WASHINGTON, D.C. (Dec. 7, 1999) -- The Biotechnology Industry Organization (BIO) will present alternatives to proposed guidelines for reporting adverse patient events in gene therapy clinical trials when an advisory committee of the U.S. National Institutes of Health (NIH) meets Wednesday through Friday to consider the issue.
Gene therapy has strong potential for improving the lives of millions of people who suffer from cancer, heart disease, cystic fibrosis and other deadly and debilitating illnesses. Because it is so new, gene therapy is subject to greater oversight than virtually all other drug development technologies. In addition to regulation by the U.S. Food and Drug Administration (FDA), gene therapy clinical trials are reviewed by the NIH's Recombinant DNA Advisory Committee (RAC) in an open, public forum.
"BIO fully supports this extra level of scrutiny because werecognize the need for public discussion of the social and ethical implications involving new and advanced technologies," said BIO President Carl B. Feldbaum.
BIO has identified several problems with RAC's proposed guidelines. First, RAC inappropriately recommends immediate reporting ofserious adverse events unrelated to the treatment. Second, the proposal creates a new format for reporting adverse events that conflicts with current regulations. Third, the reports requested by NIH would contain confidential commercial and patient information, precluding RAC from disclosing them to the public.
As an alternative, BIO proposes the following: Companiessponsoring gene therapy clinical trials voluntarily would provide RAC with the serious, related and unexpected adverse event reports currently sent to the FDA. Companies also would send RAC safety data summarized for the FDA in annual progress reports on clinical trials. This would give RAC the same information at the same time and in the same format as the FDA.
In addition, the NIH and industry would have to agree on how the data will be used. NIH would be responsible for ensuring patients' privacy rights are protected and companies' trade secrets remain confidential. The reporting structure then would be reviewed after one year.
The complete text of BIO's recommendations will be submitted to the RAC during the committee's public meetings Wednesday through Friday, Dec. 8 -10. H. Stewart Parker, CEO of Targeted Genetics Corp. and a member of BIO's board of directors, will present BIO's position Friday. BIO's comments also are posted online.
BIO represents more than 850 companies, academic institutions and state biotech centers in 47 states and 26 nations. BIO members are involved in the research and development of health care, agricultural, industrial and environmental biotechnology products.