California Life Sciences Celebrates Rare Disease Week, Promotes Innovation and Access for Patients

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FOR IMMEDIATE RELEASE: Feb 28, 2025

South San Francisco – California Life Sciences (CLS) celebrates what has been a critically important and fulfilling rare disease week spent advocating for access to innovative treatments for patients with rare diseases from coast-to-coast. With the enactment of HR/ACR 33 which establishes today, February 28, 2025, as Rare Disease Day in California, CLS joined rare disease patients and advocates throughout the week in Sacramento and Washington, DC, sharing their stories with policymakers to drive education and awareness of policies — at all levels of government — that pave the way for the next generation of rare disease treatments.

For continued progress it is critical to maintain the incentives that have long supported the development of hundreds of new rare disease treatment options. There are several ways the U.S. Congress can ensure scientists and researchers have the tools needed to keep the hope of future treatments alive for the patients living with one of the 95% of rare diseases that have no FDA approved treatment options.

  • First, we need long-term reauthorization of the Rare Pediatric Disease Priority Review Voucher program. A multi-year reauthorization will prevent uncertainty about the future of incentives that are crucial to the development of new therapies for rare conditions.
  • Second, we need to fix the premature eligibility timelines for rare-only drugs in the government price setting program created by the Inflation Reduction Act. Right sizing the current orphan drug exemption will ensure there is an economically viable path forward for orphan drugs.
  • Third, we need to restore the Orphan Drug Tax Credit to the original level as established in the 1983 Orphan Drug Act. This long-standing incentive to offset the complex and expensive process of developing a rare disease drug is particularly important for small and mid-size innovators.

To meet the needs of rare disease patients, policymakers must prioritize elevating the patient voice to foster innovation and ensure patient access. Across California policymakers can support rare disease patients of today and tomorrow in the following ways:

  • First, policymakers can change the trajectory of a child’s life by ensuring critical newborn health screenings are inclusive of more rare diseases like Duchenne’s Muscular Dystrophy.
  • Next, elevating patient voices by supporting the creation of the Patient Advisory Committee within the Office of Healthcare Affordability to ensure patient needs and experiences are considered in the future health care policy making.
  • Lastly, we are calling on policymakers to fund the Rare Disease Advisory Council (RDAC). Establishing the council was a crucial first step to addressing the unique challenges faced by rare disease patients. However, investing in a well-funded RDAC demonstrates a commitment to the rare disease community.

Despite significant progress in the fight to treat rare diseases, there are still many patients with unmet needs. With medical breakthroughs on the horizon right here in California, now is not the time to whittle away at the enabling infrastructure for developing rare disease treatments. The events this week remind us of the progress we’ve seen, and the opportunities ahead to improve policies that strengthen our ability to bring lifesaving treatments to communities who need it most.

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For media inquiries, please contact:
Katie Kiss
Senior Director, Marketing
California Life Sciences
[email protected]