Qualitative data, content validity and performance-based (PerfO) outcomes

What is it about?

There is increasing interest in the appropriate development and validation pathways for Clinical Outcome Assessments that are not Patient Reported Outcomes (i.e. CinRO, ObsRO and PerfO). PerfOs may present a particular challenge in cases where they may be intended to measure concepts which are difficult to observe or report, and/or be employed in populations unable to reliably self-report. Such tests may be used in e.g. a diagnostic, or surrogate context, where content validity is not relevant. Furthermore, such tests may be initially developed as tools to measure neuropsychological concepts independent of disease or condition and it is only in the event of later application as measures of treatment benefit which require thought as to conceptual relevance. In this context, the use of qualitative data may be limited or flawed in its ability to inform content validity.

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Why is it important?

As part of the PDUFA VI re-authorization, FDA plan a focus on Capturing the Patient Voice in Drug Development. Under PDUFA VI, FDA will engage in a multi-stakeholder approach that will lead to development of methods and approaches to ensure patients not only become active participants but informants to industry drug development and agency review. In this context, FDA will hold a series of four public workshops, and develop four key guidance documents on needed methods and approaches. The development and validation of PerfO will be included in this guidance and it is critical that such guidance reflects those contexts in which the use of qualitative data may be challenging or inappropriate.