GVS advice Tezacaftor/ivacaftor (Symkevi®) in combination with ivacaftor (Kalydeco®) extension of further conditions

The National Health Care Institute has completed its assessment whether the further conditions of tezacaftor/ivacaftor (Symkevi®) and ivacaftor monopreparation (Kalydeco®) could be extended. The application for the extension of the further conditions is related to specific patients aged 6 to 11 with cystic fibrosis. The National Health Care Institute advises the Minister to extend the List 2 conditions. 

This is a corrected version of the previously sent letter report dated 15 November 2021 in which the size of the additional costs has been corrected. 

Indication for which reimbursement is requested

The application for the extension of the further conditions is related to patients aged 6 to 11 with cystic fibrosis who are homozygous for the F508del mutation or heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A-G, 5945L, 5977F, R1070W, D1152H, 2789+5G–A, 3272-26A-1G and 3849+10kbC-, T. 

National Health Care Institute's advice

Tezacaftor/ivacaftor (Symkevi®) in combination with ivacaftor monopreparation (Kalydeco®) has already been included on list 1B with further conditions. Based on the new research results, we advise the Minister to extend the List 2 conditions of tezacaftor/ivacaftor (Symkevi®) and ivacaftor monopreparation (Kalydeco®) for the use in cystic fibrosis patients aged 6 to 11 years who are homozygous for the F508del mutation or heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A-G, 5945L, 5977F, R1070W, D1152H, 2789+5G–A, 3272-26A-1G and 3849+10KBC, T. 

Extension further condition for tezacaftor/ivacaftor

Only in combination with ivacaftor for the treatment of cystic fibrosis (CF) patients aged 6 and older who are:
1.    homozygous for the F508del mutation in the CFTR gene or
2.    heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A-G, 5945L, 5977F, R1070W, D1152H, 2789+5G–A, 3272-26A-1G and 3849+10kbC-, T.

Extension further condition for ivacaftor

Only in combination with tezacaftor/ivacaftor for the treatment of cystic fibrosis (CF) patients aged 6 and older who are:
1.    homozygous for the F508del mutation in the CFTR gene or
2.    heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A-G, 5945L, 5977F, R1070W, D1152H, 2789+5G–A, 3272-26A-1G and 3849+10kbC-, T.

This report is a summary of recommendations by the National Health Care Institute. The original text is in Dutch.