All Times EDT
Keywords: patient-focused drug development, PRO
FDA recently developed a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making. Stakeholders are encouraged to incorporate patient experience data in study design and analysis. In this talk, I will discuss some current challenges on using patient-reported outcomes, for example, selecting the fit-for purpose PROs, analysing ordinal variable, assessing multi-component/multiple endpoints, utilizing high frequency data, and handling missing data, etc. Regulatory perspectives on these issues as outlined in FDA PFDD Guidance will be briefly introduced. A few cases studies will be presented to illustrate how some challenges can be resolved via innovative trial designs.