Perform Patient Preference Studies
The timing of patient preference studies depends on the stage of therapy development you are hoping to influence.
- Early patient preference studies focused on understanding unmet medical needs, burden of disease, and general patient benefit-risk perspectives can provide crucial information during the therapy discovery phase to academic and industry researchers.
- Influence symptoms to be targeted for treatment development.
- Attract interest of academic and industry researchers.
- Determine priority of therapies in early development from a patient perspective.
- In the preclinical phase of therapy development, when researchers have completed initial analyses of the possible benefit-risk of a potential therapy, patient preference studies can obtain information about more specific preferences and can use this data to develop clear COAs.
- Ask about more specific benefit-risk scenarios that may influence clinical trial design and patient subpopulations to target for enrollment.
- Develop a COA or choose one that already exists. You can learn more about COAs in NCATS Toolkit section on Help Industry with Clinical Trial Design.
- Determine information that may be important to patients during their decision process of whether or not to enter a specific clinical trial.
- During Phase 2 and 3 clinical trials, patient preference studies can be focused on actual benefits and risks seen among trial participants.
- The data from studies performed now can be submitted to the FDA to be included in the marketing review process.
- After therapy approval, patient preference studies can be used to detect any difference between stated preferences (what a person believes they will choose) and revealed preferences (what a person actually chooses).
Engage with the FDA
Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project Report Medical Device Innovation Consortium (MDIC) (link)
FDA-led Patient-Focused Drug Development (PFDD) Public Meetings U. S. Food and Drug Administration (FDA) (link)
Enhancing Benefit-Risk Assessment in Regulatory Decision-Making U. S. Food and Drug Administration (FDA) (link)
Tips for Success
Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) (link)