FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit
The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:
“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”
It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.
That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:
“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”
Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.
Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:
“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”
He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”
Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.
“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”
For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.
We will be writing more as the conference unfolds so stay tuned!
The Stem Cellar 
Great article. Gives basis for further hope. Our Congressional people need to hear more about the very high cost of R&D in bringing new medicines to market. Putting too much restraint on drug pricing can also remove incentives for innovation. Investor’s lose confidence to fund innovation when Congress intervenes, to legislate price controls. The patient and family presence is happening, witness the fight being waged with Duchenne’s muscular dystrophy and Jenn McCrery and her two boys. It is happening.
Like Richard said….you better believe it’s happening! Here’s our group of patients who’ve been speaking up loudly for the past 4 years. http://www.patientsforstemcells.org/home/
We are all patients who’ve had quality of life improvements being treated with our own cells for different diseases/conditions in different clinics around the world. There are NO cures out there for most diseases like cancer, auto immune, cardiac, ALS, parkinson’s, COPD, diabetes, MS, etc. The current state of affairs in America is that my doctor cannot innovate within the practice of medicine to help me even when there is no FDA approved medication left for me to try. Most patients find themselves as “No Option Patients!” Medical tourism to receive stem cell therapy, is becoming the norm. Patients are sharing their stories with other patients. The word is out in most disease communities that success is being seen being treated with your own cells.
I read an article today that describes what a lot of patients are doing…”refusing 2 accept the status quo!” When your doctor say’s to you, there is nothing left for us to try, so many patients are becoming their own advocates and figuring out treatment for themselves. I assume many doctors are frustrated as well.
Unfortunately, Canada, the UK, Japan, Korea and Jordan are leading the world in regenerative medicine, while the US lags behind trying to figure out who’s going to be in charge, putting all of it’s eggs in one glacially slow FDA basket.