The Real Cost of Duchenne Muscular Dystrophy: $120,000 PA

In Advocacy, Market Access by Cameron

Duchenne Muscular Dystrophy can cost the state around $120,000, and the patient $71,000 a year

Duchenne Muscular Dystrophy (DMD), an x-linked neuromuscular condition, is caused by the lack of the protein dystrophin caused by a mutation in the dystophin gene, and affects 1 in 3,600 boys.

Patients with DMD have muscle weakness from early childhood, requiring wheelchair use from usually their early teens. eventually, during mi to adult life, the condition results in fatal respiratory or cardiac complications.

A recent study, supported by GSK, has looked at data from DMD patients from across the EU  and US to determine the economic burden and total cost of illness. From the 770 patients surveyed for this report, the authors found that their mean annual direct cost of illness was estimated to be anywhere between $23,920 and $54,270. This puts the direct cost of DMD between 7 and 16 times greater than per capita health spending in the countries involved.

It is cheaper to be a DMD patient in Italy than the US to the tune of $40,000

But this is just the tip of the iceberg. The overall societal burden is estimated to be anywhere in-between $80,0000 and $121,000, a number that grows increasingly with disease progression. The rsult is a household burden reaching well beyond $50,000 a year.

The report also sheds light on the differences between countries when it comes to care for rare diseases. Whilst the pateints in Italy and the US must pay nearly $1,500 a year for medications, UK patients need only hand over $100 per year.

US patients also have the highest burden associated with ‘investments’. Here, investments indicate medical and nonmedical aids and devices (as well as investments in and modifications made to homes) that are not reimbursed.

But whilst the US may well be handing out fewer reimbursements, US patients apparently loose less in terms of leisure time. Living in Germany however will cost patients over $6,000 in loss of leisure time.

Interesting Findings:
It is cheaper to be a DMD patient in Italy than the US, to the tune of $40,000
The UK also has the lowest cost of for patients in terms of investments to their home and devices for which they receive no reimbursement.
The UK has by far the lowest cost of medicines for patients, at just $100 pa. Where as patients in Italy and the US can find themselves paying closer to $1,500 for medications.
US patients had the highest cost associated with visits to physicians and healthcare practitioners: $18,210 compared to the UK in second place with $8,230.
The cost of seeing a specialist in either Germany or Italy can be nearly ten times less than in the UK or US.
 The UK has the highest costs associated with late stage DMD treatment. Over $20,000 more than next highest spender, the US.
The UK spends more than double on non-medical community services, such as home help, personal assistance and transport, than any of the other countries in this report: over $19,000

DMD Expenditure


Find the report, published in Neurology, here.