July 14, 2000 Meeting Minutes

The meeting of the Advisory Committee to the Director (ACD), National Institutes of Health, was convened via conference call on Friday, July 14, 2000, in Room 151, Building 1, National Institutes of Health (NIH), Bethesda, Maryland. The entire meeting was open to the public beginning at approximately 12:15 p.m. Dr. Ruth L. Kirschstein presided as Acting Chair.

Members Present:

  • William R. Brody, M.D., Ph.D.
  • Christine K. Cassel, M.D.
  • Steven Chu, Ph.D.
  • Yank D. Coble, Jr., M.D.
  • Victor J. Dzau, M.D.
  • Rebecca Eisenberg, J.D.
  • Elaine V. Fuchs, Ph.D.
  • Ting-Kai Li, M.D.
  • Jane A. Menken, Ph.D.
  • Shirley M. Tilghman, Ph.D.
  • Phillip L. Williams

Members Absent:

  • Thomas R. Cech, Ph.D.
  • Eric S. Lander, Ph.D.
  • Philip Needleman, Ph.D.
  • Ruth J. Simmons, Ph.D.
  • Larry L. Smarr, Ph.D.
  • Arthur D. Ullian
  • Donald E. Wilson, M.D.

NIH Employees Present:

  • Vida Beaven, Ph.D., OD/NIH
  • John Burklow, OCLP
  • Sarah Carr, OBA
  • Donna J. Dean, Ph.D., OD/NIH
  • Robert Lanman, OGC
  • Janice C. Ramsden, OD/NIH
  • P. Pearl O'Rourke, M.D., OSP
  • Amy Patterson, M.D., OBA
  • Lana Skirboll, Ph.D., OSP

I. Call to Order

Dr. Ruth L. Kirschstein, Acting Chair, called the meeting to order and welcomed the members participating in the conference call.

II. Interim Report of the Working Group on NIH Oversight of Clinical Gene Transfer Research

Dr. Christine K. Cassel, Chair of the Working Group on NIH Oversight of Clinical Gene Transfer Research, summarized the recommendations stated in the Interim Report (Appendix A) for discussion by, and consideration of, the members of the ACD.

Dr. Kirschstein explained the difference in the governance of an Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC). The IRB is governed by the Federal Policy for the Protection of Human Subjects and reviews human subjects research conducted with federal funds. The IBC is governed by the NIH Guidelines for Research Involving Recombinant DNA Molecules and is responsible for and authorized by the research institution to review and approve potentially biohazardous lines of research.

Currently, to undertake human gene transfer studies, investigators must obtain IRB and IBC approvals of the protocol and then submit a copy of the proposed research protocol to the Office of Biotechnology Activities (OBA)/Recombinant DNA Advisory Committee (RAC), which is advisory to the NIH Director. Protocols must comply with the policies and procedures for the conduct of clinical research involving gene transfer as set forth in the NIH Guidelines. Protocols that present novel scientific, ethical or safety issues are reviewed by RAC at a meeting which is open to the public.

Investigators must receive authorization from the Food and Drug Administration (FDA), as well as approval from the IRB and IBC, in order to initiate a gene transfer clinical trial. Those investigators governed by the NIH Guidelines are required to submit their proposals to OBA/RAC, but neither submission to RAC nor RAC discussion are necessary prior to the initiation of such a trial.

There was some concern expressed as to whether an investigator may appeal a RAC recommendation. In response to this concern, Dr. Kirschstein stated that the protocol could be resubmitted to the RAC, which would require more data and a re-review. Dr. Skirboll added that the RAC does not have the authority to hold up initiation of the trial and that the final decision is under FDA authority and requires IRB approval.

In response to a concern related to considerable variation in review procedures by IRBs, Dr. Cassel said that the IRBs and IBCs should take RAC considerations seriously.

Some concern was raised as to whether the lack of expertise regarding gene transfer studies by members of an IBC would result in delay of the start of the protocol. Dr. Cassel responded by saying that there would be a requirement for the IBC to submit to the RAC, in writing, a reason for the delay. She went on to explain that the only intervention on the part of NIH would be to withhold funding. There is a safety factor built in since the FDA is represented on the RAC. Dr. Kirschstein added that the Office of Human Research Protection (formerly the Office of Protection from Research Risks) also could be involved and reminded the group that the RAC is subject to the same advisory rules as the ACD.

Dr. Skirboll stated that this recommendation should encourage earlier submission of a protocol and went on to explain that, if a protocol is submitted eight weeks before a RAC meeting, there is no problem; however, if it is submitted later than eight weeks before the meeting, it would have to wait for review until the next RAC meeting. She added that there is a fifteen day turnaround time between the arrival of a protocol in OBA and informing the investigator of OBA's plans for RAC review.

Dr. Kirschstein told the members of the ACD that, while most research related to recombinant DNA and gene transfer is funded by NIH, many of the protocols are not, but are performed by industry. There was some discussion as to whether all protocols could be submitted to OBA. Some concern was expressed as to the increased workload. Dr. Kirschstein explained that the OBA staff is being doubled as well as being provided with an increased budget.

Dr. Cassel reported that even though some protocols are funded by industry, most of the funding still comes from institutions receiving their funds from NIH. Dr. Kirschstein replied that, if a gene transfer protocol is performed in an institution which does not receive NIH funds for recombinant DNA research, there is no requirement that it be reviewed by RAC.

There was some concern related to whether the required oversight is appropriate. Dr. Cassel stated that this issue is addressed in the appendices of the final draft report. Dr. Kirschstein told the ACD members that she, other NIH staff and Dr. Cassel would review the oversight statement and develop a revision if it is not strong enough.

A motion was made and passed unanimously to accept the Interim Report prepared by the Working Group.


The meeting ended at approximately 12:50 p.m. on July 14, 2000.


I hereby certify that the foregoing minutes are accurate and complete.

Ruth L. Kirschstein, M.D.
Acting Chairperson
Advisory Committee to the Director
National Institutes of Health



William R. Brody, M.D., Ph.D.
Johns Hopkins University
Baltimore, MD 21218-2688
Christine K. Cassel, M.D.
Professor and Chairperson
Department of Geriatrics and Adult Development
The Mount Sinai School of Medicine
New York, NY 10029-6574
Thomas R. Cech, Ph.D.
Distinguished Professor
Department of Chemistry and Biochemistry
University of Colorado
Boulder, CO 80309-0215
Steven Chu, Ph.D.
Physics Department
Stanford University
Stanford, CA 94305-4060
Yank D. Coble, Jr., M.D.
Jacksonville, FL 32205
Victor J. Dzau, M.D.
Department of Medicine
Brigham and Women's Hospital
Boston, MA 02115
Rebecca S. Eisenberg
Professor of Law
Stanford Law School
Crown Quadrangle
Stanford, CA 94305-8610
Elaine V. Fuchs, Ph.D.
Amgen Professor of Basic Sciences
The University of Chicago
Department of Molecular Genetics and Cell Biology
Chicago, IL 60637
Eric S. Lander, Ph.D.
Professor of Biology
Massachusetts Institute of Technology
Director, Whitehead Institute/MIT
Center for Genome Research
Cambridge, MA 02139-1561
Ting-Kai Li, M.D.
Distinguished Professor
Indiana University School of Medicine
Indianapolis, IN 46202-5124
Jane A. Menken, Ph.D.
Population Program
Institute of Behavioral Science
University of Colorado at Boulder
Boulder, CO 80309-0484
Philip Needleman, Ph.D.
Chief Scientist
Monsanto Company
St. Louis, MO 63167
Ruth Simmons, Ph.D.
Smith College
Northampton, MA 01063
Larry L. Smarr, Ph.D.
National Center for Supercomputing Applications
University of Illinois
Champaign, IL 61820
Shirley M. Tilghman, Ph.D.
Lewis Thomas Laboratory
Department of Molecular Biology
Princeton University
Princeton, NJ 08544
Arthur D. Ullian
Chairman, Task Force on Science, Health Care and The Economy
Boston, MA 02110
Phillip L. Williams
Vice Chairman (Retired)
The Times Mirror Company
Pacific Palisades, CA 90272
Donald E. Wilson, M.D.
Vice President for Medical Affairs
Dean, School of Medicine
University of Maryland, Baltimore
Baltimore, MD 21201-1559

Executive Secretary
Ruth L. Kirschstein, M.D.
National Institutes of Health
Bethesda, MD 20892

This page last reviewed on February 12, 2011