Innovative caregiver burden study for health technology assessment


Vitaccess is partnering with a pharmaceutical company on an international, patient-centric real-world evidence study. This observational longitudinal study is designed to identify and increase understanding of the impact of living with a rare autoimmune, neuromuscular disease.

The client is 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 is a gap in research on the health-related quality of life of the caregivers of patients living with the disease.



    • To provide detailed and robust RWE on caregiver burden and health-related quality of life that can be used for HTA in the UK and other countries. Linked data collection from caregivers and patients will identify the correlation between caregiver burden and disease severity, condition, and characteristics of the patient.


    • 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.

    • The study was conducted online, with 75 caregivers enrolled from the UK, Spain, Italy, Germany, and France (15 per country). Linked caregivers were recruited via patients enrolled in the study who had previously consented to additional contact.

  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 increases 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 allows an in-depth understanding of caregiver burden and demonstrates 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 allows caregiver utility values to be derived that are linked to patient health states and so can be easily integrated into health-state models used for economic analysis.

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