatory-explainer-everything-you-need-to-know
- https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-first-targeted-treatment-rare-duchenne-muscular-dystrophy-mutation
- https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis
- https://www.sobi.com/en/press-releases/fda-approves-gamifantr-emapalumab-first-and-only-treatment-primary-haemophagocytic
- https://everylifefoundation.org/everylife-foundation-applauds-the-introduction-of-the-creating-hope-reauthorization-act-of-2024-urges-congress-to-act-swiftly/
- https://everylifefoundation.org/rare-advocates/rare-disease-week-2023/rare-disease-week-attendee-corner-resource-library/
- https://barragan.house.gov/2024/02/16/reps-barragan-mccaul-eshoo-bilirakis-trahan-and-burgess-introduce-creating-hope-reauthorization-act-of-2024/
- https://go2.bio.org/NDkwLUVIWi05OTkAAAGRUWwN3He6UwcMgj6JPxSbfXsZVID4_Y6uRRKiT8pGHe6WB5QJmmT2XoWthIeLAtt-r7S7vhA=?_gl=1*1fc1arn*_gcl_au*NDUwNjAyMDczLjE3MDk1NjY5Mzg.&_ga=2.168779745.171284454.1709566938-1724185872.1709566938
Disclosure: Josie Cooper is executive director of the Alliance for Patient Access.
This article was also published at healthpolicytoday.org
Corresponding Author
Josie Cooper
Executive Director
Institute for Patient Access
2020 K Street NW, Suite 505
Washington, DC 20006
Telephone: (202) 951-7095
Email: jcooper@woodberryassociates.com