Health Care Evaluation Group, School of Health and Population Sciences, Primary Care and Clinical Sciences Building, University of Birmingham, Edgbaston, Birmingham B15 2TT, UK.
J Clin Epidemiol. 2011 Jul;64(7):711-7. doi: 10.1016/j.jclinepi.2010.12.010. Epub 2011 Mar 31.
There is increasing interest in conducting "Real-World" trials that go beyond traditional assessment of efficacy and safety to examine market access and value for money questions before marketing authorization of a new pharmaceutical product or health technology. This commentary uses practical examples to demonstrate how high-quality evidence of the cost-effectiveness of an intervention may be gained earlier in the development process.
Issues surrounding the design and analysis of "Real-World" trials to demonstrate relative cost-effectiveness early in the life of new technologies are discussed. The modification of traditional phase III trial designs, de novo trial designs, the combination of trial-based and epidemiological data, and the use of simulation model-based approaches to address reimbursement questions are described.
Modest changes to a phase III trial protocol and case report form may be undertaken at the design stage to provide valid estimates of health care use and the benefits accrued; however, phase III designs often preclude "real-life" practice. Relatively small de novo trials may be used to address adherence to therapy or patient preference, although simply designed studies with active comparators enrolling large numbers of patients may provide evidence on long-term safety and rare adverse events.
Practical examples demonstrate that it is possible to provide high-quality evidence of the cost-effectiveness of an intervention earlier in the development process. Payers and decision makers should preferentially adopt treatments with such evidence than treatments for which evidence is lacking or of lower quality.
人们越来越感兴趣的是开展“真实世界”试验，这些试验不仅评估疗效和安全性，还在新药或新医疗技术获得营销许可之前，研究市场准入和资金效益问题。本文使用实际案例展示了如何在新干预措施开发过程的早期阶段，获得高质量的成本效益证据。
讨论了在新技术生命早期展示相对成本效益的“真实世界”试验设计和分析的问题。描述了传统 III 期试验设计、全新试验设计、试验和流行病学数据的组合以及基于模拟模型的方法在解决报销问题中的应用。
在设计阶段可以对 III 期试验方案和病例报告表进行适度修改，以提供对医疗保健使用和所获得效益的有效估计；然而，III 期设计通常排除了“真实生活”实践。相对较小的全新试验可用于解决治疗依从性或患者偏好问题，尽管采用活性对照、招募大量患者的简单设计研究可能会提供长期安全性和罕见不良事件的证据。
实际案例表明，在开发过程的早期阶段提供干预措施的成本效益的高质量证据是可能的。支付者和决策者应优先采用具有此类证据的治疗方法，而不是缺乏证据或证据质量较低的治疗方法。
