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Abstract
Objective: Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A. Effective enzyme replacement therapies are available that are administered intravenously. However, a new oral treatment is being developed as an alternative option for patients with amenable mutations. This study was designed to understand the value that people place on the different features of treatments for Fabry disease.
Research design and methods: A discrete choice experiment (DCE) was designed to assess the importance of different aspects of treatments for Fabry disease. The attributes included overall survival, mode of administration, treatment related reactions, treatment related headaches and risk of antibody formation. Attributes were combined using a published orthogonal array into choice sets. A research panel was used to survey the UK general public. The mixed logit model was used to estimate strength of preference for the attributes and marginal rates of substitution (MRSs). Disutilities were estimated from the DCE data for changes in each attribute.
Results: The sample (n = 506) was broadly representative of UK demographics. The logit model revealed that all attributes were significant predictors of choice. Participants were significantly more likely to choose a treatment which meant an increase in their life expectancy by 1 year (odds ratio = 1.574; 95% CI = 1.504–1.647) and significantly less likely to choose self-administered intravenous (IV) treatment compared to an every other day tablet (OR = 0.426 95% CI = 0.384–0.474). Estimated disutilities were −0.0543 (self-administered infusion), treatment related headaches 12 times a year (−0.0361) and infusion reactions six times a year (−0.0202).
Conclusions: The survey revealed a significant preference for oral treatment compared with IV even in the context of a treatment that can extend overall survival. MRSs were used as a basis for estimating disutilities associated with changes in attribute levels which could be used to weight QALYs. It is possible that other important treatment attributes are missing from this research which may have provided further insights. It would also be useful to extend this research to include Fabry disease patients so their preferences can be assessed against the societal perspective.
Transparency
Declaration of funding
This project was supported by Amicus Therapeutics Inc.
Author contributions: A.J.L. designed the survey, helped to interpret the results and led the writing of the paper. K.G. helped to design the survey, undertook the data collection, supported data analysis and interpretation and supported the writing of the paper. S.A. undertook all statistical analysis of the data. D.H. supported the design of the study, and interpretation of the results. She also supported the writing of the paper. A.M. conceived the study, supported the survey design and data interpretation. All authors read and approved the final manuscript.
Declaration of financial/other relationships
A.J.L. and K.G. have disclosed that they work for Bladon Associates Ltd., a private consultancy which was paid a fixed fee for work on this project. S.A. has disclosed that he was engaged by Bladon to provide statistical services on the project and was paid a fixed fee. D.H. received no compensation for her time on the project. D.H. has disclosed that she receives support for research and travel and honoraria for advisory boards and speaking from Genzyme, Shire, Amicus and Protalix. A.M. has disclosed that he is an employee of Amicus Therapeutics UK Ltd. which owns migalastat – a treatment for Fabry disease.
The CMRO peer reviewer on this manuscript received an honorarium for the review work, but has no financial or other relationships to disclose.
Acknowledgements
The authors would like to acknowledge the efforts of the study participants who completed the survey. We would also like to acknowledge the support of Amicus Therapeutics Inc who supported the project.
Notes
* Fabrazyme® is a registered trade name of Genzyme, US.
† Replagel® is a registered trade name of Shire Human Genetic Therapies AB, Sweden.
