NCATS Toolkit for Patient-Focused Therapy Development

NCATS is dedicated to engaging the patient community throughout the translational science process. The NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) was created to provide a collection of online resources that can help patient groups advance through the process of therapy development and provide them with the tools they need to advance medical research.

Launched in September 2017, the Toolkit includes resources that have been developed primarily for the rare diseases community to facilitate therapeutics research and development. Since early 2016, NCATS has worked with a diverse group of partners in the rare diseases community to conduct an extensive landscape analysis of available tools. These resources were defined, characterized and organized in a centralized portal that can be helpful to all patient groups regardless of how far along in the research and development process they might be.

Tools include:

  • How to establish a patient registry;
  • How to drive patient-focused discovery and pre-clinical research and development;
  • How to work with NIH and the Food and Drug Administration; and
  • How to conduct post-market surveillance.

Learn more about the NCATS Toolkit for Patient-Focused Therapy Development.

NCATS Rare Diseases Research Video

Right-click to download a transcript (4KB)

NCATS’ latest video features an overview of the Center’s robust rare diseases research programs, initiatives and resources. Narrated by Petra Kaufmann, M.D., M.Sc., former director of the NCATS Office of Rare Diseases Research, the goal is to increase awareness about NCATS’ work to address the unique struggles rare disease patients and their caregivers face.