真实世界研究(杜文民)

• This was a naturalistic, real-world, clinic-based, outpatient study involving 336 general practitioners/primary care physicians in 65 cities in India.
敏感性 分析 模型 外在的 真实性 贝叶氏 方法
选择的人群 霍索恩效应 观察时间短 样本量太小 观察范围窄
内在的 真实性
局限性
真实世界 研究
临床 试验
内在的 真实性
真实世界研究的作用?
公共健康管理水平 康促进 - 卫生行政和药品行政决策
• RCT应该用于评价有效性（efficacy ），而 RWS用于决定效应性（effectiveness ） • RWS 提供RCT没有的补充资料 • RWS 能够作为属于药物流行病学重要的工 具用于决定临床实践中真实的效益、风险 和治疗价值
• Saskatchewan • Group Health Co-operative of Puget Sound • GPRD • Medicaid
• Fixed combination perindopril 4 mg/amlodipine 5 mg once daily for 60 days. The primary outcomes were the mean change in BP from baseline and the proportion of patients achieving adequate BP control (</=140/90 mmHg, or </=130/80 mmHg in patients with diabetes mellitus) in the intent-to-treat (ITT) population. Secondary analyses included incidence of adverse events (ITT) and treatment adherence rate (completers).
• Adults aged 40-70 years with newly diagnosed/untreated stage 2 hypertension (BP >/=160/100 mmHg), hypertension uncontrolled with monotherapy (BP >140/90 mmHg), or hypertension inadequately managed with another combination therapy.
The STRONG study
• STRONG(Safety & efficacy analysis of coversyl amlodipine in uncontrolled and Newly diagnosed hypertension) • Management of Hypertension with the Fixed Combination of Perindopril and Amlodipine in Daily Clinical Practice: Results from the STRONG Prospective, Observational, Multicenter Study. • Bahl VK, Jadhav UM, Thacker HP • Am J Cardiovasc Drugs. 2009;9(3):135-42
药物流行病学研究方法
真实世界研究 (RWS)
队列和病例对照研究
病例报告和病例系列
--- RCT 设计 ---
--- RWS 设计 ---
患病人群 纳入 / 排除标准 随机化 治疗 对照 暴露
患病人群
治疗的适应症 进行治疗 对照
研究方案的治疗和随访
专门的治疗和随访
结果?
结果?
关于真实世界研究的结论
真实世界研究 (Real world Study)
上海市药品不良反应监测中心 杜文民
评价研究的矛盾
观察性研究 局限性
选择偏移 观察偏移 混杂因素
内在的 真实性
?
外在的 真实性
选择的人群 霍索恩效应 观察时间短 样本量太小 观察范围窄
局限性
临床 试验
评价研究的矛盾
观察性研究
局限性
选择偏移 观察偏移 混杂因素
• In total, 1250 patients comprised the ITT population: 32.6% with newly diagnosed hypertension; 40.5% with hypertension uncontrolled with monotherapy; and 26.9% with hypertension inadequately managed with another combination therapy. Mean SBP/DBP decreased significantly from baseline (167.4 +/- 15.2/101.4 +/- 9.1 mmHg) over 60 days (-41.9 +/- 34.8/-23.2 +/- 21.8 mmHg; p < 0.0001).
• Target BP was achieved in 66.1% of patients in the total population, 68.3% of untreated patients, 68.4% of patients uncontrolled with monotherapy, and 59.9% of patients inadequately managed with combination therapy. In 161 patients with SBP >180 mmHg at baseline (newly diagnosed: n = 50; uncontrolled on monotherapy: n = 53; inadequately managed on combination therapy: n = 58), BP was reduced by 63.2 +/- 32.5/29.0 +/- 21.9 mmHg (p < 0.0001) at day 60. The fixed combination was safe and well tolerated. All 1175 patients completing the 60-day study (94%) adhered to their treatment regimen.
患病人群 靶人群
药物相关人群
研究方案 用于治疗
合格人群
研究队列
治疗靶人群
结果
?
治疗过的人群
?
药物相关问题
观点 患病人群 期望的治疗效果 问题 可获得治疗 的比例？
靶人群
代表性?
各个亚组患者 的治疗效果 合格人群 代表性? 研究人群 推荐性? 潜在的治疗效果
治疗靶人群
依从性? 接受治疗的人群
真实的治疗效果