Authors: Marieke Schurer, MSc; Melanie van Riemsdijk, PharmD, PhD. Kintiga, Ghent, Belgium.

Abstract

OBJECTIVES

In health technology assessments (HTA), inclusion of caregiver health-related quality of life (HRQoL) and associated costs is essential to capture the full societal impact of disease and treatment, as interventions often generate “spillover effects” beyond the patient, particularly for parents of children with (degenerative) rare diseases. Ignoring these effects can systematically undervalue interventions and lead to suboptimal resource allocation. Yet it is not formally included by many HTA authorities. This study assessed recent changes in global HTA practices regarding inclusion of caregiver HRQoL in cost-effectiveness
analyses and evaluated their impact following the publication of Pennington et al. (2022).

METHODS

A targeted review of HTA methodological guidelines and recent assessments was conducted across major agencies in Europe, North America, and Asia-Pacific. We examined whether caregiver HRQoL is included in the reference case or in scenario/sensitivity analyses. Additionally, HTA reports for orphan drugs licensed for patients aged 0-18 years (2022-2026) were analysed to assess real-world application and impact on outcomes.

RESULTS

Since 2022, several agencies, including ZIN, HIQA, NICE, and ICER, updated their guidance. ZIN now recommends inclusion of caregiver HRQoL in scenario analyses where relevant, while ICER permits a societal co-base alongside the healthcare perspective. Analysis of UK and Dutch assessments identified 13 NICE appraisals including caregiver HRQoL for paediatric orphan drugs. Of these, only two were evaluated by ZIN, and neither incorporated caregiver HRQoL. Inclusion of caregiver HRQoL in NICE assessments increased incremental QALYs and reduced ICERs, indicating improved cost-effectiveness. Final results, including outcomes for the other countries, will be shared in November.

CONCLUSIONS

Global HTA guidance on caregiver HRQoL remains heterogeneous. While some jurisdictions increasingly incorporate caregiver outcomes, others limit inclusion to sensitivity analyses or exclude them entirely. Recent NICE assessments demonstrate growing uptake, yet methodological guidance on consistent incorporation remains limited and is a recognized priority for further development.