The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to conditionally include atidarsagene autotemcel (Libmeldy®) in the basic health insurance package for a specific group of patients. This medicine can be used in certain patients with the rare and hereditary metabolic disorder metachromatic leukodystrophy (MLD).
Current state of affairs: advice sent to the Minister
The National Health Care Institute has sent this advice to the Minister of VWS. The Minister makes the final decision on whether to conditionally include the medicine in the basic health insurance package.
Follow-up to previous advice
On 8 May 2023, the National Health Care Institute advised the Minister of VWS to designate atidarsagene autotemcel (Libmeldy®) as a potential candidate for conditional inclusion of orphan medicinal products, conditionals and exceptionals for a specific group of patients. This advice follows on from that advice.
Go to the advice ‘Possible candidate for the conditional inclusion of atidarsagene autotemcel (Libmeldy®)’ of 8 May 2023.
Atidarsagene autotemcel is intended for certain children with the rare and hereditary metabolic disorder metachromatic leukodystrophy (MLD)
Atidarsagene autotemcel is the active substance. The brand name is (Libmeldy®). It is a gene therapy for certain children with a rare hereditary metabolic disorder called metachromatic leukodystrophy (MLD). In the Netherlands, 2 to 3 children per year are diagnosed with this disorder. Due to a genetic defect, the protection of their central nervous system is affected and this slowly damages their nerve cells and brain. Children with MLD develop a mental disability and are increasingly less able to move. Without treatment, children die. The complete registered indication can be read in the drop-down menu.
Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterised by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of ARSA enzymatic activity in children:
- with the pre-symptomatic late-infantile (PSLI) or early-juvenile (PSEJ) forms;
- with the early symptomatic early juvenile (ESEJ) form, who still have the ability to walk independently and before the onset of cognitive decline.
National Health Care Institute’s advice on reimbursement of atidarsagene autotemcel
The National Health Care Institute advises the Minister of Health, Welfare and Sport to have atidarsagene autotemcel (Libmeldy®) conditionally included in the basic health insurance package for a period of 14 years for the group of patients with the early symptomatic, early juvenile form who still have the ability to walk independently and before the onset of cognitive decline. After this period, the National Health Care Institute will reassess and advise the Minister once again on the reimbursement of atidarsagene autotemcel. For the treatment of pre-symptomatic children, atidarsagene autotemcel already complies with the established medical science and medical practice.
For more information, please visit the page 'Conditional inclusion of orphan drugs, conditionals and exceptionals'.
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.
Explanation of lock procedure for expensive medicinal products
The minister has placed atidarsagene autotemcel 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 insurance package until:
- there is a positive package advice from the National Health Care Institute;
- there are arrangements and safeguards for appropriateness in place;
- a price reduction has been successfully negotiated with the marketing authorisation holder.
For more information, see the page ‘Lock procedure for expensive medicinal products’.