From science, hope for young patients with SMA, in Italy 16 the centers for the administration of the first therapy in the world

"The conclusion in very short times of the Spinraza authorization process"Comments the Director General of AIFA, Mario Melazzini"it is a victory of Science and of the Italian regulatory system. Finally, SMA's little patients, in the past the subject of unscrupulous speculations of charlatans, have available a therapy, supported by robust scientific evidence and whose costs are borne by the National Health Service at the conclusion of a negotiated procedure conducted in very short times. The country system confirms the ability to offer concrete answers to the demand for health, also, as in this case, for innovative high-cost therapies for rare genetic diseases ".

A few months after the approval of the first therapy for spinal muscular atrophy (SMA), Spinraza, there are already 16 hospital centers, distributed in 11 Italian regions, authorized to administer it. 135 patients have already started receiving the treatment and 190 those considered eligible who are waiting for the first administration, according to the data of the AIFA Monitoring Registers, updated to 12 March 2018.

A real revolution in the treatment of a rare genetic disease whose symptoms occur shortly after birth. The smallest patient enrolled in the AIFA Registry has one month of life, while the average age of all patients is around 4 years.

Spinraza's reimbursement was the culmination of a long scientific, clinical and regulatory journey. A real challenge for the National Health Service, represented by the definition of the criteria for access to treatment with this innovative medicine with high therapeutic value and very high cost. More than 115 thousand euros per single dose is the price set to the public, with respect to which AIFA has contracted a very significant reduction, as part of a confidential negotiating agreement.

The drug is available in Italy thanks to the national fund of innovative drugs (non-oncology), established by the December budget law 2016 and which provides a loan of 500 million euros in three years, up to 2019.

"The basic objective of our activities and fundraising that we carry forward is to support each region in the provision of at least one specialized structure" adds Daniela Lauro, president of Families Sma, the association that brings together patients suffering from the disease and their parents. "We are just over half of the path and to complete the Sma Families project, it offers all the specialized centers a support to 360 degrees, through the contribution of a team of experts that intervenes in different fields: medical-scientific, legislative, logistic, psychotherapeutic ".

 

From science, hope for young patients with SMA, in Italy 16 the centers for the administration of the first therapy in the world

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