The US Food and Drug Administration (FDA) is caught between a rock and a hard place. And CIRM is going to try and help them get out from under that.
As things stand today, if the FDA approves a therapy quickly and a patient later dies from it, then they are widely criticized. If they take a long time to approve a therapy and people die waiting for that treatment, then they are just as widely criticized.
So maybe it’s time to help them change that, by creating a new pathway that allows for a faster, more efficient, but equally safe, process of approving stem cell therapies.
This was a topic that CIRM’s President and CEO, Dr. Randy Mills, took on at last week’s World Stem Cell Summit. He highlighted our mission – accelerating stem cell therapies to patients with unmet medical needs – as the driving force behind everything we do, including regulatory reform:
“We have had the current FDA regulatory structure for cell therapy in place for 15 years, and in that 15 years not one stem cell therapy has been approved. The scoreboard is not lying, there’s a zero on it. Not one therapy has been approved. There is an issue here, we can’t ignore that fact and so we made it part of our proposed new Strategic Plan to try and remove this burden.
“There is an excessively long translational pathway to get an Investigational New Drug (IND) approval from the FDA (a necessary step to proceed with testing a therapy in a clinical trial). For non-cell therapies it takes 3-4 years to get an IND. For cell therapies it takes 6-8 years, twice as long.”
Mills says many potential therapies have been abandoned, or even stopped before they even got started, simply because the regulatory hurdles are so many and the costs so high.
“We are not anti-regulation, we are not anti-FDA, and we are not calling for the removal of rules and regulations around stem cell therapies, that would be bad for patients and research. These therapies have risks and we are not proposing any strategy that puts things on the market without any testing or safety data. But right now we are being so careful about safety to ensure patients are not put at risk while those same patients are dying from their disease.”
Chaohong Fan, MD, PhD, a Medical Officer at the FDA was in the audience and said the people at the FDA really want to help, that they feel it’s part of their mission.
Mills said he had no doubts that the people at the FDA are committed and passionate about what they do. He says it’s not that people at the FDA aren’t working, it’s that the process isn’t working, and needs to be transformed.
“At CIRM we are saying doing nothing is not OK. It’s not OK. So we are going to be working with patients and patient advocates, companies, researchers and the FDA to make change, to make it easier for patients to get access to the therapies they need.”