The budget impact decreases each year as single-dose therapy precludes long-term acquisition and administration costs of treating prevalent patients. Inclusion of AVXS-101 on a formulary is estimated to offset payers' SMA care costs by -$15K by the fifth year. Additionally, installment plan for AVXS-101 payments will reduce the budget impact further.
Cost-effectiveness was dependent on assumptions made about survival, costs, and utilities; and whether the motor function milestones were sustained over lifetime. Given the relatively short-term effectiveness data available for these therapies, a registry to collect long-term data of infantile-onset SMA patients is recommended.
A full description of the different types of Smith fractures following Thomas classification is given, together with the guidelines of treatment of each type. Conservative treatment is usually sufficient for types I and III, provided that reduction is anatomic and stable. The indication for internal fixation is given for all type II and unstable type III fractures. An exact description of the volar buttressing technique with the AO-T-plate is presented.
lifetime horizon was developed to evaluate the cost-effectiveness of regorafenib from a third-party payer perspective. The primary model outputs were total costs, qualityadjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER). Only direct medical costs were considered. Clinical efficacy data were obtained from the pivotal clinical trial (RESORCE). One-way sensitivity and probability sensitivity analyses were conducted to examine the robustness of the base-case findings. Results: In the base-case analysis, the lifetime total costs were higher for regorafenib plus BSC at U200,593 compared with U113,358 for BSC. The QALYs gained were 1.30 and 0.79 for patients treated with regorafenib plus BSC and BSC only, respectively. The ICER of regorafenib plus BSC vs BSC was U172,670/QALY, which is under the threshold of 3 times per capita GDP of China in 2017 (U178,980). One-way sensitivity analysis showed that ICER was sensitive to the increased overall survival in regorafenib-treated patients. Findings from the probabilistic sensitivity analysis indicated that the likelihood of regorafenib plus BSC being cost-effective was 53%. Conclusions: Compared with BSC, HCC treatment with regorafenib is more expensive but also with greater QALYs gains. On balance, regorafenib is an attractive cost-effective option for the 2 nd line treatment of HCC in China.
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