McMahon Alex D
Robertson Centre for Biostatistics, Boyd Orr, Building, University of Glasgow, Glasgow, G12 8QQ, Scotland, UK.
Pharmacoepidemiol Drug Saf. 2003 Oct-Nov;12(7):551-8. doi: 10.1002/pds.883.
There has been a resurgence of controversy about the usefulness of observational data to study the efficacy of drugs. Nearly every week a researcher makes some criticism of clinical trials or justifies observational research into intended effects, with other researchers offering a contradictory viewpoint. Literature reviews are not useful in this regard because the contradictory studies will not usually be carried out. Some methods are discussed which may have potential utility in the study of intended effects. There may be a marginal role for statistical techniques such as propensity scores and confounder scores. More promising techniques may include ecological analyses, restriction of subjects and blinded prospective review. Because it is currently unknown when the observational study of drug efficacy is possible, we should arguably always carry out a study of the determinants of prescribing first, and possibly consider using the various techniques that are outlined in this article.
关于观察性数据在研究药物疗效方面的有用性，争议再度兴起。几乎每周都有研究人员对临床试验提出一些批评，或为针对预期效果的观察性研究进行辩护，而其他研究人员则提出相反的观点。在这方面，文献综述并无用处，因为相互矛盾的研究通常不会进行。本文讨论了一些可能在研究预期效果方面具有潜在效用的方法。诸如倾向得分和混杂因素得分等统计技术可能发挥有限的作用。更有前景的技术可能包括生态分析、受试者限制和盲法前瞻性审查。由于目前尚不清楚何时可以进行药物疗效的观察性研究，我们可以说应该始终首先开展一项关于处方决定因素的研究，并可能考虑使用本文概述的各种技术。
