Although the change removes one layer of regulation, gene therapy trials will still need to meet US Food and Drug Administration (FDA) safety and ethical requirements and, as with all clinical research, regulatory approvals must be in place before a gene therapy trial can proceed.
'There is no longer sufficient evidence to claim that the risks of gene therapy are entirely unique or unpredictable – or that the field requires special oversight that falls outside our existing framework for ensuring safety', wrote NIH Director Dr Francis Collins and FDA Commissioner Dr Scott Gottlieb in the The New England Journal of Medicine.
Gene therapies, used to introduce a new gene, delete or edit abnormal DNA, became a safety concern following a teen's death in 1999. The field has advanced significantly especially in recent years, encompassing genome editing approaches such as CRISPR/Cas9. The FDA approved three gene therapies for clinical use in the USA in 2017 (see BioNews 916)
Previously trial protocols, annual reports, and reports of serious adverse events had to be submitted to both the NIH and the FDA, leading to duplicated work and increased administrative burden on researchers. The NIH have, therefore, set out new plans to simplify the safety management in US gene therapy trials by introducing changes to the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules.
As oversight of gene therapy trial will now be handled by the FDA, the NIH is calling on the Recombinant DNA Advisory Committee (RAC) to re-focus its attention on the 'scientific, safety, and ethical issues associated with emerging biotechnology'.
However, not everyone supports the changes.
'This is not the right time to be making any moves based on the idea that we know what the risks are,' said Professor Mildred Cho, a biomedical ethicist at Stanford University and a member of RAC. 'We really don't understand how [gene therapies] work.'