Rare Disease Month Developments – Part 3: The Ugly (just kidding) – See You at Rare Disease Week

If Part 1 of our Rare Disease Month series highlighted The Good, and Part 2 addressed The Bad, Part 3 turns to The Ugly. And by “Ugly,’ we mean something entirely different – members of Hyman, Phelps & McNamara will be well represented at Rare Disease Week (RDW) on Capitol Hill next week, convened for the 15th consecutive year by the EveryLifeFoundation for Rare Diseases.

For the uninitiated, RDW brings together patients, advocates, policymakers, regulators and industry stakeholders for several days of programming focused on advancing policy solutions for the rare disease community. This year’s events arrive amid continued attention on regulatory flexibility, patient access, and opportunities to improve development pathways for therapies targeting rare conditions.

Honoring Frank Sasinowski’s Rare Disease Leadership

On the morning of Wednesday, February 25th, during the RDW Legislative Conference, our very own Frank Sasinowski will be honored with the EveryLife Foundation’s “Abbey” Lifetime Achievement Award, recognizing decades of leadership and contributions to rare disease drug development and regulatory policy. Named for pioneering advocate Abbey Meyers, the award is not presented annually but is given only when EveryLife determines that an individual has distinguished themselves through a career of service to the rare disease community. The most recent prior recipient was Dr. Francis Collins, who served as the 16th Director of the National Institutes of Health (NIH), in 2021.

Frank’s influence across the rare disease space is well known to readers of this blog (and beyond). Over the course of his career, he has helped shape how FDA evaluates therapies intended for small patient populations, advancing regulatory flexibility and championing development approaches tailored to rare diseases. His work has contributed to hundreds of individual product approvals and to the evolution of regulatory approaches that have enabled patients with rare diseases to gain access to therapies that might otherwise never have reached them. This award is extremely well-deserved, and we are proud to call Frank one of our own.

The recognition also coincides with a transition in leadership at the EveryLife Foundation. Frank recently concluded nine years of service on the Foundation’s Board of Directors, including service as Chair and Vice Chair during that period, stepping down at the end of his term in December 2025.

Passing the Torch: Continued Leadership at EveryLife

As Frank concludes nearly a decade of board leadership, the work continues with new voices stepping into governance roles. Earlier this year, James Valentine, Director at Hyman, Phelps & McNamara, began his term as a Board Member of the EveryLifeFoundation.

James’s appointment reflects a continuity familiar to many in the rare disease community. Having worked together for many years on regulatory and policy initiatives, this transitionunderscores a broader theme of this year’s RDW: progress in rare disease policy depends not only on institutional change but on sustained leadership passed from one generation of advocates and advisors to the next.

Legislative Conference Panel: Opportunities to Reshape the Regulatory Environment

RDW programming also includes a number of policy and regulatory discussions worth following closely. On Wednesday, February 25, James will participate in the panel “Opportunities to Reshape the Regulatory Environment,” alongside Annie Kennedy of the EveryLife Foundation, Ryan Fischer of the Foundation for Angelman Syndrome Therapeutics, and Emily Milligan of the Barth Syndrome Foundation.

The panel will address current regulatory challenges and emerging opportunities to improve development pathways and patient access for rare disease therapies. Sessions such as these have increasingly become important venues for dialogue among patient organizations, policymakers, and regulatory experts working toward practical solutions.

Rare Disease Congressional Caucus Briefing: “Rare Disease: The Time is Now”

On Thursday, February 26th, James will moderate the Rare Disease Congressional Caucus briefing titled “Rare Disease: The Time is Now.” The briefing will follow the Senate Special Committee on Aging’s hearing examining FDA regulatory processes, rare disease treatment delays, and opportunities to improve patient access to innovation. The alignment of these events underscores growing Congressional focus on how regulatory systems can better support timely development and availability of therapies for rare disease patients.

Other RDW Events to Watch

Other important events next week include FDA’s Rare Disease Day public meeting on Monday, February 23rd, exploring how FDA can better engage and collaborate with patients and their communities to support and accelerate development of medical products for rare diseases, and the NIH’s Rare Disease Dayevent on Friday, February 27th, bringing together researchers, clinicians, patients and advocates to discuss scientific progress and future research priorities.

A Week for Advancing Stakeholder Dialogue

RDW remains one of the few opportunities each year for patients, sponsors, regulators, advocates, and policymakers to convene in a single forum focused exclusively on rare disease challenges and solutions. The discussions taking place next week are expected to help advance ongoing stakeholder dialogue across regulatory and legislative fronts.

These authors, along with our colleagues across Hyman, Phelps & McNamara, congratulate our colleague, Frank Sasinowski, on this well-deserved lifetime achievement recognition and recognize James Valentine’s continued leadership and engagement in advancing rare disease regulatory policy discussions. We also extend our appreciation to the many sponsors, patient organizations, and advocates with whom we have the privilege to work, whose ongoing efforts continue to drive the development of therapies for patients and families across the rare disease community.