GVS assessment of amikacin liposome inhalation suspension (Arikayce® liposomal) for the treatment of NTM lung infections

Registered indication

Amikacin liposome inhalation suspension (ALIS; Arikayce® liposomal) is registered for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC) in adult patients with limited treatment options, who do not have cystic fibrosis.

Account should be taken of the fact that patients for whom ALIS is registered have an evident unmet medical need. The five-year mortality of patients with this NTM lung disease is estimated to be approximately 27%. In addition, these patients experience a reduced quality of life.

ALIS should be used in conjunction with other antibacterial agents active against Mycobacterium avium Complex lung infections.

Assessment

One randomised controlled open-label phase III study directly investigated the desirable and undesirable effects of ALIS in the treatment of patients with the above indication, when added to an antibacterial treatment regimen. The National Health Care Institute, advised by the Scientific Advisory Board (WAR), has concluded that, when added to an antibacterial treatment regimen, amikacin liposome inhalation suspension has added value in the treatment of non-tuberculous mycobacterial lung infections caused by the Mycobacterium avium Complex in adults with limited treatment options, who do not have cystic fibrosis.

Appropriateness

The marketing authorisation holder (MAH) proposes that treatment with ALIS should be linked to a discussion of the patient within the context of the Multidisciplinary Consultation Meeting offered by the NTM Centre of Expertise every 2 weeks. This proposal is primarily motivated by a wish to establish, with greater certainty, whether the patient is a candidate for treatment with ALIS. And to secure the correct advice and ‘supervision’, especially in terms of managing the adverse effects. In addition, the occupational group and the patients’ association have indicated that it is important to centralise treatment in centres of expertise.

Zorginstituut's advice

Amikacin liposome inhalation suspension (Arikayce® liposomal) is not interchangeable with any product in the GVS. Based on the considerations mentioned in the report, we recommend to include ALIS in List 1B and List 2 of the Healthcare Insurance Regulations. 

Condition regarding amikacin liposome suspension

Only for an insured person with a non-tuberculous mycobacterial lung infection caused by Mycobacterium avium Complex, who does not have cystic fibrosis, and who has not achieved culture conversion after at least 6 months of treatment involving a guideline-based antibacterial regimen in accordance with the guidelines accepted by the relevant professional practitioners in the Netherlands.

Parallel Procedure CBG-ZIN pilot

In the Parallel Procedure CBG-ZIN pilot, the reimbursement process was started while the registration process had not yet been completed. Medicinal products that go through this parallel procedure, rather than the current sequential procedures, will become available to patients more quickly.

The EMA registration of amikacin liposome inhalation suspension (ALIS) was published on the EMA website on 18 November 2020. This is normally the time when a reimbursement dossier can be submitted. Due to the parallel procedure, the National Health Care Institute can now rule on the reimbursement immediately after registration. The National Health Care Institute has since completed its assessment. 

This report is a summary of recommendations by Zorginstituut Nederland. The original text is in Dutch.