Most expensive medicine in the world soon to be covered in basic health insurance package – NL Times

Young children with the life-threatening muscle disease, spinal muscular atrophy (SMA) can receive the medicine Zolgensma from November 1 as part of the basic healthcare package.

The drug has not yet been reimbursed due to the high price of around two million euros per treatment, making it the most expensive medication in the world. The Netherlands together with Ireland and Belgium have forced a reduction from manufacturer Novartis.

Through this collaboration, the medicine will become more affordable and accessible and this is wonderful news for the patients, Health State Secretary Paul Blokhuis said. The minister wants to bring down prices of other expensive medication in the future.

SMA is a serious illness that usually manifests shortly after birth and progressively affects muscles and the nervous system. Half of SMA patients die in the first year of their life. The disease is caused by a missing gene as a result of which an important protein is insufficiently produced.

It is incredibly special that for the first time there is a drug that tackles the cause of this serious condition and does not just fight the symptoms, directors of Muscle Disease Netherlands Marcel Timmen said.

Zolgensma is a one-off gene therapy that tackles SMA at the root by inserting the missing gene. This can be done even before children develop symptoms because the illness will be included next year in the initial blood screening of children after they are born. A baby can be treated before symptoms show. Unnecessary, irreparable damage can then be prevented, Timmen said. Zolgensma offers children with SMA and their parents a whole new perspective on life.

Between ten to twenty children were predicted to be healed every year in the Netherlands through this therapy.

The financial details of the deal were not revealed. The National Health Care Institute has previously announced that the drug will only be included in the basic health insurance package if the price would at least be reduced by half.

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Most expensive medicine in the world soon to be covered in basic health insurance package - NL Times