Kings College London School of Medicine, London, SE5 9RS, UK.
Expert Rev Pharmacoecon Outcomes Res. 2010 Jun;10(3):229-38. doi: 10.1586/erp.10.19.
Lenalidomide represents the first drug in a novel class of agents known as IMiDs. It has both direct antimyeloma activity and an indirect effect acting through the microenvironment. In the relapsed/refractory setting, lenalidomide has been demonstrated to be highly active, producing partial and complete responses that translate into improved survival. Generally, the drug is well tolerated and more recently this agent has been used in combination with steroids, chemotherapy agents and other novel agents that have further enhanced its efficacy in clinical trials. However, the cost of this and other novel agents is significantly greater than previously used chemotherapy protocols, which in turn means that they have fallen under the scrutiny of regulatory bodies such as NICE. It is important that researchers understand the instruments used by these bodies to come to decisions regarding cost-effectiveness if patients are not to be disadvantaged by not being given access to these active new agents. This article outlines the models used by health economists and assesses their potential shortcomings. It also suggests alternative methods and identifies areas of research where improvements might be achieved.
来那度胺是首个作用于被称为 IMiDs 的新型药物类别中的药物。它具有直接的抗骨髓瘤活性和通过微环境发挥的间接作用。在复发/难治性环境中，来那度胺已被证明具有高度活性，可产生部分和完全缓解，从而转化为生存改善。通常，该药物具有良好的耐受性，最近该药物已与类固醇、化疗药物和其他新型药物联合使用，这进一步提高了其在临床试验中的疗效。然而，这种药物和其他新型药物的成本明显高于以前使用的化疗方案，这反过来意味着它们受到了监管机构（如 NICE）的审查。如果不给患者提供这些有效的新型药物，那么让患者无法获得这些药物可能会使他们处于不利地位，因此研究人员了解这些机构用来做出成本效益决策的工具非常重要。本文概述了卫生经济学家使用的模型，并评估了它们的潜在缺陷。它还提出了替代方法，并确定了可能取得改进的研究领域。
