Palace Jacqueline, Bregenzer Thomas, Tremlett Helen, Oger Joel, Zhu Feng, Boggild Mike, Duddy Martin, Dobson Charles
Department of Clinical Neurology, John Radcliffe Hospital, Oxford, UK.
BMJ Open. 2014 Jan 17;4(1):e004073. doi: 10.1136/bmjopen-2013-004073.
In 2002, the UK's National Institute for Health and Care Excellence concluded that the multiple sclerosis (MS) disease modifying therapies; interferon-β and glatiramer acetate, were not cost effective over the short term but recognised that reducing disability over the longer term might dramatically improve the cost effectiveness. The UK Risk-sharing Scheme (RSS) was established to ensure cost-effective provision by prospectively collecting disability-related data from UK-treated patients with MS and comparing findings to a natural history (untreated) cohort. However, deficiencies were found in the originally selected untreated cohort and the resulting analytical approach. This study aims to identify a more suitable natural history cohort and to develop a robust analytical approach using the new cohort.
The Scientific Advisory Group, recommended the British Columbia Multiple Sclerosis (BCMS) database, Canada, as providing a more suitable natural history comparator cohort. Transition probabilities were derived and different Markov models (discrete and continuous) with and without baseline covariates were applied.
MS clinics in Canada and the UK.
From the BCMS database, 898 'untreated' patients with MS considered eligible for drug treatment based on the UK's Association of British Neurologists criteria.
The predicted Expanded Disability Status Scale (EDSS) score was collected and assessed for goodness of fit when compared with actual outcome.
The BCMS untreated cohort contributed 7335 EDSS scores over a median 6.4 years (6357 EDSS 'transitions' recorded at consecutive visits) during the period 1980-1995. A continuous Markov model with 'onset age' as a binary covariate was deemed the most suitable model for future RSS analysis.
A new untreated MS cohort from British Columbia has been selected and will be modelled using a continuous Markov model with onset age as a baseline covariate. This approach will now be applied to the treated UK RSS MS cohort for future price adjustment calculations.
2002年,英国国家卫生与临床优化研究所得出结论,多发性硬化症(MS)疾病修正疗法,即干扰素-β和醋酸格拉替雷,短期内不具有成本效益,但认识到从长期来看减少残疾可能会显著提高成本效益。英国风险分担计划(RSS)得以设立,以便通过前瞻性收集在英国接受治疗的MS患者的残疾相关数据,并将结果与自然病史(未治疗)队列进行比较,从而确保成本效益高的医疗服务提供。然而,在最初选定的未治疗队列和由此产生的分析方法中发现了缺陷。本研究旨在确定一个更合适的自然病史队列,并使用新队列开发一种稳健的分析方法。
科学咨询小组推荐加拿大不列颠哥伦比亚多发性硬化症(BCMS)数据库作为更合适的自然病史对照队列。推导了转移概率,并应用了有无基线协变量的不同马尔可夫模型(离散和连续)。
加拿大和英国的MS诊所。
从BCMS数据库中,根据英国神经学家协会标准,898名被认为符合药物治疗条件的“未治疗”MS患者。
收集预测的扩展残疾状态量表(EDSS)评分,并与实际结果进行比较时评估拟合优度。
1980年至1995年期间,BCMS未治疗队列在中位6.4年期间贡献了7335个EDSS评分(连续就诊记录了6357次EDSS“转变”)。以“发病年龄”作为二元协变量的连续马尔可夫模型被认为是未来RSS分析最合适的模型。
已选定来自不列颠哥伦比亚的一个新的未治疗MS队列,并将使用以发病年龄作为基线协变量的连续马尔可夫模型进行建模。这种方法现在将应用于接受治疗的英国RSS MS队列,以进行未来的价格调整计算。