癌癥研究和藥物研發(fā)可獲得更理想的療法,，但是根據(jù)一項(xiàng)由Sloan-Kettering癌癥中心的研究人員發(fā)表的報(bào)告指出,，這些測(cè)試癌癥藥物的臨床試驗(yàn)存在著很大的缺陷。
這項(xiàng)發(fā)表于2月1 日Clinical Cancer Research的研究報(bào)告,，解釋了為什么許多實(shí)驗(yàn)性藥物無通過最后的大型且昂貴的臨床測(cè)試,。
研究人員發(fā)現(xiàn)，在他們審查的2003 年6月至2005 年6月間，共70項(xiàng)第II階段研究中,，只有9項(xiàng)研究清楚地定義用于評(píng)估對(duì)患者益處之標(biāo)準(zhǔn),。這些研究通常包含30到50 名患者，目標(biāo)是評(píng)估是否應(yīng)繼續(xù)進(jìn)行第III階段的大型臨床試驗(yàn),，這是決定該藥物是否能用于癌癥患者之最后測(cè)試,。
研究人員指出，這是臨床試驗(yàn)測(cè)試中不斷衍生出的問題,，因?yàn)榧词巩?dāng)藥物改變了,，研究人員仍然使用一樣的老方法來測(cè)量這些藥物的效果。因此研究人員建議,，當(dāng)制藥公司或?qū)嶒?yàn)室針對(duì)新的物質(zhì)進(jìn)行臨床試驗(yàn)時(shí),，必須設(shè)定新的評(píng)估標(biāo)準(zhǔn)，才能更有效地評(píng)估藥效及藥物的安全性,。
(資料來源 : Bio.com)
英文原文摘要：
Setting the Bar in Phase II Trials: The Use of Historical Data for Determining "Go/No Go" Decision for Definitive Phase III Testing
Andrew J. Vickers1,2, Vennus Ballen1 and Howard I. Scher1
Authors' Affiliations: Departments of 1 Medicine and 2 Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, NY
Requests for reprints: Andrew J. Vickers, Memorial Sloan-Kettering Cancer Center 1275 York Avenue, New York, NY 10021. Phone: 212-639-4910; Fax: 212-639-4953; E-mail: [email protected] .
Purpose: Phase II trials aim to determine whether a cancer treatment is sufficiently promising to justify phase III study. Whether an agent is declared promising in a phase II trial depends on prespecified "null" and "alternative" rates of an outcome of interest such as tumor response. In some cases, the null must be determined with reference to historical data. We sought to determine the proportion of phase II trials that require historical data to establish the null and to determine how these historical estimates were derived.
Experimental Design: We conducted a systematic review of phase II trials published in the Journal of Clinical Oncology or Cancer in the 3 years to June 2005. Data were extracted following a prespecified protocol.
Results: We retrieved 251 papers, of which 117 were found to be ineligible; 70 of 134 included trials (52%) were defined as requiring historical data for design. Nearly half (32, 46%) of these papers did not cite the source of the historical data used, and just 9 (13%) clearly gave a single historical estimate as the rationale for the null. Trials that failed to cite prior data appropriately were significantly more likely to declare an agent to be active (82% versus 33%; P = 0.005). No study incorporated statistical methods to account for either sampling error or possible differences in case mix between the phase II sample and the historical cohort.
Conclusions: Many phase II trials require historical data to determine null response rates. Simple guidelines may improve design and reporting of such trials.
原文出處：Clinical Cancer Research 13, 972-976, February 1, 2007. Published Online First February 2, 2007;
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