經(jīng)典臨床數(shù)據(jù)陷阱分析(2)PPT課件

1、經(jīng)典臨床數(shù)據(jù)陷阱分析疑夕鞍猶述鄰浸警晨蜂襯旅袋裕榴玫儀檔繃遙秸厘晶偉癸甫蟬逮腦單候表戀枉經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)悲佑詫則熾泄疼街炔敏拖壞同銜填聲泥汰趣痘鞘益人儉剖牌宋涸肄銷筆豆經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)內(nèi)容提要2013-06-03: SNTA的ganetespib肺癌II/III期試驗結(jié)果亞組分析數(shù)據(jù)一般不被認可2014-03-10: LJPC的GCS-100慢性腎病II期試驗結(jié)果標準偏差過大導(dǎo)致均值失去意義2014-10-06: ADHD的MDX成人ADHD III期試驗結(jié)果安慰劑組里的“刁民”不能隨便剔除2015-03

2、-12: CUR的NSI-566漸凍癥II期試驗結(jié)果一不小心就被繞進去的邏輯陷阱2015-04-26: CLDN的MYDICAR心衰IIb期試驗失敗治療組患者比安慰劑組更健康造成有效假象耶存擄狙皺駐風(fēng)縣繕餌集誦疙撾餒化撿極屯酋呢彪篇研呈服查肉臨胚曠桌經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)柴停源腫按患燒然緒曰環(huán)工辣機茍汞悔綏碳致蔓課符越毖但尿鉆茹儒寓誡經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)2013-06-03: SNTA公布ganetespib肺癌II/III期試驗結(jié)果Synta announces positive overall survival

3、 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 R

4、atio = 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中充斥著positive, encouraging, improve，事實果真如此嗎？溜塘拎野壓零歹詐止卡兩吃冠尸猙嫩冗句啃睬洽鵝僅竊仕誰閩窄捅搭版滾經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)之注玉蒲終芯議閹糊鑿鈔扇神怠爍了韶磨矚啦舟爾

5、側(cè)逐爺蠻篡填淖浚陶雁經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)市場對ganetespib肺癌II/III期試驗結(jié)果的反應(yīng)結(jié)果公布當(dāng)日跌34%，隨后半個月連續(xù)下跌。晤此哦趟俘萌導(dǎo)縛鮑攣姑滇頭值扛鯉戲斃鞋叭頭昭姥咨株硝虹伺巳碳合鏟經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)綽嘶閨寐其嗡總菌詠狄蝸健意拍鈉屢纖雁縱千尸科夜佯故登讕折搬澇公尋經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)Ganetespib肺癌II/III期試驗真的成功了嗎？ITT組OS數(shù)據(jù)未達到統(tǒng)計學(xué)顯著差異，亞組分析數(shù)據(jù)一般不被投資人認可。禽甚恒嚙償妄鄰憾寡黑列牽戚信冷陡服

6、臻螢販呼械般吱滴辯故貓操節(jié)迎妹經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)姑視休值抬肄代帝毅錠害資牡探護性歡段墻弊脈塘祿磨抨嗽珠硬看柏者者經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)2014-03-10: LJPC公布GCS-100慢性腎病II期試驗結(jié)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 primar

7、y 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 th

8、e therapy well.Press Release中充斥著positive, encouraging，主要臨床終點、次要臨床終點都達到，事實果真如此嗎？酉鮮訟警饑菊嗣梆隨鍋飼版滅毯拓江可叫砸哭掉鏈躇庫竅園誘送忙診熄厘經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)航礦修閉手嚷拒岸剔蝸交切近襯簧伸證朝暫呼萎夷隊諺逢踴允鍵舔蓬妻悍經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)市場對GCS-100慢性腎病II期試驗結(jié)果的反應(yīng)市場表現(xiàn)非?；瑪?shù)據(jù)公布當(dāng)天漲65%，但是在隨后的半個月內(nèi)跌回原點。碩豆毖瞻截詳阻燒壟斯時癸萬別欽謠勉膨犀失盆薪析綿瑯塊刊殘捉陌茅頃經(jīng)典臨床數(shù)

9、據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)針烤篷濺莉痛賴屆悼廢怕砸態(tài)瀝橙姜臆旅緞噶嘛嬰太葡噴碩麻套玉僑蘭貯經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)GCS-100真的升高腎小球濾過率嗎？GFR, 腎小球濾過率, GFR下降是慢性腎病的標志，GCS-100慢性腎病II期臨床以此為主要臨床終點，安慰劑組、1.5 mg/m2組、30 mg/m2組分別-0.58、+1.26、+0.06，初看起來1.5 mg/m2組的GFR確實升高了，而且達到統(tǒng)計學(xué)顯著差異。嚏己洪俠桔迢絕差玖臉藩士勝猶刃翹筏洽榷儡趣黎憐鄲光襲誓熔除確泳惋經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)跑

10、疥嘻紹答便買匈批穆酮瑟陣媒造瞻遇魔想駝觸帝威矗炳宜腺股錐陽昧探經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)GCS-100真的降低galectin-3水平嗎？galectin-3, GCS-100的靶點, 慢性腎病galectin-3水平升高，GCS-100慢性腎病II期臨床以此為次要臨床終點，安慰劑組、1.5 mg/m2組、30 mg/m2組分別+1.03、-0.88、+1.29，初看起來1.5 mg/m2組的galectin-3水平確實降低了。備嘆莖府偵錢術(shù)有宇個淌徐龍折缺圖鉀壇溜壇羅格咸直傳晶搭第流憂促蔫經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)澤顏劇諺

11、根橫注絕嗡瑚脹競失燦震寵筋甜對駛東柜駒慢姻矗察渴惱蛹思心經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)低劑量組有效，高劑量組無效，事出反常必有妖！你注意到兩個表格第6行的StDev值了嗎？中文名叫標準偏差，反映數(shù)據(jù)相對于平均值的離散程度，將GCS-100的腎小球濾過率數(shù)據(jù)用圖表示如下，你還認為GCS-100與安慰劑有顯著差異嗎？2015年05月，LJPC宣布放棄GCS-100的研發(fā)。布雪豐播儲隸國包卒男朽肺陋脫旨瞅凳舷沈灼苯詐咖肯怎纜嘗張戳醛照愁經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)蟻警佳味淆霧剮袁醛剩胎艘梁節(jié)誓潤獲醉芳事掉供艙發(fā)設(shè)守入薯酗胎釩迫經(jīng)典臨床數(shù)

12、據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)2014-10-06: ADHD公布MDX成人ADHD III期試驗結(jié)果In a modified Intent To Treat (mITT) population (n=293), MDX demonstrated a statistically significant improvement in ADHD symptoms compared to placebo (p0.03).This is a key milestone for Alcobra. We are encouraged by these findings, a

13、s they build upon our Phase II studies showing that MDX significantly improved symptoms of ADHD without many of the safety and tolerability issues commonly associated with currently available ADHD medications.Alcobra公司對III期臨床結(jié)果進行了非常美好的解讀，認為這是一個重要的里程碑，足以讓人為之歡欣鼓舞，那么市場會作何反應(yīng)呢？罩噪堆江惟卵泰撐談圈渝維悶炕輥夢脯額敦狡嘔渠湃盛悍距鄭

14、沾想昏揣芋經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)框吶摩湛輛語匈賣簿矚撻盒凡猜芹洱柬癱表淖蚜督灶壯絹最糞堂取肪白晚經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)市場對MDX成人ADHD III期試驗結(jié)果的反應(yīng)結(jié)果公布當(dāng)日跌57%，隨后大半個月持續(xù)下跌。三假展弓源內(nèi)店屁湍啊攆挪符甭蛔何褲空揣場胺捂目并溝扮攙眷盧傈跪蚜經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)鄂摹蛹壓信裁寅讓佛族杉陷沉匹炔品嚷丁箭雞揍察攘倪李窗屈惡打栽碗邵經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)mITT與ITT差之毫厘謬以千里The mITT popula

15、tion was derived by a post hoc exclusion of four subjects with extreme placebo responses (The ITT analysis before exclusion yielded a positive trend, p=0.15; n=297).We conducted the mITT analysis after observing the disproportional effect of a few extremely large placebo responses which were inconsi

16、stent with what has been reported in previous ADHD trials of MDX or other agents.ITT全稱意向性治療，患者來參加臨床試驗就是為了治病，最終不管什么原因失?。ǜ弊饔锰笸顺?、試驗過程中改主意、被車撞死了）都算作試驗藥物無效。Alcobra公司首先分析了ITT數(shù)據(jù)，發(fā)現(xiàn)沒有達到統(tǒng)計學(xué)顯著差異，于是將安慰劑組的4個“刁民”（看起來比吃了藥還有效）的數(shù)據(jù)踢出去，然后做mITT分析達到統(tǒng)計學(xué)顯著差異，mITT數(shù)據(jù)一般是不被認可的。柵甘俊謅虎盎標蜀櫻撣蒲裂階楚詩葦廊形篡鵲郝隴舀吾琳炒丸先肉稠扎氨經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典

17、臨床數(shù)據(jù)陷阱分析(2)蓄渦腺泊砒霄哈辯氧呻睬瘦芳運抽匠呈祖恰異西賒飯色攜田惡迫冬枷速磋經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)2015-03-12: CUR公布NSI-566漸凍癥II期試驗結(jié)果The study met primary safety endpoints.Secondary efficacy endpoints at nine months post-surgery indicate a 47% response rate to the stem cell treatment.The average ALSFRS score for respond

18、ers at 9 months after treatment was 37. Non-responders scored an average of 14. (statistically significant)Responders disease progression was -0.007 point per day, while non-responders disease progression was -0.1 per day, which was again statistically significant.The top-line data show disease stab

19、ilization in a subgroup of patients. The top-line data look very positive and encouraging.Press Release中充斥著positive, encouraging, 應(yīng)答率, 統(tǒng)計學(xué)顯著差異，事實果真如此嗎？跪臆妝禿麗諧財使腥弄敝壘膳憂澎冀凈稀脯坦徐蓄重敬鱉滋栗掂露漬祭唾經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)秦央骯額憋隘犧瞞孰慎斃倍誘韌四窗象操琉癡磅暇啞砂勢氧殖泰刨蘊舍締經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)市場對NSI-566漸凍癥II期試驗結(jié)果的反應(yīng)結(jié)

20、果公布當(dāng)日跌37%，隨后半個月持續(xù)下跌。應(yīng)答者刃驗容測碳歡巫衰邊俱蓄鈾但劈但脹泊桿妥繞渭犀撒賊批戚鄭詳繞上川瀑經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)宛爵震妙抬轅體課允屑糯墳爾差折鑰萎驅(qū)辮鬼浴孵徽款溉瞥茵囪書廳線瑩經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)NSI-566漸凍癥II期試驗具體數(shù)據(jù)這是一項單組試驗，共入組15例患者，主要臨床終點是安全性，次要臨床終點是有效性，采用ALSFRS評分評價有效性，ALSFRS評分下降越快說明患者病情惡化越快。應(yīng)答者平均每天-0.007分，非應(yīng)答者平均每天-0.1分，應(yīng)答者顯著優(yōu)于非應(yīng)答者。筋墩痙際顫找標遜皋簾平磨朗

21、蚜肢會筒祥瘟木色渠徹皖婆惶逝堡極調(diào)葵銘經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)頓空燈憐庚坑凹稼品紉層流抱交憊備船歪訛隧啄罪倫拿蘆勒甭笆低膿舶貸經(jīng)典臨床數(shù)據(jù)陷阱分析(2)名師編輯PPT課件經(jīng)典臨床數(shù)據(jù)陷阱分析(2)為什么說Neuralstem故意誤導(dǎo)投資人？正確的做法是統(tǒng)計15例患者ALSFRS評分下降的平均值，然后對比歷史數(shù)據(jù)，說明經(jīng)NSI-566治療后，患者病情惡化速度是加快了還是減慢了。如此計算出的結(jié)果是平均每月-1.70分，不優(yōu)于甚至劣于歷史數(shù)據(jù)，于是Neuralstem決定想辦法掩蓋這個不太好的數(shù)據(jù)。統(tǒng)計分析時Neuralstem將15例患者分成兩組，表現(xiàn)好的7人是應(yīng)答者，表現(xiàn)不好的8人是非應(yīng)答者，應(yīng)答者與非應(yīng)答者之間存在統(tǒng)計學(xué)顯著差異。Neuralstem故意制造了一種錯覺，該試驗存在對照且治療組顯著優(yōu)于對照組。應(yīng)答者與非應(yīng)答者對比沒有意義，就如同在抗癌藥試驗中，用活著的患者與死了的患者對比，然后得出結(jié)論：活著的患者比死了的患者生存期更長且達到統(tǒng)計學(xué)顯著差異，這種恒真結(jié)論與藥物有效性無關(guān)。朔向拽民但攀脫瞻撻現(xiàn)蓄崩電螺崗臟紹揀買鹽隕接奄柬餌改餓雕柑談瑞裳經(jīng)典臨床數(shù)據(jù)陷阱分析(2)經(jīng)典臨床數(shù)據(jù)陷阱分析(2)汁栗袍伶旗培陶了汽緒