Supplementary materials: How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison

<b>These are peer-reviewed supplementary materials for the article '</b><b>How does risdiplam compare with other </b><b>treatments for Types 1–3 spinal muscular </b><b>atrophy: a systematic literature review and </b><b>indirect treatment comparison</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Supplementary figure 1</b><b>Supplementary figure 2</b><b>Supplementary figure 3</b><b>Supplementary figure 4</b><b>Supplementary figure 5</b><b>Supplementary figure 6</b><b>Supplementary Table 1:</b> PICOS framework and additional search criteria for the SLR.<b>Supplementary Table 2: </b>List of data sources used in the SLR.<b>Supplementary Table 3:</b><b> </b>Key deviations from statistical analysis plan and justification<b>Supplementary Table 4: </b>Clinical trials excluded from the SLR.<b>Supplementary Table 5:</b> Availability of endpoints of interest in Type 1 SMA.<b>Supplementary Table 6: </b>Baseline characteristics of FIREFISH pre- and post‑matching with STR1VE‑US (MAIC analysis).<b>Supplementary Table 7: </b>STC model fit statistics of FIREFISH versus STR1VE-US.<b>Supplementary Table 8: </b>BSID-III covariate estimates (STC FIREFISH vs STR1VE-US).<b>Supplementary Table 9: </b>CHOP-INTEND covariate estimates (STC FIREFISH vs STR1VE-US).<b>Supplementary Table 10: </b>Availability of endpoints of interest in Types 2 and 3 SMA.<b>Supplementary Table 11: </b>Analyses of HFMSE outcomes in SUNFISH and CHERISH at Month 12.<b>Aim: </b>To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). <b>Patients &amp; methods:</b> Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. <b>Results:</b> In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. <b>Conclusion:</b> Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.