The Alliance for Health Policy Releases Insights Report Highlighting Rare Disease Policy Gaps and Opportunities for 2025 and Beyond

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The Alliance for Health Policy Releases Insights Report Highlighting Rare Disease Policy Gaps and Opportunities for 2025 and Beyond

Educational programing and mapping stakeholders in rare disease also part of larger effort

Washington, D.C. — The Alliance for Health Policy today released a new insights report on rare disease policy as part of a program designed to gather policy experts and inform future educational programming. Rare diseases collectively affect an estimated 25 to 30 million people in the United States, about 1 in 10 Americans, and is an area of bipartisan interest. 

A broad range of experts were interviewed for the insights report, identifying key challenges and opportunities in rare disease policy, which sits at the intersection of high patient need, highly complex science, and financial and health system constraints. The report identifies several critical themes, including:

• Access to Care: Timely and affordable access to therapies and specialists remains a significant challenge. Geographic barriers, delays in diagnostics, and treatment inequities require renewed attention.
• Insurance and Affordability: High out-of-pocket costs and complex insurance structures pose significant barriers to care, exacerbating the financial strain on families managing rare diseases. Balancing the urgency of care with the costs of drug development is essential.
• Rapid Diagnostics and Telehealth: Advances in technology offer opportunities to enhance diagnostic speed and access. Telehealth is particularly promising in overcoming geographic and financial obstacles.
• Genetic Testing and Cell and Gene Therapy: While genetic testing and counseling provide critical tools for diagnosis and treatment, including potentially curative solutions through cell and gene therapy, concerns about privacy and data protection must remain a policy priority.
• Incentivizing Research and Innovation: Encouraging investment in rare disease therapies requires robust incentives for pharmaceutical companies to tackle the high costs and limited market potential of such treatments.
• Regulatory Landscape Opportunities: The FDA’s accelerated approval pathway and Rare Disease Innovation Hub are crucial mechanisms in fostering investment and accelerating the delivery of therapies that can improve patient outcomes.

As part of the larger effort, the Alliance also convened trusted thought leaders from Congress, the administration, patient advocacy groups, industry leaders, healthcare providers, and academia to advance solution-driven conversations around rare disease policy. These experts focused on setting priorities for 2025 educational programming, mapping stakeholders engaged in addressing complex issues in rare diseases, and preparing members of Congress and their staff with needed information in this complex field. A report sharing these nonpartisan, cross-sector policy considerations will be released in early 2025.

“We know that the rare disease policy landscape is distinct and raises unique questions for policymakers,” said Claire Sheahan, President and CEO of the Alliance for Health Policy. “By bringing together diverse voices and fostering informed discussions, we aim to illuminate some of the areas most suited for educational focus. The insights memo is our ‘listen first’ approach to harnessing the collective wisdom of Alliance community experts to inform a robust nonpartisan approach to teaching about rare disease policy.”

This initiative on rare diseases builds on the Alliance’s long history of fostering nonpartisan collaboration across sectors to engage and educate the policy community on the most pressing health policy issues.

Read the full report:

About the Alliance for Health Policy

The Alliance for Health Policy is a nonpartisan, nonprofit organization dedicated to advancing the understanding of health policy issues. Through educational events and resources, the Alliance informs policymakers, the media, and the public about health policy issues and promotes evidence-based solutions.

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