Forum on Drug Discovery, Development, and Translation: 2018 Annual Report (2019)

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Action Collaboratives 1 Action collaboratives are ad hoc, participant-driven activities associated with the Forum that foster collaboration and information sharing among Forum members and external thought leaders and stakeholders. Action collaboratives engage experts with similar interests and responsibilities to analyze in-depth, high-priority issues and Action collaboratives are ad hoc, to advance the identified goals of the Forum and recommendations highlighted in participant-driven activities National Academies’ Consensus Study Reports. associated with the Forum that Advancing the Science of Patient Input in Medical Product R&D foster collaboration and There is growing momentum to incorporate patient input into medical product R&D information sharing among Forum and regulatory decision-making processes. Converting traditionally anecdotal patient members and external thought input to rigorous, credible evidence for use by a broad range of stakeholders— including academic and clinical researchers, medical product developers, patient/ leaders and stakeholders. disease advocacy groups, and regulatory decision makers—could better align medical product development and regulation with patient perspectives on disease experience, burden, management, and treatment. Many efforts have been launched to advance a science of patient input. However, there is a need to identify key gaps in the knowledge base and other barriers that impede progress and to develop an approach for addressing them. This action collaborative launched in 2017 to foster the development of a strategic research framework to advance the science of 1 Each action collaborative is an ad hoc activity associated with the Forum at the National Academies. The work each produces does not necessarily represent the views of any one organization, the Forum, or the National Academies and is not subjected to the review procedures of, nor is it a publication or product of, the National Academies. 14 

Action Collaboratives patient input. During the first phase of work, collaborative participants catalogued current efforts and progress in the science of patient input and identified gaps in the knowledge base and other barriers that impede progress. In the second phase, collaborative participants laid the groundwork for a public workshop hosted by the Forum on May 9, 2018, titled Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda. Topics raised during this discussion- based workshop will help inform the third phase, which will focus on the development of a research framework for advancing the science of patient input. Clinical Trial Site Standards Harmonization Mobilized in 2012, this action collaborative set out to explore opportunities to improve clinical trial site functioning with the goal of increasing productivity in medical product development. To date, the collaborative has drawn together a group of diverse stakeholders for four in-person meetings (December 2012, August 2013, March 2014, and September 2018). Collaborative participants published a National Academy of Medicine Discussion Paper in October 2017, which summarizes their perspectives on a proposed launching point for harmonizing the requirements applied to clinical trial sites and the development of standards. In 2018, the A working group reviews and discusses potential baseline clinical trial site standards during an action collaborative meeting in September 2018. 15 

collaborative collected, analyzed, and assessed a set of baseline site standards necessary for launching the majority of phase II, III, and IV clinical trials, and which could be considered for broad application across clinical trial sites by research sponsors. Improving the Drug Development Process Through Examining Late-Stage Failures The development of new drugs is inherently complex and costly, with sources documenting up to $2.6 billion and more than 9 years in investment to bring a new treatment to market. The high cost is driven, at least in part, by the failure rate for new compounds entering late-stage development, but also encompasses opportunity costs due to time and resources not spent on developing an alternate treatment candidate, as well as investment in clinical trial infrastructure. Most importantly, these failures have an impact on patients—those who participate in the clinical trials associated with the testing of a failed drug candidate and those who will not benefit from having access to treatments that could have been developed instead. The goal of this action collaborative is to examine the contributing factors to late-stage failures and develop a set of key considerations for stakeholders to improve the probability of success in late-stage product development. In 2018, collaborative participants conducted structured, qualitative interviews with subject-matter experts; analyzed and synthesized responses from these interviews; considered information compiled from a literature survey; and held a meeting on October 3, 2018, to discuss and inform the key considerations. In 2019, collaborative participants will continue to prepare and publish a written summary of the key considerations and points identified by this activity. 16 

Action Collaboratives Sharing Clinical Trial Data Sharing clinical trial data can help facilitate more efficient and effective development of better medicines, diagnostics, and procedures for the ultimate benefit of patients. At the same time, sharing data presents risks, burdens, and challenges that need to be addressed by a broad set of stakeholders. These opportunities and challenges were laid out in the IOM report Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk, which calls on stakeholders to foster a culture of sharing and offers a blueprint for action within and across sectors. Four National Academies forums and Action collaboratives engage roundtables, including the Forum, provided momentum and a framework for initiating experts with similar interests the consensus study that produced the report and continue to work together to support coordination and collaboration among stakeholders engaged in data-sharing and responsibilities to analyze initiatives through convening and other activities. in-depth, high-priority issues and This action collaborative, in collaboration with Brigham and Women’s Hospital to advance the identified goals of and Harvard University’s Multi-Regional Clinical Trials Center, helped inform the establishment of Vivli—a global data repository, Cloud-based analytics platform, the Forum and recommendations and in-depth search engine. The public launch of Vivli took place at the National highlighted in National Academies’ Academies on July 19, 2018. The action collaborative also worked to establish Consensus Study Reports. data-sharing goals for nonprofit funders of clinical trials. On November 30, 2017, the collaborative convened a meeting of leaders of nonprofit disease advocacy organizations, philanthropic organizations and foundations, federal government, academia, industry, and others to review a draft of the Statement of Data- Sharing Goals for Nonprofit Funders of Clinical Trials, discuss the associated risks and challenges with implementing the goals, and explore the next steps for operationalizing them. In 2019, collaborative participants will continue to work toward publication of this statement of data-sharing goals. . 17