Build Relationships With Key Partners

With Government

The ability to connect with FDA in meaningful ways continues to increase thanks to those patient group leaders who blazed the trail before you. The different opportunities to connect to the FDA are covered in more detail in subsequent sections, but a few resources that provide overviews are highlighted here. In addition, you will want to identify the NIH Institutes and Centers (ICs) that may be focused on research related to your disease. Building relationships with relevant NIH ICs increases your awareness of grants, special workshops, and other opportunities that may help you accelerate developing a therapy for your disease. 

  • FDA: The FDA recognizes that patient perspectives are primary, and that these perspectives might differ significantly from what would be expected by clinicians and researchers who do not experience the challenges of living with the disease. This is particularly true in the context of rare diseases. Therefore, the FDA recommends early and continued contact as your group begins the process of therapy development.
    • Resources with information about ways to connect to the FDA include:
      • For Patients is a website developed by the FDA Patient Affairs staff to help patients and patient group leaders learn about therapy approvals, public meetings, special opportunities to connect with the FDA, and more. 
      • Learn About FDA Patient Engagement is a summary of organizations and opportunities at the FDA that incorporate patient involvement. Opportunities include the FDA Patient Network, a Patient Engagement Initiative, and the Patient Representative Program. 
      • Developing Products for Rare Diseases & Conditions explains the mission of the FDA Office of Orphan Products Development (OOPD) as well as the different programs that may be available to help with the development of therapies for rare diseases. The contact information for the OOPD is also provided.
      • FDA Patient Affairs Staff coordinates and supports patient engagement activities across the FDA to facilitate awareness and collaboration with patients, patient groups, and the FDA. Staff can be contacted directly at (301) 796-8460 or .
    • To identify what is most important to patients, the FDA has multiple patient focused initiatives. Each of these programs is described in more detail in sections of Discovery: Collect Patient Experience Data. For example, the FDA:
      • Hosts internal Patient-Focused Drug Development (PFDD) meetings.
      • Assists with organization of external PFDD meetings.
      • Supports the development of benefit-risk analysis in the form of clinical outcome assessments (COAs).
      • Offers grants for natural history studies and clinical trials focused on rare diseases. 
    • Other resources to learn more about engaging with the FDA include:
    • It is important to remember that although the FDA is open to and encourages the involvement of patient groups and patients, it is a federal regulatory agency focused on public health safety and has rules and regulations it must follow when considering approval of a therapy. 
  • NIH: The different ICs at NIH may provide opportunities to connect your researchers and clinicians with specialists at the NIH. Many of the ICs offer grants or engagement opportunities for patient groups. Finding the ICs that may be involved in research for your disease early in the process will allow you to use their resources to their fullest.
    • List of NIH Institutes, Centers, and Offices can help you learn more about the 27 ICs at NIH. Each has its own specific research agenda, often focusing on particular diseases or body systems.
      • Many rare and complex diseases lacking treatments affect multiple systems and therefore may fall under more than one IC. 
      • If you are uncertain whether you found the right IC, use the contact information to call and find out. 
      • Find NIH Clinical Center Trials is a registry of publicly supported clinical studies conducted mostly at the NIH Clinical Center. Searching for studies for your disease or similar, more common diseases may help you identify NIH researchers and Institutes with knowledge about your disease.
    • The National Center for Advancing Translational Sciences (NCATS) was established to transform and accelerate the translational research process. NCATS collaborates with researchers, the public, and other stakeholder groups to design new approaches and technologies that ultimately will deliver more treatments to more people more quickly. NCATS:
      • Complements other NIH ICs, the private sector, and the nonprofit community.
      • Focuses on research that can broadly be applied to other diseases. 
      • Supports multiple programs focused on rare diseases through ORDR including NCATS Toolkit. 


With Academic Researchers and Clinicians
PubMed National Library of Medicine (NLM) (link)
GeneReviews National Library of Medicine (NLM) (link)
NORD Rare Disease Information National Organization for Rare Disorders (NORD) (link)
UpToDate Wolters Kluwer (link)
Research Portfolio Online Reporting Tool (RePORTER) National Institutes of Health (NIH) (link) National Library of Medicine (NLM) (link)
Find a Genetic Counselor National Society of Genetic Counselors (NSGC) (link)
Find a Genetics Clinic American Medical College of Genetics and Genomics (AMCG) (link)
Rare Diseases Clinical Research Network (RDCRN National Center for Advancing Translational Sciences (NCATS) (link)
With Government
FDA For Patients Website U. S. Food and Drug Administration (FDA) (link)
Learn About FDA Patient Engagement U. S. Food and Drug Administration (FDA) (link)
Developing Products for Rare Diseases & Conditions U. S. Food and Drug Administration (FDA) (link)
FDA Patient Affairs U. S. Food and Drug Administration (FDA) (link)
Working with Regulators: A Focus on the FDA Cancer Policy Institute at the Cancer Support Community (link)
List of NIH Institutes, Centers, and Offices National Institutes of Health (NIH) (link)
Find NIH Clinical Center Trials National Institutes of Health (NIH) (link)
NCATS Patient/Community Engagement & Health Information National Center for Advancing Translational Sciences (NCATS) (link)
Tips for Success
Best Practices for Effective Patient Group Engagement Clinical Trials Transformation Initiative (CTTI) (link)