Shawn McKown, MA
RWS Life Sciences
Clinical outcome assessments (COAs), defined by the FDA as tools (generally questionnaires) that “measure a patient’s symptoms, overall mental state, or the effects of a disease or condition on how the patient functions,” are widely utilized within global clinical trials to assess concepts of interest and determine whether clinical benefit has been demonstrated. COAs are categorized into four types of measures:
- Patient-reported outcome (PRO), completed only by the patient
- Clinician-reported outcome (ClinRO), completed by a trained health-care professional
- Observer-reported outcome (ObsRO), completed by someone other than the patient or health professional (e.g., a parent or caregiver)
- Performance outcome (PerfO), based on standardized task(s) performed by the patient.
While use of these classifications has become widespread, it is also relatively new. Until recently, regulatory bodies and industry users referred primarily to PRO measures rather than the broader category of COAs.
The spread of this broader COA concept, which encourages us to consider PRO measures as one of several COA types rather than as the primary focus, has led to a gap in recommendations associated with translation, cultural adaptation, and linguistic validation processes. Existing translation guidance developed by FDA and industry stakeholders applies specifically to PRO measures, and does not address the process requirements for the translation of ObsRO, ClinRO, or PerfO measures. Translation companies and academic groups performing cultural adaptations or linguistic validation currently do not have access to consensus recommendations specific to these COA types, leading to inconsistent approaches across the industry. Lack of existing guidance is of particular concern for pharmaceutical sponsors and clinicians seeking to use these instruments in clinical trials or practice across the world.
In order to address this gap, our team in the ISOQOL Translation and Cultural Adaptation Special Interest Group (TCA SIG) sought to develop recommendations specific to each of the three COA types that currently lack such documentation through a consensus approach. Our recommendations are designed to be reasonable and actionable, and to address the existing gap in process recommendations and good practices.
To develop these good practice recommendations, the ISOQOL TCA SIG examined the characteristics and requirements of each COA type by means of a literature review, completion of newly developed surveys by industry experts, and group discussion and analysis. Our findings indicate that while recommended translation process steps generally align across all COA types (including PRO measures), the substantial differences between respondent categories (i.e., patients, clinicians and observers) across COA types require targeted approaches to cognitive interviewing procedures. As a result, our team developed specific process recommendations for each non-PRO COA type, which will assist in further aligning procedures across service providers, COA instrument developers, and industry sponsors.
This newsletter editorial represents the views of the author and do not necessarily reflect the views of ISOQOL.
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