Advisory report on possible candidate for conditional inclusion of atidarsagene autotemcel (Libmeldy®)
The National Health Care Institute advises the Minister of Health, Welfare and Sport (VWS) to designate atidarsagene autotemcel (Libmeldy®) as a possible candidate for conditional inclusion (CI) (procedure: conditional inclusion of orphan drugs, conditionals and exceptionals). Atidarsagene autotemcel can be used in certain patients with the rare and hereditary metabolic disease metachromatic leukodystrophy (MLD).
Recommendations from the National Health Care Institute
On 22 February 2023, the parties submitted an application for conditional inclusion of atidarsagene autotemcel (AA, Libmeldy®). Libmeldy is a new gene therapy for certain children with the rare hereditary metabolic disease metachromatic leukodystrophy (MLD). In the Netherlands, 2 to 3 children per year are diagnosed with this disease. Due to a fault in the genes, they have a reduced protection of the central nervous system and their nerve cells and brains are slowly damaged. Children with MLD develop mental disability and are progressively less able to move. Without treatment, these children die. The fully registered indication can be read in the drop-down menu.
In its advisory report of 27 September 2022, the National Health Care Institute concluded that gene therapy AA can only be reimbursed under conditions for the treatment of children with MLD who do not yet show any symptoms of the disease (pre-symptomatic). For children who already show symptoms of the disease (early-symptomatic), there is insufficient evidence whether treatment with AA has an effect and thus AA does not (yet) meet the 'established medical science and medical practice'. The application for conditional inclusion submitted therefore concerns AA for the treatment of early symptomatic children with the early juvenile form of MLD.
Based on the data in the dossier and the advice of the Scientific Advisory Board (WAR), the National Health Care Institute has concluded that treatment with AA for this indication meets the criteria for conditional inclusion. Those criteria are as follows:
- AA is registered by the European Medicines Agency (EMA) and has the status of an orphan drug;
- there is an unmet medical need;
- the marketing authorisation holder is the dossier’s lead applicant. The co-applicants are the treating physicians, an independent research institute and the patient association;
- the package question can be answered on the basis of the data collected by the registry study;
- the National Health Care Institute can answer the package question within 14 years.
Based on these conclusions, the National Health Care Institute recommends that AA should be designated as a potential candidate for conditional inclusion. If the Minister adopts the advice of the National Health Care Institute, phase 2 of the procedure begins. In this phase, the parties must develop their plans further and document the arrangements for a careful and successful conditional inclusion procedure in an agreement.
April 2023: no agreement on price after negotiations
The VWS usually uses phase 2 to reach a financial arrangement with the marketing authorisation holder. However, the Minister of VWS announced in April 2023 that negotiations on AA had been held with Belgium and Ireland. This was done within the framework of the Beneluxa collaboration. Because no agreement has been reached on the price, AA for the pre-symptomatic patients will not be admitted to the basic health care package. This also means that conditional inclusion for the early symptomatic patients is currently not possible. If the marketing authorisation holder is still willing to agree to an acceptable price and the parties have drawn up an agreement, the National Health Care Institute will prepare an additional advisory report on the inclusion of AA in the conditional inclusion.
Temporary inclusion to the basic health care package
Since 1 January 2012, health care that does not meet 'the established medical science and medical practice', in other words health care that is not proven to be effective, can still be temporarily admitted to the basic health care package. We call this ‘conditional inclusion’. The condition is that the study group collects data on the effectiveness and cost-effectiveness of a medical intervention or medicinal product during the period of conditional inclusion. These data will help to determine, at the end of the conditional inclusion period, whether a medical intervention or medicinal product can be a permanent part of the basic health care package.