Juan Da Silva, President of the Spanish Cystic Fibrosis Foundation and the Spanish Cystic Fibrosis Federation.
The pediatric access to Kaftrio, a treatment for patients with cystic fibrosis, has not yet been approved in Spain through public funding. Last May, the Interdepartmental Commission on the Prices of Medicines (CIMP) proposed to use half of the currently funded Kaftrio dose for children over 12 years of age with children aged 6 to 11 years, a decision that the Spanish Federation of Cystic Fibrosis as “inadequate” .
Approval could come at the end of this September, when the Interagency Commission publishes the new drugs and marketing authorization indications. This will be the first approval with Cesar Hernandez as the new Director General of the National Health System (SNS) General Services Portfolio and of Pharmacy, replacing Patricia LaCruz. The Spanish Cystic Fibrosis Federation assures that they see the change of address “with very good eyes”. “Hernández’s entry assures us of an interest in access to innovation in Spain,” said Juan Da Silva, president of the Spanish Cystic Fibrosis Foundation and the aforementioned Spanish federation, who highlighted Hernández’s career. “He knows the processes very well and has a lot of experience,” he says.
However, he explains that the only concern is that the relay delays negotiations: “Everything indicates that will not be the case, but we have a little concern that the new appointment interferes with the approval of Caftrio.”
“Adult” doses adapted to children
Currently, in the absence of pediatric indication approval, the norm is to adapt the adult dose to children. “It may be a transitional option until pediatric approval arrives, but it can by no means remain the only option,” Da Silva says. So the president hopes that “At the next meeting, the Interdepartmental Commission reaches an agreement on financing for the pediatric indication and that it may be available in November’.
The approval of the generic drug with this specific use in children was approved by the Ministry, according to Da Silva, “with conditions that were not agreed with the laboratory.” In this case, Vertex Pharmaceuticals. “The pharmacist is making accusations because he disagrees. They are saying there is a pediatric indication available that is most appropriate,” he explains.
The indication has already been approved by the EMA
On the other hand, the President of the Federation claimed that This indication has undergone a safety process and has been approved by the European Medicines Agency (EMA) last January. “We were confident that the approval for Spain would arrive in May, so the drug would be available in June or July: again we are facing a delay compared to Europe,” explains Da Silva, adding that “our country is following the lag behind its neighboring countries when it comes to innovation penetration.
“Spain follows the tail of Europe in terms of the arrival of innovations”
The ministerial review is only part of the process for approval of the market of a drug. Once published, the laboratory in question must accept the proposal. After all procedures are completed, the drug becomes available. In this case, as the laboratory submitted claims, approval is still up in the air. “It should not be normal for the laboratories to present one proposal and the Commission another. All it does is prolong the process,” Da Silva condemns, although he ends with a message of hope. “Everything points to approval coming in September.”
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