Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies

Marshall, Deborah A.; Gerber, Brittany; Lorenzetti, Diane L.; MacDonald, Karen V.; Bohach, Riley Jewel; Currie, Gillian R.

doi:10.1007/s40273-023-01308-0

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2024

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Cited by 5 publications

References 98 publications

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Scoping Review of Economic Analyses of Rare Kidney Diseases

Angell,

Wang,

Gadsden

et al. 2024

Kidney International Reports

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Scoping Review of Economic Analyses of Rare Kidney Diseases

Angell,

Wang,

Gadsden

et al. 2024

Kidney International Reports

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Systematic scoping review of socioeconomic burden and associated psychosocial impact in patients with rare kidney diseases and their caregivers

Palagyi,

Sengupta,

Moorthy

et al. 2024

Kidney International Reports

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Comparative evaluation of access to emicizumab (Hemlibra®) for people with haemophilia A in community and hospital pharmacies in France

Cabon,

Chamouard,

Freyssenge

et al. 2024

European Journal of Public Health

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Since June 2021 in France, patients with haemophilia A with anti-factor VIII inhibitors and patients with severe haemophilia A without anti-factor VIII inhibitors have the choice between the community and the hospital pharmacy for dispensing emicizumab (Hemlibra®). This study aims to investigate patient-centred access to treatment by evaluating and comparing the dimensions of the Penchansky and Thomas model, between community and hospital pharmacies. The evaluation of access to treatment was based on the dimensions of the Penchansky and Thomas model: accessibility, availability, acceptability, accommodation and affordability. These were assessed using appropriate and specific indicators in the study context and calculated for patients choosing community pharmacy or hospital pharmacy for emicizumab dispensing. Geographical data collected as part of the national ‘PASO DOBLE DEMI’ study were used for this analysis. The findings reveal that dispensing emicizumab in community pharmacies improves accessibility by significantly reducing travel time. The availability of healthcare services is also improved due to the territorial coverage of community pharmacies. The extended opening hours and low waiting time also contribute to better access to emicizumab in community pharmacies. The dimension of acceptability must be improved, and further investigations are needed to address the affordability dimension. Several months after emicizumab became available in French community pharmacies, access to treatment has significantly improved, reducing the burden of this rare chronic disease for patients and their careers. These results suggest that this innovative model could be extended to other medicines and even other therapeutic areas.

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Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies

Cited by 5 publications

References 98 publications

Scoping Review of Economic Analyses of Rare Kidney Diseases

Scoping Review of Economic Analyses of Rare Kidney Diseases

Systematic scoping review of socioeconomic burden and associated psychosocial impact in patients with rare kidney diseases and their caregivers

Comparative evaluation of access to emicizumab (Hemlibra®) for people with haemophilia A in community and hospital pharmacies in France

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