This is the tile of my recent paper Journal of Medical Economics With co-authors Ki-Sin Thanh, Sanjana Muthukrishnan, Jincy Paulose, Ver Bilano and Nicolas Kuypers. The gist is below:
Objective
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with a significant economic burden. Despite the availability of multiple treatments, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. The objective of this study is to evaluate the economic value of eptacopan compared to terminal complement inhibitors (specifically, complement component C5 inhibitors or C5I) – including eculizumab and ravulizumab – among patients with PNH who are either (i) C5I-experienced or (ii) complement-inhibitor-naïve.
methods
Cost per responder was analyzed based on treatment efficacy from clinical trials comparing eptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion freedom. Treatment costs were estimated as pharmaceutical wholesale acquisition costs and treatment administration costs, accounting for discontinuation. The outcomes evaluated included the number of treatments needed to achieve response and the cost per responder during the 24-week treatment period.
Result
At 24 weeks, the number of treatments required to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs 1.69 with C5is). Cost per responder was lower for iptacopan compared to the C5i comparator for both C5i-experienced ($264,337 for iptacopan versus $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilars, with a 10%–30% discount). eculizumab cost) and complement-inhibitor-naive patients ($256,754, versus $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilars).
conclusion
In both C5i-experienced and complement-inhibitor-naïve patients, treatment with eptacopan resulted in higher response rates and lower costs per responder compared with C5is.
You can read the full paper Here.
