Cystic Fibrosis finally reimbursed

Edition 28 December 2017, by Anastasiia Myronenko

After long and challenging negotiations between the manufacturer and the Dutch Ministry of Health, Welfare and Sport, the patients suffering from cystic fibrosis will finally be compensated for the cost of the medication.

Cystic fibrosis is a rare, but very dangerous and incurable genetic disease. It is caused by mutations in genes and results in difficulties of forming and transporting of the body mucus. It is vitally important, since the mucus helps to get rid of bacteria and small foreign particles that we inhale (for instance, dust). It moisturizes and protects the body tissues. Additionally, it transports enzymes to the intestines and makes sure that the fats are well digested. If, due to cystic fibrosis, the mucus gets too thick, such organs as lungs, pancreas, liver and intestines get heavily affected. Therefore, the life expectancy among people diagnosed with cystic fibrosis reaches on average 35 to 45 years. According to the World Health Organization, it strikes 1 in 2000- 3000 newborns in the European Union and 1 in every 3500 births in the United States of America. In the Netherlands it is diagnosed in about 1,500 people, 800 of which are adults. Even though it doesn’t look like much, for people who have to face it, life is a constant struggle.

The symptoms of the illness include, among others, such severe conditions as persistent coughing, at times with phlegm; wheezing or shortness of breath; an excessive appetite but low weight gain; very salty-tasting skin. Symptoms are different for every patient, regardless of age, partly because of the variety of mutations in the gene – more than 1,000 mutations. Therefore, each case should be treated with extra attention and seriousness. The treatment is purely symptomatic, as until today the scientists did not manage to find a cure for the illness.

In the Netherlands the most common drug to treat patients with cystic fibrosis is Orkambi, manufactured by Vertex Corporation. According to their calculations, the treatment for the disease costs around 170,000 Euros a year per patient. It is undoubtedly a large amount of money. Therefore, its inclusion in the basic insurance package remained undetermined. Starting from early this year, there were multiple negotiations between the Ministry of Health, Welfare and Sport and the manufacturer of the medication. At the beginning of October, Edith Schippers, the Dutch Minister of Health, Welfare and Sport (VWS) at that time, claimed that she “made great efforts to reach a positive outcome to make the treatment accessible for the patients”. Unfortunately, the price demanded by Vertex remained “unacceptably high”. The Dutch Cystic Fibrosis Foundation (NCFS) commented on the situation: “Since the resumption of negotiations, CF patients are in great uncertainty about their fate and that is devastating. It is unacceptable that the chance of a possible major improvement in their health is being taken away. We are talking about people’s lives”.

However, at the end of October the Minister of Health, Ms. Schippers, and the manufacturer seemed to reach consent. As a result, from the 1st of November 2017 Orkambi will be included in the basic insurance package and reimbursed. The price that both parties agreed on remained confidential, though. The Minister said that “the agreement with the manufacturer is beneficial for both the policyholders and the health care affordability in general. It is a pity that the price should remain confidential, but without that confidentiality there would have been no deal”. Ms. Schippers also added that she was happy with the fact that the patients who benefit from the medication can now afford to use it. “Their patience was heavily tested. The agreement is, of course, in the first place in the interests of the afflicted and their families”.