Community Congress is a membership-based program dedicated to bringing patient organizations, industry leaders, and other rare disease stakeholders together. Community Congress acts as a coalition of collaborators with shared priorities, providing strategic guidance and insight on policy issues and initiatives.
It comprises four permanent working groups that meet at least quarterly to advance federal and state legislative and regulatory policy solutions for the rare disease community.
All working groups utilize a legislative policy grid to organize and prioritize legislative matters. For additional explanation, please see Legislative Priorities Reference Guide.
2026 Working Groups
Public Policy
Mission: Focused on responding to and shaping legislative policy impacting rare diseases in order to close the innovation gap.
Maynard Friesz
Vice President
Cure SMA
Gina Cioffi Loud
Lead Manager, Public Affairs
Global Rare Diseases
Chiesi Group
Areas of Focus
- Initiatives for policy change at the state and federal level
- Incentives for therapy development (Orphan Drug Tax Credit)
- Appropriations priorities that support funding for research, newborn screening and other federal rare disease initiatives
- Responsible for formulating and updating the federal legislative priority grid each year
Examples of policy activity informed by the Public Policy Working Group:
Newborn Screening & Diagnostics
Mission: Focused on reducing and eliminating the diagnostic odyssey.
Danae Bartke
Executive Director
HCU Network America
Andrew Hebert
Director, Therapeutic Area Policy and Advocacy
Takeda
Areas of Focus
- State efforts to screen for all RUSP conditions
- Modernize the newborn screening system
- Expand coverage of and access to a wide range of genetic tests and genetic counseling services
Examples of policy activity informed by the Newborn Screening & Diagnostics Working Group:
Regulatory
Mission: Focused on enhancing regulatory infrastructure and processes, as well as advancing regulatory science for rare disease products.
Ryan Fischer
Chief Operating Officer
Foundation for Angelman Syndrome Therapeutics (FAST)
Jewell Martin
Director for US R&D and Regulatory Policy
BioMarin Pharmaceutical
Areas of Focus
- Concordance within and across FDA centers and divisions
- Earlier engagement between regulatory and access stakeholders
- Monitor implementation of user fee agreements and relevant legislation
- Strengthen and protect the accelerated approval pathway
- Enhance consistency, transparency, and flexibility of the regulatory review process
Examples of policy activity informed by the Regulatory Working Group:
Access & Value
Mission: Focused on ensuring patient access to safe and efficacious, approved treatments at the earliest moment possible.
Parisa Sanandaji
Executive Director and Head of Global Patient Advocacy & Policy
Stoke Therapeutics
Brigid Brennan
Board Director, General Counsel, and Director of Advocacy
Friedreich’s Ataxia Research Alliance
Areas of Focus
- Timely and equitable access to rare disease care and FDA-approved therapies
- Patient and rare disease expert representation in payer decision-making
- Protections from inappropriate utilization management practices
- Patient-centered value assessment development and adoption
Examples of policy activity informed by the Access & Value Working Group:
- IVI and EveryLife Foundation’s Rare Disease Initiative Final Report on Valuing Rare Disease Treatments in Healthcare
- A Blueprint for Developing Patient-Centered Core Impact Set (PC-CIS)
- Position Paper – Value for Whom? Incorporating Patient Perspectives into Value Assessment
- ICD Code Roadmap
- Accelerated Approval Resources
- DUR and P&T Wishlist
- ERISA RFI
Community Congress Meetings
All meetings are members only. If you are interested in joining a meeting and are not yet part of the working group or a community congress member, please contact communitycongress@rareadvocates.org.
2026 Winter Calls – Concluded
- Public Policy – Concluded.
- Newborn Screening – Concluded.
- Regulatory – Concluded.
- Access & Value – Concluded.
2026 Spring Calls – Concluded
- Newborn Screening – Concluded.
- Regulatory – Concluded.
- Public Policy – Concluded.
- Access & Value – Concluded.
2026 Midyear Virtual Webinar – Concluded
Concluded.
2026 Fall Calls:
- Regulatory Working Group – Tuesday, September 22, 2-3 PM ET
- Newborn Screening – Friday, October 2, 1-2 PM ET
- Access & Value – Thursday, October, 15, 2-3 PM ET
- Public Policy – Tuesday, November 10, 1-2 PM ET
2026 Annual In-Person Meeting:
Annual in-person meeting will take place on Thursday, December 10 in Washington, DC.
Active Ad-Hoc Discussions
FDA Guidance Book Club
- Topic: Review of the Draft Guidance for Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products
- Overview: Will be discussing key takeaways from the latest guidance from the FDA
- Upcoming Meeting: Thursday, August 27, 3:00–4:00 PM ET
PDUFA VIII Reauthorization Ad-Hoc
- Topic: FDA User Fee Reauthorization & Rare Disease Priorities
- Overview: This ad-hoc series focuses on the upcoming PDUFA VIII reauthorization process, including discussions related to regulatory flexibility, patient-focused drug development, Rare Disease Innovation Hub activities, early policy priorities, and opportunities for community engagement throughout the legislative cycle.
- Upcoming Meetings: Ongoing throughout 2026
ICD-10-CM Coding Ad-Hoc
- Topic: Rare Disease ICD-10-CM Classification & Coding
- Overview: This ad-hoc group is focused on ongoing discussions related to ICD-10-CM coding challenges for rare diseases, including the CDC request for comments on organizing principles for ultra-rare conditions and gene-level specificity, the MONDO proposal, and broader efforts to improve rare disease code infrastructure and classification processes.
- Upcoming Meetings: Additional discussions anticipated Spring/Summer 2026
