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Evan Muse, M.D., Ph.D., giving a presentation.

NCATS-Funded Researchers Apply Precision Medicine to Digital Health

Investigators from the Scripps Translational Science Institute advance precision medicine to combat a dangerous heart condition through improved screening and diagnostic approaches.

grdr boy on swing

New Funding Opportunities Available for Rare Diseases Research

NCATS has released two new funding opportunities to support research on rare diseases through the Rare Diseases Clinical Research Network.


Potential Gene-Editing Treatment Advances to Clinical Trials

RDCRN-supported researchers charted how several rare diseases progressed in patients over time, helping to enable a potential gene-editing therapy to advance to clinical trials.

Work with Us

NCATS aims to address common barriers in clinical and translational research that scientists nationwide face every day, with the goal of delivering more treatments to more patients more quickly.

Contact the Division of Clinical Innovation (link sends e-mail)

Clinical Innovation

NCATS supports innovation in new technologies and other clinical and translational approaches that may greatly accelerate the process of transforming laboratory discoveries into new treatments for patients. 

Find NCATS Programs & InitiativesAccess NCATS Expertise & Resources

Clinical & Translational Science Awards (CTSA) Program

The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care.

Rare Diseases Clinical Research Network (RDCRN)

The RDCRN is designed to advance medical research on rare diseases by facilitating collaboration, study enrollment and data sharing.

Patient Registries

Patient and contact registries are key tools that help researchers identify potential clinical study participants, including health volunteers.

Clinical Research Toolbox

NCATS supports clinical research tools that facilitate trial design, patient recruitment and partnerships for commercialization.

Clinical Trials

NCATS programs support a broad range of clinical research, including clinical trials. NCATS authorization (PDF - 133KB) limits specific support for clinical trials only through the end of Phase IIA.

Phase II clinical trials are designed to test drugs for efficacy (or effectiveness) and side effects in a limited number of patients. Phase IIA trials provide data for exposure-response in patients, while Phase IIB trials provide data for dose-ranging in patients.

Addressing Clinical Research Challenges

Researchers nationwide face common barriers in clinical and translational research that can delay the development of new interventions for patients in need. These challenges include:

  • Difficulties in recruiting, mentoring and retaining a critical mass of qualified clinical and translational investigators
  • Increased research costs and complexity
  • Low patient recruitment and retention in clinical research studies
  • Inflexible study designs
  • Increased regulatory burdens

NCATS supports innovative programs, such as the Clinical and Translational Science Awards (CTSA) Program and Rare Diseases Clinical Research Network (RDCRN), to help address these challenges.