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Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
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A

Data Sources and Methods

The Institute of Medicine (IOM) Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee (RAC) was tasked with evaluating the necessity for the additional oversight of individual gene therapy protocols by the RAC. The specific goals of this review were to specify the scientific, safety, and ethical concerns that may justify a special level of oversight for this and potentially other areas and determine whether gene transfer research raises issues of concern that warrant extra oversight by the RAC, considering the current regulatory context. The IOM committee was instructed that if it concluded that this particular function of the RAC should remain intact, it should then describe the criteria that the RAC should use to select protocols for public review. The IOM committee reviewed information collected from a variety of sources, including scientific literature, previous evaluations and progress reports, open-session meetings and conference calls, public testimony and input, and other publicly available resources.

COMMITTEE EXPERTISE

To complete its task, the IOM formed a committee of 11 experts to conduct an 8-month study to respond to the statement of task. The committee was composed of members with expertise in clinical medicine, molecular biology, virology, molecular genetics, high-risk clinical trials, gene transfer technologies, biomedical ethics, law, public policy, and patient advocacy. Appendix C provides biographical information for each committee member.

Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
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MEETINGS AND INFORMATION GATHERING ACTIVITIES

The committee deliberated from June 2013 through October 2013 to conduct this expert assessment. During this period, the committee held three 2-day meetings, and committee members also participated in multiple conference calls. Two of the committee meetings were open-session, which allowed committee members to hear input from a wide range of stakeholders and members of the public. Experts in the field of clinical gene transfer research shared their perspectives on the role of the RAC, how and why some protocols are chosen for individual protocol review, the degree of harmonization among oversight bodies, the oversight of gene transfer compared with other cutting-edge areas of science, and the importance of public education and trust in the oversight process. Experts in the field of financial and scientific investment in gene transfer research presented their perspectives on whether individual protocol review by the RAC brings expertise and transparency to the oversight process and whether additional oversight has a significant impact on financial investment in potential gene transfer products. Investors also compared gene transfer oversight in the United States to that in Europe. Patient advocacy groups shared their perspectives on acceptable risk and oversight of clinical gene transfer research.

Each open-session meeting included a public comment period in which the committee invited input from any interested parties. All open-session meetings were held in Washington, DC. A conference call number and online public input tool were provided to allow opportunity for input from individuals unable to travel to the meetings. A link to the public input tool was made available on the National Academies’ website from April 2013 through October 2013, and all online input was catalogued in the study’s public access file. All information provided to the committee from outside sources or through the online input tool is available by request through the National Academies’ Public Access Records Office. The agendas for the two open-session committee meetings are reproduced below.

Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×

MEETING ONE

Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee

June 4, 2013
Keck Center, Room 109
500 Fifth Street, NW
Washington, DC

1:00 p.m. Welcome and Introductory Remarks
Larry Gostin, J.D., Committee Chair
 
SESSION 1:
THE STATE OF GENE TRANSFER RESEARCH OVERSIGHT
 
1:10 p.m. The Charge to the Committee: A Discussion with the Sponsor
Kathy Hudson, Ph.D.
Deputy Director for Science, Outreach, and Policy Office of the Director, National Institutes of Health
 
2:10 p.m. The RAC Process
Jacqueline Corrigan-Curay, J.D., M.D.
Acting Director, Office of Biotechnology Activities, National Institutes of Health
 
2:40 p.m. Committee Discussion
 
3:00 p.m. Presentation—The Role of the FDA in Gene Therapy Products
Daniel Takefman, Ph.D.
Chief, Gene Therapy Branch, Division of Cellular and Gene Therapies, Office of Cellular, Tissue, and Gene Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration
 

3:30 p.m.

Committee Discussion
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
 
3:45 p.m. Break
 
4:00 p.m. Presentation—Perspectives on RAC and Gene Therapy
Xandra Breakefield, Ph.D.
Former President, American Society for Gene and Cellular Therapy Professor of Neurology, Harvard Medical School Geneticist, Department of Neurology and Radiology, Massachusetts General Hospital
 
4:20 p.m. Committee Discussion
 
4:45 p.m. Presentation—Patient Perspective
Edward R. B. McCabe, M.D., Ph.D.
Senior Vice President and Chief Medical Officer, Office of Medicine and Health Promotion, March of Dimes
 
5:00 p.m. Follow-Up Discussion with Sponsor
 
5:15 p.m. Public Comment Period
 
5:30 p.m. Closing Remarks
Larry Gostin, J.D., Committee Chair

MEETING TWO

Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee

August 6, 2013
Keck Center, Room 100
500 Fifth Street, NW
Washington, DC

9:00 a.m. Welcome and Introductory Remarks
Larry Gostin, J.D., Committee Chair
 
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×

SESSION 2:
FINANCIAL AND SCIENTIFIC INVESTMENT

 
9:10 a.m. Panel Discussion
Session Objectives: Understand the current state of regulation of gene transfer research and compare its regulatory landscape to other areas of science. Discuss any models that exist for gene transfer oversight, including those that add value or may no longer be necessary. Explore the investigator experience of individual gene transfer protocol review by the RAC.
 
  Moderator: Howard Federoff, M.D., Ph.D.
Committee Member
 
  Panelists: Barry Byrne, M.D., Ph.D.
Director of the University of Florida Powell Gene Therapy Center
Professor of Pediatrics, Molecular Genetics, and Microbiology
Associate Chair of Pediatrics
University of Florida
 
    Helen Heslop, M.D.
Professor in the Department of Medicine
Director of Adult Stem Cell Transplant Program
Baylor College of Medicine
 
    Elizabeth Hohmann, M.D.
Chair and Director of Partners Human Research Committee
Partners Healthcare
 
    Carl June, M.D.
Richard W. Vague Professor in Immunotherapy
Director of the Translational Research Program
University of Pennsylvania
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
    Margaret Riley, J.D.
Professor of Law
Professor of Medicine
University of Virginia
 
10:30 a.m. Panel Discussion
Session Objectives: Explore the state of clinical gene transfer oversight from the perspective of those who analyze the regulatory context when making decisions about whether to invest finances or scientific resources in the field.
 
  Moderator: Alta Charo, J.D.
Committee Member
 
  Panelists: Jeffrey Chulay, M.D.
Chief Medical Officer and Vice President of Regulatory Affairs
Applied Genetic Technologies Corporation
 
    Todd Foley, M.B.A.
Managing Director
MPM Capital
 
    Manuel Litchman, M.D.
Vice President and Global Program Head, CTL019
Oncology Global Development
Novartis Pharmaceuticals Corp.
 
SESSION 3:
PATIENT ADVOCACY EFFORTS AND PERSPECTIVES
 
11:15 a.m. Panel Discussion
Session Objectives: Discuss the patient, consumer, and public perspective on oversight of clinical gene transfer protocols and how patients who may benefit from future gene therapies view the relevant regulatory landscape.
 
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
  Moderator: Sharon Terry
Committee Member
 
  Panelists: Nicholas Dainiak, M.D., FACP
Clinical Professor of Medicine
Chairman of Medicine
Yale University School of Medicine
 
    Jennifer Farmer, M.S., CGC
Executive Director
Friedreich’s Ataxia Research Alliance
 
    Margie Frazier, Ph.D., LISW-S
Executive Director
Batten Disease Support and Research Association
 
12:30 p.m. Lunch
 

SESSION 4:
OVERSIGHT OF CONTROVERSIAL SCIENCE

1:45 p.m. Panel Discussion
Session Objectives: Explore the policy implications of emerging sciences and the underlying reasons for establishing layers of oversight. Understand overlapping ethical, legal, and social issues that warrant elevated scrutiny of gene transfer research and other areas of scientific research. Discuss assessments of oversight in gene transfer research and other areas.
 
  Moderator: Jeffrey Kahn, Ph.D., M.P.H.
Committee Member
 
  Panelists: Alexander Capron, L.L.B.
Professor of Law and Medicine
University of Southern California
 
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
    Ellen Wright Clayton, M.D., J.D.
Professor of Pediatrics
Professor of Law
Vanderbilt University
 
    Hank Greely, J.D.
Professor of Law
Stanford University
 
    Peter Palese, Ph.D.
Professor and Chair of the Department of Microbiology
Mount Sinai Icahn School of Medicine
 
    Steven Rosenberg, M.D., Ph.D.
Chief of Surgery
National Cancer Institute
National Institutes of Health
 
3:00 p.m. Public Comment Period
Harry Malech, M.D.
President-Elect, American Society of Gene and Cell Therapy
 
3:15 p.m. Concluding Remarks
Larry Gostin, J.D., Committee Chair

Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 95
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 96
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 97
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 98
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 99
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 100
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 101
Suggested Citation:"Appendix A: Data Sources and Methods." Institute of Medicine. 2014. Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. doi: 10.17226/18577.
×
Page 102
Next: Appendix B: Historical and Policy Timelines for Recombinant DNA Technology »
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Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion.

Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

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