Approaches to Model Patient And Carer Utilities and Disutilities of Adverse Events in Economic Models for Rare Disease Treatments in NICE Appraisals
Author(s)
Lea Wiedmann, MSc, MSc, John Cairns, MA, MPhil;
London School of Hygiene and Tropical Medicine, Department of Health Services Research and Policy, London, United Kingdom
London School of Hygiene and Tropical Medicine, Department of Health Services Research and Policy, London, United Kingdom
Presentation Documents
OBJECTIVES: Providing robust economic modelling evidence for rare disease treatments (RDTs) is difficult due to challenges in the generation and interpretation of clinical evidence for rare diseases. The objective was to systematically analyse approaches used to model patient and carer utilities and disutilities of adverse events in appraisals of RDTs published by the National Institute for Health and Care Excellence (NICE).
METHODS: We analysed NICE appraisal documents for RDTs issued between 2011-2023. Data from documents were extracted and analysed, specifically: approaches to model patient and carer health-related quality of life (HRQoL), approaches to model disutilities of adverse events (AEs), and the impact of AEs on the results of the cost-effectiveness analyses (CEA). Sources of patient and carer HRQoL used in economic models were mapped against NICE’s hierarchy of preferred HRQoL methods.
RESULTS: A total of 110 indications were identified. In 39.1% of indications EQ-5D data was not available from a relevant study, and in 16.4% of indications, EQ-5D was not considered appropriate. In contrast, in 44.5% of indications, EQ-5D was available and used. Patient utilities were derived from a variety of sources, including clinical studies, published literature, previous NICE appraisals, vignettes, statistical mapping and expert input. Approaches to model patient utilities were also discussed in three-thirds of final appraisal documents suggesting a considerable influence on the reimbursement recommendation. The impact on carer HRQoL was considered in 30.9% of indications and was typically accounted for quantitatively using a carer disutility approach. Disutilities of adverse events and corresponding costs were modelled for most indications, but usually their impact on the CEA results was minimal or not mentioned.
CONCLUSIONS: Findings reflect challenges in the generation and interpretation of robust evidence for RDTs, emphasise the need for improving methods to include carer HRQoL, and show the limited impact of AEs on the CEA results.
METHODS: We analysed NICE appraisal documents for RDTs issued between 2011-2023. Data from documents were extracted and analysed, specifically: approaches to model patient and carer health-related quality of life (HRQoL), approaches to model disutilities of adverse events (AEs), and the impact of AEs on the results of the cost-effectiveness analyses (CEA). Sources of patient and carer HRQoL used in economic models were mapped against NICE’s hierarchy of preferred HRQoL methods.
RESULTS: A total of 110 indications were identified. In 39.1% of indications EQ-5D data was not available from a relevant study, and in 16.4% of indications, EQ-5D was not considered appropriate. In contrast, in 44.5% of indications, EQ-5D was available and used. Patient utilities were derived from a variety of sources, including clinical studies, published literature, previous NICE appraisals, vignettes, statistical mapping and expert input. Approaches to model patient utilities were also discussed in three-thirds of final appraisal documents suggesting a considerable influence on the reimbursement recommendation. The impact on carer HRQoL was considered in 30.9% of indications and was typically accounted for quantitatively using a carer disutility approach. Disutilities of adverse events and corresponding costs were modelled for most indications, but usually their impact on the CEA results was minimal or not mentioned.
CONCLUSIONS: Findings reflect challenges in the generation and interpretation of robust evidence for RDTs, emphasise the need for improving methods to include carer HRQoL, and show the limited impact of AEs on the CEA results.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE61
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology, SDC: Pediatrics, SDC: Rare & Orphan Diseases