Statement from Pam Gavin, President and Chief Executive Officer
NORWELL, Mass., October 14, 2026 — The National Organization for Rare Disorders (NORD®) applauds the U.S. Food and Drug Administration (FDA) for launching an initiative to develop a new regulatory framework tailored to the scientific and practical challenges of rare disease drug development and review.
This is an important and welcome step forward for the rare disease community. We congratulate FDA leadership for recognizing that extraordinary advances in rare disease science must be matched by continued evolution in how therapies are evaluated. We are particularly encouraged by the agency’s responsiveness to the needs of the rare disease community, including concerns NORD and other patient organizations raised directly with the Acting Commissioner in June. For patients and families who have waited years, sometimes generations, for treatment options, this commitment offers meaningful hope for progress.
Since its founding in 1983, NORD has championed policies that advance rare disease research and therapeutic development. That same year, the Orphan Drug Act helped transform the landscape of rare disease innovation by establishing incentives to develop treatments for conditions long overlooked. More than four decades later, scientific advances have created unprecedented opportunities, underscoring the need for a regulatory framework that keeps pace with innovation and addresses the distinct challenges of rare diseases.
Small patient populations, limited natural history data, disease heterogeneity, and the absence of established biomarkers or clinical endpoints can make traditional approaches to clinical development difficult or, in some cases, infeasible. A regulatory framework that consistently accounts for these realities, while maintaining rigorous standards for safety and effectiveness, is essential to translating scientific discoveries into treatments.
NORD particularly welcomes the FDA’s emphasis on strengthening consistency and predictability across review divisions, incorporating patient perspectives throughout development and review, drawing upon external scientific and clinical expertise, and building the institutional knowledge necessary to support sound regulatory decision-making. Sustained leadership, adequate staffing and resources, and consistent implementation across the agency will be essential.
What makes this initiative especially important is the opportunity to move beyond case-by-case regulatory accommodations toward a framework designed around the scientific realities of rare diseases. When traditional clinical trial approaches are not feasible, we need scientifically rigorous ways to evaluate other forms of evidence. This is not about lowering standards. It’s about ensuring that the methods used to demonstrate safety and effectiveness are appropriate for the diseases and treatments being evaluated.
NORD looks forward to participating in the FDA’s stakeholder engagement process and working alongside the agency and the broader rare disease community to help shape this important initiative.
We also look forward to welcoming FDA leadership, including the Acting Commissioner and representatives of the FDA’s Rare Disease Innovation Hub, to NORD’s 2026 Rare Diseases and Orphan Products Breakthrough Summit®, October 25–27 in Washington, D.C. The Summit will provide an important early opportunity for the rare disease community to engage directly with FDA leadership and explore the framework and its implications in greater detail.
The measure of success will ultimately be whether this commitment translates into more consistent regulatory practices, more effective pathways for developing therapies, and more treatment options for people living with rare diseases.
NORD stands ready to work alongside the FDA and the rare disease community to turn this commitment into meaningful progress and ultimately, more safe and effective treatments for the millions of individuals and families who are counting on it.
