The Lymphangiomatosis & Gorham's Disease Alliance (LGDA) is proud to be among the signatories of an important letter presented to the incoming presidential administration, aimed at supporting the rare disease community. As a key advocate within the EveryLife Foundation for Rare Diseases' initiative, LGDA is helping to shape policies that crucially affect the estimated 30 million Americans living with rare diseases.
This advocacy letter calls for the preservation and enhancement of programs crucial for the development of treatments for rare diseases, particularly focusing on the Orphan Drug Tax Credit and the Rare Pediatric Disease Priority Review Voucher Program. These programs have been instrumental in fostering the development of therapies for rare diseases, which often face significant research and financial challenges due to their low prevalence.
Orphan Drug Tax Credit (ODTC)
The Orphan Drug Tax Credit has been a cornerstone in promoting the development of rare disease treatments since its enactment in 1983 as part of the Orphan Drug Act. The ODTC allows drug manufacturers to claim a tax credit for a significant portion of the clinical testing expenses incurred in the development of orphan drugs (drugs intended for the treatment of diseases affecting fewer than 200,000 people in the U.S.). This incentive is crucial because it helps mitigate the high costs and risks associated with the development of drugs for small patient populations. Despite its success, the credit was reduced in 2017, impacting the financial viability of potential treatments. Advocacy efforts are focused on restoring the ODTC to its original strength to continue encouraging the development of orphan drugs.
Rare Pediatric Disease Priority Review Voucher Program (PRV Program)
Similarly impactful is the Rare Pediatric Disease Priority Review Voucher Program, which provides additional incentives for companies to invest in treatments for rare pediatric diseases. Under this program, companies that develop approved drugs for certain rare pediatric conditions are awarded a voucher that can be used to expedite the FDA review process for another drug. This can be tremendously valuable, not only speeding access to life-saving treatments but also enhancing the commercial value of developing drugs for rare diseases. Despite broad bipartisan support, this program lapsed in late 2024, and urgent calls for its reauthorization are being made to ensure that new treatments continue to emerge for vulnerable patient groups.
The Impact of Advocacy
LGDA’s advocacy emphasizes the need for these programs, reflecting our commitment to the health and well-being of individuals with rare diseases. By addressing the policy and process hurdles that can delay the development and approval of vital therapies, LGDA aims to facilitate quicker access to treatment options and support ongoing innovation in this vital area of healthcare.
Our letter also highlights the broad economic impact of rare diseases, pointing out the pressing need for strategic policy responses that consider the full scope of challenges faced by patients and families. The rare disease community deserves comprehensive health insurance and access to the latest approved therapies without undue burdens, which these programs can help facilitate.
As we continue to advocate for these essential policies, we invite our community and policymakers to join us in this important mission. For more details on how you can get involved and support our advocacy efforts, please visit our blog and sign up for updates.
LGDA remains dedicated to supporting every individual affected by rare diseases through advocacy, education, and research. Together, we can build a future where no disease is too rare to deserve attention and care.
