Cost-Utility Analysis of Luxturna versus the Standard of Care Treatment from a Narrow Danish Societal Perspective
Authors
Morgan, Alexandria Marie ; Zheng, Shida
Term
4. term
Publication year
2020
Submitted on
2020-06-12
Pages
68
Abstract
Background: Luxturna is the first approved gene therapy for a rare inherited eye disease (IRD) caused by two faulty copies (biallelic) of the RPE65 gene. The disease often begins in childhood and can lead to blindness. Although Luxturna is approved by the EMA and FDA, the Danish Medicines Council did not recommend it due to high cost and uncertain long-term benefits. This thesis assesses whether Luxturna is cost-effective compared with current standard care in Denmark. Methods: We conducted a cost-utility analysis, an economic evaluation that compares costs and health gains. Using a decision model (Markov model, which simulates how patients move between health states over time), we estimated the extra cost per QALY (quality-adjusted life year, a measure that combines length and quality of life) gained with Luxturna versus standard care from a Danish narrow societal perspective, which includes productivity losses (e.g., reduced ability to work). The model followed patients from age 15 for the rest of their life. Clinical effectiveness came from phase III trials and a U.K. economic evaluation by Viriato et al. based on the natural history of RPE65-mediated IRD. Health states were defined using American Medical Association Guides, and utility values came from Lloyd et al. (2019). Costs were taken from analyses by the Danish Medicines Council and AMGROS; indirect costs used data from Statistics Denmark. Results: In the base case, the incremental cost-effectiveness ratio (ICER) was 521,990.97 DKK per QALY gained. Deterministic sensitivity analysis showed the ICER was most affected by the time horizon, discount rate, whether productivity loss was included, and assumptions about long-term treatment effects. Probabilistic sensitivity analysis indicated Luxturna had a 25.9% chance of being cost-effective at a willingness-to-pay (WTP) threshold of 325,000 DKK per QALY, and a 77.9% chance at 745,000 DKK per QALY. Conclusion: Luxturna is likely to be cost-effective if the WTP threshold is 745,000 DKK per QALY (based on Swedish experience for rare diseases), but not cost-effective at 325,000 DKK per QALY (a commonly cited Danish threshold; Denmark has no official threshold). Further work is needed to define appropriate WTP thresholds for orphan drugs in Denmark.
Baggrund: Luxturna er den første godkendte genterapi til en sjælden arvelig øjensygdom (IRD) forårsaget af to defekte kopier (biallelisk) af RPE65-genet. Sygdommen starter ofte i barndommen og kan føre til blindhed. Selvom EMA og FDA har godkendt Luxturna, valgte Medicinrådet ikke at anbefale den på grund af høj pris og usikre langtidseffekter. Dette speciale undersøger, om Luxturna er omkostningseffektiv sammenlignet med nuværende standardbehandling i Danmark. Metoder: Vi gennemførte en cost-utility analyse, en økonomisk evaluering der sammenligner udgifter og helbredsmæssige gevinster. Med en beslutningsmodel (Markov-model, som simulerer hvordan patienter bevæger sig mellem helbredstilstande over tid) beregnede vi den ekstra omkostning per QALY (quality-adjusted life year, et mål der kombinerer livslængde og livskvalitet) opnået med Luxturna versus standardbehandling, set fra et snævert samfundsperspektiv, der inkluderer produktivitetstab (fx nedsat arbejdsevne). Modellen fulgte patienter fra 15-års-alderen og resten af livet. Den kliniske effekt kom fra fase III-forsøg og en britisk økonomisk evaluering af Viriato m.fl. baseret på den naturlige sygdomsudvikling ved RPE65-medieret IRD. Helbredstilstande blev defineret efter American Medical Association Guides, og nytteværdier kom fra Lloyd m.fl. (2019). Omkostningerne blev hentet fra analyser fra Medicinrådet og AMGROS; indirekte omkostninger brugte data fra Danmarks Statistik. Resultater: I basisscenariet var den inkrementelle omkostningseffektivitetsratio (ICER) 521.990,97 DKK per QALY. Deterministisk følsomhedsanalyse viste, at ICER især påvirkes af tidshorisonten, diskonteringsraten, om produktivitetstab indgår, og antagelser om langtidseffekt. Probabilistisk følsomhedsanalyse viste, at Luxturna havde 25,9% sandsynlighed for at være omkostningseffektiv ved en betalingsvillighed (WTP) på 325.000 DKK per QALY og 77,9% ved 745.000 DKK per QALY. Konklusion: Luxturna er sandsynligvis omkostningseffektiv, hvis WTP-tærsklen er 745.000 DKK per QALY (baseret på svensk erfaring for sjældne sygdomme), men ikke omkostningseffektiv ved 325.000 DKK per QALY (ofte nævnt dansk tærskel; der findes ingen officiel tærskel). Der er behov for videre arbejde med at fastlægge passende WTP-tærskler for orphan drugs i Danmark.
[This apstract has been rewritten with the help of AI based on the project's original abstract]
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