Topics for Discussion- Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities; Public Meeting

Our goal is to convene a rare diseases meeting gathering patient and caregiver perspectives that is valuable to patients and supportive of medical product development. Thus, we want the discussion questions to allow an efficient and productive dialogue. These draft discussion questions may be modified based on patient and caregiver input we receive prior to the meeting. We sincerely appreciate your interest and hope that this meeting will lead to additional future endeavors to support medical product development for rare diseases.

Topic 1: Commonalities in symptom management and treatment considerations  

1. What are you currently doing to help manage the symptoms of your rare disease or condition? (Examples may include, but are not limited to, prescription medicines, over-the-counter products, devices, non-drug therapies, or support services)

2. What factors do you take into account when making decisions about how to manage your symptoms?
2a. What potential benefits factor most into your decisions?
2b. How do you weigh potential benefits versus potential common side effects?
2c. How do you weigh potential benefits versus potential less common but serious risks?

Topic 2: Commonalities in clinical study considerations and registry considerations

3. What factors have you or would you consider when thinking about participating in a clinical study or registry (Examples may include, but are not limited to, side effects, travel, time commitment, or trial design)?