The module will review definitions of patient reported outcomes and the contexts in which they are used, including applications to health quality improvement, clinical trials and drug safety. The module will provide an overview of methodologic issues to consider when using patient reported outcomes or research with patient reported outcomes, and the concepts that are assessed by government agencies such as FDA and NIH. Measurement concepts such as content validity will be introduced, and limitations of discussed.
After the overview, the module will walk through all issues related to statistical analysis of patient reported outcomes after the instrument has been validated, calibrated, approved and finalized. Concepts related to development will be introduced only as they relate to ultimate analysis of the data (development will be covered more thoroughly in a separate module). The use of multiple endpoints and composite endpoints will be covered, as well as approaches to continuous, categorical and time to event analysis. Approaches to analysis of missing data will be introduced.
Three case studies will be used to illustrate analysis of PRO data in different contexts. The first case study will describe the work being done by PROMIS, and the status of the outcome measures available for measuring health care quality. The second case study will describe work being done in the United Kingdome National Health Service (NHS) in PROMs, and analysis of data within a health system. The final case study will illustrate the use of PROs in clinical trials, in support of FDA labeling claims. All modules will discuss statistical analysis, and interpretation of statistical results.
By the end of the Patient Reported Outcomes – Analysis and Interpretation module, you will be able to:
- describe what patient-reported outcomes are
- what general statistical issues to consider when analyzing data collected from PRO instruments
- understand three case studies demonstrating use of PRO instruments.