Medical Costs Related to Enzyme Replacement Therapy for Mucopolysaccharidosis Types I, II, and VI in Brazil: A Multicenter Study

Fernanda Hendges de Bitencourt; Taiane Alves Vieira; Carlos Eduardo Steiner; Jordão Correa Neto; Raquel Boy; Ida Vanessa Doederlein Schwartz

doi:10.1016/j.vhri.2015.08.002

Medical Costs Related to Enzyme Replacement Therapy for Mucopolysaccharidosis Types I, II, and VI in Brazil: A Multicenter Study

Value Health Reg Issues. 2015 Dec:8:99-106. doi: 10.1016/j.vhri.2015.08.002. Epub 2015 Sep 29.

Authors

Fernanda Hendges de Bitencourt¹, Taiane Alves Vieira², Carlos Eduardo Steiner³, Jordão Correa Neto⁴, Raquel Boy⁵, Ida Vanessa Doederlein Schwartz⁶

Affiliations

¹ Federal University of Rio Grande do Sul, Porto Alegre, Brazil; Health Technology Assessment for Clinical Genetics Group, Federal University of Rio Grande do Sul, Porto Alegre, Brazil.
² Federal University of Rio Grande do Sul, Porto Alegre, Brazil; Bioethics Service, Hospital de Clínicas of Porto Alegre, Porto Alegre, Brazil.
³ Department of Medical Genetics, University of Campinas, Campinas, Brazil.
⁴ Pontifical Catholic University of Campinas, Campinas, Brazil.
⁵ Department of Pediatrics, State University of Rio de Janeiro, Rio de Janeiro, Brazil.
⁶ Medical Genetics Service, Hospital de Clínicas of Porto Alegre, Porto Alegre, Brazil; Department of Genetics, Federal University of Rio Grande do Sul, Porto Alegre, Brazil. Electronic address: [email protected].

PMID: 29698178
DOI: 10.1016/j.vhri.2015.08.002

Abstract

Background: Mucopolysaccharidosis (MPS) type I (MPS I), MPS type II (MPS II), and MPS type VI (MPS VI) are lysosomal storage disorders for which enzyme replacement therapy (ERT) is available.

Objective: The objective of this study was to evaluate the frequency of medical interventions in a cohort of patients with MPS I, II, and VI on ERT to estimate the impact of direct medical costs associated with the treatment of MPS and compare its frequency with that observed among patients not on ERT.

Methods: This was a multicenter study using a retrospective design including a convenience sampling of Brazilian patients with MPS I, II, and VI. Data on the number and type of medical appointments, hospital admissions, medications used, and surgical procedures performed per patient were obtained through a review of medical records, as were data on ERT. These variables were then compared between patients undergoing ERT and those not on ERT.

Results: Thirty-four patients (27 on ERT) were included in the study. Overall, between-group differences were found in median absolute frequencies of hospital admissions and surgical procedures per year, both of which were higher in the non-ERT group. Furthermore, we observed a high rate of failure to record medication dosage regimens.

Conclusions: Our findings suggest that Brazilian patients with MPS I, II, and VI who are on ERT undergo fewer medical interventions, which can lead to a reduction in direct medical costs to the publicly funded health care system. The cost of ERT, however, is extremely high and probably outweighs this reduction.

Keywords: enzyme replacement therapy; health technology assessment; mucopolysaccharidosis type I; mucopolysaccharidosis type II; mucopolysaccharidosis type VI; rare disorders.