Build Relationships With Key Partners

With Other Groups

An umbrella group is an association of groups or institutions who formally pool their resources. Many umbrella organizations, especially in the rare disease community, hold conferences and webinars and can assist with learning about therapy development and ways to combine multi-stakeholder voices to advocate for medical research more effectively. In addition, knowing the different groups involved in your disease space and engaging in the opportunities they offer is one of the best ways to empower your patient voice and become involved in driving research for your disease.

  • Types of opportunities: Groups may offer a variety of resources in different formats. Learning about the groups, their missions, and the types of resources they offer can help you determine which groups may be most important to help accelerate your own mission. The most common types of resources include: 
    • Conferences: Although in-person attendance will be more expensive than watching a livestream, conferences, especially those by umbrella groups, offer ways to meet other groups and network. Scholarships may be available to attend a conference, so don’t be afraid to ask if you are unable to attend due to financial constraints. Advantages of attending include:
      • Learning from other patient groups who are further along in the process than you are.
      • Brainstorming with other patient groups who are facing the same challenges.
      • Mentoring patient groups who may not be as far along in the process as you are. 
      • Gaining insight into what your group can offer to key stakeholders via the conference speakers who may include:
        • Researchers.
        • Clinicians.
        • Representatives from both small and large pharmaceuticals.
        • NIH researchers.
        • FDA staff.
    • Webinars: As you likely know, webinars are seminars available over the internet. Webinars usually are focused on a single topic and last about an hour; they may be easier to incorporate in your busy schedule and may better fit your current needs. 
      • Some webinars are interactive and will take questions, most often through a chat box within the webinar platform.
      • Most webinars are recorded and archived on the group’s website.
    • Web-based materials: Umbrella groups often develop resources (sometimes labeled as toolkits) that cover several facets of a single topic. They may provide templates, checklists, and links to other helpful resources. The group may have special programs that are focused on therapy development or disease research. Taking the time to explore their websites to see all they have to offer can save you time in the long run. 
  • Rare disease umbrella groups: Umbrella organizations focused on rare and genetic diseases and nonprofits focused on therapy development may be a good place to get started. Such groups include:
    • Global Genes : A non-profit organization with a mission to eliminate the challenges of rare diseases by building awareness, educating the global community, and providing critical connections and resources that equip advocates to become activists for their disease. Global Genes holds focused conferences and webinars and offers multiple resources, including RareToolkits, through their website. 
    • National Organization for Rare Disorders (NORD) : A federation of voluntary health organizations helping people with rare diseases through programs of education, advocacy, research, and patient service. NORD offers a membership program for patient groups in rare disease. NORD also holds conferences and webinars, offers special programs to assist with building registries or funding a research project, and provides information on specific rare and genetic diseases. 
    • EveryLife Foundation for Rare Diseases : A nonprofit organization dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. In addition to their many advocacy programs and activities, EveryLife holds an Annual Scientific Workshop that brings together thought leaders to address urgent issues impacting the development process for rare disease treatments. 
    • FasterCures : This “action” tank  center of the Milken Institute is driven by a singular goal: to save lives by speeding up and improving the medical research system. FasterCures focuses on cutting through the roadblocks that slow medical progress by expanding the science of patient input, fostering policies to support biomedical innovation, and spurring cross-sector collaboration in research to get better outputs. FasterCures offers a variety of programs, webinars, reports, and other opportunities to learn more about the therapy development process and practices that can help you speed up the time to your goal. 
    • Genetic Alliance: A non-profit organization that aims to improve health through the authentic engagement of communities and individuals. The organization is committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities. Genetic Alliance’s network includes disease-specific advocacy organizations, universities, private companies, government agencies, and public policy organizations.
  • Disease specific groups: Other patient groups focused on specific diseases can also present opportunities to build relationships that will be invaluable through the therapy development process. 
    • Patient groups for the same disease: In some cases, there is more than one patient group for the same disease. This may occur when the missions of the groups are different. For example, one group may be more focused on supporting patients and their families, while another may be more focused on one specific therapy research effort. 
      • Work together with other patient groups focused on your disease, so that patient data is shared and preferably joined in one place and you aren’t duplicating efforts.
    • Larger parent disease groups:  There may be a larger patient group that includes your disease and other related or similar diseases.The larger group may have more resources available, such as lawyers, an IRB, or industry, government, and research connections. 
      • Working with these larger groups when possible may allow you access to these resources, their experience and expertise, and save both time and money. In return, your efforts may allow them to focus on other diseases that do not have a specific patient group. 
    • Patient groups for related disease: You may find a group focused on a disease with similar symptoms, cause, or disease process. 
      • Forging a relationship can prevent needless competition for scarce researchers, funding, and industry interest.
    • Patient groups willing to mentor: Many who have been involved in the therapy development process are happy to share their journey with others. Although no two paths are exactly the same, you can take advantage of the time and effort that will be saved by listening to their experiences. 
      • Watch for news about a patient group who has a therapy headed to clinical trials or just approved, and then contact their lead staff members to set up a time to talk about their process.


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)