Forum on Drug Discovery, Development, and Translation, Board on Health Sciences Policy, Institute of Medicine. "6 Suggesting an Agenda for Transforming Elements of the Clinical Trials Enterprise." Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press, 2012.
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Envisioning a Transformed Clinical Trials Enterprise in the United States
Regulators and federal agencies that support research
• Enhance collaboration in developing the learning health system among NIH, FDA, and other HHS components, with overarching attention by the HHS Secretary to clinical research;
• Make large databases accessible to researchers, with registries online;
• Place the organization of CER efforts under the purview of the HHS Secretary;
• Develop more public-private research partnerships; and
• Relax principal investigator requirement, harmonize regulations, show greater flexibility, and promulgate regulatory “safe harbors.”
Illustrative actions that could be taken by all stakeholder groups include the following suggestions made during the course of the workshop:
• strengthening the “four labs” of innovation, traditional, health care delivery, and community engagement;
• agreeing on national leadership of the CTE;
• establishing a national research agenda, to be reviewed and modified every several years;
• creating a central data repository, to merge treatment results from all providers;
• merging research organizations’ information systems on compliance and preparing standard contracts, to reduce study costs; and
• creating a standard international nomenclature, to make study results from different countries compatible.
Moving, within one decade, from relatively few decisions being evidence-based to nearly all decisions being evidence-based—and being entered into a researchable database—will require traversing a great distance. Individual workshop participants variously mentioned at least five discrete steps that could be applied to each medical treatment or intervention: (1) Results of previous experience with the treatment or intervention could be obtained (through clinical trials or observational data) for sufficient numbers of patients in order to permit valid statistical analyses of the treatment’s efficacy or effectiveness; (2) demographic, disease, and biologic factors affecting the success of the treatment could be identified; (3) a protocol could be developed for applying these research results to the care of individual patients; (4) the bulk of clinicians could use the protocol when appropriate; and (5) patients and families would understand the advantages of evidence-based care, participate in the studies necessary for medical advances, and join with clinicians in demanding clinical care that is based on medical evidence. The foundation of all these steps is the CTE.