The current 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. Novel clinical trial designs that target genetically identified participants represent a potentially disruptive paradigm shift that could reduce overall health care costs associated with drug development, improve patient outcomes, and further realize the goals of precision medicine. An ad hoc committee will plan and conduct a one-day public workshop that will examine and discuss 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 could include 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. Discussions will be held among a broad array of stakeholders, which may include patients, academic researchers, health care providers, and representatives from the biopharmaceutical sector.
Objectives:
• To explore how clinical trials with genetically identified participants can enable more efficient and effective drug development and advance precision medicine.
• To highlight ongoing genetics-based clinical trials across a variety of diseases, examining best practices and lessons learned.
• To learn about the logistical challenges and successes associated with genetics-based clinical trial design.
• To examine possible mechanisms to engage participants and improve enrollment into clinical trials based on genetic characteristics.
The planning committee will develop the workshop agenda, select and invite speakers and discussants, and may moderate the discussions. Proceedings of the workshop will be prepared by a designated rapporteur in accordance with institutional policy and procedures.