The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to not reimburse elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®) from the basic healthcare package just yet. This medicinal product can be used for certain patients with rare variants of cystic fibrosis. Missing information from the marketing authorisation holder does not allow us to determine at this time what a good price is for this medicinal product. The National Health Care Institute has requested the marketing authorisation holder to provide this information.
Current state of affairs: advisory report sent to the Minister
The National Health Care Institute has sent this advisory report to the Minister of VWS. The Minister makes the final decision whether or not to reimburse the medicinal product from the basic healthcare package.
Elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor is intended for certain people with rare variants of cystic fibrosis
Elexacaftor/tezacaftor/ivacaftor and ivacaftor are the active substances. The brand names are Kaftrio® and Kalydeco®. The medicinal product is available as tablets that you swallow or as granules that you first mix with soft food or liquid and then swallow.
The medicinal product can be used for people with cystic fibrosis (CF). This is a hereditary chronic disease in which the CFTR protein works less well. This makes the body’s mucus tougher. Accumulation of the thick and sticky mucus in the lungs causes coughing, a reduced pulmonary function and recurrent bouts of pneumonia. In addition, people with cystic fibrosis often suffer from reduced nutrient uptake and reduced metabolism. This is caused by the accumulation of tough mucus in the pancreas and intestines. As a result, children with cystic fibrosis often have a delayed growth.
Elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor is already reimbursed for patients aged 2 years and older with cystic fibrosis who have at least one F508del mutation in the CFTR gene. The manufacturer has requested an expansion of the reimbursement, namely for people with cystic fibrosis with a rare mutation that still produces CFTR protein.
Advice from the National Health Care Institute on the expansion of the reimbursement conditions for elexacaftor/tezacaftor/ivacaftor and ivacaftor
The National Health Care Institute advises the Minister of Health, Welfare and Sport to not yet reimburse elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®) from the basic healthcare package. This is because the economic analysis provided by the marketing authorisation holder is of insufficient quality. As a result, the National Health Care Institute cannot assess what a socially acceptable price would be for the health benefits of patients. That is necessary for reimbursement from the basic healthcare package, in order to prevent an excessively high price from displacing other good care. The marketing authorisation holder has had opportunities to correct incomplete information and to provide missing information, but has not done so. The National Health Care Institute has also concluded that the medicinal product has an added therapeutic value for patients. The National Health Care Institute also recognises that the outcome of the advice is disappointing for patients and healthcare providers. The National Health Care Institute therefore calls on the marketing authorisation holder to submit sound information after all. This would allow a new assessment to start as soon as possible, to give patients clarity about the reimbursement of the medicinal product.
For more information on the GVS and lists 1A, 1B and 2, see the page on ‘Reimbursement of outpatient medicines’.
More information or questions?
If you have any questions about this advice, please send your question to the National Health Care Institute via warcg@zinl.nl. If you have questions about the reimbursement of a medicinal product, the personal expenses or whether you should pay a contribution, please ask your health insurance provider.
How did the advice come about?
The Scientific Advisory Board (WAR) advises the National Health Care Institute about the assessment. The Minister makes the final decision whether or not to reimburse the medicinal product from the basic healthcare package.
Explanation about the reimbursement of medicinal products
Elexacaftor/tezacaftor/ivacaftor is an extramural medicinal product. ‘Extramural’ means medicines for home use that can be obtained at the pharmacy with a prescription from a physician. They are only reimbursed from the basic healthcare package if they are listed in the GVS.