FDA Proposes Rule on Gene Therapy, Xenotransplantation

FDA Consumer, May, 2001

The Food and Drug Administration has proposed new requirements that would give consumers increased access to information on clinical trials involving gene therapy or xenotransplantation. The rule, proposed Jan. 17, would ensure that people participating in clinical trials involving these technologies are fully informed of the risks, as well as the opportunities for curing a disease or reducing its symptoms.

Gene therapy and xenotransplantation have the potential to alleviate suffering and save lives. But they also hold unique risks for individuals who participate in the studies. Gene therapy is an experimental technique that treats disease by replacing defective genes or inserting new ones. Xenotransplantation refers to transplanting tissues or organs from animals into humans.

The FDA is proposing to make available information about side effects, including deaths, seen during clinical trials. But the names of study participants and proprietary commercial information would remain confidential.

The proposed rule also would ensure that FDA policies for public access to information on gene therapy and xenotransplantation studies are compatible with those of other government agencies that oversee these types of research. The 90day comment period for the rule ended April 17. Comments will be considered in the FDA's development of a final rule.