GVS advice elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®)
The National Health Care Institute has completed its assessment whether elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor monopreparation (Kalydeco®) (hereafter: triple therapy) is interchangeable with a product that is included in the Medicine Reimbursement System (GVS). Triple therapy is indicated for patients with for the treatment of patients with cystic fibrosis (CF). The National Health Care Institute advices the Minister not to include triple therapy in the Health Insurance Regulation, unless some conditions are met, including a price reduction of at least 75%.
Registered indication
Patients with cystic fibrosis (CF) can be divided into different subgroups based on their genetic profile. CFTR modulators are medicinal products that focus specifically on the CFTR gene that plays a crucial role in CF.
Kaftrio® is a combination tablet. Each film-coated tablet contains 100 mg elexacaftor, 50 mg tezacaftor and 75 mg ivacaftor. Kaftrio® is indicated in a combination schedule with ivacaftor (Kalydeco®) 150 mg tablets, for the treatment of patients aged 12 years and older with CF who are homozygous for the F508del mutation in the CFTR gene (the homozygous subgroup) or who are heterozygous for the F508del mutation with a minimal function mutation (the heterozygous subgroup).
The National Health Care Institute's advice
For technical reasons, elexacaftor/tezacaftor/ivacaftor (Kaftrio®) could be placed on List 1B. The aforementioned combination treatment has a therapeutic added value compared to standard care. However, there are uncertainties about the long-term effects, the budget impact is high and cost-effectiveness is very unfavourable.
The National Health Care Institute therefore recommends the minister not to include elexacaftor/tezacaftor/ivacaftor (Kaftrio®) in combination with ivacaftor (Kalydeco®) in List 1B of the GVS unless the following conditions are met:
- A price reduction of at least 75% for the triple therapy in combination with ivacaftor monopreparation. The National Health Care Institute advises the minister to renegotiate with the marketing authorisation holder for a price agreement for the entire portfolio, so for all CTFR modulators. The National Health Care Institute also advises the minister to negotiate about future indication extensions.
- Reaching agreement with the physicians association on the appropriate use of the efficient treatment application.
- The collection of long-term data by the physicians association to evaluate the effectiveness and safety of the triple therapy in combination with ivacaftor monopreparation. The National Health Care Institute will use these data to monitor long-term effects.
The National Health Care Institute also advises the minister to enter into a European collaboration for (price) negotiations and data collection.