Your organization’s activities, for example
- Natural history studies
- Patient-reported outcomes studies
- Patient surveys on risks, benefits, and preferences related to treatments
Impact of the disease and its treatment
- Chief complaints (most bothersome signs and symptoms)
- Burden of living with and managing a disease or condition
- Impacts from a disease or condition on activities of daily living and functioning
Perspectives about current and potential treatment approaches
- Expectations of benefits
- Tolerance for harms or risks
- Patient preference
- Unmet medical needs
Clinical trial considerations
- E.g., views on various therapies, such as gene therapy
- Burden of participating in clinical studies
- Risk tolerance
