Hilibrand Alan S, Spindler Kurt, O'Keefe Regis J
The Rothman Institute, 925 Chestnut Street, Philadelphia, PA 19107. E-mail address:
Cleveland Clinic Sports Health Center, 5555 Transportation Boulevard, Garfield Heights, OH 44125.
J Bone Joint Surg Am. 2015 Apr 1;97(7):e35. doi: 10.2106/JBJS.N.00159.
Orthopaedic surgery is expensive and will be carefully scrutinized in the U.S. under health-care reform. Unfortunately, although the orthopaedic literature is replete with clinical outcomes studies, there is still a paucity of meaningful clinical outcomes data that are free from bias. It is possible that orthopaedic procedures may be among the most cost-effective medical treatments for the aging population. However, it is only through the collection of patient-generated outcomes data in prospective randomized and observational studies that orthopaedic surgery can be shown to provide high value (defined as high-quality outcomes at a relatively low cost) to society.The burden of musculoskeletal disease in the U.S. is high; nearly half of all adults describe themselves as having a chronic musculoskeletal condition, and approximately one-quarter of all health-care dollars are spent treating musculoskeletal disease. For this reason, treatment for osteoarthritis, the costliest condition in the elderly population, has drawn great scrutiny from insurers and the government. In the absence of clinical outcomes data that prove the value of orthopaedic interventions, there will be pressure to reduce payments or even deny treatments for these conditions if they are perceived to be too expensive or lack outcomes data supporting their use.Multicenter trials are expensive; this paper analyzes challenges to, and opportunities for, funding. Although National Institutes of Health (NIH) funding has dropped nearly 20% over the past ten years in inflation-adjusted dollars, it has begun a gradual reorientation toward clinical research, which comprised almost 50% of its budget in 2013. The Patient Protection and Affordable Care Act focused more attention on clinical outcomes research, with the establishment of the Patient-Centered Outcomes Research Institute (PCORI), which will ultimately fund $750 million of comparative effectiveness research annually. Another new funding source within the Centers for Medicare & Medicaid Services (CMS) is the Center for Medicare & Medicaid Innovation (CMMI), which recently funded several major initiatives, including a $1 billion health-care innovations grant program in 2012.The purpose of this article is to promote increased participation in prospective orthopaedic multicenter trials, which can answer clinical questions that affect the care of millions of patients. Some have enrolled large numbers of patients and have demonstrated the cost-effectiveness of surgery, provided subgroup analyses to define the optimal timing of surgery, and identified which patients are most and least helped by surgery. These studies have the power to establish new standards of care and prove the value of orthopaedic surgery.
骨科手术费用高昂，在美国医疗保健改革的背景下将受到严格审查。不幸的是，尽管骨科文献中充斥着临床疗效研究，但仍然缺乏无偏差的有意义的临床疗效数据。骨科手术有可能是针对老年人群最具成本效益的医疗治疗手段之一。然而，只有通过在前瞻性随机和观察性研究中收集患者产生的疗效数据，才能证明骨科手术能为社会提供高价值（定义为以相对较低的成本获得高质量的疗效）。
美国肌肉骨骼疾病的负担很重；几乎一半的成年人称自己患有慢性肌肉骨骼疾病，并且所有医疗保健费用的约四分之一用于治疗肌肉骨骼疾病。因此，骨关节炎的治疗，这是老年人群中最昂贵的疾病，受到了保险公司和政府的严格审查。在缺乏能证明骨科干预措施价值的临床疗效数据的情况下，如果这些疾病被认为过于昂贵或缺乏支持其使用的疗效数据，就会面临减少支付甚至拒绝治疗的压力。
多中心试验成本高昂；本文分析了资金方面的挑战和机遇。尽管美国国立卫生研究院（NIH）的资金在过去十年中按通胀调整后的美元计算下降了近20%，但它已开始逐步重新转向临床研究，临床研究在其2013年的预算中占近50%。《患者保护与平价医疗法案》更加关注临床疗效研究，成立了以患者为中心的疗效研究所（PCORI），该研究所最终每年将为比较疗效研究提供7.5亿美元的资金。医疗保险和医疗补助服务中心（CMS）内的另一个新的资金来源是医疗保险和医疗补助创新中心（CMMI），该中心最近资助了几项重大举措，包括2012年的一项10亿美元的医疗保健创新资助计划。
本文的目的是促进更多人参与前瞻性骨科多中心试验，这些试验能够回答影响数百万患者护理的临床问题。一些试验已经招募了大量患者，并证明了手术的成本效益，进行了亚组分析以确定手术的最佳时机，并确定了哪些患者从手术中获益最大和最小。这些研究有能力建立新的护理标准并证明骨科手术的价值。
