The US Food and Drug Administration (FDA) has announced a fast-track review process for gene therapies and other regenerative medicine treatments.
The new draft guidelines are an attempt to bring effective gene therapies to market more quickly. So far, the FDA has only approved two such treatments, which treat leukaemia and lymphoma. The updated guidelines will also speed up approval of stem cell based treatments that could treat serious or otherwise untreatable illnesses.
'These concepts are no longer the stuff of science fiction,' said Dr Scott Gottlieb, commissioner of the FDA. 'We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body… where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness.'
In addition to the fast-track process, the FDA has clarified several points to help researchers and drug developers understand whether their treatments are considered higher risk, requiring pre-market regulation, or whether they fall into a lower-risk category.
Researchers and therapy developers have welcomed the new guidelines.
'This is an incredibly significant development for the gene therapy, regenerative medicine sector,' Michael Werner, a lawyer and co-founder of the Alliance for Regenerative Medicine, told the New York Times. 'This should really open the door to a lot more gene therapies coming on the market quickly.'
However, Dr Michael Carome, director of non-profit organisation Public Citizen's health research group, told the newspaper that there was 'excessive hype' around the guidelines. 'We are talking about rushing to market very complex biologics products where we are still in the infancy of this field,' he said.
Clinical trials will remain an essential element of the accelerated approvals process, the FDA said. At the same time, the agency plans to crack down on illegitimate clinics offering unproven and often risky procedures.
This is overdue, said Dr Leigh Turner, an associate professor at the University of Minnesota's Centre for Bioethics in Minneapolis, Minnesota. The market for unproven treatments with no basis in evidence is 'quite large, quite active and [it's been there] a long time without meaningful oversight', he told Regulatory Focus. Many of these clinics purport to use stem cells derived from fat tissue. These procedures will now be brought under the FDA's remit.
Dr Peter Marks, director of the FDA's centre for biologics evaluation and research, said at a news conference: 'Our goal here is not to flood the market with products. It's to get products on that are safe and effective, and to clear up the field so that people know what they have to do.'