Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting Resource Library

Bringing the Patient Voice Forward in Rare Disease Drug Development

The U.S. Food and Drug Administration (FDA) recognizes that patients and caregivers have unique insights into the experience of living with a disease—including the symptoms, daily challenges, treatment experiences, and outcomes that matter most to them. Incorporating this perspective into drug development can help inform research and support the development of treatments that address meaningful patient needs.

Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings are part of the FDA’s patient-focused drug development initiative. These meetings provide an opportunity for patients, caregivers, healthcare professionals, researchers, industry, and other stakeholders to share and learn from the patient experience. The discussions can help identify what matters most to patients and caregivers and provide important context for drug development and regulatory decision-making.

NORD has partnered with rare disease organizations and other stakeholders to help organize and facilitate EL-PFDD meetings focused on specific rare diseases and patient communities. This resource library brings together those meetings and the resources generated from them, including meeting recordings, presentations, reports, and other materials when available.

What You’ll Find in the Library

Each EL-PFDD meeting offers an opportunity to explore the patient experience, including:

• Disease burden: How the disease affects daily life, physical and emotional well-being, relationships, work, school, and other activities
• Symptoms and impacts: Which symptoms and challenges are most significant to patients and caregivers
• Treatment experiences: What patients value about current treatments, as well as their limitations and challenges
• Unmet needs: Where existing treatment options fall short and where additional therapies are needed
• Patient priorities: The outcomes and changes that patients and caregivers consider most meaningful
• Perspectives for drug development: Insights that can help researchers, drug developers, and regulators better understand what matters to the people they seek to serve

Why EL-PFDD Meetings Matter

Patient experience data can provide important context throughout the drug development process. By bringing patients and caregivers together to share their experiences directly, EL-PFDD meetings can help make the patient perspective more visible in discussions about research, clinical development, treatment benefit, and regulatory decision-making.

Explore the library below to learn from the experiences and priorities shared by rare disease communities through EL-PFDD meetings NORD has helped organize with our partners.