The EveryLife Foundation for Rare Diseases, along with 209 undersigned organizations representing leading rare disease patient advocacy groups, has provided a set of priority policies to support the rare disease community as President Trump begins his term as the nation’s 47th President. These policy priorities, informed by the needs of the rare disease community, reflect a shared commitment to ensuring that the estimated 30 million Americans living with one or more of the 10,000 identified rare diseases have access to timely diagnoses, expert clinical care, and optimal treatment options.
The letter emphasizes that the nation’s rare disease community deserves access to comprehensive, affordable health insurance that enables timely diagnoses, recommended clinical care, and access to prescribed therapies. It highlights the critical role that programs like Medicaid and the Children’s Health Insurance Program (CHIP) play in supporting individuals with rare diseases and disabilities, noting that Medicaid covers almost half of all children with special healthcare needs. This coverage is a vital component in facilitating diagnosis, care, and treatment for the rare disease community.
The letter also underscores the collaborative efforts of patients, families, researchers, clinicians, the diagnostic and biopharmaceutical industry, and other committed stakeholders. Together, these groups drive evidence-based policy solutions and work alongside regulatory agency leaders to ensure that the public health infrastructure serves all Americans, regardless of the rarity of their condition.
