Advice - reimburse avalglucosidase alfa (Nexviadyme®) for the treatment of Pompe disease
The National Health Care Institute has advised the Minister for Medical Care to reimburse avalglucosidase alfa (Nexviadyme®) from the basic healthcare package, after price negotiations. This medicinal product can be used in the treatment of patients with Pompe disease. The reason for this advice was the placing of the medicinal product in the so-called ‘lock procedure for expensive medicinal products'.
Avalglucosidase alfa is intended for certain people with Pompe disease
Avalglucosidase alfa is the active substance; the brand name is Nexviadyme®. The medicine is administered into the bloodstream by infusion. It is a form of therapy that is known as enzyme replacement therapy. It eliminates enzyme deficiencies in muscle cells and prevents further muscular weakness.
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.
Advice from the National Health Care Institute on the reimbursement of avalglucosidase alfa
The National Health Care Institute advises the Minister for Medical Care to reimburse avalglucosidase alfa (Nexviadyme®) from the basic healthcare package, after price negotiations. We also recommend negotiating prices at the same time for the various medicines for Pompe disease, namely avalglucosidase alfa and alglucosidase alfa (Myozyme®), as well as cipaglucosidase alfa if our assessment for that medicinal product yields a positive recommendation. This means that the price of alglucosidase alfa will also have to be renegotiated.
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 a package advice. 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 avalglucosidase 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’.