Long-Term Follow Up of Participants in Human Gene Transfer Research

June 1-2, 2004
Millennium Hotel
1313 Nicollet Mall
Minneapolis, MN 55403-2697

Following are the materials presented at the Long-Term Follow Up workshop held in Minneapolis, Minnesota. To access the slides or other materials which were presented at the workshop, simply click on a specific part of the agenda. If there were no slides or materials available during a portion of the program, there will not be a link.

This workshop on long-term follow up of participants in human gene transfer research was co-sponsored by the American Society of Gene Therapy, the Center for Biologics Evaluation & Research, (CBER/FDA), the Office of Biotechnology Activities, (OBA/NIH), Biotechnology Industry Organization (BIO), and Pharmaceutical Research and Manufacturers of America (PhRMA).

Audience
The target audience for this workshop included all individuals involved in the conduct or oversight of gene therapy clinical trials, such as:

• Academic Investigators
• Clinical Trial Principal Investigators
• Research Nurses
• Members of IRBs, IBCs, RAC
• Regulatory Affairs Groups
• Management officials from entities and institutes involved in sponsoring or conducting such trials

Purpose of the Workshop
To discuss the scientific, clinical, ethical, social and legal issues related to the long-term follow up of patients who participate in gene therapy clinical trials

Goals

• To assist the audience in understanding the current FDA requirements for long term follow up of participants in gene therapy clinical trials
• To hold a public discussion of the issues and publish a concise report that summarizes the key issues surrounding the long-term follow up of patients participating in gene therapy clinical trials.

Organizing Committee
Philippe C. Bishop, MD, Sarah Carr, Barrie J. Carter, PhD, John Cutt, PhD, Michael Egan, Terence Flotte, MD, Mark Frankel, PhD, Beth Hutchins, PhD, Richard A. Knazek, MD, Donald B. Kohn, MD, Alex Kuta, PhD, Bob Martin, Manal Morsey, MD, PhD, MBA, Susan P. Nemeth, PhD, Philip D. Noguchi, MD, Amy Patterson, MD, Sara Radcliffe, Alex Rakowsky, MD, Cynthia Rask, Stephen Rose, PhD, Stephanie L. Simek, PhD, Melissa Tice, Alice E. Till, PhD, Marie A. Vodicka, PhD, Carolyn A. Wilson, PhD

Day One (June 1, 2004)

Long Term Follow Up: Overview of Current Requirements and Workshop Goals

Stephanie Simek, PhD
FDA/CBER/OTRR

Long-Term Follow-Up in Bone Marrow Transplantation

Mary Horowitz, MD, MS
Scientific Director, International Bone Marrow Registry/Allogeneic Bone Marrow Registry, Robert A Uihlein Professor of Medicine, Medical College of Wisconsin

Use of the Gaucher Registry to Track Outcomes

Neal Mantick
Director, Global Registry Programs, Genzyme

Scientific Issues

Barrie J. Carter, PhD, Targeted Genetics Corporation &
Carolyn Wilson, PhD, FDA/CBER

Clinical Issues

Terence R. Flotte, MD, University of Florida
Daniel Rosenblum, MD, FDA/CBER

Scientific Issues Breakout Sessions

Co-Moderators Session 1:
Barrie J. Carter, PhD, Targeted Genetics Corporation
Stephanie Simek, PhD, FDA/CBER/OTRR

Co-Moderators Session 2:
Beth Hutchins, PhD, Canji, Inc.
Carolyn Wilson, PhD, FDA/CBER

Clinical Issues Breakout Sessions

Co-Moderators Session 1:
Stephen Rose, PhD, OBA/NIH
Daniel Rosenblum, MD, FDA/CBER

Co-Moderators Session 2:
Terence R. Flotte, MD, University of Florida
Philip Noguchi, MD, FDA/CBER

Legal / Social / Ethical Issues Breakout Session

Legal Issues Co-Moderators:
Lewis A. Grossman, JD
Professor of Law, Washington College of Law, American University
Of Counsel, Covington & Burling

Privacy Issues in Long-Term Follow Up
Hilary Schock, JD, MBA
Merck & Co., Inc

Ethics & Social Issues Co-Moderators:
William L. Allen, JD
Associate Professor & Director, Bioethics Program, CHFM, University of Florida

Ethical and Social Issues
James F. Childress, PhD
Kyle Professor of Religious Studies, Professor of Medical Education
University of Virginia

HIPAA Privacy Rule and Long-Term Follow-up in Research
Lora Kutkat, MS
Office of Science, Policy, Office of the Director, NIH

Long Term Animal Models Issues Breakout Session

Co-Moderators:
Theresa Chen, PhD
Pharm/Tox Branch, FDA/CBER/OCTGT

Cynthia Dunbar, MD
Head, Molecular Hematopoiesis Section, Hematology Branch, NHLBI, NIH

Day Two (June 2, 2004)

Reports from all Breakout Sessions

1. Scientific Issues
2. Clinical Issues
3. Legal, Social and Ethical Issues
4. Long-Term Animal Models Issues

What Information Would Bring About a Reassessment of the Requirements for Long-Term Follow Up in a Particular Class of Human Gene Transfer Clinical Trials?

Co-moderators:
Barrie J. Carter, PhD, Targeted Genetics Corporation
Terence R. Flotte, MD, University of Florida
Daniel Rosenblum, MD, FDA/CBER
Carolyn Wilson, PhD, FDA/CBER

Concluding Remarks

Stephanie Simek, PhD, FDA/CBER/OTRR