Our Policy Goals
Close the innovation gap for the 95% of rare diseases that have no FDA-approved treatment.
Eliminate the diagnostic odyssey for rare disease patients.
Improve the regulatory process and advance regulatory science for rare disease therapies.
Ensure patient access to safe and efficacious therapies and cures at the earliest moment possible.
Empower patients to develop an impactful voice in policymaking, drug development, and regulatory decision-making.
Accelerating Diagnosis
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Newborn Screening
Join the fight to protect and advance one of America’s most successful public health programs.
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Genetic Testing
Better access to genetic testing and stronger protections against genetic discrimination mean faster answers for patients and families.
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Diagnostic Odyssey
In rare disease, it can take more than 6 years and 17 medical visits to get an accurate diagnosis.
Advancing Therapy Development
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Research Funding
Advocating for federal biomedical research funding that powers progress in understanding rare diseases and enables the discovery of new therapies.
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Accelerated Approval
Ensuring tools like accelerated approval are fully utilized so patients living with rare diseases can access promising new treatments sooner than the traditional approval process allows.
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Rare Disease Innovation Hub
Strengthening the infrastructure behind this collaboration between the FDA's Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health to streamline and accelerate treatments for rare diseases.
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Patient-Focused Drug Development (PFDD)
Ensuring patients' experiences, perspectives, needs, and priorities are included in drug development and evaluation.
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FDA User Fees
Ensuring the needs of the rare disease community are included in the reauthorization of FDA user fees.
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Orphan Drug Incentives
Advocating for tax credits, grants, and market exclusivity that make developing treatments for rare diseases possible.
Ensuring Access
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Medicaid
Protecting Medicaid coverage, since cuts to eligibility, benefits, or access would directly harm the rare disease community's ability to get care.
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Prescription Coverage & Reimbursement
Supporting timely access to recommended treatments by removing coverage barriers that delay care, raise costs, and lead to worse health outcomes.
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Drug Pricing
Advocating for lower prescription drug costs in ways that account for the unique complexities of rare disease therapy development.
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Value Assessment
Ensuring value assessment frameworks, which weigh a therapy's effectiveness and cost against alternatives, include patient experience data and the outcomes that matter most to patients.
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Care Access
Expanding access to specialty care, including out-of-state care, to prevent delayed diagnoses and worsening health outcomes.
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Navigating the Access Environment
Helping patients overcome systemic barriers to early diagnosis, specialized care, and life-saving therapies.
Activating in Your State
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State Policy Priorities
Change starts at home. Find out more about the issues impacting the rare disease community in your state or region.
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Find Your Legislators
Start building relationships with local, state, and federal lawmakers.
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Advocate in Your State
Advocacy begins at home. Discover ways you can share your story in your home state.
Take Action
Encourage Your Member of Congress to Support the Rare Disease Innovation Hub
With appropriate resources, the Rare Disease Innovation Hub can continue to transform how rare disease community stakeholders, clinical and scientific experts, and product developers engage with the FDA.
