Patient advocates and researchers are heralding the creation of a federal Rare Disease Innovation Hub, designed to unite their efforts to improve patients’ health.
While each rare disease individually impacts fewer than 200,000 people in the United States, more than 10,000 rare diseases collectively affect roughly 30 million people. That’s approximately one in 10 individuals.
A Long-Awaited Resource
The Food and Drug Administration’s Rare Disease Innovation Hub is poised to become a pivotal resource for the community by offering:
- Connectivity: By enhancing relationships between patients, advocacy groups, trade organizations, researchers, pilot programs, centers of excellence and regulators, the Hub promises progress across a broad range of rare disease objectives.
- Coordination: Increased communication and streamlining of rare disease research efforts has the potential to accelerate the development of new treatments and perhaps even the discovery of cures. Bringing together various research groups could incubate solutions that may not be possible without that central point of connectivity.
- Attention: The Hub’s creation will ensure that treatment development remains a priority within government and across the broader research community. Upwards of 90% of rare diseases lack an approved treatment.
- Regulatory Support: Half of new drugs approvals in 2023 addressed a rare disease. This exciting pace of innovation is best supported by a flexible regulatory infrastructure designed to protect patients while not unnecessarily delaying access to novel treatments. The FDA predicts the Hub will “advance regulatory science by considering such things as novel endpoints, biomarker development, innovative trial design and the use of real-world evidence.”
Patient Advocacy Remains Crucial to Public Awareness
About half of Americans impacted by rare diseases are children, including those suffering from various kinds of cancer. While these are the patients who stand to benefit most from the supercharged innovation the Hub is designed to generate, all patients and their respective advocacy organizations must continue their awareness efforts relative to the unique needs of the rare disease community.
Ongoing promotion of rare disease patients’ interests is essential to ensuring they remain top of mind and support for new treatments doesn’t waiver. The establishment of the new Rare Disease Innovation Hub represents a monumental step forward in this effort.
