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the added benefits, for example, finding others who have the same challenges you are experiencing but that may not be published in the medical literature.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