Statistical Methods for Cancer Clinical Trials

描述（由申请人提供）：这个雄心勃勃的计划项目的总体目标仍然是为癌症临床试验开发创新的，变革性的统计方法，这些方法有可能加速新疗法和治疗策略的成功引入。生物学、基因组学、统计学和计算科学的进步为个性化癌症治疗的发展带来了巨大的希望。我们的多机构，跨学科的研究人员团队将利用这些进展来创建新的临床试验设计和数据分析方法，解决当前统计方法的许多关键限制，并最大限度地提高个性化癌症药物临床试验的有效性。此外，我们将促进将这些方法转化为实践，包括根据新方法开展试点动物和人体研究。该计划将通过五个相互关联的研究项目实现这些目标，这些项目由三个机构的统计，计算和临床科学方面的互补专业知识的研究人员进行。前四个项目的重点是开发新的试验设计和分析方法，整合生物标志物，以有效发现新的个性化治疗;创建分析生物标志物和患者报告结果的现有数据的方法，以告知和改进未来研究的设计;开发方法，最大限度地发挥药物基因组学的力量，以确定生物标志物和候选个体化治疗;并创造新的方法来发现和验证癌症治疗的顺序，个性化的决策策略。第五个项目将把这些方法整合到胰腺癌的新的临床前和临床研究中。我们的综合方法涉及一个充满活力和协调的过程，用于实施，交流和传播结果，包括开发专业，公共使用的软件和相关教程;研讨会和其他外联机制;以及计划赞助的研讨会和活动，以加速在实践中采用这些方法。提出的临床试验设计和分析创新有可能影响癌症临床试验的方式进行个性化药物的发现和验证的范式转变。这种全面的、多机构的努力将导致癌症临床试验实践的重大创新，从而改善癌症患者的健康状况。

DESCRIPTION (provided by applicant): The overarching goal of this ambitious program project continues to be to develop innovative, transformative statistical methods for cancer clinical trials that have the potential to hasten successful introduction of new therapies and treatment strategies into practice. Advances in the biologic, genomic, statistical, and computational sciences hold great promise for the development of personalized cancer treatments. Our multi-institutional, interdisciplinary team of investigators will leverage these advances to create new clinical trial designs and data analysis approaches that resolve many of the key limitations of current statistical methods and that maximize the effectiveness of clinical trials for personalized cancer medicine. In addition, we will foster translation of these methods into practice, including carrying out pilot animal and human studies based on the new methodology. The program will achieve these objectives through five, interrelated research projects carried out by investigators with complementary expertise in the statistical, computational, and clinical sciences from three institutions. The first four projects focus on developing new trial designs and analysis methods that integrate biomarkers for efficient discovery of new, personalized treatments; on creating methods for analysis of existing data on biomarkers and patient reported outcomes to inform and improve the design of future studies; developing methods for maximizing the power of pharmacogenomics for identifying biomarkers and candidate individualized therapies; and creating new methods for discovering and validating sequential, personalized decision-making strategies for cancer treatment. The fifth project will integrate the methods into novel preclinical and clinical studies of pancreatic cancer. Our comprehensive approach involves an energetic and coordinated process for implementation, communication, and dissemination of results, including development of professional, public-use software and associated tutorials; workshops and other outreach mechanisms; and program-sponsored symposia and events, to accelerate the adoption of the methods in practice. The proposed clinical trial design and analysis innovations have the potential to effect a paradigm shift in the way cancer clinical trials are conducted for discovery and validation of personalized medicine. This comprehensive, multi-institutional effort will lead to significant innovations in cancer clinical trial practice that will result in improved health of cancer patients.