Innovative caregiver burden study for health technology assessment
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Background
Vitaccess partnered with a pharmaceutical company on an international, patient-centric real-world study. This observational longitudinal study was designed to identify and increase understanding of the impact of living with a rare autoimmune, neuromuscular disease.
The client was seeking recommendation from HTA bodies across Europe for a new product to treat this disease. Some HTA bodies will include impact on caregiver burden within the value assessment of new health technologies; however, there was a gap in research on the health-related quality of life of the caregivers of patients living with the disease.
Objectives
- To provide detailed and robust real-world evidence on caregiver burden and health-related quality of life that could be used for HTA in the UK and other countries. Linked data collection from caregivers and patients was to be used to identify the correlation between caregiver burden and disease severity, condition, and characteristics of the patient.
Approach
- Scope: EU4 + UK.
- For the study design stage, our work included:
- Targeted literature review of qualitative and quantitative research
- Interviews with two caregivers recruited via patient advocacy groups
- Selection of validated patient-reported outcome instruments to measure caregiver impact
- Testing of PROs and bespoke questions with caregivers.
Key deliverables
- Literature review
- Synthesis of evidence and presentation of recommended PROs
- Ethics submissions (including development of protocol synopsis)
- ePRO adaptations and linguistic validation of the study survey into 5 languages
- Caregiver recruitment, study design and implementation, and data analysis
- Journal publication to disseminate study results
Client benefit
The innovative data linkage increased the value of data for HTA bodies by demonstrating the relevance and validity of caregiver burden and utility data to the target patient population within the assessment. Granular data collection allowed an in-depth understanding of caregiver burden and demonstrated robustness by confirming intuitive patterns and relationships.
The study was tailored to the needs of cost-effectiveness modelling. The direct link between caregiver burden data and patient data allowed caregiver utility values to be derived that were linked to patient health states and so could be easily integrated into health-state models used for economic analysis.
