Cystic fibrosis sufferers plead for government help to obtain life-saving medication
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    Cystic fibrosis sufferers plead for government help to obtain life-saving medication

    Testing lung function © Virginia Commonwealth University

    Mucovereniging, the association representing sufferers of cystic fibrosis and their families, has called on the medical insurance authorities to begin immediately to reimburse patients who buy the new “miracle drug” for the condition.

    Cystic fibrosis is an inherited disease that affects the lungs and digestive system, affecting the secretions of mucus and digestive fluids, making them more viscous and liable to cause blockages that can lead to infection.

    The new drug is trade named Orkambi (medical name (lumacaftor/ivacaftor) which, according to the European Medicines Agency, “is used in patients who have a genetic mutation called the F508del mutation. This mutation affects the gene for a protein which is involved in regulating the production of mucus and digestive juices. Orkambi is used in patients who have inherited the mutation from both parents.”

    The drug was licensed for prescription in Europe on the basis of two studies of children over the age of 12 and one study of children under 11.

    The three studies reported significant improvements in lung function in patients using Orkambi compared to placebo.

    However discussions between the Belgian government and the drug’s manufacturer, Vertex Pharmaceuticals based in Boston in the US, have been going on since 2016 without producing an agreement which would allow the Belgian system to reimburse users. Some 1,319 people in Belgium suffer from the condition, although not all of them are suitable candidates for treatment with Orkambi.

    Here at the Mucovereniging, we are bitterly disappointed that an agreement remains elusive,” the association said in a statement last July, when the latest talks broke down.

    Orkambi is after all an innovative and life-enhancing medication of a new generation, which works on the origins of cystic fibrosis. Examples from other countries and from Belgian patients who have tested Orkambi show that this drug can make a world of difference.”

    Discussions on the price of the drug for the health insurance agency Inami are at the moment stalled, but the association hopes for a breakthrough by the end of the year.

    On the one hand we are of course delighted that there are new treatments appearing which can improve the health of our patients,” said Stefan Joris, director of the association.

    On the other hand we have to regret that Belgian patients do not have access. Unless an agreement can be reached rapidly on a medication which is even today available in neighbouring countries, we fear for the future.”

    Alan Hope
    The Brussels Times