Clinician-Reported Outcome Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force

Abstract

A clinician-reported outcome (ClinRO) assessment is a type of clinical outcome assessment (COA). ClinRO assessments, like all COAs (patient-reported, observer-reported, or performance outcome assessments), are used to 1) measure patients' health status and 2) define end points that can be interpreted as treatment benefits of medical interventions on how patients feel, function, or survive in clinical trials. Like other COAs, ClinRO assessments can be influenced by human choices, judgment, or motivation. A ClinRO assessment is conducted and reported by a trained health care professional and requires specialized professional training to evaluate the patient's health status. This is the second of two reports by the ISPOR Clinical Outcomes Assessment-Emerging Good Practices for Outcomes Research Task Force. The first report provided an overview of COAs including definitions important for an understanding of COA measurement practices. This report focuses specifically on issues related to ClinRO assessments. In this report, we define three types of ClinRO assessments (readings, ratings, and clinician global assessments) and describe emerging good measurement practices in their development and evaluation. The good measurement practices include 1) defining the context of use; 2) identifying the concept of interest measured; 3) defining the intended treatment benefit on how patients feel, function, or survive reflected by the ClinRO assessment and evaluating the relationship between that intended treatment benefit and the concept of interest; 4) documenting content validity; 5) evaluating other measurement properties once content validity is established (including intra- and inter-rater reliability); 6) defining study objectives and end point(s) objectives, and defining study end points and placing study end points within the hierarchy of end points; 7) establishing interpretability in trial results; and 8) evaluating operational considerations for the implementation of ClinRO assessments used as end points in clinical trials. Applying good measurement practices to ClinRO assessment development and evaluation will lead to more efficient and accurate measurement of treatment effects. This is important beyond regulatory approval in that it provides evidence for the uptake of new interventions into clinical practice and provides justification to payers for reimbursement on the basis of the clearly demonstrated added value of the new intervention.

Keywords: clinical trials; clinician-reported outcomes; end points; outcome assessments.

Fig. 1

Direct to indirect assessments of treatment benefit. ADAS-COG, Alzheimer’s Disease Assessment Scale—Cognitive; ALS, amyotrophic lateral sclerosis; BPRS, Brief Psychiatric Rating Scale; COA, clinical outcome assessment; CT, computed tomography; ETDRS, Early Treatment Diabetic Retinopathy Study; EXACT-PRO, the EXAcerbaCTtions of chronic pulmonary disease tool; FVC, forced vital capacity; FEV1, forced expiratory volume in 1; FLU-PRO, InFLUenza Patient-Reported Outcome; HgbA1c, glycated hemoglobin; HIV, human immunodeficiency virus; MRI, magnetic resonance imaging; SDMT, Symbol Digit Modalities Test.