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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. These activities 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 Academiesâ Consensus Study Reports. Clinical Trial Site Standards Harmonization35 The cost and duration of clinical trials have escalated dramatically in the past 20 years, hampering the introduction of new therapies; driving recruitment to developing countries; and raising questions about generalizability, data quality, and participant protections. While some sites in the United States are extremely efficient in launching new trials and in identifying participants, many still suffer from Action collaboratives are long delays in contracting and Institutional Review Board approvals, and enrollment ad hoc, participant-driven targets are frequently missed. The protections of research participants are variably activities associated with the applied, with the processes for consent and adherence to Good Clinical Practice suboptimal at some sites, and standards for training and site-level preparedness forum that foster collaboration are variable. The consequences are borne by NIH, foundations, and industry alike, and information sharing with the ultimate impact on the public. among forum members and This action collaborative seeks to accelerate progress in the improvement of the external thought leaders national clinical trials infrastructure. To date, the action collaborative has drawn and stakeholders. together a group of diverse stakeholders for four in-person meetings (December 2012, August 2013, March 2014, and September 2018). Action collaborative participants published a discussion paper,36 which summarized their perspectives on the concept of clinical trial site accreditation and laid out a framework and principles for developing clinical trial site standards. In addition, the action collaborative has undertaken an activity to collect, analyze, and prioritize a set of core site standards that could be considered for broad application by research sponsors. 30
Action Collaboratives Left to right: Current Forum Co-Chair, Robert Califf, former Forum Director Anne Claiborne, and former Forum Co-Chairs Russ Altman and Steven Galson during the June 2015 forum meeting. Science of Patient Input37 There is growing momentum to incorporate patient input into medical product R&D and regulatory decision-making processes. Converting traditionally anecdotal patient input to rigorous, credible evidence for use by a broad range of stakeholdersâincluding academic and clinical researchers, medical product developers, patient/disease advocacy groups, and regulatory decision makersâcould 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 critical need to identify gaps in the knowledge base and other barriers that impede progress and develop a research agenda for addressing them. This action collaborative was established to identify gaps in the knowledge base and other barriers that impede progress in advancing the science of patient input. Stakeholders came together for a meeting of the action collaborative on July 30, 2019, to discuss and prioritize gaps and barriers that should be included in a research agenda to advance the science of patient input. In 2021, action collaborative participants will work toward the publication of this activity. 35 For more information and updates on this project, see https://www.nationalacademies.org/our-work/clinical- trial-site-standards-harmonization-an-action-collaborative. 36 The discussion paper âItâs Time to Harmonize Clinical Trial Site Standardsâ (2017) published in NAM Perspectives is available at https://nam.edu/its-time-to-harmonize-clinical-trial-site-standards. 37 For more information and updates on this project, see https://www.nationalacademies.org/our-work/advancing- 31 the-science-of-patient-input-in-medical-product-rd-an-action-collaborative.
IInnovation in Drug Research and Development for Highly Prevalent Chronic Diseases38 Half of all Americans live with at least one chronic disease,39 such as heart disease, cancer, stroke, or diabetes. These and other chronic diseases are the leading cause of death and disability in the United States and are a leading driver of health care costs, with approximately 90 percent of health care expenditures related to chronic conditions.40,41 Yet, investment in these leading causes of death and disability, other than cancer, has not reflected this burden. The 2020 outbreak of coronavirus disease 2019 (COVID-19) may further exacerbate the health disparities associated with high- impact prevalent chronic diseases. The goal of this action collaborative is to examine and highlight the bottlenecks to drug R&D for highly prevalent chronic diseases and develop a set of key considerations and potential strategies for spurring innovation. In 2021, action collaborative participants will develop and distribute a structured, qualitative questionnaire to key stakeholders. Results will be synthesized and used to inform next steps for the action collaborative. 38 For more information and updates on this project, see https://www.nationalacademies.org/our-work/ innovation-in-drug-research-and-development-for-highly-prevalent-chronic-diseases-an-action-collaborative. 39 Broadly defined by the National Center for Chronic Disease Prevention and Health Promotion as âconditions that last 1 year or more and require ongoing medical attention or limit activities of daily living or both.â See https://www.cdc.gov/chronicdisease/about/index.htm. 40 See https://www.cdc.gov/chronicdisease/index.htm. 41 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/content/dam/rand/pubs/tools/TL200/TL221/RAND_TL221.pdf. 32
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