Supplementary materials: Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

<b>These are peer-reviewed supplementary materials for the article '</b><b>The impact of willingness-to-pay threshold </b><b>on price reduction recommendations for </b><b>oncology drugs: a review of assessments </b><b>conducted by the Canadian Agency for </b><b>Drugs and Technologies in Health</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Supplemental Table 1: </b>Overview of the patients from the placebo arms of DMD clinical trials<b>Supplemental Table 2: </b>Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by clinical trial corticosteroid type<b>Supplemental Table 3: </b>Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline age groups and corticosteroid type<b>Supplemental Table 4: </b>Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline corticosteroid duration and corticosteroid type<b>Supplemental Table 5: </b>Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline timed rise from supine and corticosteroid type<b>Supplemental Table 6: </b>Unadjusted change in motor function outcomes from baseline to 12, 24, 36, and 48 weeks among the daily deflazacort and prednisone groups of the DMD clinical trial placebo arms<b>Supplemental Table 7: </b>Comparison of change in motor function outcomes from baseline to week 48 between the daily deflazacort and prednisone groups in subgroups of the pooled DMD clinical trial placebo arms<b>Aim: </b>To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. <b>Methods:</b> Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. <b>Results: </b>Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (&gt;3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.