Möller Hans-Jürgen
Department of Psychiatry, Ludwig-Maximilians-University München, Nussbaumstrasse 7, 80336, Munich, Germany.
Eur Arch Psychiatry Clin Neurosci. 2008 Aug;258(5):257-70. doi: 10.1007/s00406-008-0812-0. Epub 2008 Jul 11.
In recent years, so-called "effectiveness studies" have gained increasing importance in the context of evidence-based medicine. These studies supposedly follow less restrictive methodological standards than phase III studies in terms of patient selection, co-medication and other design issues, and their results should therefore be better generalisable than those of phase III studies. Effectiveness studies, like other types of phase IV studies, can therefore contribute to the knowledge about antipsychotics or other psychopharmaceuticals and supply relevant information in addition to that gained from phase III trials. However, the less restrictive design and inherent methodological problems of phase IV studies mean that their results cannot falsify the results of phase III studies. The greater complexity of phase IV studies, for example the greater variance caused by the different kinds of confounders, means that their results have to be interpreted with great care and especially with a high degree of awareness of problematic design issues, such as insensitive primary outcome criteria, biased randomisation, unblinded treatment conditions, inclusion of chronic refractory patients, etc. Some recently published effectiveness studies on antipsychotic treatment of schizophrenia will be discussed under these methodological aspects. The main conclusions of these trials will be questioned on the basis of their severe methodological pitfalls.
近年来,在循证医学背景下,所谓的“有效性研究”变得越来越重要。据说,这些研究在患者选择、合并用药及其他设计问题方面,遵循的方法学标准比III期研究宽松,因此其结果应比III期研究更具普遍性。所以,有效性研究与其他IV期研究类型一样,能够增进对抗精神病药物或其他精神药物的了解,并提供除III期试验所得信息之外的相关信息。然而,IV期研究设计宽松及固有的方法学问题意味着其结果无法证伪III期研究的结果。IV期研究更为复杂,例如由各类混杂因素导致的更大变异性,这意味着对其结果的解读必须格外谨慎,尤其要高度意识到存在问题的设计问题,如不敏感的主要结局标准、有偏倚的随机化、未设盲的治疗条件、纳入慢性难治性患者等。将在这些方法学层面讨论近期发表的一些关于抗精神病药物治疗精神分裂症的有效性研究。基于这些试验严重的方法学缺陷,将对其主要结论提出质疑。