Innovative caregiver burden study for health technology assessment
Background
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.
Objective
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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.
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Approach
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Scope: EU4 + UK.
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For the study design stage, our work included:
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Targeted literature review of qualitative and quantitative research
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Interviews with two caregivers recruited via patient advocacy groups
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Selection of validated patient-reported outcome instruments to measure caregiver impact
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Testing of PROs and bespoke questions with caregivers.
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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.
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Key deliverables
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Literature review
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Synthesis of evidence and presentation of recommended PROs
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Ethics submissions (including development of protocol synopsis)
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ePRO adaptations and linguistic validation of the study survey into 5 languages
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Caregiver recruitment, study design and implementation, and data analysis
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Journal publication to disseminate study results
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