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Co-Sponsored by:

U.S. Food and Drug Administration (FDA) and
The Society for Immunotherapy of Cancer (SITC)

Location:

Walter E. Washington Convention Center
801 Mt. Vernon Place NW
Washington, DC  20001

Background:

The FDA and SITC intend to engage with leaders in academia, industry, international regulatory, and patient groups. Numerous studies have been reported in the literature in an effort to improve upon conventional response criteria for tumor measurement based efficacy endpoints for cancer immunotherapeutics as conventional response criteria may not fully capture the benefit of this product class. This workshop will evaluate current practices and identify opportunities to advance strategies that can provide rigorous tumor measurement-based endpoint data—accounting for the unique mechanism of action of cancer immunotherapeutics—suitable to support regulatory, payer, clinician, and patient decision making.

Based on greater understanding of the molecular underpinnings of cancer and the host immune response to cancer, immunotherapeutics are now demonstrating efficacy across a wide breadth of cancer types. In oncology, improvements in overall survival remains a gold standard for demonstrating the efficacy of drugs; however, tumor measurement-based endpoints, e.g., response rate and progression-free survival, facilitate assessment of direct effects of treatment on the cancer. Trials evaluating tumor assessment based endpoints can inform benefit-risk assessments of a drug earlier than a trial evaluating treatment effects on mortality. Based on a unique mechanism of action, conventional tumor response criteria do not appear to fully capture the benefit of immunotherapeutics. For example, treatment with immune checkpoint inhibitors (anti-CTLA-4 monoclonal antibody (mAb), anti-PD-1 mAb, and anti-PD-L1 mAb) may result in an atypical response pattern characterized by initial progression of disease based on appearance of new lesions or an increase in the size of existing tumors followed by either subsequent stable disease or regression of overall tumor burden. Tumor response criteria for cancer immunotherapeutics containing modifications to conventional criteria that account for this atypical pattern of response exist but pose unique challenges for use in clinical trials intended to support marketing approval. The purpose of this workshop is to provide an open public forum for a broad representation of stakeholders (1) to explore methods for analysis, presentation, and interpretation of tumor measurement-based efficacy endpoints and (2) to discuss considerations for use of immune-modified response criteria in development programs of cancer immunotherapeutics intended to support marketing approval.

Workshop Objectives:

• To provide a forum for open discussion among academia, industry, international regulatory bodies, payers, and patient groups to evaluate current methods for assessing efficacy of cancer immunotherapeutics and identify opportunities for advancing immune-modified, tumor measurement-based efficacy endpoint data to support regulatory, clinician, and patient decision making;
• To ensure the leading experts in cancer immunotherapeutics have an opportunity to evaluate international efforts to standardize immune-modified response criteria in cancer trials;
• To seek feedback from stakeholders regarding use of immune-modified response criteria in the assessment of tumor measurement-based endpoints in development programs of cancer immunotherapeutics.

Workshop Information:

Videos and slides from the FDA-SITC Workshop have been posted online and are now available on the SITC Program Materials page.

• Immediate free access to attendees of the workshop;
• Free access to SITC members after 3 months (February);
• Free access to the public after 6 months (June 1).

Contact:

FDA
Valerie Vashio: [email protected]
Oncology Program, Office of Hematology and Oncology Products

SITC
Cari Grulke: [email protected]
Meetings Coordinator