The socioeconomic burden of facioscapulohumeral muscular dystrophy.

Abstract

BACKGROUND:

Promising genetic therapies are being investigated in facioscapulohumeral muscular dystrophy (FSHD). However, the current cost of illness is largely unknown.

OBJECTIVE:

This study aimed at determining the socioeconomic burden of FSHD.

METHODS:

Adult patients with FSHD from the Dutch FSHD registry were invited to complete a questionnaire on medical consumption, work productivity and health-related quality of life (HR-QoL) using the EQ-5D-5L. Associated costs were calculated from a societal perspective. A generalized linear model was fitted to the data to investigate whether level of mobility was related to annual costs of illness.

RESULTS:

172 patients with FSHD completed the questionnaire (response rate 65%). The per-patient annual direct medical costs of FSHD were estimated at €12,077, direct non-medical costs at €9179 and indirect costs at €5066, adding up to a total cost of illness of €26,322 per patient per year. The direct costs of illness were €21,256, approximately five times higher than the mean per-capita health expenditures in the Netherlands. Major cost-driving factors were formal home care and informal care. A decreased level of mobility was associated with higher direct costs of illness. HR-QoL was significantly reduced in patients with FSHD with a median health utility value of 0.63.

CONCLUSIONS:

We show that FSHD is associated with substantial direct and indirect socioeconomic costs as well as a reduction in HR-QoL. These findings are important for health care decision makers and aids in allocation of research funds and evaluation of the cost-effectiveness of novel therapies.

• Authors: Anna M Blokhuis, Johanna C W Deenen, Nicol C Voermans, Baziel G M van Engelen, Wietske Kievit, Jan T Groothuis
• Journal: Journal of neurology (J Neurol) Vol. 268 Issue 12 Pg. 4778-4788 (Dec 2021) ISSN: 1432-1459 [Electronic] Germany
• PMID: 34043041 (Publication Type: Journal Article)
• Copyright: © 2021. The Author(s).

Topics

• Adult
• Cost of Illness
• Cross-Sectional Studies
• Humans
• Muscular Dystrophy, Facioscapulohumeral (epidemiology, therapy)
• Quality of Life
• Socioeconomic Factors
• Surveys and Questionnaires