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About NCATS Toolkit

Our toolkit (NCATS Toolkit) provides information and resources about the growing role of patient voices from rare disease communities in treatment development.

About NCATS Toolkit 

The tips, strategies and many of the resources are presented from the perspective of rare disease patient group leaders with the goal of empowering these groups to actively participate in each stage of the process. The level of participation of patient groups in the therapy development process varies for many reasons, including the level of resources and experience of the group. One of the goals of NCATS Toolkit is to help even young, newly forming groups with limited resources and experience find ways to have a voice in the process.      

What is the NCATS Toolkit? 

NCATS Toolkit is a website that provides rare disease patient group leaders, like you, with reliable information about the stages of the therapy development process and the ways your group can become involved. The ultimate goal is to help your group become a driving force behind the development of therapies and/or a cure for your disease(s). In fact, of the 7,000-plus recognized rare diseases, only about 5% have a U.S. Food and Drug Administration (FDA)–approved treatment. Involving the patient voice in the therapy development process is recognized by the FDA, researchers and pharmaceutical industry as a way to increase the pace of therapy development for rare diseases. 

Where do I start? 

Getting Started provides information that can help a patient group become involved in the therapy development process. The remaining sections provide a more in-depth look into the different stages of treatment development, including what happens after a therapy is approved by the FDA. The tips and strategies in these sections highlight ways patient groups, like your own, can add vital information and resources to the process. You may also find the Glossary helpful. 

The organization of the NCATS Toolkit may make the process of therapy development seem like a series of steps to be followed one after the other, but it is not a straight line from start to finish. In fact, at any given time, different potential therapies for your disease may be at different stages of development. Restarting the process is not uncommon, as not every promising therapy is found to be safe and effective. In addition, not every task or research tool will be needed for every disease. Therefore, we suggest skimming through the website to decide which areas may be important to the development of a therapy for your disease, and then reading those sections and their listed resources in depth. 

Do you perform research? 

NCATS Toolkit is solely an information website and is not involved in research.

Is there information for patients and their families? 

NCATS Toolkit focuses on patient groups, and the tips and strategies were written with patient group leaders in mind. However, the information about how a therapy is developed for a rare disease may be helpful to any person or family affected by a rare disease. If you are a patient who has a genetic or rare disease or a family member or caregiver and would like to support the efforts for the development of a therapy for your disease, the NCATS Toolkit has two topic areas that can help get you started: “Finding a Group” and “Starting a Patient Group.” In addition, NCATS has another program, the Genetic and Rare Diseases Information Center (GARD), that is directed towards individuals, families and caregivers: About GARD. The GARD website provides access to information about rare or genetic diseases. If you can’t find the information you need on the GARD website, you may contact a GARD Information Specialist, who can direct you to disease information, research studies, clinical trials, patient advocacy organizations (supportive resources), medical journal articles and genetic services.

Where do the resources and content come from? 

The resources on the NCATS Toolkit are collected from rare disease patient groups, academia, industry and government agencies, such as the National Institutes of Health (NIH) and the FDA. The content is developed using the listed resources for that topic area and by engaging with key stakeholders at conferences and through one-on-one meetings and informal conversations.      

The information and resources in the NCATS Toolkit are intended to help you better target your conversations and efforts. NCATS Toolkit is not intended to replace talking with your own Board of Directors and Medical/Scientific Advisory Boards, other patient group leaders, rare disease umbrella organizations, the FDA, researchers, industry and other stakeholders. 

Can I contribute resources?

NCATS Toolkit welcomes your patient group to submit resources that you found helpful at any stage of the therapy development process by emailing NCATS Toolkit.

Learn more about our list of goals, what kinds of resources might helpful, and our review criteria for evaluating resources to be included in the NCATS Toolkit.

What is the history of the website? 

NCATS Toolkit was created in response to requests from rare disease patient groups to develop a resource repository to help other patient groups become more involved in the therapy development process. Leading a working group of NCATS staff and various patient groups for nearly two years, NCATS Toolkit co-chairs Ronald J. Bartek (co-founder of the Friedreich’s Ataxia Research Alliance) and Annie Kennedy (senior vice president of legislation and public policy for Parent Project Muscular Dystrophy) identified and evaluated existing resources. The original NCATS Toolkit was launched September 8, 2017. In 2019, in response to feedback from patient groups, content and resources were added and the website was restructured. NCATS Toolkit was relaunched on Rare Disease Day 2020.

What’s next for the NCATS Toolkit?

The role of the patient groups in therapy development continues to expand. NCATS Toolkit is meant to be a living website that will add new content as it becomes available and as the therapy development process adapts to scientific advancements and regulatory changes.

Who oversees the development of the website?

Dr. Eric Sid oversees the NCATS Toolkit management and development team.

Eric Sid, M.D., M.H.A.  
Program Officer  
National Institutes of Health  
NCATS Division of Rare Diseases Research Innovation


What can the NCATS Toolkit do better?

Provide Your Feedback 

Please use our feedback form to submit comments on existing information or resources. You can also let us know if there are topics or areas you would like to see added. Your feedback will help us improve the NCATS Toolkit for the whole rare disease community.

Request Assistance or Submit Resources

You can use the contact form for the GARD Information Center to reach an Information Specialist if you need help navigating the NCATS Toolkit. You can also suggest the addition of a resource your group found helpful. Or you may wish to share resources your group developed, such as a meeting agenda template or a checklist for reviewing clinical trials to post on your website. Thank you in advance for helping us keep the NCATS Toolkit up to date.

Last updated on April 20, 2024