Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches: Workshop Summary (The Learning Healthcare System ... on Value & Science-Driven Health Care)

Recent scientific and technological advances have accelerated our understanding of the causes of disease development and progression, and resulted in innovative treatments and therapies. Ongoing work to elucidate the effects of individual genetic variation on patient outcomes suggests the rapid pace of discovery in the biomedical sciences will only accelerate. However, these advances belie an important and increasing shortfall between the expansion in therapy and treatment options and knowledge about how these interventions might be applied appropriately to individual patients. The impressive gains made in Americans' health over the past decades provide only a preview of what might be possible when data on treatment effects and patient outcomes are systematically captured and used to evaluate their effectiveness. Needed for progress are advances as dramatic as those experienced in biomedicine in our approach to assessing clinical effectiveness. In the emerging era of tailored treatments and rapidly evolving practice, ensuring the translation of scientific discovery into improved health outcomes requires a new approach to clinical evaluation. A paradigm that supports a continual learning process about what works best for individual patients will not only take advantage of the rigor of trials, but also incorporate other methods that might bring insights relevant to clinical care and endeavor to match the right method to the question at hand. The Institute of Medicine Roundtable on Value & Science-Driven Health Care's vision for a learning healthcare system, in which evidence is applied and generated as a natural course of care, is premised on the development of a research capacity that is structured to provide timely and accurate evidence relevant to the clinical decisions faced by patients and providers. As part of the Roundtable's Learning Healthcare System series of workshops, clinical researchers, academics, and policy makers gathered for the workshop Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches . Participants explored cutting-edge research designs and methods and discussed strategies for development of a research paradigm to better accommodate the diverse array of emerging data resources, study designs, tools, and techniques. Presentations and discussions are summarized in this volume. Recent Scientific And Technological Advances Have Accelerated Our Understanding Of The Causes Of Disease Development And Progression, And Resulted In Innovative Treatments And Therapies. Ongoing Work To Elucidate The Effects Of Individual Genetic Variation On Patient Outcomes Suggests The Rapid Pace Of Discovery In The Biomedical Sciences Will Only Accelerate. However, These Advances Belie An Important And Increasing Shortfall Between The Expansion In Therapy And Treatment Options And Knowledge About How These Interventions Might Be Applied Appropriately To Individual Patients. The Impressive Gains Made In Americans' Health Over The Past Decades Provide Only A Preview Of What Might Be Possible When Data On Treatment Effects And Patient Outcomes Are Systematically Captured And Used To Evaluate Their Effectiveness. Needed For Progress Are Advances As Dramatic As Those Experienced In Biomedicine In Our Approach To Assessing Clinical Effectiveness. A Paradigm That Supports A Continual Learning Process About What Works Best For Individual Patients Will Not Only Take Advantage Of The Rigor Of Trials, But Also Incorporate Other Methods That Might Bring Insights Relevant To Clinical Care And Endeavor To Match The Right Method To The Question At Hand. The Institute Of Medicine Roundtable On Value & Science-driven Health Care's Vision For A Learning Healthcare System, In Which Evidence Is Applied And Generated As A Natural Course Of Care, Is Premised On The Development Of A Research Capacity That Is Structured To Provide Timely And Accurate Evidence Relevant To The Clinical Decisions Faced By Patients And Providers. As Part Of The Roundtable's Learning Healthcare System Series Of Workshops, Clinical Researchers, Academics, And Policy Makers Gathered For The Workshop Redesigning The Clinical Effectiveness Research Paradigm: Innovation And Practice-based Approaches. Participants Explored Cutting-edge Research Designs And Methods And Discussed Strategies For Development Of A Research Paradigm To Better Accommodate The Diverse Array Of Emerging Data Resources, Study Designs, Tools, And Techniques. Presentations And Discussions Are Summarized In This Volume.--publisher's Description. Evidence Development For Healthcare Decisions: Improving Timeliness, Reliability, And Efficiency -- Ebm And Clinical Effectiveness Research -- Clinical Effectiveness In Research: Past, Present, And Future / Harvey V. Fineberg -- The Path To Research That Meets Evidence Needs / Carolyn M. Clancy -- Cases In Point: Learning From Experience -- Hormone Replacement Therapy / Joann E. Manson -- Drug-eluting Coronary Stents / Ashley B. Boam -- Bariatric Surgery / David R. Flum -- Antipsychotic Therapeutics / Philip S. Wang ... [et Al.] -- Cancer Screening / Peter B. Bach -- Taking Advantage Of New Tools And Techniques -- Innovative Approaches To Clinical Trials / Robert M. Califf. Distributed Data Networks / Richard Platt -- Moving To The Next Generation Of Studies -- Large Data Streams And The Power Of Numbers / Sharon-lise T. Normand -- Observational Studies / Wayne A. Ray -- Enhancing The Evidence To Improve Practice: Experimental And Hybrid Studies / A. John Rush -- Accommodating Genetic Variation As A Standard Feature Of Clinical Research / Isaac Kohane -- Phased Introduction And Payment For Interventions Under Protocol / Wade M. Aubry -- Research Networks / Eric B. Larson And Sarah Greene -- Aligning Policy With Research Opportunities -- Course-of-care Data / Greg Pawlson -- Pharmaceutical Industry Data / Peter K. Honig. Innovative Analytic Tools For Large Clinical And Administrative Databases / Sebastian Schneeweiss -- Adaptive And Bayesian Approaches To Study Design / Donald A. Berry And Frank T. Mcgraw -- Simulation And Predictive Modeling / Mark S. Roberts And David M. Eddy -- Emerging Genomic Information / Teri A. Manolio -- Organizing And Improving Data Utility -- The Electronic Health Record And Care Reengineering: Performance Improvement Redefined / Robert A. Paulus ... [et Al] -- Administrative Databases In Clinical Effectiveness Research / Alexander M. Walker -- Clinical Effectiveness Research: The Promise Of Registries / Alan J. Moskowitz And Annetine C. Gelijins. Regulatory Requirements And Data Generation / Mark B. Mcclellan -- Ensuring Optimal Use Of Data Generated By Public Investment / J. Stanford Schwartz -- Building The Research Infrastructure / Alan M. Krensky -- Engaging Consumers / Kathy Hudson, Rick E. Borchelt, And Shawna Williams -- Organizing The Research Community For Change -- Increasing Knowledge From Practice-based Research -- Opportunities For Action (panel Discussion) / Mark B. Mcclellan ... [et Al.] -- Common Themes -- Issues For Possible Roundtable Follow-up -- Appendixes -- A. Workshop Agenda -- B. Biographical Sketches Of Participants -- C. Workshop Attendee List. Leighanne Olsen And J. Michael Mcginnis, Editors ; Institute Of Medicine Of The National Academies Includes Bibliographical References. Also Available Online. Mode Of Access: World Wide Web. "Recent scientific and technological advances have accelerated our understanding of the causes of disease development and progression, and resulted in innovative treatments and therapies. Ongoing work to elucidate the effects of individual genetic variation on patient outcomes suggests the rapid pace of discovery in the biomedical sciences will only accelerate. However, these advances belie an important and increasing shortfall between the expansion in therapy and treatment options and knowledge about how these interventions might be applied appropriately to individual patients. The impressive gains made in Americans' health over the past decades provide only a preview of what might be possible when data on treatment effects and patient outcomes are systematically captured and used to evaluate their effectiveness. Needed for progress are advances as dramatic as those experienced in biomedicine in our approach to assessing clinical effectiveness. In the emerging era of tailored treatments and rapidly evolving practice, ensuring the translation of scientific discovery into improved health outcomes requires a new approach to clinical evaluation. A paradigm that supports a continual learning process about what works best for individual patients will not only take advantage of the rigor of trials, but also incorporate other methods that might bring insights relevant to clinical care and endeavor to match the right method to the question at hand. The Institute of Medicine Roundtable on Value & Science-Driven Health Care's vision for a learning healthcare system, in which evidence is applied and generated as a natural course of care, is premised on the development of a research capacity that is structured to provide timely and accurate evidence relevant to the clinical decisions faced by patients and providers. As part of the Roundtable's Learning Healthcare System series of workshops, clinical researchers, academics, and policy makers gathered for the workshop Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches. Participants explored cutting-edge research designs and methods and discussed strategies for development of a research paradigm to better accommodate the diverse array of emerging data resources, study designs, tools, and techniques. Presentations and discussions are summarized in this volume."--Publisher's description