Advice - reimburse cipaglucosidase alfa (Pombiliti®) in combination with miglustat (Opfolda®) for the treatment of Pompe disease
The National Health Care Institute has advised the Minister of Health, Welfare and Sport to reimburse cipaglucosidase alfa (Pombiliti®) in combination with miglustat (Opfolda®) from the basic healthcare package, after price negotiations. These medicinal products can be used in certain patients with Pompe disease. The reason for this advice was that cipaglucosidase alfa was placed in what is known as the ‘lock procedure’ for expensive medicinal products.
Cipaglucosidase alfa in combination with miglustat is intended for certain people with Pompe disease
Cipaglucosidase alfa is one of the two active substances; its brand name is Pombiliti®. The medicine is administered into the bloodstream by infusion. The other active substance is miglustat; its brand name is Opfolda®. This medicine is a capsule that you swallow. The patient has to take the capsule before the infusion is administered.
The medicinal product can be used in people with Pompe disease. Pompe disease is an inherited muscular disease in which the enzyme alpha glucosidase does not work properly. As a result, certain sugars (glycogens) are not broken down and accumulate in the muscle cells, preventing the muscle cell from doing its job properly. Eventually, this makes the muscles weaker and weaker. Pompe disease can occur in both children and adults. Adults with Pompe disease have difficulty running or climbing stairs, for example. Because their muscles become increasingly weakened, patients are unable to stand up and walk independently after a certain time. They may also get respiratory problems.
Advice from the National Health Care Institute reimbursing cipaglucosidase alfa in combination with miglustat
The National Health Care Institute advises the Minister of Health, Welfare and Sport to reimburse cipaglucosidase alfa (Pombiliti®) in combination with miglustat (Opfolda®) from the basic healthcare package, after price negotiations. The National Health Care Institute also recommends including miglustat (Opfolda) in List 1A of the GVS (Medicine Reimbursement System). The List 2 conditions for miglustat should also be amended, on the condition that a lower price can be achieved after price negotiations.
Conditions for reimbursement of miglustat
The following conditions apply for reimbursement.
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 advises the National Health Care Institute about the assessment. The final decision as to whether or not reimbursement from the basic health care package will take place lies with the Minister.
Lock procedure for expensive medicinal products
The Minister has placed cipaglucosidase alfa 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’.