Presentation (CTI)

OBJECTIVES: Observer-Reported Outcomes (ObsROs) are tools based on reports of observable signs, events or behaviors related to a patient’s health condition by someone other than the patient or health professional. They are typically used when patients are unable to reliably self-report, particularly in pediatric or cognitively impaired populations. Identifying fit-for-purpose tools requires aligning regulatory expectations, understanding label claim precedents, and evaluating available validity evidence. This study aimed to develop a practical, sequential strategy for identifying ObsROs suitable for specific contexts of use and to illustrate its application to a pediatric population with attention-deficit/hyperactivity disorder (ADHD) using interconnected regulatory and evidentiary databases.
METHODS: We first reviewed regulatory guidance using PROINSIGHT to identify expectations for ObsRO use in pediatric ADHD. We then examined FDA and EMA label claims in PROLABELS to determine which ObsROs supported regulatory decisions in this indication. Finally, we assessed psychometric validity evidence in PROQOLID, evaluating conceptual relevance and measurement properties within the intended context of use to evaluate whether candidate ObsROs met fit-for-purpose criteria.
RESULTS: We identified 28 ObsROs relevant to pediatric ADHD. PROLABELS contained 28 pediatric ADHD labels referencing ObsROs, while PROINSIGHT included two pertinent guidelines. Integrating regulatory guidance, label precedents, and validity evidence narrowed the list to the most fit-for-purpose ObsROs: the Conners ADHD/DSM-IV™ Scale for Teachers (CADS-T) and the Swanson, Kotkin, Agler, M Flynn, and Pelham Scale (SKAMP).
CONCLUSIONS: A structured pathway that begins with regulatory guidance, incorporates label-based precedents, and integrates available validity evidence offers a transparent method for identifying fit-for-purpose ObsROs. Applying this strategy in pediatric ADHD enabled the selection of tools aligned with regulatory expectations and supported by adequate psychometric evidence. This approach provides a reproducible framework for future outcome selection, improves confidence in endpoint justification, and supports greater consistency across clinical development programs where ObsROs are required in relevant therapeutic areas.