Perform Patient Preference Studies

Timing

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).

Resources