It is the most expensive, unique, and organized treatment approved by the Institute. The child will receive the medicine at Karolinska sjúkrahúsið in Stockholm. It is assumed that one single administration of this medicine could cost well over a third of a million ISK.

The treatment is classified as gene therapy and is provided for this genetic disease which until five years ago was not possible to treat. SMA is a disease that causes progressive weakness due to nerve damage. The prognosis of children born with this disease has been extremely poor and for many of them it was not expected that they would live for only a few years. There are already medicines in use that are given to keep the disease down. Two such medicines are registered in Iceland and have already been given to several children in Iceland. This has opened up the possibility of keeping their disability caused by the disease to a minimum. Recently, treatment applications were made abroad for a young child who was diagnosed with the disease SMA at a newborn screening.

In May 2020, the European Medicines Agency approved a treatment with a medicine called Zolgenesma, which is a single-dose administration for the purpose of curing the gene defect that causes the disease SMA. This is called gene therapy, i.e. it is given a healthy gene that is transported by what is called a virus vector that is responsible for restoring the medical gene as it can repair the defective DNA. There are strict conditions for the use of this medicine. It is not available here in Iceland and treatment abroad with it is only available at the largest pediatric hospitals, where the knowledge and experience of treatment of this patient group is available and the conditions set for the administration can be met. It is necessary to administer the medicine during the first months of children who are diagnosed with SMA at or shortly after birth. Children who benefit from this treatment can be expected to be born in Iceland on average every two to three years. There are five different manifestations of the SMA disease but the medicine is only used for one of them, i.e. SMA 1, which are children who are diagnosed with the disease either during a newborn screening or in the first weeks of their lives.

In this case, the assessment of these treatments is based on a professional and economic assessment, also called Health Technology Assessment. The cost of the treatment is then assessed in the context of the improvement in the quality of life and the patient’s life expectancy, along with savings in the cost of medical care, nursing and care that these individuals would have been expected to need during their lifetime. The assessment of these treatments clearly shows that even if the cost can be very high, the benefits can be even greater.

says Sigurður Helgi Helgason, CEO of Iceland Health.