It has been months of negotiation with the pharmaceutical company Novartis until finally, the Interministerial Commission on Drug Prices (CIPM) agreed, last October 28th, to include in the pharmaceutical provision of the National Health System (SNS) the gene therapy onasemnogene abeparvovec for the treatment of spinal muscular atrophy (SMA) in its most severe expression, type 1, and in pre-symptomatic patients.
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This gene therapy will be indicated in patients with type 1 spinal muscular atrophy, the most severe type, and in presymptomatic patients
The therapy is being financed by the Spanish Health Service as of Wednesday, December 1st, by means of a mixed model consisting of a pay-for-results and a pay-for-volume agreement. It is estimated that some 30 children a year will benefit from the treatment. The prices involved in these negotiations are not usually known, but everything points to the fact that this is a very high-priced drug. The European Medicines Agency (EMA) gave its conditional authorization for the use of this therapy in March 2020. It finally reaches Spanish patients 20 months later.
Developed by Novartis and marketed under the name Zolgensma, the drug is administered intravenously in a single dose. Its administration increases the expression levels of the SMN protein in the motor neurons of children suffering from this disease. It is the second drug included in the SNS, following the funding of Spinraza since 2018, for this disease and the first gene therapy in this indication.
It is administered intravenously in a single dose and allows to increase the expression levels of the SMN protein in motor neurons
Spinal muscular atrophy is a rare disease caused by an alteration in the SMN1 gene which leads to progressive muscle weakness, as the lack of SMN protein causes the progressive death of motor neurons in the spinal cord. In patients with type 1 disease, the baby is born with severe weakness and its life expectancy does not exceed two years. This rare pathology represents the most frequent genetic cause of infant mortality.
The financing system, based on payment by results and by volume, means that payments to the pharmaceutical company are conditional on the achievement of results in the patients treated, and the price decreases as the number of patients treated with this therapy increases. According to the Ministry of Health, this mixed financing model “addresses clinical and financial uncertainty in the interest of patient access to the drug and the sustainability of the SNS”.
The Ministry of Health has been financing the therapy since Wednesday through a mixed model consisting of a payment-by-results and a payment-by-volume agreement
Eligibility criteria have also been agreed upon to identify patients in whom, based on the evidence from clinical trials, clinical benefit is expected. That is an improvement in the quality of life and life expectancy of the patients who receive it. Its results will be measured through Valtermed, the SNS information system for measuring health outcomes in the use of drugs. Treatment must be initiated and administered in healthcare centers and supervised by a physician with experience in the management of SMA patients.
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First published in iSanidad, a third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.
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Author: Olivia McCall, Biotech – Born2Invest