National Academies Press: OpenBook

Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk (2015)

Chapter: Appendix A: Study Approach

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Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
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Appendix A

Study Approach

STUDY PROCESS

During the course of its deliberations, the committee gathered information through a variety of mechanisms: (1) three 1.5-day workshops held in person in Washington, DC, in October 2013, February 2014, and May 2014 and one virtual workshop held in April 2014, all of which were open to the public; (2) release in January 2014 of a document presenting a framework for discussion, which invited public feedback on a set of issues relevant to this report and is described in greater detail in the section below; (3) reviews of the scientific literature and commissioning of two papers on special topics, including de-identification of clinical trial data (see Appendix B) and drug regulation in selected developing countries;1 and (4) personal communication between committee members and staff and individuals who have been directly involved in or have special knowledge of the issues under consideration.

FRAMEWORK FOR DISCUSSION AND PUBLIC FEEDBACK

As directed by the committee’s charge, a document presenting a framework for discussion (“the Framework”) was publicly released in January 2014. The Framework articulated the committee’s preliminary

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1 The commissioned paper, “The Interaction Between Open Trial Data and Drug Regulation in Selected Developing Countries,” was used by the committee in support of its analysis in this report. This paper is available on this report’s website (www.iom.edu/datasharing).

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
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observations on guiding principles that underpin the responsible sharing of clinical trial data, a nomenclature for data sharing, and a description of a selected set of data sharing activities. The Framework did not contain conclusions or recommendations but rather served to elicit feedback from a variety of stakeholders to inform the second phase of this study and the conclusions and recommendations contained in this final report. The committee invited comments on a set of specific topics for public feedback on difficult issues that were likely to be complex and on which the public and stakeholders were likely to have differing perspectives.

In addition to the public release of the Framework, several medical journals wrote editorials on the committee’s work and encouraged their readership to send comments. In response to these efforts, the committee received 85 written comments from a variety of individuals and organizations, including academic researchers from across the globe, industry (pharmaceutical, device, and biologic) representatives (from both individual companies and trade associations), clinicians and health care organizations, patient/disease advocacy representatives, and others. Staff collected and compiled all comments for the committee’s review, calling particular attention to cross-cutting themes and unique perspectives.

CLINICAL TRIALS LITERATURE REVIEW

Search Parameters:

  • Date range: 2000-present
  • International, English only

Databases:

  • OVID Medline
  • OVID Embase
  • Scopus
  • Web of Science
  • Grey literature reports (NIH, FDA, EMA [European Medicines Agency], WHO)

Email Alerts:

  • LexisNexis—major newspapers (New York Times, wall Street Journal, washington Post)
  • Alerts received on a weekly schedule

Search Strategy:

(“Clinical Trials” or “Clinical Trials as Topic” or “Clinical trial (topic)”)

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×

AND

(“Data sharing” or “Information dissemination”2 or “Data-sharing” or “Data transparency”)

AND (with the following key words searched individually)

  • “Resource constraints” or “Resource limitations”
  • “Implementation”
  • “Incentives” or “Disincentives” and “Academic”/or “Outcome research” or
  • “Changing norms”
  • “Protection of human subjects” “/or” “/”
  • “Patient privacy” or “Patient confidentiality” or Privacy
  • “Intellectual property”
  • Legal or Jurisprudence or “Legal issues” or “Legal aspects” or Law
  • “Scientific standards” or “Rogue analyses”
  • “Data quality” or “Quality control”
  • “Informed consent”
  • “Competition law” or “Antitrust”
  • “Liability”
  • “Data exclusivity”
  • “Infrastructure”
  • “Governance”
  • “Resource poor setting”
  • “Public health”
  • “Risks”
  • “Benefits”
  • “Challenges”

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2 Note that “information dissemination” is the MeSH (Medical Subject Headings) term for data sharing.

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×

COMMITTEE MEETING AGENDAS

Meeting One: October 22-23, 2013

The National Academies
2101 Constitution Avenue, NW
Washington, DC 20418

October 22, 2013 (Day 1)
CLOSED SESSION (9:30 AM-2:20 PM)

October 22, 2013 (Day 1)
OPEN SESSION (2:30 PM-4:45 PM)

OPEN Session Objectives

  • Review statement of task with sponsors
  • Receive testimony from invited speakers and the public on attributes of responsible data sharing activities

National Academy of Sciences Building, NAS 125
2101 Constitution Avenue, Washington, DC

2:30 PM Welcome and Introductory Remarks (begin open session)
Bernard Lo, Committee Chair
The Greenwall Foundation
2:40 PM The Charge to the Committee: A Discussion with the Sponsors
Objective: Receive remarks from invited sponsor representatives to discuss background, purpose, and context for the study, including needs the study could address. Provide opportunity for the committee and sponsors to clarify the study scope and task through question and answer and open discussion.
  • Kathy Hudson, U.S. National Institutes of Health
  • Richard Moscicki, U.S. Food and Drug Administration
  • Elizabeth (Betsy) Myers, Doris Duke Charitable Foundation
  • John Orloff, Novartis
  • Nicola Perrin, The wellcome Trust
4:30-4:45 PM Closing Remarks (end open session)
Bernard Lo, Committee Chair
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×

October 22, 2013 (Day 1)
CLOSED SESSION (4:45 PM-5:45 PM)

October 23, 2013 (Day 2)
OPEN SESSION (8:00 AM-5:45 PM)

8:00 AM Welcome and Introductory Remarks (begin open session)
Bernard Lo, Committee Chair
8:15-8:45 AM Clinical Trial Data and Challenges to Data Sharing
Robert Califf, Duke University (by webEx)
Discussion
8:45-9:15 AM Preparing for Responsible Sharing of Clinical Trial Data
Michelle Mello, Harvard University
Discussion

SESSION 1: CLINICAL TRIAL DATA TYPES AND SHARING ACTIVITIES

Objectives: Characterize the spectrum of “data” generated in the conduct of clinical trials and review existing and proposed data sharing activities. Explore the benefits, risks, and burdens associated with sharing different types of data.

9:15-9:45 AM Clinical Trial Data: What types of data (and associated materials) might be shared? Who holds these data? Under what circumstances are they now shared, and why are they shared? What analyses are possible using summary vs. analytic data sets vs. clinical study reports (CSRs) vs. participant-level data?
Panel discussion: Each panelist to briefly introduce himself/herself and provide 5 minutes of prepared comments, followed by a moderated discussion.
  • Pat Teden, Teden Consulting (by webEx)
  • Deborah Zarin, U.S. National Institutes of Health
Moderator: David DeMets, University of wisconsin–Madison
9:45-10:00 AM BREAK
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
10:00 AM-12:00 PM Selected Data Sharing Activities: What are the drivers and goals of proposed and existing data sharing activities? What data are shared, with whom, and how? What are some of the barriers to and risks, burdens, and benefits of data sharing, and how do different data sharing activities address these issues?
Panel discussion: 8 minutes of prep Each panelist to briefly introduce himself/herself and provide red comments, followed by a moderated discussion.
  • Adam Asare, Immune Tolerance Network Trial Share
  • Hans-Georg Eichler, European Medicines Agency
  • Charles Hugh-Jones, Project Data Sphere
  • Frank Rockhold, GlaxoSmithKline
  • Joseph Ross, Yale University Open Data Access Project
Moderator: Joanne waldstreicher, Johnson & Johnson
12:00-1:00 PM Lunch

SESSION 2: PRINCIPLES FOR RESPONSIBLE SHARING OF CLINICAL TRIAL DATA

Objectives: Explore the perspectives of those conducting, sponsoring, or participating in clinical trials and those disseminating and using clinical trial data. Identify interests, values, and concerns to consider in the development of guiding principles for sharing clinical trial data.

Panel discussion: Each panelist to briefly introduce himself/herself and provide 5 minutes of prepared comments, followed by moderated discussion.

1:00-2:20 PM Research Community Perspectives
  • Kay Dickersin, Johns Hopkins Bloomberg School of Public Health
  • Louis Fiore, vA Boston Healthcare System
  • Ben Goldacre, London School of Hygiene and Tropical Medicine (webEx)
  • Rebecca Kush, CDISC
  • Eric Perakslis, Center for Biomedical Informatics, Harvard Medical School
  • Lesley Stewart, University of York
  • Madhukar Trivedi, University of Texas, Southwestern
Moderator: Jeffrey Drazen, New England Journal of Medicine
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
2:20-2:45 PM Research Participant Perspectives
  • Deborah Collyar, Patient Advocates In Research
  • Sharon Hesterlee, Parent Project Muscular Dystrophy
Moderator: Deven McGraw, Manatt, Phelps & Phillips, LLP
2:45-3:00 PM BREAK
3:00-3:50 PM Study Sponsor Perspectives
  • Elaine Collier, National Center for Advancing Translational Sciences
  • Nicole Hamblett, Cystic Fibrosis Therapeutics Development Network-Seattle Children’s Hospital
  • Richard Kuntz, Medtronic (by webEx)
  • Justin McCarthy, Pfizer
Moderator: Arti Rai, Duke University School of Law
3:50-4:30 PM Scientific and Medical Journal Perspectives
  • Catherine DeAngelis, Journal of the American Medical Association (former)
  • Charlotte Haug, Norwegian Journal of Medicine
  • Elizabeth Loder, British Medical Journal
Moderator: Steven Goodman, Stanford University
4:30-5:30 PM Public Comment Period
5:30-5:45 PM Closing Comments and Discussion (end open session)
Bernard Lo, Committee Chair

MEETING TWO: FEBRUARY 3-4, 2014

The National Academies
Keck Center, Room 208
500 Fifth Street, NW
Washington, DC 20001

Workshop Objectives:

  • Seek public comment on the discussion framework document released in January 2014.
  • Discuss the elements and activities of data sharing outlined in the discussion framework document, and review the completeness of the set of selected models as a heuristic framework for the committee’s analytic process to be undertaken as part of the study.
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • Identify key benefits of sharing and risks of not sharing clinical trial data, and key challenges and risks of sharing clinical trial data.
  • Discuss the landscape of laws, regulations, and policies under which data sharing occurs, focusing on competition and intellectual property laws and protection of clinical trial research participants.
  • Discuss incentives for data sharing and challenges in the implementation and ongoing conduct of data sharing activities.
  • Seek public comment on potential strategies and approaches to facilitate responsible data sharing.

February 3, 2014 (Day 1)
OPEN SESSION (1:00 PM-5:00 PM)

1:00 PM Welcome and Introductory Remarks (begin open session)
Bernard Lo, Committee Chair
The Greenwall Foundation
1:05 PM Overview of the Framework for Discussion
Bernard Lo, Committee Chair

SESSION 1: CLINICAL TRIAL DATA ELEMENTS AND SHARING ACTIVITIES: PUBLIC FEEDBACK

Objectives: Identify the key purposes, benefits, risks, and challenges of each model described in the discussion framework. where relevant, explore how each model’s benefits and burdens are differentially experienced by research sponsors and investigators, study participants, regulatory agencies, patient groups, and the public. Consider whether other models of sharing might be included in the analytic framework.

Series of Panel Discussions

1:20-1:45 PM Model 1Open Access
  • Atul Butte, Stanford University School of Medicine
  • John wilbanks, Sage Bionetworks
Moderator: Ida Sim, University of California, San Francisco, School of Medicine
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
1:45-2:10 PM Model 2Controlled Access to Individual Company, Institution, or Researcher Data
  • Joe Ross, Yale University School of Medicine
  • Ira Shoulson, Georgetown University
Moderator: Steve Goodman, Stanford University School of Medicine
2:10-2:35 PM Model 3Controlled Access to Pooled or Multiple Data Sources
  • Laurie Ryan, Alzheimer’s Disease Neuroimaging Initiative
  • Jessica Scott, GlaxoSmithKline
Moderator: Steve Goodman, Stanford University School of Medicine
2:35-3:00 PM Model 4Closed Partnership/Consortium
  • Lynn D. Hudson, Critical Path Institute
Moderator: Ida Sim, University of California, San Francisco, School of Medicine
3:00-3:25 PM Moderated Discussion and Public Response: Invited panelists have the opportunity to present benefits, risks, and challenges of other models from their perspective. What, if any, changes or additions to the descriptions of the models might be considered? Are there other models substantially different from those the committee has proposed that could be included?
Moderator: Steve Goodman, Stanford University School of Medicine
3:25-3:40 PM BREAK
3:40-4:45 PM Guiding Principles for Clinical Trial Data Sharing: Invited discussants to consider the suggested guiding principles for data sharing. Discuss how the principles can be operationalized to balance the benefits and risks of data sharing.
  • Barbara Bierer, Brigham and women’s Hospital
  • Susan Bull, The Ethox Centre, University of Oxford
  • Phil Fontanarosa, JAMA
Moderator: Patricia A. King, Georgetown University Law Center
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
4:45-5:00 PM Brief Preliminary Public Comment Period
5:00 PM Closing Remarks (end open session)
Bernard Lo, Committee Chair

February 4, 2014 (Day 2)
OPEN SESSION (9:00 AM-5:30 PM)

9:00 AM Welcome and Introductory Remarks (begin open session)
Bernard Lo, Committee Chair

SESSION 2: LEGAL, REGULATORY, AND POLICY CONTExT

Objectives: Discuss the landscape of laws, regulations, and policies under which data sharing occurs, focusing on protection of clinical trial research participants and competition and intellectual property laws.

Legal, Regulatory, and Policy Context: Protection of Research Participants

9:05-9:35 AM International Legal and Policy Context
Mark Barnes, Ropes & Gray LLP and Harvard Multi-Regional Clinical Trials (MRCT) Network
9:35-10:20 AM Discussion Panel: Informed Consent: Issues and barriers for retrospective data sharing (trials already conducted or under way). Current legal framework—United States (Common Rule and the U.S. Food and Drug Administration [FDA]) and international. Suggestions to facilitate sharing while guarding principles and requirements for informed consent for prospective data sharing (trials not yet conducted or initiated). Legal and policy framework needed to facilitate prospective data sharing. Principles and elements of the consent document and process. Operational and institutional issues, especially Institutional Review Board (IRB)/Ethics Committee Review.
  • David Forster, western IRB
  • Pearl O’Rourke, Harvard University
Moderator: Elizabeth G. Nabel, Harvard Medical School, Brigham and women’s Hospital
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
10:20-10:30 AM Erika Von Mutius, University of Munich
10:30-10:45 AM BREAK
10:45-11:45 AM Discussion Panel: Privacy: Current legal framework of privacy protections—global legal/regulatory structure, with an emphasis on European Union (EU) and United States and high-level description of other non-EU/U.S. jurisdictions. How can a global infrastructure or common global approach to data sharing address or take into account disparate data privacy protection requirements and different cultural standards? Privacy risks presented by data sharing (including to patients, researchers, and institutions). Current de-identification and re-identification technology and standards. Defining “de-identified” and “anonymized” data; purposes and uses of identifiable/nonanonymized data—when/for what scientific or other purposes are identifiable data required? Fair information practices and approaches to privacy protection.
  • Mark Barnes, Ropes & Gray LLP
  • Barbara Evans, University of Houston Law School
  • Robert Gellman, Privacy and Information Policy Consultant
  • Bradley Malin, vanderbilt University
Moderator: Deven McGraw, Manatt, Phelps & Phillips, LLP
11:45 AM-12:30 PM LUNCH

SESSION 3: INCENTIVES FOR SHARING AND IMPLEMENTATION OF DATA SHARING ACTIVITIES

Objectives: Discuss how recognition and promotion structures and processes can provide incentives or disincentives to share data. Identify these incentives and norms in academia, industry, government, and other sectors as relevant. Explore potential strategies to lower disincentives or other barriers to data sharing. Discuss potential negative or unintended consequences of sharing data and explore potential strategies to mitigate these consequences or challenges.

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
12:30-1:15 PM Discussion Panel: Scientific Standards and Data Integrity/Quality: The impact of secondary analyses of data. What methods should be in place to ensure that potential consequences are balanced? Strategies to provide an understanding of how different analyses may lead to different conclusions. Approaches to address potential negative consequences and support the scientific integrity of the original and derivative works. Standards and expectations for secondary use. Provide examples where data sharing made a positive difference in understanding and where data sharing led to detrimental outcomes or analyses that did not meet scientific standards.
  • Peter Doshi, Johns Hopkins University
  • John Ioannidis, Stanford University School of Medicine
Moderator: Jeffrey Drazen, New England Journal of Medicine
1:15-2:15 PM Legal, Regulatory, and Policy Context: Intellectual Property and Competition Law: Speakers to address: Intellectual property law and patent issues, data exclusivity rules and regulatory landscape, definition of “commercially confidential information,” and antitrust considerations for data sharing.
  • Jorge Contreras, American University, washington College of Law
  • Trevor Cook, wilmerHale
  • Aliza Y. Glasner, Georgetown University
  • Benjamin Roin, Petrie-Flom Center, Harvard Law School
Moderator: Arti Rai, Duke University School of Law
2:15-3:00 PM Discussion Panel: Cultural and Financial Incentives for Data Sharing—Recognition and Promotion: Recognition and promotion norms in academia—including academic promotion/tenure structures; approaches to academic credit for clinical trialists—and their impact on incentives to share data. Industry staffing/promotion structures; cultural issues relating to data sharing.
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • Ann Bonham, Association of American Medical Colleges (AAMC)
  • Michael Rosenblatt, Merck
  • Ira Shoulson, Georgetown University
Moderator: Joanne waldstreicher, Johnson & Johnson
3:00-3:15 PM BREAK
3:15-4:00 PM Discussion Panel: Resource Considerations and Implementation Barriers: Benefits, risks, and challenges associated with having staff to answer inquiries and questions from secondary users of data. Handling of data queries/requests; allocation of responsibilities for housing data and maintaining needed records. Issues pertaining to sharing of data in settings of limited resources (e.g., developing or resource-poor countries or small companies/biotech).
  • Atul Butte, Stanford University School of Medicine
  • Matt Gross, SAS
  • Kenneth I. Moch, Chimerix
  • Janet wittes, Statistics Collaborative
Moderator: Tim Coetzee, National MS Society

SESSION 4: OVERARCHING AND CROSS-CUTTING ISSUES

Objectives: Discuss and explore the practical implications of the proposed guiding principles in light of the panel discussions held during this public workshop. Discuss selected cross-cutting questions and issues posed by the committee in the discussion framework. Suggest strategies and practical approaches to facilitate responsible data sharing.

4:00-4:45 PM Discussion Panel: Cross-Cutting Proposed Guiding Principles and Discussion Framework Questions
Panelists to discuss:
  • − Because most large clinical trials are global in nature, how can clinical trial data be shared in that global context? How can different national regulations for research participants’ privacy protections; approval of drugs and devices; data exclusivity; and intellectual property laws, resources, and health priorities be taken into account?
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • − How might strategies and approaches regarding data sharing take into account clinical trials conducted in resource-poor settings; trials designed by citizen-scientists using data they contribute directly; and trials designed through participatory research?
  • − How might different types of clinical trial data, and different uses of shared data, be prioritized for sharing? What would be the rationale for placing a higher priority on certain types of data or analyses? What might be the advantages and disadvantages of distinguishing highest-priority sharing of clinical trial data from subsequent sharing activities?
  • − What might be the advantages and disadvantages to various stakeholders of sharing different types of data sets, at different points in time after the completion of a clinical trial?
  • − Should programs or approaches calling for or requiring new data sharing apply only to new trials undertaken from the date of a new program forward, or retroactively apply to clinical trials started before the data sharing program was initiated?
  • − What might be done to minimize the risks to patients and to public health from the dissemination of findings from invalid analyses of shared clinical trial data?
  • − What measures should be deployed to minimize the privacy and confidentiality risks to trial participants? For example, are current anonymization or de-identification methodologies sufficient?
  • − Under what circumstances are identifiable data needed to fulfill articulated purposes of a data sharing activity? Under what circumstances might re-identification of trial participants be beneficial (for the participants or the public)? Have there been there examples of instances of re-identification of trial participants (e.g., for safety reasons to warn a patient of a potential risk, or for questionable and potentially unethical reasons), and what were the impacts?
  • − What incentives and protections might be established to encourage clinical trial sponsors and clinical investigators to continue to conduct clinical trials in the future, without unduly restricting the sharing of certain types of data? How do we protect or provide incentives for researchers to share data?
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • − What is the appropriate responsibility of the primary investigator(s) or research institution(s) to support secondary users in their interpretation of shared data, and what infrastructure or resources are needed to enable such ongoing support? For those with experience in data sharing, what is the burden of providing such support to help others understand and use the provided information?
  • − What would be appropriate outcome measures to assess the usefulness of different models of clinical trial data sharing, and how can they be used to guide improvements in data sharing practices?
  • Susan Bull, The Ethox Centre, University of Oxford
  • John Ioannidis, Stanford University School of Medicine
  • Ira Shoulson, Georgetown University
Moderator: Bernard Lo, Committee Chair
4:45 PM Public Comment Period
5:15 PM Closing Comments (end open session)
Bernard Lo, Committee Chair

MEETING THREE: APRIL 9, 2014
Virtual WebEx

April 7, 2014 (Day 1)
CLOSED SESSION (10:30 AM-5:00 PM)

April 8, 2014 (Day 2)
CLOSED SESSION (10:30 AM-5:00 PM)

April 9, 2014 (Day 3)
CLOSED SESSION (11:00 AM-12:00 PM)

OPEN SESSION (12:00 PM-1:30 PM)

12:00-1:30 PM Clinical Trial Data Sharing: Product Liability

Objective: Discuss the product liability litigation concerns of requiring drug and device manufacturers to make clinical trial data public.

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×

Panel discussion:Each panelist will provide an 8- to 10-minute presentation, followed by moderated discussion with the committee.

  • Loren Brown, DLA Piper
  • George Fleming, Fleming, Nolan, and Jez L.L.C.
  • william Sage, University of Texas, School of Law
Moderator: Arti Rai, Committee Member

CLOSED SESSION (1:30 PM-3:30 PM)

MEETING FOUR: MAY 5-7, 2014

Public Workshop
Keck Center, Room 100
The National Academies
500 Fifth Street, NW
Washington, DC 20001

May 5, 2014 (Day 1)
OPEN SESSION (9:30 AM-5:00 PM)

Overall Workshop Objectives:

  • Discuss the benefits, risks, and challenges of data sharing with medical product developers outside of large pharmaceutical companies, including small biotechnology/venture capital, diagnostics and other devices, and disease- and condition-specific organizations.
  • Discuss incentives and disincentives in the global clinical trial landscape, particularly within research institutions, including universities, organizations that carry out data sharing, funders, journals, and other organizations involved in clinical trials.
  • Discuss guiding principles and characteristics for the optimal infrastructure and governance for sharing clinical trial data.
9:30 AM Welcome and Introductory Remarks
Bernard Lo, Committee Chair
The Greenwall Foundation
9:40-10:00 AM Introductory Presentation: Vision for the Future of Clinical Trials: Implications for Data Sharing
Bernard Munos, M.B.A. (confirmed), InnoThink Center for Research in Biomedical Innovation
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×

SESSION 1: STRATEGIES AND PRACTICAL APPROACHES FOR RESPONSIBLE SHARING OF CLINICAL TRIAL DATA: PERSPECTIVES OF TRIAL SPONSORS AND INVESTORS

Objectives: Hear from investors and sponsors of clinical research (e.g., biotechnology, diagnostic, device, and patient-supported research trials), who will discuss the benefits, risks, and challenges of sharing clinical trial data from their perspective and how those may align with or differ from those associated with large drug trials. Identify strategies and practical approaches to overcome challenges and barriers to responsible data sharing identified by these sponsors.

10:00-11:15 AM Discussion Panel
Small Biotechnology/Venture Capital
  • Jonathan Leff (confirmed), Partner and Chairman, Deerfield Institute
Device/Diagnostic Companies
  • Rick Kuntz, M.D., M.S. (confirmed), Senior vice President and Chief Scientific, Clinical and Regulatory Officer, Medtronic, Inc.
  • Steven Gutman, M.D. (confirmed), Strategic Advisor, Myraqa
Disease- and Condition-Specific Organizations
  • Robert N. McBurney, Ph.D. (confirmed), Chief Executive Officer, Accelerated Cure Project for MS
Moderator: Sharon Terry, Committee Member
11:15 AM BREAK

SESSION 2: STRATEGIES AND PRACTICAL APPROACHES FOR INCENTIVIZING RESPONSIBLE SHARING OF CLINICAL TRIAL DATA: PERSPECTIVES OF INVESTIGATORS AND LEADERS OF ACADEMIC MEDICAL CENTERS

Objectives: Understand current norms and attitudes toward clinical trial data sharing. Identify new and current incentives that might facilitate clinical trial data sharing and practical steps within the broad clinical trial enterprise (including major research fields, international and limited-resource settings, data coordinating centers). Discuss incentives and disincentives in the global clinical trial landscape and the academic research model and strategies for overcoming disincentives.

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
11:30 AM-12:30 PM Discussion Panel: Clinical Trial Investigators and Leaders of Academic Medical Centers and Data Coordinating Centers
  • Rory Collins, Ph.D., Professor of Medicine and Epidemiology, University of Oxford and Chief Executive, UK Biobank (via webEx, May 6)
  • Steve Cummings, M.D. (confirmed), Professor Emeritus, Department of Medicine (General Internal Medicine), UCSF, and Director, San Francisco Coordinating Center
  • Clay Johnston, M.D., Ph.D. (confirmed by webEx), Dean, School of Medicine, University of Texas at Austin, former Director of UCSF Clinical and Translational Science Institute
  • Quarraisha Abdool Karim, Ph.D., M.S., Associate Professor, Columbia University and Associate Scientific Director, CAPRISA (Center for the AIDS Programme of Research in South Africa) (via webEx, May 6)
  • Paula K. Shireman, M.D. (confirmed), vice Dean for Research, University of Texas Health Science Center San Antonio
Moderator: Bernard Lo, Committee Chair
12:30 PM LUNCH

SESSION 3: STRATEGIES AND PRACTICAL APPROACHES FOR RESPONSIBLE SHARING OF CLINICAL TRIAL DATA: GOVERNANCE AND INFRASTRUCTURE

Objectives: Identify guiding principles and characteristics for the optimal infrastructure and governance for responsible sharing of clinical trial data. Discuss optimal and practical governance models that account for the global nature of clinical trials, in which relevant laws, policies and practices vary by jurisdiction.

1:30-3:00 PM Discussion Panel: Operational Principles for the Governance for Sharing Clinical Trial Data
  • Philip E. Bourne, Ph.D. (confirmed), Associate Director for Data Science (ADDS), National Institutes of Health
  • Glenn Cohen, J.D. (confirmed), Professor of Law and Co-Director, Petrie-Flom Center for Health Law Policy, Biotechnology & Bioethics
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • Jane Kaye, D.Phil., LL.B. (via webEx, May 7), Director, Centre for Law, Health and Emerging Technologies, Oxford: (HeLEX) based in the Department of Public Health at the University of Oxford
  • Bartha Knoppers, Ph.D., LL.M., LL.B. (confirmed), Director, Centre of Genomics and Policy, McGill University
Moderator: Tim Coetzee, Committee Member
3:00-4:30 PM Discussion Panel: Characteristics for the Optimal Infrastructure of Data Sharing
  • Philip E. Bourne, Ph.D. (confirmed), Associate Director, Data Science (ADDS), National Institutes of Health
  • Rory Collins, Ph.D., Professor of Medicine and Epidemiology, University of Oxford and Chief Executive, UK Biobank (via webEx, May 6)
  • Harlan Krumholz, M.D. (confirmed), Director, Yale University Open Data Access (YODA) Project
  • Frank Rockhold, Ph.D. (confirmed), GSK Sr. vice President GCSP
  • Paula K. Shireman, M.D. (confirmed), vice Dean for Research, University of Texas Health Science Center San Antonio
Moderator: Ida Sim, Committee Member
4:30-5:00 PM Public Comment Period
5:00 PM ADJOURN

May 6, 2014 (Day 2)
OPEN SESSION (9:30 AM-11:30 AM)

SESSION 4: CONTINUATION FROM MAY 5

9:30-10:15 AM Clinical Trial Investigator Perspectives
Quarraisha Abdool Karim, Ph.D., M.S.
Associate Professor, Columbia University and Associate Scientific Director, CAPRISA (Center for the AIDS Programme of Research in South Africa) (confirmed)
10:15-10:30 AM BREAK
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
10:30-11:30 AM Characteristics for the Optimal Infrastructure of Data Sharing and Incentivizing Data Sharing
Rory Collins, Ph.D., Professor of Medicine and Epidemiology, University of Oxford and Chief Executive, UK Biobank (confirmed)

May 7, 2014 (Day 3)
OPEN SESSION (9:00 AM-9:45 AM)

9:00-9:45 AM Operational Principles for the Governance for Sharing Clinical Trial Data
Jane Kaye, D.Phil., LL.B., Director, Centre for Law, Health and Emerging Technologies, Oxford: (HeLEX) based in the Department of Public Health at the University of Oxford (confirmed)

DATA SHARING PUBLIC COMMENTS AND CONTRIBUTIONS

Contributors
Name Organization
Alves, Teresa

Health Action International Europe, International Society of Drug Bulletins, Medicines in Europe Forum, and Transatlantic Consumer Dialogue

Aquino, John

Bloomberg BNA

Azoulay, Daniel

AP-HP Hôpitaux Universitaires Henri Mondor

Barnes, Mark

Harvard MRCT (Multi-Regional Clinical Trials) Center

Beckett, William

Harvard Medical School

Berger, Philip

University of Toronto, St. Michael’s Hospital

Bierut, Laura

Washington University School of Medicine in St. Louis

Brannin, Nancy L.

Clinician

Brewer, Alina

Preeclampsia Foundation

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
Name Organization
Cantekin, Erdem

University of Pittsburgh School of Medicine

Charles, H. Cecil

Duke University

Cheah, Phaik

Faculty of Tropical Medicine, Mahidol University

Davies, Gerry

PreDiCT-TB Consortium

Detmer, Don

University of Virginia School of Medicine

Dixon, Dennis

Unknown

Espeland, Mark

Wake Forest School of Medicine

Federici, Tara

AdvaMed

Feigal, Ellen

California Institute for Regenerative Medicine

Ferguson, Stephen

Cook Group, Inc.

Francer, Jeffrey

PhRMA (Pharmaceutical Research and Manufacturers of America)

Gellman, Robert

Privacy and Information Policy Consultant

Goldacre, Ben

Author

Gutierrez, Querol

Universitat Autonoma de Barcelona

Hauze, Joyce

Xogene Services

Holbrook, Anne

McMaster

Holmes, J.

Unknown

Johnson, Lorraine

Consumers United for Evidence-Based Healthcare

Jureidini, Jon

University of Adelaide

Kalamegham, Rasika

American Association for Cancer Research

Kush, Rebecca

Clinical Data Interchange Standards Consortium

Lehman, Dale

Alaska Pacific University

Levett, Paul

George Washington University

Levit, Laura

ASCO (American Society of Clinical Oncology

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
Name Organization
Li, Rebecca

Harvard MRCT

Lin, Edward

Emory University School of Medicine

Mayer, Mark

Chief Medical Officer Roundtable

McLean, Samuel

University of North Carolina School of Medicine

Miller, Pamella

New England Journal of Medicine

Murray, Jeff

University of Iowa

NA

The Cancer Imaging Archive (TCIA)

NA

Global Health Network

NA

Harvard MRCT

NA

Novo Nordisk

NA

The Radiological Society of North America (RSNA)

NA

Roche

O’Donnel, D.

Unknown

Offermann, Margaret

Federation of America Societies for Experimental Biology

O’Neill, Onera

The Wellcome Trust

Potter, Bill

U.S. National Institutes of Health

Radecki, Ryan

University of Texas Medical School

Rivas, Maria

AbbVie

Rosenblatt, Michael

Merck and Co., Inc.

Rouse, Dwight

NIH funded clinical trialist

Sanjuan, Judit Rius

Médecins Sans Frontières/Doctors Without Borders (MSF)

Scott, James

University of Utah, School of Medicine

Scott, Jessica

GSK (GlaxoSmithKline)

Shahzamani, Azin

Genentech, Inc.

Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
Name Organization
Shorish, Yasmeen

James Madison University

Shuttes, James

Unknown

Sprosen, Tim

Clinical Trial Service Unit & Epidemiological Studies Unit (CTSU), Nuffield Department of Population Health, University of Oxford

States, David

Brown Foundation Institute of Molecular Medicine & University of Texas Health Science Center at Houston

Taichman, Darren

ICMJE (International Committee of Medical Journal Editors)

Vinson, Eric

Northwest Portland Area Indian Health Board

Wang, Yajie

CSPCC: VA (Clinical Studies Program Coordination Center: U.S. Department of Veterans Affairs) VA Palo Alto Healthy Care Center

Weitzman, Stephen A.

The Wellcome Trust

Womack, Andrew W.

BIO (Biotechnology Industry Organization)

Specific Topics for Public Feedback

Global Implementation and Practical Consideration

  • Because most large clinical trials are global in nature, how can clinical trial data be shared in that global context? How can different national regulations for research participants’ privacy protections; approval of drugs and devices; data exclusivity; and intellectual property laws, resources, and health priorities be taken into account?
  • How might strategies and approaches regarding data sharing take into account clinical trials conducted in resource-poor settings, trials designed by citizen-scientists using data they contribute directly, and trials designed through participatory research?

Timing and Prioritization

  • How might different types of clinical trial data, and different uses of shared data, be prioritized for sharing? What would be the
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
×
  • rationale for placing a higher priority on certain types of data or analyses? What might be the advantages and disadvantages of distinguishing highest-priority sharing of clinical trial data from other sharing activities?

  • What might be the advantages and disadvantages to various stakeholders of sharing different types of data sets, at different points in time, after the completion of a clinical trial?
  • Should programs or approaches calling for or requiring new data sharing apply only to new trials undertaken from the date of a new program forward, or retroactively apply to clinical trials started before the data sharing program was initiated?

Mitigating Risks

  • What might be done to minimize the risks to patients and to public health from the dissemination of findings from invalid analyses of shared clinical trial data?
  • What measures should be deployed to minimize the privacy and confidentiality risks to trial participants? For example, are current anonymization or de-identification methodologies sufficient?
  • Under what circumstances are identifiable data needed to fulfill articulated purposes of a data sharing activity? Under what circumstances might re-identification of trial participants be beneficial (for the participants or the public)? Have there been there examples of instances of re-identification of trial participants (e.g., for safety reasons to warn a patient of a potential risk, or for questionable and potentially unethical reasons), and what were the impacts?

Enhancing Incentives

  • What incentives and protections might be established to encourage clinical trial sponsors and clinical investigators to continue to conduct clinical trials in the future, without unduly restricting the sharing of certain types of data? How do we protect or provide incentives for researchers to share data?
  • What is the appropriate responsibility of the primary investigator(s) or research institution(s) to support secondary users in their interpretation of shared data, and what infrastructure or resources are needed to enable such ongoing support? For those with experience in data sharing, what is the burden of providing such support to help others understand and use the provided information?

Measuring Impact

  • What would be appropriate outcome measures to assess the usefulness of different models of clinical trial data sharing, and how can they be used to guide improvements in data sharing practices?
Suggested Citation:"Appendix A: Study Approach." Institute of Medicine. 2015. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington, DC: The National Academies Press. doi: 10.17226/18998.
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Next: Appendix B: Concepts and Methods for De-identifying Clinical Trial Data »
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Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health.

Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs.

Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research--from funders, to researchers, to journals, to physicians, and ultimately, to patients.

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