Abstract:
|
Clinical outcome assessments (COAs) are used to capture input from patients, their caregivers, and physicians concerning key symptoms and functional impacts in a scientifically rigorous manner. COAs are increasingly used to construct efficacy, tolerability, and safety endpoints in medical product development programs-especially for rare and pediatric diseases, gastrointestinal conditions, psychiatric conditions, and cancer. Patients with diseases in these therapeutic areas often present with heterogeneous symptoms making it difficult to summarize findings from one or more instruments with relevant symptoms to detect change in the underlying condition. Personalized COA-based endpoints have been proposed as a potential approach to assessing meaningful change when symptom heterogeneity is present. However, several potential psychometric and statistical issues would need to be addressed, including: (1) how the development process and required evidence might differ from non-personalized COAs; (2) how to combine, analyze, and interpret data to evaluate treatment efficacy, safety, and/or tolerability; and (3) impact on statistical power to detect treatment effects.
|