The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to expand the reimbursement conditions for elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®). This medicinal product can be used in certain patients with cystic fibrosis. This medicine has already been included in the Medicines Reimbursement System (GVS) for patients aged 6 years and older with cystic fibrosis. If the Minister adopts our advice, this medicinal product can also be reimbursed for patients aged 2 to 5 years. However, we do recommend further conditions for reimbursement.
Current state of affairs: advisory report sent to the Minister
The National Health Care Institute has sent this advice to the Minister of VWS. The Minister makes the final decision whether or not to reimburse the medicinal product from the basic health care package.
Elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor is intended for certain children with cystic fibrosis
Elexacaftor/tezacaftor/ivacaftor and ivacaftor are the active substances. The brand names are Kaftrio® and Kalyedeco®. The medicinal products are small pellets (granules) that are mixed with soft food or liquid and you swallow them. A child takes 1 sachet of granules with elexacaftor/tezacaftor/ivacaftor in the morning and 1 sachet of granules with ivacaftor in the evening every day.
The medicinal product can be used for people with cystic fibrosis. Also known as CF. This is a hereditary chronic disease in which the CFTR protein does not work properly. CFTR is the abbreviation of cystic fibrosis transmembrane conductance regulator. The CFTR protein ensures healthy and fluid mucus. Due to an error in the CFTR protein, it works less well, and the mucus becomes thick and sticky. Accumulation of the thick and sticky mucus in the lungs causes coughing, a reduced pulmonary function and recurrent bouts of pneumonia. Children with CF often have a stunted growth due to reduced nutrient uptake and reduced metabolism.
Advice from the National Health Care Institute on the reimbursement of elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor
The National Health Care Institute advises the Minister of VWS to expand the reimbursement conditions for elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®) for patients aged 2 to 5 years. However, to be eligible for reimbursement, the following conditions must be met:
- a price reduction of at least 75% of elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor;
- appropriate use arrangements for the medicinal products with the physicians association for the effective use of the treatment;
- long-term data collection on the effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor in this group of patients.
We advise the expansion of the further conditions, the so-called List 2 conditions.
Conditions for reimbursement of elexacaftor/tezacaftor/ivacaftor
Only in combination with ivacaftor for the treatment of cystic fibrosis (CF) patients aged 2 years and older who have at least one F508del mutation in the CFTR gene.
Conditions for reimbursement of ivacaftor
Only for the treatment of cystic fibrosis (CF) patients aged 2 years and older who have at least one F508del mutation in the CFTR gene, in combination with elexacaftor/tezacaftor/ivacaftor.
For more information on the GVS and the Lists 1A, 1B and 2, see page ‘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 National Health Care Institute advises the Minister after the assessment. The Minister makes the final decision whether or not to reimburse the medicinal product from the basic health care package.
Explanation about the reimbursement of medicinal products
Elexacaftor/tezacaftor/ivacaftor in combination with ivacaftor are extramural drugs. Extramural means: medicines for home use that can be obtained at the pharmacy with the prescription from a physician. They are only reimbursed from the basic health care package if they are listed in the GVS.