Mapel D, Pearson M
Lovelace Respiratory Research Institute, Lovelace Scientific Resources, Albuquerque, New Mexico 87108, USA.
Respir Med. 2002 Aug;96 Suppl C:S23-30. doi: 10.1016/s0954-6111(02)80031-2.
Healthcare payers make decisions on funding for treatments for diseases, such as chronic obstructive pulmonary disease (COPD), on a population level, so require evidence of treatment success in appropriate populations, using usual routine care as the comparison for alternative management approaches. Such health outcomes evidence can be obtained from a number of sources. The 'gold standard' method for obtaining evidence of treatment success is usually taken as the randomized controlled prospective clinical trial. Yet the value of such studies in providing evidence for decision-makers can be questioned due to the restricted entry criteria limiting the ability to generalize to real life populations, narrow focus on individual parameters, use of placebo for comparison rather than usual therapy and unrealistic intense monitoring of patients. Evidence obtained from retrospective and observational studies can supplement that from randomized clinical trials, providing that care is taken to guard against bias and confounders. However, very large numbers of patients must be investigated if small differences between drugs and treatment approaches are to be detected. Administrative databases from healthcare systems provide an opportunity to obtain observational data on large numbers of patients. Such databases have shown that high healthcare costs in patients with COPD are associated with co-morbid conditions and current smoking status. Analysis of an administrative database has also shown that elderly patients with COPD who received inhaled corticosteroids within 90 days of discharge from hospital had 24% fewer repeat hospitalizations for COPD and were 29% less likely to die during the 1-year follow-up period. In conclusion, there are a number of sources of meaningful evidence of the health outcomes arising from different therapeutic approaches that should be of value to healthcare payers making decisions on resource allocation.
医疗保健支付方在人群层面上就诸如慢性阻塞性肺疾病(COPD)等疾病的治疗资金进行决策,因此需要有在适当人群中治疗成功的证据,并将常规护理作为替代管理方法的比较对象。此类健康结果证据可从多个来源获得。获取治疗成功证据的“金标准”方法通常被视为随机对照前瞻性临床试验。然而,由于入组标准受限,这类研究在为决策者提供证据方面的价值可能受到质疑,因为入组标准限制了推广到现实生活人群的能力,研究重点狭窄,仅关注个别参数,使用安慰剂进行比较而非常规治疗,以及对患者进行不切实际的严格监测。从回顾性和观察性研究中获得的证据可以补充随机临床试验的证据,前提是要注意防范偏差和混杂因素。然而,如果要检测药物和治疗方法之间的细微差异,就必须对大量患者进行调查。医疗保健系统的行政数据库提供了获取大量患者观察数据的机会。此类数据库表明,COPD患者的高医疗成本与共病情况和当前吸烟状况有关。对一个行政数据库的分析还表明,在出院后90天内接受吸入性糖皮质激素治疗的老年COPD患者,因COPD再次住院的次数减少了24%,在1年随访期内死亡的可能性降低了29%。总之,有多种来源可提供不同治疗方法对健康结果有意义的证据,这对于在资源分配方面做出决策的医疗保健支付方应该是有价值的。