Annie Kennedy | Chief Mission Officer
EveryLife Foundation for Rare Diseases

A veteran leader in the rare disease patient focused drug development movement, Annie joined the EveryLife Foundation in 2018, where she’s led numerous, community-driven evidence development efforts including the National Economic Burden of Rare Disease study, The Guide to Patient Involvement in Rare Disease Therapy Development, and advocacy efforts leading to the establishment of FDA’s Rare Disease Innovation Hub. Annie previously held leadership roles at Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA) where she led landmark legislative, regulatory, newborn screening, transitions, and access policy efforts including the MD CARE Act, and the Patient Focused Impact Assessment Act (PFIA), which became the Patient Experience Data provision of the 21st Century Cures Act. She is a sought-after advisor to patient-centered organizations and initiatives across the nonprofit and government sectors, and in 2024 was named by Women We Admire as one of the Top 50 Leaders in Washington, DC. Annie’s community roles have included service on the Board of Directors of Cure SMA, the National Duchenne Newborn Screening Steering Committee, the NIH Strategic Planning Working Group on Engaging the Public as Partners in Clinical Research, and as a member of the NIH NCATS Advisory Council. She currently serves on the Boards of Directors of Patient Focused Medicine Development (PFMD) and the TREAT-NMD Neuromuscular Network.

Appearances:

Day 1 - World Orphan Drug Congress USA 2026 @ 09:10

Building a Patient-Centered Policy Framework for Rare Disease Drug Development

Session Objectives:

Define what a patient-centered policy framework entails in the context of rare disease drug development.
Explore mechanisms for embedding patient perspectives into every stage of the regulatory and policy decision-making process.
Examine existing policy gaps that limit timely diagnosis, research funding, and equitable access to orphan drugs.
Discuss cross-sector collaboration opportunities between patient organizations, regulators, industry, and payers to shape sustainable policy reform.
Chart actionable next steps for integrating patient input into evolving rare disease frameworks at national and global levels.