In a milestone for the field of gene therapy, the National Institutes of Health (NIH) will no longer subject all proposed gene therapy clinical trials to review by a special federal advisory committee.
“Given the progress in the field, I am confident that the existing regulatory authorities can effectively review most gene transfer protocols and that a streamlined process will reduce duplication and delays in getting gene transfer trials initiated,” said NIH Director Francis Collins in a statement today. Instead, the 40-year-old Recombinant DNA Advisory Committee (RAC) will review only a few trials that pose special risks.
Originally created to review all gene transfer experiments, the RAC in the late 1980s shifted its focus mainly to gene therapy trials. The committee selects about 20% of submitted protocols for discussion at public meetings.
As the field has matured and gene therapy has been used to treat disorders such as inherited blindness and immune deficiencies, researchers have argued that the risks are no different than those of other experimental treatments. They point out that they must already go through reviews by institutional ethics and biosafety boards and the U.S. Food and Drug Administration. RAC review is redundant and slows progress, argued the American Society of Gene & Cell Therapy.
In December 2013, an Institute of Medicine (IOM) panel agreed. It said that although NIH should continue to register trials, the RAC only needs to publicly review protocols that cannot be evaluated by standard oversight bodies and pose unusual risks, for example because they use a new vector. NIH issued a noncommittal response, making it unclear whether it would follow through.
But today, Collins announced that NIH has “decided to accept the IOM committee recommendations on RAC review of gene therapy research.” The agency will soon issue a proposed change to its oversight rules for recombinant DNA research.
Collins’s statement does not address another IOM recommendation, however—that NIH consider replacing the RAC with a similar body that would also review other types of risky clinical research. NIH is still “considering” that recommendation, says spokeswoman Renate Myles.