Additional Rare Diseases Research and Initiatives
NCATS supports rare diseases research by funding a variety of extramural research programs and taking part in initiatives and activities aimed at addressing the public health crisis presented by rare diseases.
On this page:
See our Gene Therapies and Gene Editing Programs page to learn about NCATS’ role in accelerating gene-targeted therapies for rare diseases.
Extramural Rare Diseases Research Programs
Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases
Research teams funded by Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases grants will adapt the shared molecular etiology approach from oncology to rare diseases.
Contact: P.J. Brooks, Ph.D.
Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes
The Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes grants support projects focused on collecting the data needed to move promising rare disease therapies and diagnostics to clinical trial.
Contact: Alice Chen Grady, M.D.
Multidisciplinary Machine-Assisted Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey
NCATS is funding innovative research using a variety of tools and approaches to shorten the time it takes to identify and accurately diagnose rare diseases.
Contact: Alice Chen Grady, M.D.
Other NCATS Rare Diseases Activities and Initiatives
Impact of Rare Diseases on Patients and Healthcare Systems (IDeaS) for Change
The IDeaS study, led by NCATS, provided new evidence of the potential effect of rare diseases on public health, suggesting that nationwide medical costs for individuals with rare diseases are similar to costs for people with cancer or heart failure.
Download the IDeaS study fact sheet.
Contacts: Ainslie Tisdale
Shared Molecular Etiologies Task Force
NCATS participates in the International Rare Diseases Research Consortium (IRDiRC) Task Force on Shared Molecular Etiologies Underlying Multiple Rare Diseases. The Task Force will address and document challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare diseases.
Contact: P.J. Brooks, Ph.D.