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

Medo GutićAleksandra Dimitrijević Teodora SafiyeArdea MilidragSlobodan M Jankovic

Published in: Expert review of pharmacoeconomics & outcomes research (2023)

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.

Keyphrases

replacement therapy
end stage renal disease
newly diagnosed
ejection fraction
prognostic factors
brain injury
blood brain barrier
patient reported outcomes
cerebral ischemia
life cycle