Techspec schreef op 18 november 2018 15:59:
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Ik heb niets voorgeschoteld! Je probeert mij en Pharming telkens weer negatief weg te zetten....
De $23.000 is voor een dosis van 300mG/2mL Lanadelumab en was te vinden op;
www.drugs.com/price-guide/takhzyroVerder staat er in het Icer report;
Controversies and Uncertainties
Although trials of long-term prophylaxis with C1-INH and lanadelumab showed benefits in reducing the frequency of HAE attacks with few harms, the evidence base is limited to small RCTs of short duration, leaving questions about the durability of treatment response and long-term safety. We have fewer concerns about the safety profile of C1-INHs given the longer experience with their use in both acute treatment and prophylaxis. Data on lanadelumab is extremely limited at this time and long-term safety is of particular concern because new biologic therapies frequently are found to have safety concerns in the years after they are introduced that were not detected in pre-approval trials.31 Data are also very limited in children under the age of 12 and no trials included pregnant or lactating women.
For lanadelumab, because the results of the pivotal clinical trial are promising but long-term safety data are lacking, we assigned a “promising but inconclusive” (“P/I”) rating.
Compared to on-demand treatment there is significant uncertainty about the long-term risk of serious side effects of this intervention.
There are significant uncertainties about the long-term safety and efficacy of lanadelumab, a monoclonal antibody. New biologic therapies frequently are found to have safety concerns that were not detected in pre- approval trials.
Compared to on-demand treatment there is significant uncertainty about the magnitude or durability of the long-term benefits of this intervention.
The durability of effect from long-term prophylaxis has not been established; clinical trials ranged from 4-26 weeks of follow-up.
Annual cost Lanadelumab as per Icer report;
Whole Sale Acquisition > $620,338
Net Price > $478,464
The potential budget impact analysis included the population eligible for treatment: patients in the US with HAE 1/2 who are candidates for long-term prophylactic treatment. To estimate the size of the potential candidate population, we applied an estimate of one per 50,000 individuals with HAE 1/24 to the size of the US population33 to obtain an estimated US prevalence of 6,690 individuals. As not all patients with HAE 1/2 are considered candidates for long-term prophylactic treatment, we assumed that one-third were eligible for prophylaxis!