Appendix B

Statement of Task and Workshop Agenda

Enabling Precision Medicine:
The Role of Genetics in Clinical Drug Development
A Workshop

March 8, 2017
Keck Center of the National Academies
500 Fifth Street, NW Room 100
Washington, DC 20001

Statement of Task

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 1-day public workshop that will examine and discuss successes, challenges, and best practices for effectively using 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, will select and invite speakers and discussants, and may moderate the discussions. The proceedings of the workshop will be prepared by a designated rapporteur in accordance with institutional policy and procedures.

SESSION I: OVERARCHING CONSIDERATIONS FOR IMPLEMENTING SUCCESSFUL GENETICS-BASED DRUG DEVELOPMENT

Session Objectives:

• Discuss the perspective of patients with regard to the use of genetic data in clinical drug development.
• Consider challenges and possible solutions associated with the development and use of biomarkers or genetic tests for molecularly targeted therapies.
• Explore issues that pertain to the regulatory pathway for genetic tests and biomarkers used in clinical drug development.

SESSION II: CASE STUDIES IN PRECISION DRUG DEVELOPMENT

Session Objectives:

• Explore recent examples of precision drug development in order to identify lessons that can be applied across disease fields.
• Examine clinical development plans for precision medicines, including
  • using our understanding of the underlying biology to inform trial design and product development;
  • effects on research costs and timeline; and
  • patient recruitment and stratification.

SESSION III: INTEGRATING GENETICS INTO THE DRUG DEVELOPMENT PATHWAY FOR COMPLEX DISEASES

Session Objectives:

• Illuminate novel ways to apply genetics/genomics discoveries into clinical development plans for complex diseases as a way to increase efficiency and improve outcomes.
• Survey innovative ways to engage participants in precision clinical trials, including communication, outreach, and education-based initiatives, with additional attention paid to increasing the diversity of such trials.
• Discuss additional logistical challenges associated with the execution of a successful genetics-based clinical trial for a complex disease, including
  • techniques for patient stratification, including considerations for employing genetic risk scores; and
  • timelines for developing molecularly targeted therapies, including trial execution.

SESSION IV: FINDING INNOVATIVE WAYS TO INTEGRATE GENETICS RESEARCH INTO THE DRUG DEVELOPMENT PROCESS

Session Objective:

• Explore innovative, “outside-the-box” ideas for genetics-based clinical trials that could make the therapeutic development pathway more efficient.

SESSION V: LOOKING FORWARD—RECAP AND CONCLUSIONS