Caregiver Quality of Life in Orphan Drug Health Technology Assessment Submissions in Europe: A Comparative Analysis
Author(s)
Rebecca Joanne Greaves, PhD, Ciara Wright, MSc, Roisin Stout, PhD.
Maverex Limited, Newcastle upon Tyne, United Kingdom.
Maverex Limited, Newcastle upon Tyne, United Kingdom.
OBJECTIVES: Caregivers are at risk of financial, physical, and psychological detriment as a consequence of their role. This study aimed to analyse and compare how caregiver quality of life (CQoL) is incorporated into orphan drug health technology assessment (HTA) submissions across Europe.
METHODS: A targeted literature review of HTA submissions of orphan drugs in Europe between January 2024 and May 2025 was conducted. Analysis identified CQoL measurement tools, frequency of inclusion, evidence requirements, methodological approaches, barriers to integration and the impact of CQoL on decisions.
RESULTS: Analysis of CQoL incorporation in recent European orphan drug HTA submissions revealed varying approaches and acceptance rates. In Scotland, 71% (10/14) of submissions acknowledged caregiver burden and one (1/14; 7%) included CQoL in economic analysis. All CQoL input used proxy data. England demonstrated similar incorporation rates, with 60% (16/27) of submissions including CQoL data, with 56% (9/16) of these incorporating CQoL into economic models. The main criticisms were use of proxy data and calculation methods. In Norway, 25% (4/16) of submissions included CQoL; in all four cases the CQoL evidence was rejected due to inadequate standards. In Ireland, 17% (5/30) of submissions included CQoL, with 1 rejection (1/5; 20%) due to insufficient evidence.
CONCLUSIONS: This study identified that the inclusion of CQoL in HTA assessment of orphan drugs in Europe is inconsistent and methods are not well defined or reported. CQoL was incorporated into HTA assessment of orphan drugs using a variety of qualitative, quantitative, and mixed methods of measurement. Clear processes for patient/caregiver engagement (Scotland) and consideration of CQoL in models (England) may increase the rate of CQoL inclusion. When included in economic models, CQoL impacted cost-effectiveness. Currently, HTAs risk failing to capture the impact of the role of carers in certain conditions, potentially leading to unrepresentative cost-effectiveness analyses.
METHODS: A targeted literature review of HTA submissions of orphan drugs in Europe between January 2024 and May 2025 was conducted. Analysis identified CQoL measurement tools, frequency of inclusion, evidence requirements, methodological approaches, barriers to integration and the impact of CQoL on decisions.
RESULTS: Analysis of CQoL incorporation in recent European orphan drug HTA submissions revealed varying approaches and acceptance rates. In Scotland, 71% (10/14) of submissions acknowledged caregiver burden and one (1/14; 7%) included CQoL in economic analysis. All CQoL input used proxy data. England demonstrated similar incorporation rates, with 60% (16/27) of submissions including CQoL data, with 56% (9/16) of these incorporating CQoL into economic models. The main criticisms were use of proxy data and calculation methods. In Norway, 25% (4/16) of submissions included CQoL; in all four cases the CQoL evidence was rejected due to inadequate standards. In Ireland, 17% (5/30) of submissions included CQoL, with 1 rejection (1/5; 20%) due to insufficient evidence.
CONCLUSIONS: This study identified that the inclusion of CQoL in HTA assessment of orphan drugs in Europe is inconsistent and methods are not well defined or reported. CQoL was incorporated into HTA assessment of orphan drugs using a variety of qualitative, quantitative, and mixed methods of measurement. Clear processes for patient/caregiver engagement (Scotland) and consideration of CQoL in models (England) may increase the rate of CQoL inclusion. When included in economic models, CQoL impacted cost-effectiveness. Currently, HTAs risk failing to capture the impact of the role of carers in certain conditions, potentially leading to unrepresentative cost-effectiveness analyses.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA65
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas