Cost of Illness in Patients with Duchenne Muscular Dystrophy in Portugal: The COIDUCH Study

Pedro Labisa, Valeska Andreozzi (), Melina Mota, Susana Monteiro, Rita Alves, João Almeida, Björn Vandewalle, Jorge Felix, Katharina Buesch, Hugo Canhão and Igor Beitia Ortiz de Zarate
Additional contact information
Pedro Labisa: Exigo Consultores
Valeska Andreozzi: Exigo Consultores
Melina Mota: Exigo Consultores
Susana Monteiro: Exigo Consultores
Rita Alves: Exigo Consultores
João Almeida: Exigo Consultores
Björn Vandewalle: Exigo Consultores
Jorge Felix: Exigo Consultores
Katharina Buesch: PTC Therapeutics International
Hugo Canhão: PTC Therapeutics
Igor Beitia Ortiz de Zarate: PTC Therapeutics

PharmacoEconomics - Open, 2022, vol. 6, issue 2, No 6, 218 pages

Abstract: Abstract Objective The aim of this study was to estimate the cost of illness (COI) of Duchenne muscular dystrophy (DMD) and its relation to disease progression, using age as a proxy, and according to the ambulatory status of patients. Methods We conducted a cross-sectional study of patients diagnosed with DMD identified through the Portuguese Neuromuscular Patients Association (APN). Data regarding patient and caregiver demographics, patient health status, resource utilization and cost, and informal care were collected using a custom semistructured questionnaire. Labor productivity and absenteeism losses were captured using the Work Productivity and Activity Impairment questionnaire. Costs were valued using a societal perspective. Results A total of 46 patient–caregiver pairs were included, of which eight of the patients were ambulant and 38 were nonambulant. Age had a decreasing effect on COI, independent of the patient’s disease stage. Annualized lifetime costs were at their highest in nonambulant patients around the mean age of loss of ambulation (10 years of age). The mean per patient stage-specific costs (year 2019 values) of DMD were estimated at €48,991 in the nonambulant stage and €19,993 in the ambulant stage. Direct nonmedical costs were the main cost drivers, followed by indirect costs. Conclusions Our results indicate a close relation between overall disease costs and disease progression. DMD is associated with a substantial economic burden, which appears to be larger around the time ambulation is lost (10 years of age). The availability of new therapeutic options that delay disease progression, especially loss of ambulation, may prove to be highly beneficial for not only patients with DMD but also their families and society.

Date: 2022
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DOI: 10.1007/s41669-021-00303-5

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