Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02120, USA.
JAMA. 2011 May 4;305(17):1786-9. doi: 10.1001/jama.2011.539.
Comparative effectiveness is taking on an increasingly important role in US health care, yet little is known about the availability of comparative efficacy data for drugs at the time of their approval in the United States.
To quantify the availability of comparative efficacy data for new molecular entities (NMEs) approved in the United States.
Approval packages publicly available through the online database of drug products approved by the US Food and Drug Administration (FDA).
Identification of efficacy studies that supported approval of each NME approved by FDA between 2000 and 2010.
We determined whether eligible studies were head-to-head active controlled trials and whether the results of such studies were available in the approval packages. We recorded the approved indication, whether the NME was an orphan product, whether the NME had undergone priority review, and whether the control group was a specific active comparator or standard care.
Of 197 NMEs identified that met eligibility criteria, 100 (51% [95% confidence interval {CI}, 44%-58%]) met criteria for having comparative efficacy data available at the time of market authorization. After excluding NMEs designated as orphan products (n = 37) and those approved for indications for which no alternative treatments existed (n = 17), this proportion increased to 70% (95% CI, 62%-77%). The proportions of NMEs with available comparative efficacy data varied widely by therapeutic area, from 33% (95% CI, 9%-67%) for hormones and contraceptives to 89% (95% CI, 56%-99%) for diabetes medications.
Publicly available FDA approval packages contain comparative efficacy data for about half of NMEs recently approved in the United States and for more than two-thirds of NMEs for which alternative treatment options exist. We did not investigate the extent to which available comparative efficacy information is useful for clinical guidance.
在美国医疗保健领域，比较疗效正发挥着越来越重要的作用，但人们对新药在美国获批时的比较疗效数据的可获得性知之甚少。
定量评估美国批准的新型分子实体（NME）的比较疗效数据的可获得性。
通过美国食品和药物管理局（FDA）在线药品批准数据库获得的批准文件包。
鉴定了 FDA 在 2000 年至 2010 年间批准的每种 NME 所支持的疗效研究。
我们确定了合格研究是否为头对头活性对照试验，以及这些研究的结果是否在批准文件包中可用。我们记录了批准的适应症、NME 是否为孤儿药、NME 是否进行了优先审查，以及对照组是否为特定的活性对照药物或标准护理。
在符合入选标准的 197 种 NME 中，有 100 种（51%[95%置信区间{CI}，44%-58%]）在市场授权时具有可用的比较疗效数据。排除被指定为孤儿药的 NME（n=37）和批准用于无替代治疗的适应症的 NME（n=17）后，这一比例增加到 70%（95%CI，62%-77%）。具有可用比较疗效数据的 NME 比例因治疗领域而异，从激素和避孕药的 33%（95%CI，9%-67%）到糖尿病药物的 89%（95%CI，56%-99%）不等。
美国 FDA 公开的批准文件包包含了最近在美国批准的 NME 中约一半以及有替代治疗方案的 NME 中超过三分之二的比较疗效数据。我们没有调查可用的比较疗效信息在临床指导方面的有用程度。
