Pharmacoeconomic evaluation of rare disease management in primary myelofibrosis treatment with ruxolitinib
Orphan drugs designed for rare disease management is a special group of drugs from the perspective of the pharmacoeconomic analysis being a mandatory part of any proposal for inclusion in the Restrictive lists. Thanks to unique opportunities offered by orphan drugs for rare disease management through acting at the pathogenetic level, these drugs have high social significance. But inherently high cost of these innovative drugs associated with their narrow market limited due to small target population, precludes from using the conventional pharmacoeconomic approach which involves comparison of pharmacoeconomic indicators — results of cost-effectiveness and budget impact analyses — of the innovative drug vs. current medical treatment (or palliative treatment in case of no treatment option available). As such, the authors have investigated the special pharmacoeconomic approach — “precedential” — in case of ruxolitinib in treatment of primary myelofibrosis. This approach implies comparison of the studied drug vs. other orphan and high-cost medicinal products included in the National drug lists. As a result, it has been shown that the pharmacoeconomic indicators of ruxolitinib are not higher than the same of the drugs included in the Essential Drug List .
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