In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscular dystrophy and that there is a considerable financial burden carried by affected families. Funded by GlaxoSmithKline (GSK), the aim of this study was to estimate the total cost of illness and the economic burden of Duchenne.
The research, published today in the journal Neurology, was led by Newcastle University (UK) and the Karolinska Institutet (Sweden) and carried out in collaboration with TREAT-NMD patient registries for Duchenne in Germany, Italy, the UK and the United States (DuchenneConnect). Researchers asked patients and their primary caregivers to complete a questionnaire on their experience of living with Duchenne and the impact this had on medical care, employment, leisure time and quality of life. A total of 770 patients/caregivers completed the survey, with the United States having the largest number of participants (284). Thank you to all the DuchenneConnect families who participated in this important study!
Researchers found that there are many different costs accompanying Duchenne. The analysis of the data showed that the direct cost of the illness across the countries was between eight and sixteen times higher than average health expenditure per person. The study also showed that the indirect and informal care costs were substantial, each constituting between 18% and 43% of total costs.
Professor Kate Bushby, co-author of the study and one of the founding co-ordinators of the TREAT-NMD Network, said:
Rare diseases are massively underfunded generally and the cost to society is often hidden as so many costs are borne by the family themselves. Our figures show that Duchenne imposes a severe economic burden on the family and friends of affected people, as well as society as a whole. It is essential that more money is spent trying to find ways of easing this burden on patients and families.
Holly Peay, Director of DuchenneConnect, said:
Outcomes from the study about the costs related to caring for Duchenne and the more global impact on families are important and meaningful for informing future care and support of people and families with Duchenne. Recognizing the complete economic context of Duchenne is crucial to fully understand the potential impact of new therapies, and may inform evidence-based health policies, including reimbursement for treatment costs. We appreciate the participation of DuchenneConnect’s United States families in this important study.