Nature Reviews. Drug Discovery最新文献

{"title":"Design, simulate, refine: simulation-guided clinical trials for accelerated drug development","authors":"Elias Laurin Meyer, Tim Friede, Andrew P. Grieve, Christopher Jennison, Michael Krams, Elizabeth Lorenzi, Husseini K. Manji, Tobias Mielke, Jessica R. Overbey, Kert Viele, Marc K. Walton, Franz König","doi":"10.1038/s41573-026-01481-9","DOIUrl":"10.1038/s41573-026-01481-9","url":null,"abstract":"Modern clinical trials are embracing innovative strategies including adaptive designs, biomarker-guided enrolment, master protocols and real-time decision-making. These approaches may accelerate discovery and improve outcomes, yet they also introduce additional complexity in study conduct and understanding of the design. Simulation-guided design can be a powerful tool for identifying the optimal design to address research questions and for understanding and communicating how the design functions in practice. Simulations can evaluate the operating characteristics of complex trials, including control of type I error and false discovery rate, power for sample size determination, and probabilities of adaptive decisions at interim analyses. Examining individual simulated trials alongside these summaries provides insight into how the design will perform in practice, allowing potential risks to be anticipated and the design refined. Here, we outline how trial simulation can empower clinical development teams, statisticians, data monitoring committees, regulators, sponsors, funders and patient advocates by improving cross-functional communication, enhancing understanding and providing objective justification for design choices. We also provide best-practice recommendations to ensure that simulation studies are valid, transparent, thorough, efficient and comparable — helping to create trial designs that are scientifically rigorous, ethically sound, operationally practical and ultimately capable of bringing safe and effective therapies to patients as efficiently as possible. Simulation-guided clinical trial design can enable identification of the optimal design to bring safe and effective therapies to patients as efficiently as possible, and it is particularly useful when exploring innovative strategies such as adaptive designs and master protocols. This Perspective discusses the value of trial simulations from the perspective of different stakeholders involved in drug development, presents best-practice recommendations, and highlights case studies that illustrate the impact of the approach.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 10","pages":"792-808"},"PeriodicalIF":91.2,"publicationDate":"2026-07-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148432029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"FDA new drug approvals in Q2 2026","authors":"Paul Verdin","doi":"10.1038/d41573-026-00120-7","DOIUrl":"10.1038/d41573-026-00120-7","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"585-585"},"PeriodicalIF":91.2,"publicationDate":"2026-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148422852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Addressing challenges in treating rare tumours: the strategy of China’s National Cancer Center","authors":"Shuhang Wang, Jiawei Zhou, Hanqing He, Peiwen Ma, Yale Jiang, Ning Li","doi":"10.1038/s41573-026-01484-6","DOIUrl":"10.1038/s41573-026-01484-6","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 9","pages":"739-740"},"PeriodicalIF":91.2,"publicationDate":"2026-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148416336","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"FDA approves novel cell therapy made with haematopoietic stem cells, Tregs and T cells","authors":"Asher Mullard","doi":"10.1038/d41573-026-00119-0","DOIUrl":"10.1038/d41573-026-00119-0","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"584-584"},"PeriodicalIF":91.2,"publicationDate":"2026-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148408440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CAR T cell therapy for solid cancer scores first approval in China","authors":"Asher Mullard","doi":"10.1038/d41573-026-00117-2","DOIUrl":"10.1038/d41573-026-00117-2","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"583-583"},"PeriodicalIF":91.2,"publicationDate":"2026-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148408400","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"FDA approves second IGF-1R-targeted antibody for thyroid eye disease","authors":"Asher Mullard","doi":"10.1038/d41573-026-00118-1","DOIUrl":"10.1038/d41573-026-00118-1","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"583-583"},"PeriodicalIF":91.2,"publicationDate":"2026-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148408401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"HuR degrader knocks down BRAF in cancer","authors":"Alex Eccleston","doi":"10.1038/d41573-026-00116-3","DOIUrl":"10.1038/d41573-026-00116-3","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"590-590"},"PeriodicalIF":91.2,"publicationDate":"2026-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148393481","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"R&D productivity trends for biopharma companies in Asia and emerging markets","authors":"Ajay Gautam","doi":"10.1038/d41573-026-00114-5","DOIUrl":"10.1038/d41573-026-00114-5","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 9","pages":"672-673"},"PeriodicalIF":91.2,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148365392","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Growth in clinical research capability in China and impact on the pharmaceutical sector","authors":"Huimin Guo, \u0000 Mengze Ruan, \u0000 Kenneth Kaitin, \u0000 Liming Shao","doi":"10.1038/d41573-026-00113-6","DOIUrl":"10.1038/d41573-026-00113-6","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"586-587"},"PeriodicalIF":91.2,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148365463","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Circular RNA guides exon skipping in DMD","authors":"M. Teresa Villanueva","doi":"10.1038/d41573-026-00115-4","DOIUrl":"10.1038/d41573-026-00115-4","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 8","pages":"592-592"},"PeriodicalIF":91.2,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148365391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}