真实世界研究优秀课件
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真实世界研究优秀课件
评价研究的矛盾
外在的 真实性
观察性Hale Waihona Puke Baidu究
?
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
局限性
选择偏移 观察偏移 混杂因素
临床 试验
内在的 真实性
评价研究的矛盾
观察性研究
外在的 真实性
✓ 敏感性 分析
✓ 模型
✓ 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
• 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.
局限性
真实世界 研究
局限性
选择偏移 观察偏移 混杂因素
临床 试验
内在的 真实性
内在的 真实性
真实世界研究的作用?
公共健康管理水平 有效性、效果
疾病管理 ： 医院和社区
公共健康管理： - 健康促进 - 卫生行政和药品行政决策
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
--- RCT 设计 ---
患病人群
纳入 / 排除标准
随机化
治疗
对照
研究方案的治疗和随访
结果?
--- RWS 设计 ---
患病人群
治疗的适应症
进行治疗
暴露
对照
专门的治疗和随访
结果?
关于真实世界研究的结论
• RCT应该用于评价有效性（efficacy ），而 RWS用于决定效应性（effectiveness ）
• Bahl VK, Jadhav UM, Thacker HP • Am J Cardiovasc Drugs. 2009;9(3):135-42
• This was a naturalistic, real-world, clinic-based, outpatient study involving 336 general practitioners/primary care physicians in 65 cities in India.
• 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
The STRONG study
• 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.
• 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).
?
治疗过的人群
结果
?
观点
期望的治疗效果 各个亚组患者
的治疗效果 潜在的治疗效果 真实的治疗效果
药物相关问题
患病人群 靶人群
合格人群 研究人群 治疗靶人群 接受治疗的人群
问题 可获得治疗 的比例？
代表性? 代表性? 推荐性?
依从性?
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
• 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.
• 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).
评价研究的矛盾
外在的 真实性
观察性Hale Waihona Puke Baidu究
?
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
局限性
选择偏移 观察偏移 混杂因素
临床 试验
内在的 真实性
评价研究的矛盾
观察性研究
外在的 真实性
✓ 敏感性 分析
✓ 模型
✓ 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
• 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.
局限性
真实世界 研究
局限性
选择偏移 观察偏移 混杂因素
临床 试验
内在的 真实性
内在的 真实性
真实世界研究的作用?
公共健康管理水平 有效性、效果
疾病管理 ： 医院和社区
公共健康管理： - 健康促进 - 卫生行政和药品行政决策
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
--- RCT 设计 ---
患病人群
纳入 / 排除标准
随机化
治疗
对照
研究方案的治疗和随访
结果?
--- RWS 设计 ---
患病人群
治疗的适应症
进行治疗
暴露
对照
专门的治疗和随访
结果?
关于真实世界研究的结论
• RCT应该用于评价有效性（efficacy ），而 RWS用于决定效应性（effectiveness ）
• Bahl VK, Jadhav UM, Thacker HP • Am J Cardiovasc Drugs. 2009;9(3):135-42
• This was a naturalistic, real-world, clinic-based, outpatient study involving 336 general practitioners/primary care physicians in 65 cities in India.
• 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
The STRONG study
• 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.
• 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).
?
治疗过的人群
结果
?
观点
期望的治疗效果 各个亚组患者
的治疗效果 潜在的治疗效果 真实的治疗效果
药物相关问题
患病人群 靶人群
合格人群 研究人群 治疗靶人群 接受治疗的人群
问题 可获得治疗 的比例？
代表性? 代表性? 推荐性?
依从性?
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
• 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.
• 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).