Eli Lilly & Co., Lilly Corporate Center, Indianapolis, IN 46285, USA.
J Comp Eff Res. 2012 Mar;1(2):147-56. doi: 10.2217/cer.12.9.
Comparative effectiveness research (CER) includes pragmatic clinical trials (PCTs) to address 'real-world' effectiveness. CER interest would be expected to stimulate biopharmaceutical manufacturer PCT investment; however, this does not seem to be the case. In this article we identify all industry-sponsored PCT studies from 1996 to 2010; analyze them across a variety of characteristics, including sponsor, research question, design, comparators and results; and suggest methodological and policy changes to spur future manufacturer PCT investment. Nine 'naturalistic', head-to-head versus standard of care or similar agent PCTs were identified. Two included a 'usual care' arm. Chronic care trials' length averaged 12 months (range: 6-24 months), six of which reported equivocal or no difference in effectiveness; results of two chronic and the single acute care PCTs favored the sponsor drug. None reported the sponsor drug inferior. Of seven that evaluated utilization or costs, six reported no differences and four of five studies comparing brand-generic drugs reported no difference. Whereas private investment in PCTs is in the public interest, manufacturers apparently have not yet seen the business case. To induce investment, we propose several methodological and regulatory policy innovations designed to reduce business risk by decreasing outcome variability and increasing trial efficiency, flexibility and market applicability.
比较实效研究（CER）包括实用临床试验（PCTs）以解决“真实世界”的效果问题。人们预计 CER 的兴趣将刺激生物制药制造商对 PCT 的投资；然而，事实似乎并非如此。在本文中，我们确定了 1996 年至 2010 年间所有由行业资助的 PCT 研究；分析了它们在赞助商、研究问题、设计、对照和结果等各个方面的特点；并提出了方法学和政策变革的建议，以促进未来制造商对 PCT 的投资。确定了九项“自然主义”、与标准护理或类似药物的头对头实用临床试验。其中两项包括“常规护理”手臂。慢性护理试验的平均长度为 12 个月（范围：6-24 个月），其中有 6 项报告在效果上没有差异或差异不明显；两项慢性和一项急性护理的 PCT 结果都有利于赞助商的药物。没有一项报告赞助商的药物较差。在评估利用或成本的七项研究中，有六项报告没有差异，五项比较品牌仿制药的研究中有四项报告没有差异。虽然私人对 PCT 的投资符合公共利益，但制造商显然尚未看到商业案例。为了诱导投资，我们提出了一些方法学和监管政策创新的建议，旨在通过减少结果的可变性、提高试验的效率、灵活性和市场适用性来降低商业风险。
