The exorbitant price of the only drug capable of treating people with spinal muscular atrophy, a rare and fatal genetic disease, is controversial.
Nearly 85,000 euros per bottle. This is the price of Spinraza, the only drug capable of treating people with spinal muscular atrophy, a rare and fatal genetic disease. “This drug with an uncertain risk-benefit balance is sold in the United States by Biogen at a record-breaking price: approximately 500,000 euros the first year, then 250,000 euros per year in subsequent years. At nearly 85,000 euros the bottle, this leads to an additional treatment cost of more than one million euros in the first year”, deplores the independent monthly Prescribe.
The treatment is not 100% effective
An exorbitant cost, especially since the treatment is not 100% effective.”In patients with severe infantile proximal spinal muscular atrophy, i.e. clinically manifesting before the age of 6 months, the administration of nusinersen seems to reduce mortality in the short term, without a significant effect on disability. We do not know what happens in the longer term and there are still many unknowns concerning the adverse effects. review.
Spinal muscular atrophy is the name given to a group of inherited diseases characterized by muscle weakness and atrophy. They attack the nerve cells that control the muscles, causing them to deteriorate. The arms, legs and trunk are gradually affected. It never affects intellectual functions. Nearly one child in 10,000 births is affected by this disease, which represents approximately 130 children in 2013 in France and one birth per day in the United States.
A life expectancy of less than 2 years
There are several types of spinal muscular atrophy (including 3 infantile ones). Types I and II are those which are diagnosed the earliest (before 6 months and 18 months respectively) and which are the most serious. They affect lifespan (a life expectancy of less than 2 years in the absence of assisted ventilation for type I and a 25-year survival rate of approximately 75% for type II).
“Given the uncertainty associated with the extrapolation of health results, longer-term data and in current practice are expected in order to support the hypotheses formulated and document all the results to be taken into account”, indicates HAS on his side.
Patients are also outraged in Slate.com : “As a drug promoter and actor, we know that the development of innovative drugs for rare diseases that have no therapeutic alternative is risky and expensive. Also, it is normal that these costs can be remunerated and that the innovation is rewarded. On the other hand, it is not acceptable that this generates disproportionate margins which will prevent access to treatment for many patients. The costs of research and production, like the large number of patients concerned, cannot justify such a price level.”
To market Spinraza in France, the Biogen laboratory must still negotiate the price of the drug with the Economic Committee for Health Products. The drug received its European marketing authorization in 2017.
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