Objective: To evaluate cost-effectiveness of brentuximab vedotin in patients with relapsed/refractory Hodgkin lymphoma who have received autologous stem cell transplantation, from a Scottish healthcare payer perspective.
Methods: A Microsoft Excel-based partitioned survival model comprising three health states (progression-free survival [PFS], post-progression survival, and death) was developed. Relevant comparators were chemotherapy with or without radiotherapy (C/R) and C/R with intent to allogeneic hematopoietic stem cell transplantation (alloSCT). Data were obtained from the pivotal phase II single-arm trial in 102 patients (SG035-0003; NCT00848926), a systematic literature review and clinical expert opinions (where empirical evidence was unavailable). PFS and overall survival for brentuximab vedotin were estimated using 5-year follow-up data from SG035-0003, and extrapolated using event rates observed for comparator treatments from published survival data. Resource use included drug acquisition and administration; alloSCT; treatment of adverse events; and long-term follow-up. Deterministic and probabilistic sensitivity analyses were conducted to evaluate the impact of uncertainty.
Results: In the base case, the incremental cost-effectiveness ratio (ICER) for brentuximab vedotin was £38,769 per quality-adjusted life year (QALY) vs C/R, whereas C/R with intent to alloSCT was dominated by brentuximab vedotin. ICERs for brentuximab vedotin generated by the deterministic sensitivity analysis ranged between £32,000–£54,000 per QALY. Including productivity benefits reduced the ICER to £28,881 per QALY.
Limitations: Limitations include lack of comparative data from this single arm study and the heterogeneous population. Inconsistent baseline characteristic reporting across studies prevented complete assessment of heterogeneity and the extent of potential bias in clinical and cost-effectiveness estimates.
Conclusions: Although the base case ICER is above the threshold usually applied in Scotland, it is relatively low compared with other orphan drugs, and lower than the ICER generated using a previous data cut of SG035-0003 that informed a positive recommendation from the Scottish Medicines Consortium, under its decision-making framework for assessment of ultra-orphan medicines. 相似文献
Background: QALYs are widely used in health economic evaluation, but remain controversial, largely because they do not reflect how many people behave in practice. This paper presents a new conceptual model (Load Model) and illustrates it in comparison with the QALY model.Methods: Load is the average annual weight attributed to morbidity and mortality over a defined period, using weightings based on preference judgements. Morbidity Load is attributed to states of illness, according to their perceived severity. When people are in full health, Load is zero (no morbidity). Death is treated as an event with negative consequences, incurred in the year following death. Deaths may be weighted equally, with a fixed negative weight such as ?100, or differ according to the context of death. After death, Load is zero. In a worked example, we use the standard gamble method to obtain a weighting for an illness state, for both Load and QALY models. A judge is indifferent between certainty of 1.5 years’ illness followed by death, or a 50/50 chance of 1.5 years’ full health or 1-year illness, each followed by death. The weightings calculated are applied to a hypothetical life, 72 years in full health followed by 3 years with illness then death, using both models. Three other hypothetical outcomes are also compared.Results: For an example life, the relative size of the morbidity component compared with the mortality component is much higher in the Load model than in the QALY model. When comparing alternative outcomes, there are also substantial differences between the two models.Conclusions: In the Load model the weight of morbidity, relative to mortality, is very different from that in the QALY model. Given the role of the QALYs in economic evaluation, the implications of an alternative, which generates very different results, warrant further exploration. 相似文献
Research indicates that regulatory risk increases required return on investment by investors and causes underinvestment in industries with high sunk costs. The effects of regulatory changes may be measured by estimating the abnormal returns associated with the event. The results may suggest to regulators what should be encouraged or avoided. This article utilizes a fixed effects regression to examine abnormal returns from changes in Philippine nationalization regulations. The results are consistent with extant literature. Supreme Court decisions, which increased uncertainty and regulatory risk, produced negative abnormal returns. The initial release of draft implementing rules did not produce statistically significant effects, but a succeeding draft favouring liberalization, produced positive abnormal returns. 相似文献
Many students enrolled in first year introductory statistics courses believe learning statistics is a waste of time and fear they will fail. In this study, we explored the impacts on learning outcomes for students in an introductory statistics course by allowing students to arbitrarily choose their own sequence of learning from three key learning activities, namely tutorials, Peer-Assisted Study Sessions and Computer-Managed Learning quizzes. Unlike the old regime where the learning activities followed a strict, rigid sequence, a new regime allowed students to freely choose when, where and how they engaged with the course learning activities. This allowed increased opportunities for students to receive relevant and timely feedback. Using a total of 1187 students enrolled in semester 2 of 2011, 2012 and 2013, data were collected on students’ scores from 7 assessment tasks. Our experimental design ensured as many course features as possible remained constant between the control cohorts (of 2011 and 2012) and the experimental cohort (2013), thereby avoiding potential sample selection problems. The findings showed student learning outcomes in the new regime improved significantly. Interestingly, the effects were found to be greatest in the lower percentile of the score distribution. 相似文献