methods We built a cost-effectiveness model based on meta-analyses (direct or indirect) conducted between 2015 and 2021. We gathered all the effectiveness information (American College of Rheumatology (ACR)) through a PICO-S strategy including infliximab, etanercept, certolizumab, tocilizumab, golimumab, tofacitinib and upadacitinib. Two reviewers evaluated the inclusion of the studies and assessed their quality using the PRISMA-NMA Checklist. Efficiency score was cost per number needed to treat (NNT) versus placebo (PLC). The model was designed from a hospital perspective (only direct costs) and with a 1-year horizon. Cost data (C ¼ 2021) were obtained from Spanish datasets and literature review. Using all this information, a cost-effectiveness analysis between ADA and the suitable alternatives was performed. A probabilistic sensitivity analysis (PSA) was performed.Results Two meta-analyses met the inclusion criteria and fulfilled on average 70.6% of the 32 points on the PRISMA-NMA Checklist of items. Tarp et al (2017) showed no statistically significant difference in NNT between infliximab, ADA, etanercept, certolizumab, tocilizumab and golimumab for ACR-50. Song et al (2019) showed no significant difference in NNT between ADA, tofacitinib and upadacitinib for ACR-20.Total annual cost was C ¼ 4529 ADA versus C ¼ 4650-C ¼ 10 001 for the other treatments. As no effectiveness difference was seen, a cost minimisation analysis was performed. Hence ADA was the most cost-effective treatment. In the PSA, only ADA and infliximab performed as the best alternative, with ADA showing the highest probability of being cost-effective. Conclusion and relevanceAccording to our model, ADA was the most cost-effective option for RA treatment in Spain.

studies; (d) analysing data and (e) presenting the results. A comprehensive English-language literature search of the electronic databases PubMed and Science Direct was undertaken to identify published papers. Data were collected and analysed until May 2021. Results In this review, we incorporated one retrospective and one prospective cohort study. In the ongoing prospective Long-Term Follow-Up (LTFU) study (13 patients), 100% of SMA I infants in the therapeutic-dose cohort were alive and free of permanent ventilation. It was reported that 20% of SMA I infants achieved the additional milestone of standing with assistance. The LTFU study has demonstrated that SMA I infants improved their Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) scores (!4 points). In the retrospective cohort study of SMA I (3 patients) and SMA II infants (4 patients), it was perceived that 43% of SMA patients had meaningful increases in the CHOP-INTEND score and 57% had increases in the Hammersmith Functional Motor Scale-Expanded (HFMSE) score. Conclusion and relevance Despite the limited observation period, we conclude that Zolgensma is effective since no clinical regression or waning of effect had been reported. Nonetheless, several factors might still influence the duration of Zolgensma's effectiveness. As such, further research is needed to evaluate the persistence of the Zolgensma real-world effect in SMA infants.