Rare Disease Initiative

Rare diseases represent a diverse range of medical conditions that impact small populations around the globe – with the vast majority of conditions lacking any treatments at all. The rare disease community is supported by patient advocates and champions, caregivers, healthcare providers, policy leaders and companies leveraging innovative ideas and their collective expertise and commitment to addressing unmet needs — including education, data sharing, genetic testing, technology, software, and therapeutic developments — to help rare disease patients.

Spearheaded by our Life Sciences practice, our Rare Disease Initiative works with biotech, pharmaceutical, medical technology, and diagnostic companies, their investors, partners, patient advocacy organizations, and other key stakeholders focused on developing solutions for countless rare medical conditions.

On February 5, 2026, Goodwin’s Rare Disease Initiative convened an invite-only, closed door Rare Disease Executive Roundtable in Boston, followed by our annual Rare Disease Networking Reception: two energizing events for the rare disease community that brought together senior executives and visionaries across biotech, patient advocacy, investing, and policy for a candid dialogue and to build community.

A few themes from the Executive Roundtable that resonated with us:

1) Rare Disease Executives from across the spectrum agreed that FDA engagement on rare disease is real and constructive, with signs of FDA exercising flexible approaches, but with a need for that engagement and flexibility to transcend all therapeutic areas and review divisions.

2) Commenters flagged that the concept of regulatory “acceleration” isn’t just about speed and breaking things; rather, the most practical discussions about acceleration are about identifying bottlenecks the government can address or remove, especially around using novel endpoints and biomarkers and alternative models to animal testing.

3) From the investor’s lens, rare disease remains strategically resilient, particularly with rare pediatric disease PRV reauthorization, but shifts in perceived regulatory alignment or uncertainties in written feedback can drive diligence friction and weigh heavily in financing decisions.

4) Commenters highlighted that, even though many rare disease treatments are insulated from pricing pressures (MFN, negotiations, etc.), there remains a shadow over the industry as a result of pricing pressures that creates a sense of uncertainty for investment.

5) Coverage and access remain key focus areas for patients and advocacy groups while successfully navigating the FDA remains top for biotech executives.

A highlight of our Networking Reception, which welcomed over 100 members of the rare disease community to our offices, was featuring Boston artist DaNice D Marshall, whose work captures the urgency and hope of this space. Her talent was a powerful reminder that rare disease progress is about people, families, and lived experience.

We are grateful to everyone who joined us in Boston! If you are interested in joining future Goodwin Rare Disease Initiative events, please reach out.

We want to extend a special thanks to John Bishai (Managing Director, Healthcare Investment Banking, J.P. Morgan), Heidi Bjornson-Pennell (Director, Bi[o]hub), Pamela Gavin (Chief Executive Officer, NORD) & Michael Pearlmutter (Chief Executive Officer, EveryLife Foundation for Rare Diseases) for their contributions to the Executive Roundtable, along with the many biotech, research, and patient foundation leaders who shared candid perspectives.

On Rare Disease Day 2025 Goodwin lit our offices from coast-to-coast in Boston, DC, Santa Monica, and Silicon Valley in Rare Disease Day colors!