Cost-effectiveness of enzyme replacement therapy for Fabry disease

@inproceedings{Rombach2013CosteffectivenessOE,
  title={Cost-effectiveness of enzyme replacement therapy for Fabry disease},
  author={Saskia M. Rombach and Carla E. M. Hollak and Gabor E. Linthorst and Marcel G. W. Dijkgraaf},
  booktitle={Orphanet journal of rare diseases},
  year={2013}
}
BACKGROUND The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease. METHODS Cost-effectiveness analysis was performed using a life-time state-transition model. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort consisting of males and females aged 5-78 years. Intervention with ERT (either agalsidase… CONTINUE READING
Highly Cited
This paper has 20 citations. REVIEW CITATIONS
Related Discussions
This paper has been referenced on Twitter 10 times. VIEW TWEETS

Citations

Publications citing this paper.
Showing 1-10 of 13 extracted citations

Budgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases.

Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research • 2018
View 1 Excerpt