临床数据陷阱分析.pptxVIP

临床数据陷阱分析第1页/共20页 经典临床数据陷阱分析第2页/共20页 内容提要2013-06-03: SNTA的ganetespib肺癌II/III期试验结果 亚组分析数据一般不被认可2014-03-10: LJPC的GCS-100慢性肾病II期试验结果 标准偏差过大导致均值失去意义2014-10-06: ADHD的MDX成人ADHD III期试验结果 安慰剂组里的“刁民”不能随便剔除2015-03-12: CUR的NSI-566渐冻症II期试验结果 一不小心就被绕进去的逻辑陷阱2015-04-26: CLDN的MYDICAR心衰IIb期试验失败 治疗组患者比安慰剂组更健康造成有效假象第3页/共20页 2013-06-03: SNTA公布ganetespib肺癌II/III期试验结果Synta announces positive overall survival results from GALAXY-1 Phase 2b/3 trial of ganetespib in second-line non-small cell lung cancer.Median overall survival improved 32% in the all adenocarcinoma, intent-to-treat population.In the population selected for the ongoing GALAXY-2 Phase 3 trial, median overall survival improved 67%, Hazard Ratio = 0.61 (p=0.009).The magnitude and consistency of the activity reported today are very encouraging and bode well for the outcome of the GALAXY-2 Phase 3 study.Press Release中充斥着positive, encouraging, improve，事实果真如此吗？第4页/共20页 市场对ganetespib肺癌II/III期试验结果的反应结果公布当日跌34%，随后半个月连续下跌。第5页/共20页 Ganetespib肺癌II/III期试验真的成功了吗？ITT组OS数据未达到统计学显著差异，亚组分析数据一般不被投资人认可。第6页/共20页 2014-03-10: LJPC公布GCS-100慢性肾病II期试验结果La Jolla Pharmaceutical Company reports positive, top-line results from Phase 2 clinical trial of GCS-100 in chronic kidney disease.The trial met its primary efficacy endpoint of a statistically significant improvement in kidney function.Key secondary endpoints were also met, and the effect on circulating galectin-3 levels was consistent with the effect on eGFR.Our experience with GCS-100 in this trial was very encouraging, and my patients tolerated the therapy well.Press Release中充斥着positive, encouraging，主要临床终点、次要临床终点都达到，事实果真如此吗？第7页/共20页 市场对GCS-100慢性肾病II期试验结果的反应市场表现非常滑稽，数据公布当天涨65%，但是在随后的半个月内跌回原点。第8页/共20页 GCS-100真的升高肾小球滤过率吗？GFR, 肾小球滤过率, GFR下降是慢性肾病的标志，GCS-100慢性肾病II期临床以此为主要临床终点，安慰剂组、1.5 mg/m2组、30 mg/m2组分别-0.58、+1.26、+0.06，初看起来1.5 mg/m2组的GFR确实升高了，而且达到统计学显著差异。第9页/共20页 GCS-100真的降低galectin-3水平吗？galectin-3, GCS-100的靶点, 慢性肾病galectin-3水平升高，GCS-100慢性肾病II期临床以此为次要临床终点，安慰剂组、1.5 mg/m2组、30 mg/m2组分别+1.03、-0.88、+1.29，初看起