MEMORANDUM November 11, 2020 To: Hannah Dueck National Cancer Institute (NCI) From: Carly S. Cox Science and Technology Policy Institute (STPI) Through: Bill Brykczynski Acting Director, STPI CC: Amana Abdurrezak and Brian Zuckerman STPI Subject: Expert Panel Report of the Cancer Systems Biology Consortium Program Evaluation The National Cancer Institute (NCI) Division of Cancer Biology (DCB) asked the Science and Technology Policy Institute (STPI) to facilitate an expert panel to evaluate the Cancer Systems Biolgoy Consortium (CSBC) ahead of its 2020 renewal request. To fulfill this request, STPI selected panelists, issued invitations, convened the panel for meetings to discuss program evaluation materials, facilitated a meeting between CSBC investigators and panelists, and supported the panel in its deliberations. Based on the NCI provided program information, conversations with CSBC resources, and information gathered at the CSBC Annual Meeting, the panel formulated answers to the five program evaluation questions charged to them by NCI. STPI summarized the panel’s answers to these questions in a draft document and completed a round of revisions with the panel and NCI. A finalized summary of the panel’s response to the program evaluation questions is provided in the attached document. ReleasedAttachment: “Expert Panel Report of the CancerMarch Systems Biology Consortium 2021 Program Evaluation” 1 Expert Panel Report of the Cancer Systems Biology Consortium Program Evaluation Edison Liu, Chair The Jackson Laboratories Kenneth Buetow Arizona State University Teresa Przytycka NIH National Center for Biotechnology Information John Quackenbush Harvard T.H. Chan School of Public Health Cynthia Reinhart-King Vanderbilt University October 2020 This document is under review and is subject to modification or withdrawal. It should not be cited in other publications. Released March 2021 1 Released March 2021 Contents A. Introduction .........................................................................................................1 B. Summary of Evaluation Questions ......................................................................2 C. Panel Responses to Evaluation Questions ...........................................................4 1. Question 1: Should NCI continue to support a dedicated program in cancer systems biology? ...........................................................................................4 Panel Identified Sub-Question - What is the expectation or impact of a dedicated program for systems biology research? ........................................4 2. Question 2: What impact is CSBC having on the field of cancer biology? ..5 3. Question 3: What is the role of CSBC in supporting a strong and stable research community? .....................................................................................8 4. Question 4: Are the current funding mechanisms (U24, U54, and U01) appropriate to achieve the program goals? ....................................................8 5. Question 5: Are there additional training and outreach activities that should be pursued to achieve program goals? ........................................................10 Appendix A. Expert Panel Biographies .......................................................................... A-1 Abbreviations ...................................................................................................................B-1 Released March 2021 iii A. Introduction The Cancer Systems Biology Consortium (CSBC) is a network of research institutions and principal investigators working to “advance the mechanistic understanding of cancer using systems biology approaches that build and test predictive models of disease initiation, progression, and response to treatment.”1 CSBC was launched in September of 2016 and supports the following: • 17 Individual U01 Research Projects • 13 U54 CSBC Research Centers • 1 U24 Coordinating Center In support of the 2020 CSBC renewal request, The National Cancer Institute (NCI) requested an independent analysis of the program. NCI chose to convene an expert panel to provide insight into the CSBC program evaluation. As part of the process, NCI charged the panel with five questions: 1. Should NCI continue to support a dedicated program in cancer systems biology? 2. What impact is CSBC having on the field of cancer biology? 3. What is the role of CSBC in supporting a strong and stable research community? 4. Are the current funding mechanisms (U24, U54, and U01) appropriate to achieve the program goals? 5. Are there additional training and outreach activities that should be pursued to achieve program goals? The panel identified an additional question to answer as a component of Question 1: What should a dedicated systems biology program look like moving forward? NCI asked the Science and Technology Policy Institute (STPI) to facilitate the panel and its evaluation process. To fulfill this request, STPI selected panelists, issued invitations, convened the panel for meetings to discuss program evaluation materials, facilitated a meeting between CSBC investigators and panelists, and supported the panel in its deliberations. The panel is composed of five members with backgrounds in a mixture of cancer and systems biology fields. Short biographies of the panelists are provided in Appendix A. NCI provided the panel with summary CSBC program information, but not individual grant applications or review documents. To collect information important to their charge, Releasedthe panelists met and interacted with CSBC investigatorsMarch during a virtual Investigator 2021 Panel 1 Dueck, H. and Hughes, S. Communication to Panel. June 22, 2020. 1 on August 31, 2020 and during the online CSBC Annual Meeting on September 16–17, 2020. During the Annual Meeting, panelists attended sessions presenting CSBC funded research and showcasing training and education initiatives at U54 centers. The panel met during the latter part of the Annual Meeting on September 17, 2020 to develop answers to the evaluation questions. A summary of the observed strengths of the Consortium and recommendations for the program in the future is provided below. B. Summary of Evaluation Questions 1. Question 1: Should NCI continue to support a dedicated program in cancer systems biology? Panel Identified Sub-Question: What is the expectation or impact of a dedicated program for systems biology research? The panel recognizes the success of the CSBC program given its short period of operations. The panel agrees that the CSBC fosters a productive research community that is actively generating important data, resources, and computational tools that are of great value to the field. The panel also considers the CSBC to have enormous potential to produce clinically relevant models and data to inform cancer treatment and prevention. To date, however, its optimal impact has not yet been realized. In the next iteration of the CSBC, the panel recommends increased collaboration and outreach with clinical and biological researchers to translate CSBC resources into human impact. This will require specific mechanisms to encourage formal interactions toward concrete translational goals, but done in a way that will not dilute efforts to expand fundamental knowledge of the disease. The NCI should consider opportunities to expand the Consortium, bringing in new ideas and new innovators. 2. Question 2: What impact is CSBC having on the field of cancer biology? The panel recognizes that the CSBC has created a productive platform for the cancer systems biology community to make collective action that is having significant impact in the field. The panel agreed that the CSBC is producing high-impact publications, tools, and resources that otherwise would not have been generated. The panel observed, however, that the interoperability and usability of these data and tools are suboptimal. Therefore, the panel recommends that the U54 centers and especially the U24 coordinating hub develop data and tool standards for harmonizing and integrating data produced by CSBC. Despite its relatively short lifespan, the panel also recognizes the potential of future CSBC research to have clinical and translational applications. As such, the panel recommends the next iteration of the U54 Request for Application (RFA) and U01 Program Announcement with Receipt/Referral and/or Review (PAR) more effectively implement Releasedthe incorporation of experimentation into Marchcycles of computational modeling 2021 with the explicit goal of developing predictive disease models. 2 3. Question 3: What is the role of CSBC in supporting a strong and stable research community? The panel observed that the CSBC is generating a stable research community that is making great strides to train future generations of cancer systems biology researchers. However, the panel considered interconnectedness to be a critical component of a strong research community, which they did not observe to be sufficiently present across the Consortium. The panel suggested that the U24 Collaboration and Pilot Projects Hub focus on a defined set of research questions that could be used to promote collaboration within the Consortium. Additional competitive funding mechanisms were suggested to engage systems biology researchers outside of the Consortium and to expand the CSBC community. 4. Question 4: Are the current funding mechanisms (U24, U54, and U01) appropriate to achieve the program goals? The panel finds that the U54 CSBC Research Centers