Family from Alagoas tries in court to access medicine costing R$16 million to treat 5-year-old boy | Alagoas

Family seeks R$16 million medicine for child from Palmeira dos Índios

The family of a 5-year-old boy from Palmeira dos Índios, in Agreste de Alagoas, is fighting in court to gain access to a medicine that costs R$16 million to treat a rare disease, Duchenne muscular dystrophy, a degenerative disease with no cure. The problem is that, in addition to the high cost, the medicine is not available in the public health network, since there is no registration of it with the National Health Surveillance Agency (Anvisa).

Because of this, Richard’s life is filled with complementary therapy sessions. On the afternoon of this Thursday (9) he had to travel with his family from Palmeira dos Índios to Maceió to do one of the weekly physiotherapy sessions. Upon entering the place, the boy immediately loosened up and felt at home. He jumped on the trampoline, climbed a vertical ladder and assembled a structure with blocks. Ariani Cavalcante, a physiotherapist who accompanies the boy, explains that, although it may seem like a play session, the exercises are essential to maintain muscular vigor.

“The objective is to strengthen muscles to prevent atrophy. We do a series of movements without load, keeping him standing, squatting, things that a child of his age usually does. In the sessions we work on both muscular and cognitive issues, for this we use a lot of colorful things so that he also has this stimulus”, says the physiotherapist.

Neurologist Patrícia Pereira explains that the lives of patients with Duchenne disease must be divided between conventional treatments, where medicines and complementary therapies are used, but the cost of medication ends up making treatment unfeasible.

“These are still very expensive drugs, which are not accessible in the Unified Health System, but which are indicated for these specific cases depending on the genetic test. These diseases can even be diagnosed, with tests that are also very expensive, but are essential to begin treatment”, argues the neurologist.

And it was precisely for this reason that Richard’s family filed a lawsuit to seek access to the medicine that cannot be found in Brazil. Elevidys is a drug that is not yet registered with Anvisa and that Richard’s family is seeking access to. The drug is used through intravenous infusion, that is, it is applied once to the patient through the vein. This infusion contains a viral vector with a modified microdystrophin gene. This gene is taken to the muscle fibers and begins to be translated into microdystrophin protein.

Last year the use of the medication was approved by the Food and Drug Administratio, the FDA, the American government body similar to Anvisa. Approval in the United States was accelerated, as the drug showed sustained clinical benefit over 4 years in patients treated with it. At the end of April, a public hearing discussed the matter in the Chamber of Deputies here in Brazil, but there is still no prediction whether the drug will be registered with Anvisa nor whether it will be among the high-cost medicines that are made available by the Health Unic System.

According to Daniela Ávila, the family’s lawyer, the legal action is still awaiting the first degree decision. “The judge asked NATJus (Judicial Support Centers) to comment on our request. After that, it will be assessed by the first degree judge. We are very hopeful that this medication will be approved because studies indicate the improvement in the child’s quality of life is great. So we believe that although there is still no registration with Anvisa, this only happens due to slowness and not due to the inefficiency of the medicine”, argues the lawyer.

Without access to medicine, the boy’s family continues to look for ways to make treatment viable, since even if he is medicated he will continue to need complementary therapies and other costs which, even if they are cheaper, are still very high.

“The cost of his treatment is very high. In addition to the medication, we will have to stay in a place, possibly in São Paulo, for three months, there is all the cost of hospital, doctors, treatment after applying the medication. That’s why we’re knocking on every door, like in court, and also at the same time asking for this support, this help. Because if we win in court, there will also be all the treatment on the outside that is expensive”, said Leonice Barbosa, Richard’s mother.