most expensive disease in the world . Fortunately , only one dose is needed . In the case of Nusinersen and Risdiplam , it would cost between 400,000 and 800,000 euros per year ( and , of course , per patient ).
Why these sky-high prices ? We will never know . Pharmaceutical companies usually negotiate the price of the drug independently in each country and with zero transparency . This leads them to consider the drug as a commodity , the price of which is not necessarily linked to its development and production cost . Rather , it is the price one is willing to pay for the life and well-being of patients in every part of the world .
By the way , none of the three drugs cures . They only slow down the progression of the disease – which is no small thing .
Future for SMA and other rare diseases
What will happen in the future with these rare disease treatments ? Well , obviously , the market will eventually drive prices down . Treatments such as gene therapy have already been used for other diseases , and will undoubtedly continue to appear and , more importantly , to be perfected . Ultimately , the universality of their use will favour lower prices .
On the other hand , we can pin our hopes on drug repurposing , i . e . the use of existing drugs for other diseases . This has the enormous advantage of greatly reducing costs and shortening research times .
In conclusion , it is indisputable that SMA is a devastating disease , and that SMA patients will always have the right to complain about the bad luck we were born with . But at least I hope I have shown that this disease has a special charm at the molecular level which , for those of us who suffer from it , also gives us a certain right to call it “ the most beautiful disease in the world ”.
This article was originally published in The Conversation . Read the original article here .
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