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PRO139 A patient-centric approach to cost of illness research: A case example of patient engagement to identify the hidden costs associated with blindness due to inherited retinal dystrophies
Objectives
The impact of technologies beyond direct health costs and benefits may be of interest to Health Technology Assessment (HTA) bodies, especially for highly specialised technologies. Cost of illness studies are commonplace but including patient engagement can help ensure that all relevant indirect variables have been considered and burden of disease is appropriately captured. This is especially crucial for rare diseases where limited information is available and many costs may lie outside the health system. This study aimed to gather insights from patients and carers to identify costs associated with blindness due to inherited retinal dystrophies.
Methods
Following a targeted literature review, 5 adult patients with retinitis pigmentosa (RP) were invited via a patient organisation to participate in an advisory board. Patients underwent a facilitated, two-step concept elicitation exercise. First, they individually identified costs relevant for different patient age groups. Next, they participated in a group mapping exercise to discuss and confirm these costs. These costs are now being further explored and ratified in a second phase of work involving 1:1 interviews with carers of children and adults with RP.
Results
Patients identified the most significant areas of financial burden as: travel to appointments, specialist equipment, loss of employment for patients and carers, and domestic resources (including childcare, cooking, cleaning, home adaptations). Patients emphasised the significant impact on mental health resulting from a lack of treatment options and practical and emotional support, difficulty accessing welfare and benefits, loss of relationships, and financial hardship.
Conclusions
Incorporating patient engagement provided a more holistic and patient-centric view of the costs and impact of blindness. These insights helped contextualise financial costs identified from the literature to inform quantitative analysis on the costs of blindness and provided a more in-depth understanding of the wider practical, emotional and societal impact of blindness to inform HTA submissions.
Authors
F Glen, R Sidhu, A Pulfer, A Rylands
Journal
Value in Health
Therapeutic Area
Ophthalmology
Center of Excellence
Patient-Centered outcomes, Real-world Evidence & Data Analytics
Year
2019
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