The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to reimburse exagamglogene autotemcel (Casgevy®; exa-cel) from the basic healthcare package after price negotiations. This medicine can be used for certain patients with a form of hereditary anaemia. The reason for this advice was the placing of the medicinal product in the so-called ‘lock procedure for expensive medicinal products’.
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.
Exa-cel is intended for certain people with hereditary anaemia
Exa-cel is the active substance. The brand name is Casgevy®. The medicinal product is a single injection.
It can be used in patients aged 12 years and older with transfusion-dependent β-thalassemia (TDT) for whom a stem cell transplant is appropriate, but no donor is available. In a stem cell transplant, a patient is given healthy stem cells via the blood. TDT is a hereditary form of anaemia. Patients with TDT do not produce enough haemoglobin. Haemoglobin is an iron-rich protein in red blood cells. Haemoglobin enables organs to absorb oxygen from the lungs. Because of the lack of haemoglobin, patients become anaemic. This may cause them to feel tired and weak, and look pale. Serious complications may also occur. More than half of the patients die before they are 50 years old.
Advice from the National Health Care Institute on the reimbursement of exa-cel
The National Health Care Institute concludes that exa-cel (Casgevy®) has added value for patients with TDT. The National Health Care Institute advises the Minister of VWS to reimburse exa-cel for this patient group after price negotiations. And that agreements are made for appropriate use.
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) and the Insured Package Advisory Committee (ACP) advise the National Health Care Institute when issuing package advice. The final decision on whether reimbursement from the basic healthcare package will be made lies with the minister.
Explanation of the package lock procedure for expensive medicinal products
The minister has placed exa-cel 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 healthcare 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 marketing authorisation holder.
For more information, see the page ‘Lock procedure for expensive medicinal products’.