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.
The global Clarivate™ clinical outcome assessment team is comprised of health psychologists and outcomes researchers who have extensive methodological and commercial experience. Our deep and broad knowledge of therapy areas such as oncology, immune disorders, respiratory, psychiatry, CNS, autoimmune, infectious diseases, pain, and women’s health enables sponsors to make informed decisions when selecting, developing and validating clinical outcome assessments (COAs) for use in clinical trials.
In this article, we highlight two case studies which address challenges faced by sponsors looking to incorporate COAs into clinical trials for a number of therapy areas. Data and insights gleaned from these projects have most recently been presented at industry conferences and events, including ISPOR.
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 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.
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.
The 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.
Last year alone the Clarivate COA team conducted over 300 interviews with patients, caregivers or healthcare professionals. We developed or explored over 100 COAs and achieved ethical approval in multiple countries including the U.S., U.K., Mainland China and Japan. These incorporated a variety of methodologies including literature reviews, patient preferences, exit interviews and FDA dossier development.
Patient focused drug development in alopecia areata clinical trials
The Clarivate COA team worked with a sponsor to develop COAs for evaluating key clinical trial endpoints in alopecia areata clinical trials.
The team interviewed patients and clinical experts to conceptualize the disease experience and develop content valid PROs and ClinROs, with accompanying photo guides to assess disease-defining signs/symptoms. Quantitative data from clinical trials informed the psychometric performance of the COAs and thresholds for interpreting within-patient meaningful change. All data were collated in FDA COA Evidence Dossiers.
Several years of evidence generation culminated in the FDA’s approval of the first systemic treatment for alopecia areata with data from the COAs developed 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. The work, said lead investigator Brett King, Associate Professor at Yale Dermatology, “helped change the landscape of alopecia areata forever.”
A qualitative interview study into the experiences of fatigue and depression in chronic hepatitis B
Our experts helped the client to conceptualize the experience of chronic hepatitis B (CHB)-associated fatigue and depression amongst individuals living with CHB in the U.S. Patients participated in semi-structured qualitative interviews with COA experts, designed to elicit concepts important to measure in individuals living with CHB. The results led to the expansion and refinement of a previously developed conceptual model to document the multifaceted experiences of fatigue and depression for patients living with CHB. These findings can inform a patient-centred PRO measurement strategy for clinical trials in CHB.
Clarivate experts can streamline your clinical development strategy with fit-for-purpose PROs and other COA instruments to support regulatory, communication, and reimbursement strategies. To learn more about COA, our broader evidence, value and access offerings or view our industry expert profiles, please visit our website.
About the author
Helen Kitchen is the Vice President of Clinical Outcome Assessment at Clarivate. Helen has 15 years’ experience of selecting, developing, and validating COAs, including PROs, for pharmaceutical clinical trials.
