Who Policy Impacts—and How
Policy decisions influence the lives of real people—often in deeply personal and lasting ways. In rare disease spaces, these impacts are amplified by delayed diagnoses, limited treatments, and systemic gaps in care. Understanding who is impacted helps ensure we shape policy that is inclusive, patient-centered, and equitable.
Patients and Caregivers
Access & Affordability: Policies determine insurance coverage, treatment costs, and access to specialists or genetic testing. For many, these factors dictate whether lifesaving care is even possible.
Time to Diagnosis: Many rare disease patients endure diagnostic odysseys averaging 5–7 years. Policy changes can support better screening, awareness, and reimbursement for necessary diagnostic tools.
Emotional Toll: Caregivers often navigate complex systems alone, without support. Policy should protect their rights, expand respite care, and prioritize mental health resources.
Providers and Researchers
Barriers to Innovation: Researchers face funding limitations, regulatory hurdles, and challenges in trial design—especially in ultra-rare disease studies with small populations.
Provider Gaps: Many healthcare professionals lack rare disease training. Policy can encourage continuing education, patient registries, and cross-specialty collaboration.
Reimbursement Disparities: Even when a diagnosis exists, coverage for treatments or off-label therapies can be denied, affecting care delivery and trial participation.
Systemic Inequities
Racial Disparities: Black, Brown, and Indigenous communities are less likely to be diagnosed early or enrolled in clinical trials. They’re also underrepresented in genetic studies.
Gender & LGBTQ+ Barriers: Policies often overlook how care is experienced differently across gender identities or sexual orientations. LGBTQ+ patients face discrimination, assumptions, and lack of inclusive support.
Geographic Inequities: Rural patients may travel hours for care or face broadband challenges that limit telehealth access.
Why Intersectionality Matters
No one lives a single-issue life. Health policy must account for the layered experiences of race, disability, gender identity, economic status, and more. Centering intersectionality allows for policy that reflects real life complexity—and reaches the communities often left out.
How Stakeholders Can Use Their Voice
Patients & Caregivers: Share your story, participate in advocacy campaigns, join advisory boards.
Clinicians & Researchers: Support funding legislation, provide expert testimony, improve access to trials.
Community Partners: Collaborate with coalitions, amplify underrepresented voices, offer resources.
Policy that works for everyone must be shaped by everyone.