Determine Patients’ Needs

Tips for Success

Tips for patient registries and natural history studies:

  • Anticipate popular misconceptions and be prepared to alleviate concerns or unrealistic expectations. Being in a registry:
    • Does not obligate a person to join a clinical trial if they are contacted.
    • Does not guarantee that a person will be asked or able to join a clinical trial.
    • Does not ensure a new treatment or cure will be developed.
    • Does not promise any benefit from the registry in the patient’s lifetime.
  • Address your disease community’s concerns about privacy and security early in the process.
  • Educate your disease community about short- and long-term goals of the registry.
  • Highlight the added benefits, for example, finding others who have the same challenges you are experiencing but that may not be published in the medical literature.
  • Empower your patient community to help direct the therapy development process for their disease. 
  • Keep those in the registry engaged in the process so that the data continues to grow. 
  • Celebrate achievements.

Tips for externally-led PFDD Meetings:

  • Because the information produced by externally led PFDD meetings has the potential to affect all stages of the therapy development process, many groups hold PFDD meetings long before there is a possible therapy heading to clinical trials. 
  • PFDD-style sessions can be held during annual conferences, scientific workshops, etc.
  • Begin outreach early and go beyond your own group’s membership, potentially reaching out to social media groups, clinics, and medical professionals serving the patient population, and larger umbrella organizations.
  • Raise funds or find Sponsors to provide financial travel assistance to patients and their families.
  • Organize transportation from key centers if possible.
  • Provide tips to help your group members and other patients attending a PFDD meeting share their information effectively and relatively concisely.
    • Possibly offer practice opportunities through webinars. 
  • Consider alternative ways for patients and/or their families and caregivers to participate who cannot attend the meeting and may not have access to the internet.
    • Paper copies of the questions (survey) can be mailed at the request of patients, families, and caregivers.
    • Including a self-addressed, stamped envelope to increase return of completed surveys.
  • Remember to invite medical researchers and representatives from pharmaceutical groups to be listeners at the event.
  • FDA has a plain language PowerPoint presentation reviewing both FDA-led and externally-led PFDD meetings: Patient Focused Drug Development .
    • This presentation may be helpful when discussing hosting a meeting with your Board of Directors, key staff members, Scientific/Medical Advisory Board, and other stakeholders.


Natural History Study
FAQ Orphan Products Natural History Grants Program U. S. Food and Drug Administration (FDA) (link)
OOPD Grant Application Overview U. S. Food and Drug Administration (FDA) (link)
Building a Registry
Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)