Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

ABSTRACT

Background

Cerliponase alfa is an orphan drug approved for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2).

Aim

Our goal was to assess the cost-effectiveness of cerliponase alfa in patients with CLN2 in the socioeconomic context of the Republic of Serbia in contrast to symptomatic therapy.

Method

For this study, a forty-year horizon and the perspective of the Serbian Republic Health Insurance Fund were used. Quality-adjusted life years gained with cerliponase alfa and comparator, as well as direct treatment costs, were the study’s key outcomes. The creation and simulation of a discrete-event simulation model served as the basis for the investigation. Monte Carlo microsimulation was performed on a sample of 1000 virtual patients.

Results

When compared to symptomatic therapy, cerliponase alfa treatment was not cost-effective and was linked to negative net monetary benefit regardless of when the illness signs started. 

Conclusion

Cerliponase alfa is not more economical than symptomatic therapy for the treatment of CLN2 when using typical pharmacoeconomic analysis. Cerliponase alfa has been shown to be effective but more has to be done to make it accessible to all CLN2 patients.

KEYWORDS:

• Cerliponase alfa
• cost-effectiveness analysis
• health
• insurance
• late-infantile neuronal ceroid lipofuscinosis type 2

Declaration of interest

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

S Janković, M Milosavljević and M Gutić were involved in the conception, study design, analysis, and interpretation of data. All authors took part in manuscript writing. S Janković revised manuscript critically. All authors agree for the final version of the manuscript to be published.

Supplemental data

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2023.2197213.

Additional information

Funding

This study was partially funded by Grant No. 175007 from the Serbian Ministry of Education, Science and Technological Development.