Forum on Drug Discovery, Development, and Translation: 2022 Annual Review (2023)

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Looking Forward Forum Activities in 2023 FORUM MEMBERSHIP MEETINGS The forum membership will meet in March, July, and October 2023 to continue its discussions of key problems and strategies in the discovery, development, and translation of drugs. Forum workshop planning committees, working groups, and action collaboratives will convene to discuss and act on identified priority areas, including the following activities. WORKSHOPS Advancing Regulatory Science for Defining and Evaluating In-Home Safe Disposal Systems13 The proper disposal of unused or expired prescription drugs, particularly controlled substances, is critically important to help prevent a serious risk of abuse or overdose. The Substance Use-Disorder Prevention that Promotes Opioid Recovery and Treatment for Patients and Communities Act (SUPPORT Act)14 provided FDA the authority to require that drug manufacturers equip patients with a safe drug disposal system for certain drug products if FDA determines that such disposal systems may mitigate serious risks of abuse or overdose. There is some evidence that in-home drug disposal systems increase disposal rates, particularly when coupled with patient education. However, in-home drug disposal systems are relatively new, so there are many unanswered questions. such as to what extent people are willing to use these types of disposal options, whether the disposal options are being correctly and safely used, and how well the disposal options work to achieve public health goals. The forum will host a workshop to provide an opportunity for stakeholders to consider these and other pressing questions. Preparing the Future Workforce in Drug Research and Development In 2021, the forum hosted a public workshop, Envisioning a Transformed Clinical Trials Enterprise for 2030, which served as a venue for stakeholders from across the drug R&D life cycle to reflect on the lessons learned over the past 10 years and during the COVID-19 pandemic, and consider opportunities for the future. Stakeholders considered goals and priority action items that could advance the vision of a 2030 clinical trials enterprise that is more efficient, effective, person-centered, inclusive, and integrated into the health care delivery system so that the outcomes and experiences for all stakeholders are improved. To achieve this vision, it must be supported by a workforce that is resilient, interdisciplinary, inclusive, and culturally aware. In 2023, the forum will convene a workshop to explore the opportunities and challenges for preparing the next generation of experts in the field. ACTION COLLABORATIVES 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 to advance the identified goals of the forum and recommendations highlighted in National Academies’ Consensus Study Reports. 13 For more information, see https://www.nationalacademies.org/our-work/advancing-regulatory-science-for-defining-and- evaluating-in-home-safe-disposal-systems-a-workshop. 14 For more information, see https://www.congress.gov/115/plaws/publ271/PLAW-115publ271.pdf. 6  |  Annual Report 2022 

Advancing the Science of Patient Input in Medical Clinical Trial Site Standards Harmonization16 Product Research and Development15 This collaborative seeks to accelerate progress in the There is growing momentum to incorporate patient input improvement of the national clinical trials infrastructure. into medical product R&D and regulatory decision-making To date, the collaborative has drawn together a group processes. Converting traditionally anecdotal patient input of diverse stakeholders for four in-person meetings to rigorous, credible evidence for use by a broad range of (December 2012, August 2013, March 2014, and stakeholders—including academic and clinical researchers, September 2018). Action collaborative participants medical product developers, patient/disease advocacy published a National Academy of Medicine (NAM) groups, and regulatory decision makers—could better align Discussion Paper, “It’s Time to Harmonize Clinical medical product development and regulation with patient Trial Site Standards,”17 in which they summarized perspectives on disease experience, burden, management, their perspectives on the concept of clinical trial site and treatment. Many efforts have been launched to accreditation as well as a framework and principles advance the science of patient input. However, there is a for harmonizing site standards. A few collaborative need to identify key gaps in the knowledge base and other participants also released a New England Journal of barriers that impede progress and to develop an approach Medicine Perspective18 making the case for a voluntary for addressing them. accreditation system for clinical trial sites. This collaborative was established to identify gaps in the Starting in 2017, the collaborative undertook an activity to knowledge base and other barriers that impede progress in collect, analyze, and prioritize a set of core site standards advancing the science of patient input. Stakeholders came that could be considered for broad application by research together for a meeting of the collaborative on July 30, sponsors. In 2019, a working group of the collaborative 2019, to discuss and prioritize gaps and barriers that should produced an internal document, which summarizes the be included in a research agenda to advance the science results of this activity and outlines a set of core harmonized of patient input. This collaborative laid the groundwork for a clinical trial site standards. research agenda to advance the science of patient input by hosting discussions to: In 2023, collaborative participants will work toward publication of this work. • Catalogue current efforts and progress to advance the science of patient input; • Identify critical gaps in the knowledge base and other barriers that are impeding progress; and • Prioritize gaps and barriers that should be addressed in a research agenda to advance the field. In 2023, collaborative participants will work toward publication of this work. 15 For more information, see https://www.nationalacademies.org/our-work/advancing-the-science-of-patient-input-in-medical-product-rd-an- action-collaborative. 16 For more information, see https://www.nationalacademies.org/our-work/clinical-trial-site-standards-harmonization-an-action-collaborative. 17 For the full-text article, see https://nam.edu/its-time-to-harmonize-clinical-trial-site-standards. 18 For the full-text article “Voluntary Site Accreditation—Improving the Execution of Multicenter Clinical Trials,” see https://www.nejm.org/doi/full/10.1056/NEJMp1709203. Annual Report 2022  |  7 

Innovation in Drug Research and Development Building Community Practices into a Clinical Trials for Highly Prevalent Chronic Diseases19 Network: Listening Sessions23 Half of all Americans live with at least one chronic The COVID-19 pandemic has highlighted historical and disease,20 such as heart disease, cancer, stroke, or ongoing problems with the U.S. clinical trials enterprise and diabetes. These and other chronic diseases are the leading underscored the importance of partnering with stakeholders cause of death and disability in the United States and a in community-based settings, particularly those that have leading driver of health care costs.21,22 Yet, investment in had limited opportunities to participate, contribute, and these diseases, other than cancer, has not reflected this benefit from clinical research. Stakeholders have called for burden. The 2020 outbreak of COVID-19 may further a national clinical trials network in community practices exacerbate the health disparities associated with high- that can generate actionable evidence for drug R&D and impact prevalent chronic diseases. improving patient care. The goal of this action collaborative is to examine and Over the coming year, collaborative participants will hold a highlight the bottlenecks to drug R&D for highly prevalent series of listening sessions around the United States to better chronic diseases and develop a set of key considerations understand the opportunities and challenges for community- and potential strategies for addressing the scientific, based practices, particularly those serving under-served regulatory, and economic barriers to innovation in populations, to engage in the clinical trials enterprise. drug R&D for prevalent chronic diseases and lay out The intent of these sessions will be to make meaningful actionable solutions. connections and build relationships with diverse community- based practices. Listening sessions are not intended for forum members to offer fixes, suggestions, or advice. 19 For more information, see https://www.nationalacademies.org/our-work/innovation-in-drug-research-and-development-for-highly-prevalent- chronic-diseases-an-action-collaborative. 20 Broadly defined by the National Center for Chronic Disease Prevention and Health Promotion as “conditions that last 1 year or more and require ongoing medical attention or limit activities of daily living or both.” See https://www.cdc.gov/chronicdisease/about/index.htm. 21 See https://www.cdc.gov/chronicdisease/index.htm. 22 Buttorff, C., T. Ruder, and M. Bauman. 2017. Multiple Chronic Conditions in the United States. Santa Monica, CA: RAND Corporation. See https://www.rand.org/pubs/tools/TL221.html. 23 For more information, see https://www.nationalacademies.org/our-work/building-community-practices-into-a-clinical-trials-network-listening- sessions-an-action-collaborative. 8  |  Annual Report 2022