This is reported by the outgoing State Secretary of Public Health Blokhuis. The drug is not reimbursed for all patients with cystic fibrosis. The reimbursement will only apply to a group of approximately 800 patients aged 12 years and older, with a specific variant of the disease.
For patients with a different variant and younger patients, the efficacy of the drug is being further investigated.
Kaftrio
Kaftrio, as the drug is called, ensures that people with cystic fibrosis get better lung function and have less difficulty breathing. Some patients also have fewer lung attacks, such as violent coughing and shortness of breath.
The drug has three active ingredients that target a specific protein, the CFTR protein. That protein is defective in patients with cystic fibrosis.
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Cystic fibrosis or cystic fibrosis
- 1500 people in the Netherlands have cystic fibrosis, including 650 children
- This makes it the most common rare disease in the Netherlands
- CF is an incurable, hereditary condition
- Due to a genetic abnormality, the body produces exceptionally tough mucus, causing organs such as the lungs and pancreas to malfunction
- Symptoms vary by patient and include: prolonged coughing and phlegm, recurrent respiratory infections, constipation, stunted growth and reduced fertility
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High price
Manufacturer Vertex initially asked a much too high price for Kaftrio; about 194,000 euros per patient per year. In total, the drug would cost the Netherlands 156 million euros annually. The Zorginstituut had therefore advised the cabinet to only include it in the basic package if the price would fall by at least 75 percent.
It is not known whether the price that is now being paid is actually that much lower. Price agreements made with pharmaceutical companies always remain secret. Blokhuis only writes that he has agreed an ‘acceptable price’ with the manufacturer.
Orkambi
The National Health Care Institute had also advised to make price agreements for the entire group of so-called ‘CFTR modulators’, medicines that target the CFTR protein, preferably in a European context. Whether that happened is unclear.
The ministry had to renegotiate price each time about previous medicines from this same group and from the same manufacturer, such as the infamous Orkambi drug, also very expensive. Negotiations that sometimes took a long time.
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Undesirable
The Zorginstituut also thinks this is undesirable. “With every new step, the manufacturer places a higher bill with society,” said chairman Sjaak Wijma in May. “While they build on knowledge that has been developed earlier. That should actually lead to a lower price.”
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