A new report has found that for the 30 million Americans living with rare conditions, access to the latest innovations often depends on their zip code.
The National Organization for Rare Disorders (NORD) released its 2025 state report card on 9 February, revealing that while the US earned a collective “B”, access to rare disease therapies remains dependent on state-level rules which often fail to cover expensive specialised medicines.
The path to market for orphan drugs – medications for diseases affecting fewer than 200,000 people – is increasingly shaped by state-level legislation rather than federal uniformity. These state-by-state differences create unpredictable hurdles for drug launches, insurance reimbursement and patient assistance.
“It remains unacceptable that access to life-saving care still depends on where a family lives,” says Pamela Gavin, CEO, NORD. “No one is immune to acquiring a rare disease… Policymakers must strengthen protections so patients can access treatment without facing devastating financial hardship.”
The rise of RDACs
One of the most strategic developments for pharmaceutical companies is the expansion of Rare Disease Advisory Councils (RDACs), which serve as critical platforms for companies to prove the value of orphan drugs to state-level decision-maker.
While 19 states have yet to establish an RDAC, in many jurisdictions these bodies are emerging as influential advisors on healthcare policy and funding priorities. With four additional states – Vermont, Oklahoma, Hawaii and Pennsylvania – set to introduce new councils in 2026, the number of active RDACs continues to grow.
For industry government affairs teams, RDACs are becoming essential partners. As states assume greater responsibility for healthcare decision-making, these councils provide a formal mechanism for patients and industry to help bolster access to innovative therapies.
“The Rare Disease Advisory Council is how we start making that commitment real,” notes Mary-Katherine Stone, a Democratic congresswoman from Vermont, emphasising the role councils play in closing the gaps in health service.
Future outlook
While progress in telehealth and prescription affordability has improved the national average, the “B” grade masks deep regional divides. Only four states – Colorado, Delaware, Maine, and New Jersey – earned an “A”, underscoring that for most of the US, geography remains a primary barrier to accessing high-quality care for rare disease patients.
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