RDCRN 2026 Annual Report

A Message from the Program Director

Tiina K. Urv, PhD

Rare Diseases Clinical Research Network (RDCRN)
Program Director
Division of Rare Diseases Research Innovation (DRDRI)
National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health (NIH)

There are an estimated 10,000 rare diseases that collectively impact millions of people in the United States. These disorders frequently cause substantial morbidity and mortality. Although our understanding of the causes and mechanisms of rare diseases has advanced considerably, effective treatments are available for fewer than 5% of these conditions.

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Introduction

Who We Are

We are the Rare Diseases Clinical Research Network (RDCRN), a network of research teams, patients, and advocates supported by the National Institutes of Health (NIH), promoting clinical research on rare diseases. Our rare disease research community is made up of consortia studying at least three related rare diseases at sites across the nation.

RDCRN By the Numbers October 1, 2025 – September 30, 2026

198

Diseases Studied

Patient Advocacy Groups

183

Clinical Sites

Publications

219,087

Website Visitors

Protocols Approved

Tools Available

Committees

Early Career Awardees

Patient & Advocacy Impact Stories

Discover how patient advocacy organizations, individuals, families, and community partners are helping advance rare disease research across the Rare Diseases Clinical Research Network. These stories highlight meaningful collaborations, shared experiences, and the impact of patient engagement on research, care, and outcomes.

"Throughout my journey as a rare disease patient, I have often heard the phrases of how resilient and strong we are as a patient population. While most people intend to utilize these phrases as a positive, there is something that is often missed within these assessments— how isolated you can feel while still belonging to a community."

Liz Bovee, Vascular Malformations (ARDVARC)

Read Liz's Impact Story →

"As a patient living with disseminated coccidioidomycosis and co-founder of the Mycology Advocacy, Research & Education (MyCARE) Foundation, my involvement with the Pediatric Fungal Network STudy of Rare Invasive Fungal DisEases in Immunocompromised Pediatric Patients (PFN-STRIDE) and the RDCRN has profoundly influenced both my advocacy and our organization's approach to patient-centered research."

Rob Purdie, Fungal disease (PFN-STRIDE)

Read Rob's Impact Story →

"The RDCRN research has truly impacted our patient advocacy group (PAG) in a multitude of positive ways."

Batten Disease Support, Research, and Advocacy Foundation, Batten disease, neuronal ceroid lipofuscinoses (BDCRC)

Read BDSRA's Impact Story →

"The Spastic Paraplegia Foundation (SPF) has been a founding patient advocacy partner of SP-CERN, helping connect individuals and families affected by hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS) with the RDCRN."

Spastic Paraplegia Foundation, Hereditary spastic paraplegia and primary lateral sclerosis (SP-CERN)

Read SPF's Impact Story →

Consortia Spotlights Across the RDCRN

Explore discoveries and advances from fifteen Rare Diseases Clinical Research Network consortia. Each consortium conducts research focused on specific rare diseases and patient communities, contributing to a broader understanding of rare diseases and helping improve care and outcomes.