Use of patient-reported outcome measures in cost-effectiveness models informing health technology assessments of non-oncology treatments

Abstract

ObjectiveIncluding patient-reported outcome measures (PROMs) in cost-effectiveness models (CEMs) represents an important opportunity to reflect patients' perspectives in health technology assessments (HTAs). Beyond utility measures, little is known about how PROMs are used in CEMs and their acceptance by reviewers. We elucidated how PROMs were incorporated into CEMs submitted to the UK National Institute for Health and Care Excellence (NICE) for non-oncology treatments and identified feedback from NICE and external assessment groups (EAGs).MethodsNICE HTAs for non-oncology treatments from January 2016 to August 2022 which used a PROM (other than a generic utility measure) as a key element in the CEM were reviewed. Information on how PROMs were used, rationale for their use, and feedback from NICE and EAGs were summarized.ResultsOf 428 HTAs identified, 27 (6.3%) were included in our review. PROMs were included in the CEMs via treatment-effect parameters (n = 25/27, 92.6%), model structure (n = 25/27, 92.6%), treatment-stopping rules (n = 21/27, 77.8%), and/or condition-specific utility measures (n = 18/27, 66.7%). Of the 27 HTAs, 22 (81.5%) provided justification for using a PROM in the CEM, most frequently citing the PROM validation. NICE and/or the EAG raised concerns in 20 HTAs (74.1%) regarding the use of PROMs, most commonly regarding the data source, statistical methods, and implementation of the PROM in clinical practice.LimitationsThis review did not focus on ways the patient voice may be reflected in the NICE process beyond incorporation of PROMs in CEMs. Generalizability of the findings to other HTA settings may be limited.ConclusionsNICE accepted the use of PROMs in CEMs where high-quality data relevant to the target population and appropriate statistical methods were used. Few CEMs used PROMs other than utility measures. Further research is needed to understand the value of PROMs in CEMs for HTAs.