The drug for spinal muscular atrophy has already been prescribed to 33 children

The drug Zolgensma, which treats spinal muscular atrophy, has already been prescribed to 33 children in Portugal since baby Matilda received it in July 2019, according to Infarmed.

In October 2021, the National Medicines Authority (Infarmed) approved government funding for Zolgensma to treat spinal muscular atrophy type 1, a condition that affects approximately one in every 11,000 children.

It previously approved funding for the drug for baby Matilda, whose parents made a public appeal in July 2019 for their daughter to have access to the treatment.

Access to the drug is back in the news following a TVI report broadcast on Friday that two Portuguese-Brazilian twins traveled to Portugal in 2019 to receive Zolgensma – one of the most expensive drugs in the world – as part of a treatment that cost four million euros. According to TVI, there are suspicions that this was due to the influence of the President of the Republic, Marcelo Rebelo de Sousa, who has already denied any interference in the matter.

This situation prompted the Inspectorate General of Medical Activities (IGAS) to begin an audit process to check “if all the standards are applicable to this particular case,” Infarmed told Lusa.

According to Infarmed, the medicine was prescribed to 33 children during treatments carried out at the Lisbon Norte Hospital Center, Lisbon Central Hospital, Coimbra University Hospital Central, Porto Hospital Center, São João Hospital Center and São João Hospital Center. Vila Nova de Gaia/Espinho.

Regarding the cost of treatment, Infarmed responded to Lusa by saying that “due to confidentiality clauses included in funding contracts requested by laboratories holding the drugs,” it could not disclose the price of the drugs.

“However, in order to clarify some information related to the price of the medicine Zolgensma, we emphasize that after the financing process (negotiations with the laboratory to guarantee the best conditions for SNS, providing access to all citizens who need the medicine), the price turned out to be significantly lower than stated in the latest news [dois milhões de euros]”, says the medical authority in a written response to Lusa.

It also explains that payment is based on a risk-sharing agreement based on patient type and clinical outcome, for a period of four years and after the payment of the first interest (annually) if treatment fails. If we imagine the expected improvements, there will be no room for hospital departments to continue paying for drugs.

“The cost agreed and approved by the funding decision applied to patients who had already used the medicine up to that point. There was also an early access plan, put into practice before the completion of the funding process, which included treatment without costs,” emphasizes Infarmed.