At this time, the annual AAADV Workshop is returning to an in-person meeting, on May 14-15, 2025 to be held at the North Bethesda Marriott in Bethesda, MD. The program committee of Drs. Schilsky, Canetta, Fleming, and Lyerly are pleased that former FDA commissioner, Dr. Robert Califf will be the keynote speaker, highlighting his experience and providing his perspective on the next era of drug development. Four plenary sessions will follow, each consisting of a series of focused talks from experts in the field, followed by a moderated discussion, emphasizing the regulatory perspectives involved. The first plenary session will focus on the utility of large trials that utilize multiple experimental agents from different sponsors in order to identify signals of anti-cancer activity in an efficient manner. Specifically, the session aims to review and determine what has been learned from such trials, and how to organize them even more efficiently in the future. Trials to be discussed include the Beat AML and PedAL studies sponsored by the Leukemia and Lymphoma Society, the Lung-MAP study organized by NCI with support of the Foundation for the NIH (FNIH) and Friends of Cancer Research, the NCI MATCH study and the ASCO TAPUR study. The second session will focus on global drug development and highlight the experience in multi-national clinical cancer research from one of the leading organizations, the EORTC. Additional topics will address including representative populations in international clinical trials, as well as the complexities of conducting clinical trials in which regional variations in medical practice and standards of care may exist. An additional plenary session on global cancer drug development will focus on Nigeria, which has a vision of becoming a clinical trial hub and innovation ecosystem.

The second day of the workshop will consist of two plenary sessions, the first being a session on developing and implementing complex biomarkers to guide cancer treatment. This morning session will focus on the rapidly expanding role of artificial intelligence in cancer drug and biomarker development and on network analysis to identify novel biomarkers and targets. This will be followed by talks focused on the clinical applications of transcriptomics, and AI-assisted digital pathology.

Finally, the workshop will conclude with a plenary session focusing on choosing proper primary endpoints and registrational oncology trials that a focus on indolent cancers. This topic will allow a discussion of reliable assessment of efficacy and safety in trials of indolent cancers when overall survival may take a long time to ascertain as well as on evaluating the effects of cancer treatments on how patients feel and function as a potential approval endpoint.

While it is understood that exciting therapeutic opportunities for cancers are being developed in research laboratories around the world, these new molecules often are accessible only to the elite research institutions that are in major cities. The vast majority of cancer patients do not have access to these elite institutions and do not have access to these trials. Consequently, by creating a forum for best practices, and by providing educational content to early career investigators, the AAADV Workshop aims to support researchers and clinicians in the broader medical community and provide the opportunity for clinicians and patients to access new therapeutics in their communities. The AAADV has been instrumental not only to advancing the methodology of cancer clinical trials but also to the training of a new generation of clinical investigators in both academia and industry. Consequently, a long-term goal is to expand the global educational and training infrastructure to continue to support and develop a clinical investigator workforce around the world that is expert in clinical trial design, trial conduct and analysis and has a deep understanding of regulatory requirements to advance new products to market. Through these educational activities, including annual workshops and other ongoing educational activities, the AAADV Workshop hopes to catalyze efforts by senior investigators to provide training and education, and to promote the adoption of policies that accelerate the development of new therapeutics for uncommon or rare conditions especially those occurring in children. Through ongoing educational activities and workshops, the AAADV Workshop hopes to provide a foundation in regulatory science that will support the development of products for these specific populations in a timely and cost-efficient fashion, supporting the translation of the science to benefit patients.