Supreme Court reprimands hospital for choosing compassionate use drug for child with rare disease.

The Supreme Court’s decision calls into question the bureaucratic hurdles Barcelona’s Sant Joan de Deu hospital placed on a child with a rare disease to receive a compassionate-use drug that more than thirty minors were already receiving across the country. Spain. . The judges agreed with the mother, considering that it is the competent authority, in this case the Spanish Agency for Medicines and Health Products, which must examine and consider whether the patient is fit to receive the drug. With its refusal, the private center, which is subsidized by the Catalan Health Service, “ruled out all possibilities of cooperation”, criticizes the Supreme Court in its decision.

The case, which the magistrates studied, comes from Barcelona. The patient is a boy with Duchenne muscular dystrophy, a rare disease that affects one in 5,000 children worldwide and causes loss of muscle function, and who has been treated for years in the hospital’s neurology service. Sant Joan de Déu in Barcelona. His doctor issued a report recommending treatment with the active ingredient ataluren, trade name Translarna, at the time under “conditional” authorisation, which has now been rejected by the European Medicines Agency (EMA). A larger clinical trial was awaited. Confirm its benefits.

The mother requested that her son receive this treatment for compassionate use, a formula to take advantage of drugs that are not yet sold in the country when there is no other therapeutic alternative, but the center’s medical leadership and the Catalan Health Service refused. Request processing. Although the woman presented a statement from the association of patients with this rare disease, which confirmed that 33 patients in Spain were treated with the active ingredient of this medicine.

The Supreme Court annulled the decision of the High Court of Catalonia, which excluded the decision of the mother, and confirmed the decision of the court of Barcelona, ​​which gave her in the first instance. The judges criticize that the Catalan health system refused “not only the authorization but also the previous step for the competent authority to be able to decide on the request, because it refused to process the request”. In short, it “ruled out any possibility of cooperation”.

The mother, he explains, provided enough evidence to demand the drug, and that all subsequent decisions, including the court, have given the woman an “impossible” mission to provide even more evidence. He, according to the Supreme Court, “excessively justified the existence of special authorization for other patients in the national health system”, and yet his request was not even considered. “Discriminatory and unjustified” treatment, given that the drug was “authorized” but only for patients with Duchenne muscular dystrophy.

This decision, in any case, was not the responsibility of the hospital or the Catalan Health Service: it was the “public authority responsible for the special authorization decision” in the national health system. In this case, this competence corresponds to the AEMPS, which is responsible for considering and authorizing compassionate use of drugs on an individual basis when no other treatment is available and when it is believed to benefit the patient.

The decision comes a month after the EMA’s Committee for Medicinal Products for Human Use, of which the Spanish agency is a part, ordered Translarna’s marketing authorization not to be renewed across the EU. The regulator makes the decision after failing to demonstrate the drug’s effectiveness following a review of the drug’s benefits and risks and studies that failed to show a corresponding improvement in patients taking it.

AEMPS said in a statement that the studies “did not show that the drug was effective in patients with progressive walking impairment in whom translarnat was expected to benefit more than the other patients in the study.” . In patients treated with traslarnate, the distance they could walk in six minutes after 18 months of treatment decreased by approximately 82 meters from the start of the study to 90 meters in the placebo group. This difference was not statistically significant.”

Source: El Diario

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