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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Appendix A

Workshop Agenda

LARGE SIMPLE TRIALS AND KNOWLEDGE
GENERATION IN A LEARNING HEALTH SYSTEM

An Institute of Medicine Workshop

November 26–27, 2012

Room 100
Keck Center
500 Fifth Street NW, Washington, DC

Roundtable on Value & Science-Driven Health Care
Forum on Drug Discovery, Development, and Translation

Meeting Objectives

1.   Explore accelerating the use of large simple trials (LSTs) to improve the speed and practicality of knowledge generation for medical decision making and medical product development;

2.   Consider the concepts of LST design, examples of successful LSTs, the relative advantages of LSTs, and the infrastructure needed to build LST capacity as a routine function of care;

3.   Identify structural, cultural, and regulatory barriers hindering the development of an enhanced LST capacity and discuss needs and strategies in building public demand for, and participation in, LSTs; and

4.   Suggest near-term strategies for accelerating progress in the uptake of LSTs in the United States.

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

Monday, November 26

1:00 pm

Welcome, Introductions, and Overview

Welcome, framing of the meeting, and agenda overview

o   Michael McGinnis (Institute of Medicine)

o   David L. DeMets (Planning Committee Co-Chair, University of Wisconsin School of Public Health)

o   Richard E. Kuntz (Planning Committee Co-Chair, Medtronic)

 
1:15 pm

Introduction to Large Simple Trials

Session chair: David L. DeMets (Planning Committee

Co-Chair, University of Wisconsin School of Public Health)

image   Session Objectives:

image   Set the vision for large simple trials (LSTs) as part of a learning health care system.

image   Discuss the advantages of LSTs over current trial approaches.

image   Discuss opportunities for LSTs as way to embed trials in growing digital infrastructure.

image   Presentations:

image   A vision for LSTs in the learning health system Michael S. Lauer (National Heart, Lung, and Blood Institute)

image   Opportunities and challenges for LSTs Ralph I. Horwitz (GlaxoSmithKline)

image   Session Questions:

1.   What is an LST?

2.   How would these trials fit into the larger clinical research ecosystem in a learning health care system?

3.   What need would this approach to clinical trials fill? (randomized controlled trial [RCT] cost, efficiency, generalizability)

4.   What are the advantages/disadvantages to this approach? (heterogeneity, subgroup analysis)

5.   How does the increased adoption of electronic health records (EHRs) provide an opportunity for LSTs?

6.   Are there modifications to the current design and conduct of LSTs that would enhance their value to a learning health system?

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

7.   What are some exampleas of the areas still in need of work to realize this vision (e.g., the culture shift needed to adopt potentially disruptive technologies)?

Q&A and Open Discussion

 
1:55 pm

Highlighted Examples of LSTs

Session chair: James B. Young (Cleveland Clinic)

image   Session Objectives:

image   Highlight four examples of LSTs that each exemplify a different defining characteristic of LSTs.

image   Emphasize trade-offs in trial design by discussing the pros and cons, giving examples of how these play out, and suggesting alternative approaches.

image   Foreshadow the rest of the workshop by asking LST example speakers to address their experiences (successes and failures) with stakeholder engagement, infrastructure, and policy.

image   Presentations:

image   Very large, population-based trial with broad inclusion criteria, high cost-efficiency, and hybrid design (mail-based plus in-clinic component)

image   VITamin D and omegA 3 triaL (VITAL) JoAnn E. Manson (Harvard University)

image   Trial assessing role of waiving medication copayments for improving drug adherence and health outcomes, collaboration with health insurance company (Aetna)

image   Post-Myocardial Infarction Free Rx Event and Economic Evaluation (MI FREEE) trial Niteesh K. Choudhry (Brigham and Women’s Hospital)

image   Cluster randomized trial involving pediatric practices, utilization of EHR and decision support tools for obesity interventions

image   High Five for Kids Trial/Study of Technology to Accelerate Research (STAR) Elsie M. Taveras (Harvard Pilgrim Health Care Institute)

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
 

image   Industry trial for regulatory approval with global component

image   Heart Outcomes Prevention Evaluation (HOPE) trial
P. J. Devereaux (McMaster University)

 

image   Session Questions:

1.   Please give a very brief introduction on the specifics of the trial and why it is considered an LST.

2.   How does the trial address the issues of the generalizability of the evidence produced, simplification of research processes, and cost-effectiveness?

3.   In retrospect, what were the risks and trade-offs associated with the choice of an LST design (e.g., the risk of not collecting data that could be subsequently requested)? Please discuss the pros and cons, giving examples of how these play out and suggesting alternative approaches and any design changes that you would make on the basis of the lessons learned.

4.   What were your team’s experiences (successes and failures) with the following issues, which will be discussed in further detail during the course of the workshop:

a.   Stakeholder (health system leader, clinician, patient) engagement

b.   Infrastructure (research infrastructure, health information technology)

c.   Policy (privacy, consent, institutional review board issues, regulatory issues)

Q&A and Open Discussion

 
3:15 pm Break
 
3:30 pm Partners Perspectives on LST Uptake

Session chair: Joe V. Selby (Patient-Centered Outcomes Research Institute)

image   Session Objectives:

image   Identify stakeholders relevant to the increased use of LSTs, focusing on patients, clinicians/health care systems, and payers, and the incentives that they face that could impede or advance uptake.

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

image   Engage in the issues of the most importance to stakeholders and deliberate on what it will take from each of their respective points of view.

image   Presentations:

image   Patient perspective
Nancy Roach (Fight Colorectal Cancer)

image   Health systems/clinician perspective
Alan S. Go (Kaiser Permanente)

image   Payer perspective
Lewis G. Sandy (UnitedHealth Group)

image   Session Questions:

1.   What are the top three issues for patients/clinicians/payers in considering the use of an LST approach to generate clinical evidence?

2.   What are the top three considerations for patients and clinicians in contemplating the greater integration of trials into routine care settings?

3.   What are the top three priorities for raising the awareness and participation of patients and clinicians in trials integrated into routine care?

4.   What are your priorities regarding the types of evidence that can be generated through LSTs?

5.   What are the roles for health systems and payers in (a) setting priorities, (b) dedicating staff support, and (c) providing funding for LSTs in routine care settings?

Q&A and Open Discussion

 
4:30 pm Summary and Preview of Next Day
 
5:00 pm Adjourn
 
Tuesday, November 27
 
8:00 am Coffee and light breakfast available
 
8:30 am Welcome, Brief Agenda Overview

Welcome, framing of the meeting, and agenda overview

image   David L. DeMets (Planning Committee Co-Chair, University of Wisconsin School of Public Health)

image   Richard E. Kuntz (Planning Committee Co-Chair, Medtronic)

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
 
8:45 am Infrastructure Needs

Session chair: John J. Orloff (Novartis)

image   Session Objectives:

image   Highlight infrastructure needs and barriers to the greater performance of LSTs.

image   Discuss the needs and potential approaches to merge the goals of the health care system with research, focusing on the current state and future potential of the use of EHRs as platform for LSTs.

image   Discuss establishment and sustainability of trial networks as an infrastructure to host and facilitate LSTs.

image   Presentations:

image   Aligning care and research to reduce burdens and improve integration
Richard Platt (Harvard Pilgrim Health Care Institute)

image   Point-of-care trials using EHR platforms
Ryan E. Ferguson (VA Boston Healthcare System)

image   Getting to comparable, computable data
Rebecca Daniels Kush (Clinical Data Interchange Standards Consortium)

image   Building reusable research networks
Carole M. Lannon (Cincinnati Children’s Hospital Center)

image   Session Questions:

1.   What are the current infrastructure needs for the more widespread performance of LSTs? Would you consider conducting LSTs on your network?

2.   What opportunities and challenges in the use of EHRs as a platform for LSTs currently exist? What are the priorities for change to maximize this potential going forward? How can disruption to the delivery of health care be minimized to incentivize more practicing physicians to engage in knowledge generation?

3.   What is the current state of the use of routinely collected clinical data for trials? What role will data standards play in the facilitation of LSTs? What are the priorities for change to maximize this potential going forward?

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

4.   What is the current state of reusable research networks in the United States? What is their role in LSTs? What are the major opportunities and barriers to the reusable network approach? Are there alternative community-based settings with lower infrastructure costs and greater access to patients that should be considered? Are existing research networks (including perhaps CTSA institutions or PBRNs) fit for purpose? What business models (e.g., hub and spoke) would be the most effective?

Q&A and Open Discussion

 
10:45 am Break
 
11:00 am

Policy Needs: Ethics, Trial Processes
Session chair: Robert M. Califf (Duke University)

image   Session Objectives:

image   Spotlight and differentiate real and perceived policy barriers to the greater use of LSTs.

image   Highlight examples of ways in which these have been dealt with (or overcome).

image   Anticipate potential policy issues as trials move to leverage electronic systems.

image   Suggest components of a policy framework that would facilitate LSTs.

image   Presentations:

image   Policy overview
Robert M. Califf (Duke University)

image   Ethical issues of bringing research and care closer together
Ruth R. Faden (Johns Hopkins University)

image   Trial process challenges (privacy, institutional review boards)
Deven McGraw (Center for Democracy and Technology)

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

image   Session Questions:

1.   What are the major policy barriers to the more widespread performance of LSTs? How have these barriers been overcome in the past? What are the priorities for change going forward?

2.   What are the important ethical issues to consider in bringing research and care closer together? What are the components of a new ethical framework to support a learning health system?

3.   What are the major privacy and human subjects research policy-associated considerations for LSTs? How have these challenges been overcome? What are the priorities for change going forward?

4.   What are the relevant ethical and policy considerations associated with randomization without additional consent in situations of equipoise?

Q&A and Open Discussion

 
12:00 pm

Lunch Keynote

Randomized Evaluations of Accepted Choices in Treatment (REACT) trials

Tjeerd-Pieter van Staa (Clinical Practice Research Datalink, United Kingdom)

image   Session Questions:

1.   What are the REACT trials? What was the impetus for these trials? How do they compare to LSTs?

2.   What are the stakeholder engagement-related challenges you have faced in setting up/running these trials? How have the relevant stakeholder groups responded?

3.   What are the infrastructure-related challenges and opportunities you have faced? What role has the level of EHR adoption placed in facilitating or inhibiting them? What are the most crucial non-information technology infrastructure resources?

4.   How have you addressed concerns about the accuracy and validity of data in the electronic medical record?

5.   What are the policy-related challenges that you have faced? What are the differences between the UK and

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

      U.S. systems that have facilitated or impeded these challenges?

6.   What lessons learned or best practices would you pass along to LST investigators? What would you do differently?

Q&A and Open Discussion

 
1:00 pm

Policy Needs: Medical Product Regulatory Issues
Session chair: Richard E. Kuntz (Planning Committee Co-Chair, Medtronic)

image   Presentations:

image   Trial complexity
Kenneth A. Getz (Tufts University)

image   Simplifying clinical trials
Christopher B. Granger (Duke University)

image   FDA perspective
Rachel E. Sherman (Center for Drug Evaluation and Research, U.S. Food and Drug Administration)

image   Session Questions:

1.   In general, what is the optimal role of LSTs in the medical products regulatory approval pathway? Are there areas of medical product development in which LSTs are not useful?

2.   How can an understanding of those policy/regulatory issues that drive complexity in traditional RCTs and the strategies to counteract them be applied to the adoption and use of LSTs in medical products regulatory contexts?

3.   What are the real and perceived regulatory barriers hindering the development of an enhanced LST capacity?

4.   What are some near-term strategies for accelerating progress in the uptake of LSTs in the United States?

5.   What is the current thinking from the U.S. Food and Drug Administration in terms of how and when LSTs might be used without jeopardizing the medical products development process?

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

Q&A and Open Discussion

 
2:00 pm

Break

 
2:15 pm

Strategies Going Forward
Session chair: David L. DeMets (Planning Committee
Co-Chair, University of Wisconsin School of Public Health)

image   Session Objectives:

image   Identify and discuss issues and key themes from the workshop.

image   Consider strategies and priorities for accelerating progress in the uptake of LSTs in the United States.

image   Brief Summaries and Key Stakeholder Perspectives from the Workshop:

image   Representatives from key stakeholders groups will provide an overview of key themes and issues identified from their perspectives.

image   Federal funders—Michael S. Lauer (National Heart, Lung, and Blood Institute)

image   Nongovernmental funders—Robert E. Ratner (American Diabetes Association)

image   U.S. Food and Drug Administration—Bram Zuckerman (Center for Devices and Radiological Health)

image   Centers for Medicare & Medicaid Services (CMS)—Rosemarie Hakim

image   Private payers—William H. Crown (Optum)

image   Industry—Peter Held (AstraZeneca)

image   Patients—Kate Ryan (National Women’s Health Network)

image   Clinical researchers—Elizabeth A. Chrischilles (University of Iowa College of Public Health)

image   Panel Questions:

1.   What are the themes of today’s presentations and discussions that have resonated most strongly with you?

2.   Where do you see the most opportunity for the application of LSTs? What do you see to be the biggest barriers?

Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

3.   What will it take to seize these opportunities and overcome the barriers?

4.   Based on the presentations and discussions, can you identify issues that need to be resolved by others before progress can be made? For example, as the lead of the Ethics and Processes section, can you identify critical needs in infrastructure or regulatory issues that need to be resolved before you can achieve your goals?

5.   If you were granted one wish to move LSTs forward, what would that wish be?

Q&A and Open Discussion

 
4:15 pm

Next Steps

image   Session Description: The workshop will conclude with a brief discussion and summary of next steps.

 
5:00 pm Adjourn
Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Suggested Citation:"Appendix A: Workshop Agenda." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×
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Randomized clinical trials (RCTs) are often referred to as the "gold standard" of clinical research. However, in its current state, the U.S. clinical trials enterprise faces substantial challenges to the efficient and effective conduct of research. Streamlined approaches to RCTs, such as large simple trials (LSTs), may provide opportunities for progress on these challenges. Clinical trials support the development of new medical products and the evaluation of existing products by generating knowledge about safety and efficacy in pre- and post-marketing settings and serve to inform medical decision making and medical product development. Although well-designed and -implemented clinical trials can provide robust evidence, a gap exists between the evidence needs of a continuously learning health system, in which all medical decisions are based on the best available evidence, and the reality, in which the generation of timely and practical evidence faces significant barriers.

Large Simple Trials and Knowledge Generation in a Learning Health System is the summary of a workshop convened by the Institute of Medicine's Roundtable on Value & Science-Driven Health Care and the Forum on Drug Discovery, Development, and Translation. Experts from a wide range of disciplines--including health information technology, research funding, clinical research methods, statistics, patients, product development, medical product regulation, and clinical outcomes research--met to marshal a better understanding of the issues, options, and approaches to accelerating the use of LSTs. This publication summarizes discussions on the potential of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development, including efficacy and effectiveness assessments, in a continuously learning health system.

Large Simple Trials and Knowledge Generation in a Learning Health System explores acceleration of the use of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development; considers the concepts of LST design, examples of successful LSTs, the relative advantages of LSTs, and the infrastructure needed to build LST capacity as a routine function of care; identifies structural, cultural, and regulatory barriers hindering the development of an enhanced LST capacity; discusses needs and strategies in building public demand for and participation in LSTs; and considers near-term strategies for accelerating progress in the uptake of LSTs in the United States.

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