Johnston S Claiborne, Rootenberg John D, Katrak Shereen, Smith Wade S, Elkins Jacob S
Department of Neurology, University of California, San Francisco, CA 94143-0114, USA.
Lancet. 2006 Apr 22;367(9519):1319-27. doi: 10.1016/S0140-6736(06)68578-4.
Few attempts have been made to estimate the public return on investment in medical research. The total costs and benefits to society of a clinical trial, the final step in testing an intervention, can be estimated by evaluating the effect of trial results on medical care and health.
All phase III randomised trials funded by the US National Institute of Neurological Disorders and Stroke before Jan 1, 2000, were included. Pertinent publications on use, cost to society, and health effects for each studied intervention were identified by systematic review, supplemented with data from other public and proprietary sources. Regardless of whether a trial was positive or negative, information on use of tested therapies was integrated with published per-use data on costs and health effect (converted to 2004 US dollars) to generate 10-year projections for the US population.
28 trials with a total cost of 335 million dollars were included. Six trials (21%) resulted in measurable improvements in health, and four (14%) resulted in cost savings to society. At 10 years, the programme of trials resulted in an estimated additional 470,000 quality-adjusted life years at a total cost of 3.6 billion dollars (including costs of all trials and additional health-care and other expenditures). Valuing a quality-adjusted life year at per-head gross domestic product, the projected net benefit to society at 10-years was 15.2 billion dollars. 95% CIs did not include a net loss at 10 years.
For this institute, the public return on investment in clinical trials has been substantial. Although results led to increases in health-care expenditures, health gains were large and valuable.
很少有人尝试估算医学研究的公共投资回报。作为测试一种干预措施的最后一步，一项临床试验对社会的总成本和收益可以通过评估试验结果对医疗保健和健康的影响来估算。
纳入了2000年1月1日前由美国国立神经疾病和中风研究所资助的所有III期随机试验。通过系统综述确定了每项研究干预措施的使用情况、社会成本和健康影响的相关出版物，并补充了其他公共和专有来源的数据。无论试验结果是阳性还是阴性，将受试疗法的使用信息与已发表的每次使用的成本和健康影响数据（换算为2004年美元）相结合，以生成美国人群的10年预测数据。
共纳入28项试验，总成本为3.35亿美元。6项试验（21%）使健康状况得到了可衡量的改善，4项试验（14%）为社会节省了成本。在10年时，试验项目估计额外产生了470,000个质量调整生命年，总成本为36亿美元（包括所有试验的成本以及额外的医疗保健和其他支出）。以人均国内生产总值对一个质量调整生命年进行估值，预计10年时对社会的净收益为152亿美元。95%的置信区间不包括10年时的净损失。
对于该研究所而言，临床试验的公共投资回报是巨大的。尽管结果导致了医疗保健支出的增加，但健康收益巨大且有价值。
