Appendix A

Statement of Task

An ad hoc committee will plan and host a 1.5-day public workshop that will feature invited presentations and panel discussions. Workshop participants will examine the rationale and potential for the development of targeted immunotherapy techniques for the treatment of cancer patients and consider policy challenges for development pathways and clinical implementation. Participants will be invited to discuss topics that may include

• An overview of the limitations of current preclinical models for immunotherapies and efforts to identify predictive, prognostic, or pharmacodynamic biomarkers for improved models and therapies;
• Challenges in clinical trial design and implementation, such as strategies to combine the best standard of care therapies with targeted immunotherapies or combining multiple immune therapeutic agents, expedient patient identification for inclusion in studies, modification and use of genetically altered cells, and mitigation of adverse effects for patients;
• Opportunities for greater collaboration and information exchange, including the collection, curation, distribution, and analysis of appropriate patient samples for continuing laboratory studies;
• Opportunities for greater collaboration and information exchange between bio and pharmaceutical companies, especially by expansion of precompetitive space;

• Gaps in policy surrounding the development, regulation, and clinical implementation of cell-based and gene-based immunotherapies; and
• Strategies for scaling up production of novel Food and Drug Administration–approved immunotherapies to facilitate broad patient access.

The committee will develop the agenda for the workshop sessions, select and invite speakers and discussants, and moderate the discussions. An individually authored workshop summary of the presentations and discussions at the workshop will be prepared by a designated rapporteur in accordance with institutional guidelines.