Brasília – Today, there are three medications in Brazil for the treatment of Spinal Muscular Atrophy (SMA). The first to be incorporated into the SUS was Spinraza, from Biogen; then came Risdiplam, from Roche; and Zolgensma, from Novartis.

A single-dose medication, Zolgensma was incorporated into the public health system in December 2022, under the previous administration of the federal government. In this case, the update of the Clinical Protocol and Therapeutic Guidelines (PCDT) for SMA provides for the referral of babies who are identified with the disease for immediate treatment, but this document has not yet been published. With the change in government, little progress has been made.

Information obtained by Universo Coletivo AME via the Access to Information Law (LAI), last June, shows that, to enable access to the therapy, it is still necessary to sign the Risk Sharing Agreement (ACR) between the government and the manufacturer.

“According to the data sent, some meetings were held in 2023 to discuss and adjust the technical reports, and apparently there were no updates in 2024. When we asked the Ministry about the expected approval of the ACR, the answer was: ‘The signing of the agreement depends on the negotiation of the terms of the risk-sharing contract, a process linked to the internal evaluation of such terms by the responsible areas of both parties and which does not have a legally established deadline’”, say the mothers of the coalition.

Due to the delay, it is estimated that the government is losing out on savings of around 10% of the amount spent on purchasing the medicine. If the medicines were dispensed directly by the SUS, without needing to go through the courts for authorization, the government would have access to direct sales by the manufacturer at lower prices than those practiced on the market, would be able to pay in installments and would also avoid the cost of legal proceedings. The agreement with the manufacturer also provides for monitoring of the cases treated and proof of the expected effectiveness, otherwise, payment may be suspended.

Source: Medicina SA