| dc.description.abstract | Background: Novartis submitted their health technology assessment in 2019 to the Norwegian Medicine Agency (NOMA), however, in 2020, NOMA discarded the submission by Novartis as they deemed Voretigene Neparvovec (Luxturna) to not be cost-effective. However, in 2021, NOMA conditionally reimbursed Voretigene Neparvovec and approved it into clinical practice in Norway. The condition was that all eligible patients over a four-year period that was treated was to be included in a quality registry. When the four years will pass a new decision on the reimbursement would be made. Objective: To assess whether NOMA’s conditional reimbursement can be justified based on a value of information analysis. The value of information analysis can help us infer whether the additional information acquired over the four-year period can be regarded sufficient, what information is important and whether collecting this additional information is resource efficient. Method: A replication of the cost-utility analysis submitted to NOMA by Novartis was performed. For purposes to maximize the uncertainty a willingness to pay threshold of 2,200,000 Norwegian Kroner was chosen. By running a Monte Carlo simulation to propagate uncertainty within the model the probabilistic sensitivity analysis could be stored. This output was collected and imported into RStudio to run a value of information analysis to collect EVPI, EVPPI, EVSI and ENBS results. Results: By setting the willingness to pay threshold at 2,200,000 Norwegian Kroner we estimated that Voretigene Neparvovec compared to best supportive care would result in an incremental cost-effectiveness ratio of 2,200,934 Norwegian Kroner per quality-adjusted life year gained. We aggregated the population EVPI to be 9,941,417 Norwegian Kroner. Parameters shown to cause a lot of variation within the model from EVPPI results were categorized as parameters concerning clinical efficacy and the natural history disease progression. EVSI results for clinical efficacy was estimated for a sample size range between 10 and 500 patients, ENBS was negative for the complete interval and further research was therefore considered too potentially not be worthwhile. Conclusion: Based on the results acquired by value of information analysis, the conditional reimbursement of Voretigene Neparvovec cannot be deemed justified. However, as Voretigene Neparvovec seek to treat individuals diagnosed with gene mutated RPE65 associated inherited retinal dystrophies, which is considered an orphan disease different criterion could be considered when assessing whether Voretigene Neparvovec should be included in Norwegian clinical practice. Further research is required to reduce decision uncertainty to get accurate estimates from the economic evaluation. As there are few eligible patients in Norway, cross- border co-operation could be considered. | eng |