Response to FDA
Institute for Human Gene Therapy's Response to FDA
The Institute for Human Gene Therapy at Penn filed its response on February 14, to Inspectional Observations made by the Food and Drug Administration on January 18, as the result of its investigation into the death of Jesse Gelsinger, an 18-year-old with a rare metabolic disease who was participating in an experimental gene therapy trial for the disease known as ornithine transcarbamylase (OTC) deficiency, an inherited disorder that in its most common form causes death in affected newborn males due to a genetic defect in the liver.
The IHGT immediately placed the OTCD clinical trial on hold following Jesse Gelsinger's death on September 17, 1999. The IHGT had informed the FDA of Jesse Gelsinger's deteriorating condition prior to his death and, following his death, promptly notified the FDA, the Recombinant DNA Advisory Committee of the NIH and the Institutional Review Board.
The IHGT response to the FDA continues an extensive review of research at the University, ordered by President Judith Rodin, including a comprehensive review of all aspects of research using human subjects, considering everything from the mechanics of the approval process to oversight to accountability and research outcomes; and an independent review of oversight and monitoring of clinical trials at the Institute, which will be conducted by research scientists who are unaffiliated with Penn.
"We understand that nothing less than the highest possible standards for the conduct of research are acceptable at the University of Pennsylvania," said Dr. Richard Tannen, professor of medicine and senior vice dean at the School of Medicine. "The thoughtful process that has led to our [FDA] response is an important step which, together with the other important initiatives underway, will ensure that the conduct of research at Penn does, in fact, meet those standards."
The FDA identified "important issues arising out the OTCD trial," said Dr. James M. Wilson, John Herr Musser Professor of Research Medicine and director of the IHGT, and, he said "we have taken its questions and concerns very seriously."
Dr. Wilson said, that the IHGT will continue to cooperate, fully and completely, with the FDA, the NIH and all other interested agencies in their respective reviews.
He also said that IHGT "will continue to work closely with the FDA to implement revised clinical procedures relating to monitoring and related practices and to provide additional patient safeguards to the fullest extent possible in clinical research settings."
The 28-page IHGT response to the FDA makes the following points:
The University has made a commitment to cooperate, fully and completely, with the appropriate regulatory agencies and public officials in the examination of Jesse Gelsinger's death.
From the University of Pennsylvania Almanac, Vol. 46, No. 22, February 22, 2000