EveryLife Foundation Scientific Workshop Advances Patient-Centered Approaches to Rare Disease Therapy Development

WASHINGTON, May 14, 2026 /PRNewswire/ -- On Tuesday, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened its biennial Scientific Workshop titled, "Making What Matters Count: Advancing Patient-Centered Rare Disease Therapy Development Methodologies, Tools, and Knowledge Management." This interactive workshop brought together patient advocates, biopharmaceutical companies, academic researchers, and federal agency leaders from across the rare disease ecosystem to advance practical, science-driven policy solutions.

First established in 2010, the Scientific Workshop series serves as a forum for the rare disease community to address urgent challenges at the intersection of science and policy. This year's workshop focused on strengthening the application of patient-centered approaches in rare disease therapy development amid a rapidly evolving regulatory landscape.

With significant advancements in regulatory science over the past two decades, participants examined how new and existing U.S. Food and Drug Administration (FDA) pathways, guidances, and programs are being applied across the more than 10,000 known rare diseases — highlighting both progress and persistent gaps in consistency, implementation, and access.

"Ensuring that patient experience is meaningfully and consistently integrated into therapy development requires not just policy changes, but alignment, coordination, and actionable tools," said Jamie Sullivan, Senior Vice President of Policy and Advocacy for the EveryLife Foundation. "This year's discussion was designed to help us continue to identify concrete solutions that can be implemented across the ecosystem. We are grateful to everyone who attended this week's workshop, and we appreciate the productive conversations."

Key outcomes and focus areas from the workshop included:

• Assessing the effectiveness of current regulatory pathways, guidance documents, and policy frameworks in supporting rare disease product development.
• Identifying opportunities to translate successful case studies into scalable, repeatable practices across diverse rare disease contexts.
• Developing strategies to improve knowledge management and dissemination of regulatory and scientific insights to enhance consistency in product evaluation.
• Recommending concrete policy and legislative actions to address persistent gaps and barriers.

Discussions also underscored the growing complexity of the regulatory environment, with numerous new FDA guidances, pilot programs, and initiatives introduced in recent years. While these tools offer unprecedented opportunities to accelerate development, participants emphasized that variability in awareness, interpretation, and application continues to limit their full impact, particularly for smaller or less-resourced patient communities.

In the coming weeks, the EveryLife Foundation will synthesize key findings and recommendations from the workshop to guide its ongoing policy and advocacy efforts, with the goal of advancing a more consistent, transparent, and patient-centered framework for rare disease therapy development.

About the EveryLife Foundation for Rare Diseases:

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization powered by the rare disease community to improve health outcomes by driving change through evidence-based policy, leading science-driven policy and regulatory research, activating the community to advocate for their rights and needs, and strengthening the rare disease community.

To learn more, visit EveryLifeFoundation.org and follow us on Facebook, X, Instagram, and LinkedIn.

SOURCE EVERYLIFE FOUNDATION FOR RARE DISEASES