Advice - reimburse selumetinib (Koselugo®) for the treatment of benign tumours in children with neurofibromatosis type 1 (NF1)

Selumetinib is intended for certain people with NF1

Selumetinib is the active substance. The brand name is Koselugo®. The medicinal product is a capsule that you swallow. 

The medicinal product can be used in people with neurofibromatosis type 1 (NF1). Neurofibromatosis type 1 (NF1) is a hereditary disorder. It is also called Von Recklinghausen's disease. The cause is a change in the NF1 gene. This gene produces the protein neurofibromin. Normally, it inhibits cell division. The change in the NF1 gene causes a person's cells to produce too little or non-functioning neurofibromin. This can lead to uninhibited cell growth and a higher risk of developing benign and malignant tumours. Almost all people with NF1 have neurofibromas. These are benign tumours that start in the nerves. They are often located on or under the skin. People can also develop larger benign tumours. They are called 'plexiform neurofibromas'. They originate from larger nerves or nerve networks. They grow the fastest in children. Plexiform neurofibromas can press on other tissues. This can cause pain or deformity of the body, or lead to disability. Depending on where the tumour is located, nerve failure, bladder and bowel problems and bleeding can also occur. In the most serious cases, benign tumours can lead to serious disability or life-threatening damage to organs. 

Advice from the National Health Care Institute on reimbursement of selumetinib

The National Health Care Institute advises the Minister of Health, Welfare and Sport to reimburse selumetinib (Koselugo®) from the basic health care package after price negotiations. We also recommend that selumetinib should be included in an orphan drug arrangement. An orphan drug arrangement is a set of agreements with the professional association that provides health care for a particular disorder. These agreements include, for example, which patients are eligible for selumetinib treatment and when selumetinib treatment should be discontinued. The professional association has already started to draw up these agreements. 

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, personal expenses or whether you should pay a contribution, please visit our information page on excess and personal contribution. Or ask your health insurance provider.

How did the advice come about?

The Scientific Advisory Board (WAR) and the Insured Package Advisory Committee (ACP) advise the National Health Care Institute when issuing a package advice. Based on the assessment, the National Health Care Institute sends an advisory report to the Minister of Health, Welfare and Sport. The Minister makes the final decision whether or not to reimburse the medicinal product from the basic health care package.

Lock procedure for expensive medicinal products

The Minister has placed abemaciclib for this indication in the lock procedure for expensive medicinal products. A medicinal product in this lock procedure will not be eligible for reimbursement from the basic health care package until:

• there is a positive package advice from the National Health Care Institute;
• there are arrangements and safeguards for appropriateness in place;
• price reductions have been successfully negotiated with the manufacturer.

For more information, see the page ‘Lock procedure for expensive medicinal products’.

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