One of the main criticisms of the health care system in the United States is that it spends too much while achieving too little. Reports consistently indicate that the U.S. spends a greater percentage of its gross domestic product (GDP) on health care and has higher health expenditures per capita than any other industrialized country.1 However, these high levels of spending have not necessarily produced better health outcomes or improved access to care. Evidence indicates that the U.S. achieves health outcomes similar to those of other industrialized countries that spend far less on health care.2 Furthermore, for certain outcome indicators, such as the infant mortality rate, the U.S. performs particularly poorly.3 One of the contributors to increases in health care expenditures in the U.S. is new technology in the form of new drugs and medical devices.4 These generally are more costly than previously approved technologies.5 However, under the current framework of regulation, it is difficult to evaluate whether these new technologies are actually superior to existing treatments.6 Inability to make these comparisons may lead to unjustified increases in health care expenditures in a technological climate where a “few products will be breakthroughs that improve health outcomes; [and] most will offer little, if any, advantages over existing treatments.”7 In recent years, researchers, patient groups, government officials, professional associations, and other stakeholders in the healthcare system have focused on the concepts of health technology assessment (HTA) and comparative effectiveness research (CER) as means of decreasing health care expenditures in the United States. Health technology assessment has been defined as “a form of policy research that examines the short- and long-term consequences of the application of a health-care technology.”8 Comparative effectiveness research is an activity closely related to HTA, which the Institute of Medicine defined as: the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist customers, clinicians, purchasers, and policy-makers to make informed decisions that will improve health care at both the individual and population levels.9 Health technology assessment and CER have been portrayed as potential avenues by which to obtain information to make informed, evidence-based decisions with the aim of improving health outcomes and containing healthcare costs.10 The use of such information seems particularly beneficial given that currently, “less than half of medical care in the United States is based on or supported by evidence about its effectiveness, resulting in care that is inappropriate and unnecessary.”11 In recent years, many stakeholders in the health care system have called on the federal government to establish a sustainable national program of HTA or CER.12 These efforts culminated in the establishment of the Patient-Centered Outcomes Research Institute (PCORI) in 2010. Its purpose is to fund and oversee CER at the national level.13 Although there has been an increased drive for federal support of HTA and CER in the past several years, HTA, in fact, has a long (though fragmented) history of federal funding in the U.S. dating back to the 1970s.14 Federal agencies that have played a major role in HTA in the past include the Office of Technology Assessment, the National Institutes of Health, and the Agency for Health Care Policy and Research.15 The Food and Drug Administration (FDA), in contrast, has traditionally played a more limited role in HTA and CER activities.16 Some commentators, noting that the FDA is the primary federal regulator of drugs and medical devices, have called on the agency to play a more active role in promoting HTA and CER.17 This paper explores the history of HTA and CER in the U.S. with a particular emphasis on the role of federal agencies within the executive and legislative branches. Part II gives a brief history and overview of the concepts of HTA and CER. Part III examines the roles that various federal agencies and initiatives have played in generating HTA or CER and applying their results to health care decisions. To illustrate the historical development of HTA and CER, agencies are discussed chronologically, with reference to their involvement with HTA or CER activities. Part IV discusses past challenges that the federal HTA and CER funding faced, as well as the implications of such challenges for current federal CER efforts. Part V highlights potential HTA or CER strategies that the federal government could pursue in the future. Particular focus is given to the potential role of using HTA or CER in the regulation of therapeutic drugs and medical devices.

中文翻译：

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药物和医疗器械的技术评估和比较有效性研究的历史以及联邦政府的作用

对美国医疗保健系统的主要批评之一是它花费太多而实现的却太少。报告一致表明，美国在医疗保健方面的支出占其国内生产总值 (GDP) 的比例更高，人均医疗支出也高于任何其他工业化国家。 1 然而，这些高水平的支出并不一定会产生更好的健康结果或改善获得护理的机会。有证据表明，美国取得的健康成果与其他在医疗保健上花费少得多的工业化国家相似。2 此外，在婴儿死亡率等某些成果指标方面，美国的表现尤其糟糕。3美国医疗保健支出增加 是新药和医疗器械形式的新技术。4 这些通常比以前批准的技术成本更高。5 然而，在目前的监管框架下，很难评估这些新技术是否真的优于现有的治疗方法。 6 无法进行这些比较可能会导致在技术环境中不合理地增加医疗保健支出，其中“很少有产品会成为改善健康结果的突破；[并且] 大多数与现有治疗相比几乎没有优势。”7 近年来，研究人员、患者团体、政府官员、专业协会、和医疗保健系统中的其他利益相关者已经将健康技术评估 (HTA) 和比较有效性研究 (CER) 的概念作为减少美国医疗保健支出的手段。卫生技术评估被定义为“一种检查卫生保健技术应用的短期和长期后果的政策研究形式。”8 比较有效性研究是一项与 HTA 密切相关的活动，研究所医学定义为：比较预防、诊断、治疗和监测临床状况或改善护理提供的替代方法的益处和危害的证据的生成和综合。CER 的目的是帮助客户、临床医生、购买者、呼吁该机构在促进 HTA 和 CER 方面发挥更积极的作用。17 本文探讨了 HTA 和 CER 在美国的历史，特别强调了联邦机构在行政和立法部门中的作用。第二部分简要介绍了 HTA 和 CER 的概念。第三部分研究了各种联邦机构和倡议在产生 HTA 或 CER 并将其结果应用于医疗保健决策方面所发挥的作用。为了说明 HTA 和 CER 的历史发展，按时间顺序讨论机构，参考它们参与 HTA 或 CER 活动。第四部分讨论了联邦 HTA 和 CER 资金面临的过去挑战，以及这些挑战对当前联邦 CER 工作的影响。第五部分重点介绍了联邦政府未来可能采取的潜在 HTA 或 CER 战略。特别关注使用 HTA 或 CER 在治疗药物和医疗设备的监管中的潜在作用。