Clinical outcome assessments can take years to generate but may pay big dividends in patient-focused drug development, centering the patient experience and potentially bolstering a product’s case with regulators and payers. In this article, we share how a sponsor partnered with Clarivate to incorporate COAs into clinical trials for alopecia areata.
The purpose of clinical trials is to understand the efficacy and safety of treatments for the patients who need them. To understand treatment efficacy, it is necessary to measure the impact of the treatment on patients’ signs/symptoms, functioning and overall health-related quality of life. A clinical outcome assessment (COA) is a measure that describes or reflects how a patient feels, functions or survives.
COAs include patient-reported outcome (PRO) measures, clinician-reported outcome (ClinRO) measures, observer-reported outcome (ObsRO) measures and performance outcome (PerfO) measures. Including fit-for-purpose measures in clinical trials can allow sponsors to generate critical evidence for regulators to evaluate the efficacy and safety of their product, and after approval these data can inform cost-effectiveness analyses for payer decision making.
The regulatory landscape around COAs is fast evolving
The evidence standards that a COA must meet to support key clinical trial endpoints have become increasingly stringent in recent decades, following the introduction of the United States Food and Drug Administration (FDA) Patient Reported Outcomes (PRO) draft guidance in 2006, followed by the full guidance in 2009. More recently, the FDA has developed the Patient Focused Drug Development (PFDD) Guidance Series, which provides sponsors with guidance on how to collect and submit patient experience data in medical product development for regulatory decision making. The PFDD series will eventually take the place of the PRO Guidance for Industry.
The Clarivate™ clinical outcome assessment team supports sponsors in selecting, developing and validating COAs for use in clinical trials. Qualitative data and quantitative data are required to develop/modify COAs and demonstrate their measurement properties. These data can take several years to generate, requiring methodological expertise, deep disease understanding, substantial input from patients and other key stakeholders (e.g., clinical experts), and converting regulatory guidance into practical application.
Patient focused drug development in alopecia areata clinical trials
In 2017, the Clarivate COA team began working with a sponsor to develop COAs for evaluating key clinical trial endpoints in alopecia areata clinical trials.
Over the next five years the team interviewed hundreds of patients and multiple clinical experts, seeking to conceptualize the disease experience and develop content valid PROs and ClinROs, with accompanying photo guides to assess disease-defining signs/symptoms. Quantitative data were collected in clinical trials and analysed to understand the psychometric performance of the COAs and identify thresholds for meaningful change from a patient’s perspective. These data were eventually collated in COA Evidence Dossiers, submitted as part of the New Drug Application (NDA).
Several years of evidence generation culminated in the FDA’s approval of the first systemic treatment for alopecia areata. Data from the COA development by the Clarivate team was included in the approved labelling.
Most importantly, newly approved treatments can address significant unmet needs for patients living with severe alopecia areata. These COAs helped to bring the patient voice into clinical trial measurement and improved treatment options for patients. The work, said lead investigator Brett King, Associate Professor at Yale Dermatology, “helped change the landscape of alopecia areata forever.”
To learn more about Clarivate clinical outcome assessment services and other health economics and value offerings, please visit our website.
About the author
Helen Kitchen is the Vice President of Clinical Outcome Assessment at Clarivate. Helen has 14 years’ experience of selecting, developing, and validating COAs, including PROs, for pharmaceutical clinical trials.