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the added benefits, for example, finding others who have the same challenges you are experiencing but that may not be published in the medical literature.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.

Resources

Building a Registry