OBJECTIVES

: Duchenne Muscular Dystrophy (DMD) is a rare genetic muscle wasting disorder that mainly affects boys, typically diagnosed from around the age of 2. Patients will require care from a number of specialities due to the wide ranging effect of the disease – not just from neuromuscular specialists, but respiratory specialists, cardiologists, endocrinologists, physiotherapists and more. Additionally as sufferers are mostly children and young adults, there is an impact on the family, as well as a societal costs. To understand the evidence on this burden we conducted a series of literature reviews to identify the available evidence to populate economic models.

METHODS

: To identify relevant literature, we first searched for burden of illness studies directly, recognising that evidence may already exist. We then performed searches for areas likely to impacted by DMD to find studies that may look at each aspect. Searches were performed to identify inpatient and acute care resource use, other medical costs (for example wheelchairs and medical devices), other governmental costs, impacts on the cost of living, education, productivity losses, and the impact on families.

RESULTS

: CONCLUSIONS

: Whilst there is a large volume of data on the burden (particularly the cost) of DMD, to provide inputs for economic modelling further research – either novel or involving reanalysis of existing studies, will be required.