Evolocumab and alirocumab: exploring original procurement models to manage the reimbursement of these innovative treatments

Background: PCSK9 inhibitors (evolocumab and alirocumab) pose a challenge of sustainability because the potential patients are extremely numerous and the budget impact at the drugs' full price would be prohibitive. We have studied the reimbursability of these agents by constructing a series of price-volume simulations that used a model previously employed for sofosbuvir. Methods: Our price-volume model is based on the following parameters: I) total patients candidate to the treatment; II) patients actually treated; III) treatment full cost per patient; IV) estimated nationwide budget impact in the absence of any price-volume intervention; V) price-halving population (PHP), which is the main model parameter. Treated patients ranged from 30,000 to 100,000. The full nominal yearly cost per patient was set at 10,000 (sic). Results: In 9 price-volume simulations (testing three values of PHP at 25,000 or 50,0000 or 100,000 patients), the total national expenditure varied from 204 to 721 million (sic). In the least expensive scenario (PHP = 25,000 patients), the expenditure ranged from 204 to 338 million (sic) while the average treatment cost per year was 3,382 (sic). At more than 100,000 treated patients, the treatment cost reduced to 626 (sic). On the other hand, the scenarios based on PHP = 50,000 and PHP = 100,000 patients were very unlikely to be acceptable for national health systems. Conclusions: Our study offered a pattern of different scenarios among which some national health systems in Europe could select the true decision on PCSK9 inhibitors. This decision is expected to be made over the next few months.