研究证实干扰素β-1a或醋酸格拉替雷长期治疗MS的成本效益较高

波士顿——欧洲与美洲多发性硬化症治疗和研究委员会联合会议上公布的一项6年观察研究结果显示，干扰素β-1a或醋酸格拉替雷长期治疗的成本效益较高，并且可改变复发-缓解型多发性硬化症（MS）的自然史。

这项研究由英国牛津大学医院信托基金多发性硬化症和神经脊髓炎组组长Jacqueline Palace医生及其同事进行，入选4,137例复发-缓解型MS患者，中位随访时间为6年。患者的平均年龄为38岁，平均发病年龄为31岁。大部分（76%）为女性。基线时的平均病程为7.7年。患者在过去2年内平均复发3次。基线时的扩展型残疾状态量表（EDSS）评分为3.06。该研究的主要终点是效用进展，这是一个衍生自EDSS评分的生活质量指标。

研究结果显示，治疗组患者的EDSS评分增幅低于作为对照组的自然史队列中的增幅（在使用连续马尔可夫模型和多水平模型的情况下，相对率分别为0.76和0.61）。所观察到的效用进展与这一结果一致（根据两个模型，相对率分别为0.58和0.56）。与自然史队列相比，治疗组患者的主要终点指标降低42%，高于预测的38%的降低率。一系列探讨各种偏倚的敏感性分析一致显示，治疗组患者的结局优于自然史队列。如果持续20年，则该研究观察到的治疗效果的程度将与预设的58,400美元/质量调整生命年的成本效益目标一致。

研究者总结指出，这项目前为止探讨干扰素β-1a和醋酸格拉替雷的作用和成本效益的最大型观察研究的结果证明，在真实世界环境中，这种治疗可改变复发-缓解型MS的自然史。

Palace医生声明从生产醋酸格拉替雷的梯瓦公司和生产干扰素β-1a的拜耳先灵公司及其他销售MS药物的公司获得科学会议方面的支持和咨询工作酬金。此外，她还从拜耳先灵公司及其他销售MS药物的公司获得无限制性补助金。

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By: SHARON WORCESTER

BOSTON - Treatment with interferon beta-1a or glatiramer acetate, if used long term, has the potential to be cost effective and to alter the natural history of relapsing-remitting multiple sclerosis, according to findings from a 6-year observational study.

In 4,137 patients with relapsing-remitting MS who were followed for a median of 6 years, the observed increase in the Expanded Disability Status Scale (EDSS) scores was lower than the increase seen in a natural history cohort that served as a comparator group (relative rates, 0.76 using a continuous Markov model and 0.61 using a multilevel model). The observed progression for utility – a quality of life measure derived from the EDSS scores, and the primary outcome of the study – was consistent with this finding (relative rates of 0.58 and 0.56 according to the two models, respectively), Dr. Jacqueline Palace reported at the joint meeting of the European and Americas Committees for Treatment and Research in Multiple Sclerosis.

The outcome – essentially a reduction of about 42% on the primary outcome measure, compared with the natural history cohort – was better than the 38% reduction that was predicted, said Dr. Palace, leader of the multiple sclerosis and neuromyelitis group at Oxford University Hospitals Trust in England.

A range of sensitivity analyses that examined the potential for various biases consistently demonstrated better outcomes in the treated patients, compared with the natural history cohort, Dr. Palace noted.

If sustained over 20 years, the magnitude of the treatment effect observed in this study would be consistent with a predefined cost-effectiveness target of about $58,400 per quality-adjusted life-year, she said.

Patients included in this study were adults with a mean age of 38 years and a mean age at disease onset of 31 years. Most (76%) were women. Disease duration was a mean of 7.7 years at baseline, and the patients had a mean of three relapses over the prior 2 years. Baseline EDSS score was 3.06.

“In 2002, the National Institute of Clinical Excellence [NICE], which is the U.K. body that sets the guidelines that decide what should be available under the National Health Service, concluded that beta-interferon and glatiramer acetate were not cost effective during the short-term, and thus it recommended that it couldn’t be available under the NHS at that stage,” Dr. Palace explained.

The NICE did notice, however, that if the drug effect was maintained over the long term it might be cost effective and invited the Department of Health to “try and find a way to make the drugs available in a cost-effective manner,” she said.

“This was the birth of the Risk-Sharing Scheme, and it is a way of providing disease-modifying therapies to patients cost effectively,” she said.

At the start of the scheme, the drug prices were reduced in line with a cost-effective requirement using the NICE model, which equated to about $58,400/quality-adjusted life-year..

“We were allowed to reach this target over a 20-year lifespan, and the idea was to monitor a clinical cohort of patients, measuring their EDSS scores,” she said.

The natural history dataset was used as a “virtual placebo,” and a price adjustment would be required if the outcomes of the treated patients were less than predicted to be on target for cost effectiveness.

The findings of this study – the largest observational study to measure the effect and cost-effectiveness of interferon beta-1a and glatiramer acetate – provide evidence that the treatment alters the natural history of relapsing-remitting MS in the real life setting, she concluded.

Dr. Palace reported receiving support for scientific meetings and honorariums for advisory work from Teva, which makes glatiramer acetate, and Bayer Schering, which makes interferon beta-1a, as well as other companies that market drugs for MS. She’s also received unrestricted grants from Bayer Schering and other companies that market drugs for MS.