• Those affected. "We do not understand that our lives are priced"

For little Paul (nine years old), Orkambi is "the magic pill" that could prevent one day having to have a lung transplant for the cystic fibrosis he suffers. Every week, when he goes to the hospital, he asks impatiently if he will finally be able to take it. But the response he has received to date has always been negative. Although this medicine got the positive assessment of the Spanish Agency of Medicine (AEMPS) more than three years ago, it is not yet available because the Ministry of Health and the pharmaceutical company that produces it (Vertex) are still unable to reach An economic agreement.

The same goes for Symkevi, also from Vertex and also approved by the AEMPS. They are the only two drugs that stop the deterioration caused by this disease, although they do not work for all cases. Only in "patients with two copies of the F508D mutation and heterozygous with very specific mutations, which means about 350 people who could benefit, " explains Concha Prados, pulmonologist and president of the Spanish Society of Cystic Fibrosis.

While the tug of war lengthens in the negotiations between Sanidad and Vertex, the specialist points out, "the disease of the possible subsidiaries continues to progress and the deterioration produced during this time can no longer be recovered."

It is a genetic, chronic and degenerative condition that "affects many organs, especially lung function (sinusitis, nasal polyps, chronic respiratory infections, multiple exacerbations) and the digestive system (pancreatic insufficiency and associated diabetes)." In these people there is a decrease in water, sodium and potassium that leads to the obstruction of the channels that carry secretions, which causes their stagnation, infections and inflammations that destroy areas of different organs such as the liver, pancreas, system player and fundamentally the lung. Respiratory function is reduced to the point of requiring a transplant.

Although this is not yet the case with Paul, according to the results of the latest analytics and ultrasound, "the disease progresses and apart from the pulmonary and pancreatic involvement, now cystic fibrosis is attacking his liver, causing cirrhosis," he says. His mother, Estelle Delangle. "Every day he takes about 25 pills, performs three inhaled treatments and undergoes an hour of respiratory physiotherapy, with virtually no significant progress, because they are palliative treatments, nothing more." To this, we add the weekly checks at your referral hospital and the antibiotic you have to take for six months.

Your hopes are set on Orkambi (lumacaftor / ivacaftor). «My son has two copies of the F508D mutation. With this drug, we are confident that you will breathe normally again , that you do not need respiratory physiotherapy every day, that you do not have so many infections and never need a lung transplant ... It is a pity that these two drugs are able to stop the disease, there are people who cannot access them ».

After the celebration of World Cystic Fibrosis Day on the 8th, patients are concentrated today at the door of the Vertex Pharmaceuticals laboratory and the Ministry of Health. «We claim the patient's right to have access to the best medication available at the present time . We need and request an urgent agreement that avoids more suffering, ”says Blanca Ruiz, president of the Spanish Cystic Fibrosis Federation (FEFP). "We do not understand that being so proven its effectiveness, there is such a delay for money."

In other European countries such as the Netherlands, Austria, Denmark, Germany, the Republic of Ireland, Italy, Luxembourg, Sweden and recently in Scotland (United Kingdom), their refund is approved with a negotiated and agreed price. On September 30, both organizations will meet again. «We have presented our latest proposal this month for Orkambi and Symkevi, according to the criteria requested by the authorities and including a payment for results scheme, which we hope will be considered . We believe it is possible, ”the pharmaceutical sources point to this newspaper.

From the medical point of view, the drug stabilizes and slows the evolution of the disease in a certain group of affected, precisely the one that manifests the most serious symptoms. " Reduces the fall in respiratory function and the number of exacerbations significantly , which are the most important parameters that mark the severity of the affected," argues the pulmonologist Prados. "It decreases the need for insulin in diabetic patients, the number of hospital admissions for infections, the use of antibiotics and can also prevent transplants, at least delay them," he says.

According to the criteria of The Trust Project

Know more

  • Science and Health

ClimateThe oceans, way of becoming a nightmare for man

Research: A Spanish researcher discovers a marker that predicts long survival in patients with brain tumor

EnvironmentBrazil prohibits burning for planting to stop fires in the Amazon