HTA Agencies' Expectations for Caregiver Quality of Life Considerations in CEA: A Focus on Pediatric Indications

OBJECTIVES: Caregivers play a critical role in a pediatric patient's healthcare journey. By proposing to extend the impact that the intervention would have on caregivers’ quality of life (QoL), some health technology assessment (HTA) agencies have legitimized their value. As there are no guidelines that explicitly detail how caregiver utility data should be used in cost effectiveness analysis (CEA), this study aims to identify potential expectations of HTA agencies focusing on pediatric indications.

METHODS: A systematic review of NICE appraisals for pediatric indications that included caregiver utility between January 2019 and May 2023 was performed. French, Swedish, Dutch, Canadian, US and Australian HTA appraisals for the same indication were then searched and analyzed. The data collected from these assessments were intended to answer the following questions: Why was it considered legitimate or not to include caregiver utility in this assessment? How many caregivers were considered? What was the source of the caregiver utility? How was caregiver’s utility implemented in the model?

RESULTS: Of the 16 identified pediatric products assessed by NICE, only 6 had been assessed by at least 1 other HTA agency that considered caregivers QoL. Heterogeneity in the consideration of caregivers was pronounced, and NICE appears to pioneer in doing so. Based on the analysis of the NICE appraisals, the following expectations are noteworthy: evidence that the intervention affects caregiver QoL, consistency with other appraisals in the same therapeutic area in the number of caregivers, nonlinearity in utilities when there are multiple caregivers considered, use of disutility methods.

CONCLUSIONS: Although it is possible to identify the potential expectations of HTA agencies regarding the consideration of caregiver QoL in pediatric indications, there has been much debate, particularly around the "caregiver QALY trap". Consensus on these issues would be desirable.