Institute of General Practice, Family Medicine and Preventive Medicine, Paracelsus Medical University, Strubergasse 21, 5020 Salzburg, Austria.
Eur J Public Health. 2012 Oct;22(5):625-9. doi: 10.1093/eurpub/ckr147. Epub 2011 Oct 11.
Disease Management Programmes (DMPs) are proposed to enhance the quality of care, to improve health outcomes and to reduce costs. Yet, the evidence regarding the effectiveness of such structured approaches remains uncertain. Randomized controlled trials (RCTs) represent the gold standard of evaluation for complex interventions. However, most of the evidence derives from non-randomized or even uncontrolled trials. We therefore tried to assess the impact of a randomized control group on the interpretation of DMP effectiveness.
We analyzed the data of a RCT on a DMP for diabetes type 2 by creating two scenarios. The first solely includes data of the intervention group (n=649), representing an 'uncontrolled pre-test-post-test analysis'. The second comprehends all data (n=1489) of the 'randomized controlled analysis'. HbA1c was used as the primary outcome measure for metabolic control in diabetes. Depending on either scenario, we calculated relative and absolute risk reduction regarding clinically relevant endpoints and estimated costs by extrapolating our results according to the UK Prospective Diabetes Study (UKPDS) findings.
The HbA1c reduction attributed to the DMP was 0.41% (uncontrolled analysis) vs. 0.13% (controlled comparison). Estimations of relative risk reduction for cardiovascular disease were 4.6% vs. 1.4%. The estimated numbers needed to treat (NNT) to avoid one myocardial infarction within 10 years differed from 125 (uncontrolled analysis) to 417 patients (controlled comparison), which led to a substantial scenario-dependent difference in cost estimations.
Uncontrolled pre-test-post-test evaluation might lead to crucial overestimation of DMP effectiveness. We therefore recommend randomized controlled evaluations prior to long-term implementation.
疾病管理计划（DMP）旨在提高医疗质量，改善健康结果并降低成本。然而，关于此类结构化方法的有效性的证据仍然不确定。随机对照试验（RCT）是评估复杂干预措施的金标准。然而，大多数证据来自非随机甚至非对照试验。因此，我们试图评估随机对照组对 DMP 有效性的解释的影响。
我们通过创建两种情况来分析针对 2 型糖尿病的 DMP 的 RCT 数据。第一种情况仅包括干预组的数据（n=649），代表“未对照的预测试后分析”。第二种情况包括所有数据（n=1489）的“随机对照分析”。HbA1c 用作糖尿病代谢控制的主要结局指标。根据任一情况，我们计算了临床相关终点的相对和绝对风险降低，并根据 UKPDS（英国前瞻性糖尿病研究）的结果推断我们的结果来估计成本。
DMP 导致的 HbA1c 降低为 0.41%（未对照分析）与 0.13%（对照比较）。心血管疾病的相对风险降低估计值为 4.6%与 1.4%。10 年内避免一次心肌梗死的估计治疗人数（NNT）分别为 125（未对照分析）和 417 例患者（对照比较），这导致成本估计值存在显著的依赖于情况的差异。
未对照的预测试后评估可能导致 DMP 有效性的重大高估。因此，我们建议在长期实施之前进行随机对照评估。
