The drug development process faces challenges of efficiency and overall sustainability due in part to high research costs, lengthy development timelines, and late-stage drug failures. In response to these difficulties, as well as promising drug discovery opportunities offered by large-scale sequencing initiatives, the Forum on Drug Discovery, Development, and Translation and the Roundtable on Genomics and Precision Health held a workshop in March 2016, Deriving Drug Discovery Value from Large-Scale Genetic Bioresources. Speakers and participants discussed opportunities to reinvigorate the current drug development pipeline by utilizing information from genetic bioresources, a strategy that has realized some considerable successes in early-stage drug discovery. This follow-on workshop examined later stages in the drug development pipeline, including the design and implementation of genetics-based clinical trials. Novel clinical trial designs that target genetically identified participants represent a potentially disruptive paradigm shift that could reduce costs associated with drug development, improve patient outcomes, and further realize the goals of precision medicine.
This one-day public workshop examined and discussed successes, challenges, and best practices for effectively utilizing genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. Topics covered included strategies for including genetically focused populations in clinical trial design, logistical challenges in conducting genetics-based clinical trials, and mechanisms for engaging with and educating potential trial participants.
This public workshop, hosted on March 8, 2017 featured invited presentations and discussions to:
- Explored how clinical trials with genetically identified participants can enable more efficient and effective drug development and advance precision medicine.
- Highlighted ongoing genetics-based clinical trials across a variety of diseases, examining best practices and lessons learned.
- Learned about the logistical challenges and successes associated with genetics-based clinical trial design.
- Examined possible mechanisms to engage participants and improve enrollment into clinical trials based on genetic characteristics.
Below are materials for the workshop, including an agenda and presentations.
For additional information on the workshop, including the planning committee and sponsors, please visit our project page.