The FDA is holding a 2-day stem cell meeting starting tomorrow and it promises to be a really big deal. What’s the scoop on this meeting and the attendees?
Who is likely to say what?
If the deregulatory proponents get their way, could we have stem cell clinics like Starbucks popping up in even more neighborhoods?
I have a satirical cartoon from some time ago I drew imagining such a future.
I’ve put together a “field guide” to the meeting in the form of a list of 10 bullet points.
- FDA speaks. The FDA is of course hosting this meeting and is presenting at it too. I’m very curious to hear what they have to say. I’d predict that they will present the case for their draft guidances including that fat stem cells are a drug and that their use is often non-homologous.
- Clinic picks and their customers/patients. The stem cell clinics and their favorite patients are out in force. Expect them to push for less FDA oversight of stem cells and in particular for not classifying fat stem cells as a drug or bone marrow stem cells used in a non-homologous fashion as drugs. Note that some of these clinics/businesses have been the subject of past FDA actions and others could be wondering if they might be contacted by the FDA in the future so it’ll be an interesting dance.
- Questions to think about regarding clinic talks. My advice to watchers of the meeting? Listen for unsubstantiated medical claims of safety and efficacy, impressive-sounding anecdotes, claims that publications of data are in the works, and assumptions that oversight is only harmful. Also consider who is making money off of stem cell marketing to patients and who stands to gain if there’s less oversight.
- Will the disturbing, but important topic of the 3 women who were recently blinded by a fat stem cell clinic in Florida come up? It should. At last week’s FDA science meeting on stem cells, Dr. Albini presented the cases of 3 women who were literally blinded by a Florida stem cell clinic at which fat stem cells were injected into their eyes. Interestingly, it is possible that that the so far unnamed clinic may itself be presenting at the meeting so I’m wondering if someone will have the courage to confront them about this disastrous outcome.
- J&J has its say. Johnson and Johnson, a biomedical powerhouse, is on the agenda. This m means that they are clearly interested in stem cell oversight. Will they advocate for less oversight? Or will they support the current FDA approach for the most part? What else might they say?
- Tissue banks out in force. Quite a few cell and tissue banking type of organizations are on the agenda. What might they have to say? Since presumably they have an interest in the freer use of stem cells and other materials, which creates more demand, I’d guess that at least some will advocate for less oversight, while others won’t.
- REGROW on stage? To what extent will the REGROW Act, which seeks to reduce FDA oversight, take center stage in some of the talks? This bill has largely gone off the radar this summer, but could make a comeback in the fall and this FDA meeting seems that it could be a springboard for REGROW fans.
- Will CIRM advocate for less FDA oversight again? The short answer is “yes”. How strong will CIRM’s words be at this FDA meeting? If they push fairly vigorously for less oversight how will that come off with so many stem cell clinics at the same meeting also pushing for less oversight? It could be a tightrope walk for CIRM as they are opposed to the clinics.
- Academic institutions have sway? There are some universities and academic researchers from such institutions slated to talk. What will they say? Less regulation is needed? The current system largely works but needs to be more efficient? Mixed bag? We could see more blurring of the lines.
- What happens after the meeting ends? This is the most important question to think about throughout the meeting. Will the FDA begin a period of more proportionate activity in providing oversight of the stem cell clinic arena? Will it pretty much stick with the status quo of doing next to nothing on stem cells (maybe 1 warning letter every year or two?) despite there being around 600 such clinics in the US today that largely lack FDA approval or licensing? It seems to me if the FDA is going to stick with the status quo then what’s the point of evening having these meetings so I’m hopeful that change is in the air. It might not come until 2017, but the odds are that the FDA will step up its game on stem cell clinics eventually.
Very interesting points raised, Cartoon is great! May I have the permission to use it in my PPT for doctors on Regen Medicine. thanks, Dr. Minnie
http://californiahealthline.org/news/fda-wants-to-tighten-the-screws-on-stem-cell-clinics/
C. Randal Mills, California Institute for Regenerative Medicine, CEO
FDA Must Find A Middle Ground For Sake Of Patients
We aren’t happy, as a lot of people aren’t happy, with the proliferation of these stem cell clinics — some of which are probably doing good work. But some are clearly making rather outlandish claims for which there’s no real data.
There are a couple of conditions coming together to create this storm.
One is that the need is very real. These patients are really struggling. They don’t have alternatives. They’re desperate and they need help. It’s not in the realm of possibility to talk to somebody who is suffering as badly as these patients are and to say, ‘You have to wait a few more decades for the science to catch up.’
On the other hand, we have a regulatory paradigm that only provides two pathways to put a cell therapy onto the market. One pathway is the most intense regulatory requirement anywhere in the world for any product — the biologics license application through the FDA, which takes 10 to 20 years and costs over $1 billion.
The other is through the exemptions the FDA has made, which require absolutely no pre-market approval whatsoever. You can be on the market in days, with no data.
The regulatory burden associated with one is massive and the other is almost nonexistent.
So it’s not at all surprising that we’re seeing a proliferation of these stem cell clinics popping up that are operating under the assumption that they fall under the exemption.
What the FDA is doing now is saying, ‘We’re not happy with this. We’re going to define some terms more narrowly than in the past … and make it more difficult to legally be on the market under the less burdensome regulatory pathway.’
That’s what this meeting is about.
The problem with their strategy is twofold. It doesn’t address the patients, or the need side of the equation. And I don’t think it has a chance of actually working because the FDA will acknowledge that they do not have the resources to enforce these types of regulations at the clinic level.
They would have to be essentially regulating the practice of physicians, which is well beyond their capabilities. Even if they were able to enforce it, it would just drive these patients somewhere else.
We’re advocating for the creation of some middle pathway that would bring essentially unregulated therapies into the regulatory fold, but in a manner which could be complied with.
I would rather know these clinics are being regulated and collecting data than have them operating under the radar screen of the FDA. I would like there to be a formal pre-market review of these therapies before they’re put on the market. I would like there to be safety and efficacy data.
I’m going to try hard to get the FDA to see that just plugging this hole won’t make the problem go away.
Thinking that they’re going to strengthen the regulation and that patients are going to be satisfied that there’s absolutely no chance for help is naive.
There isn’t a lot of evidence to suggest these types of procedures are overly risky. It’s not that they don’t have risk, but everything in medicine does. If you’re a patient who has absolutely no alternative, you’re probably willing to take the chance.
Questions to think about regarding academic and big pharma-backed “patient advocacy” group talks. My advice to watchers of the meeting? Listen for unsubstantiated claims of cancer danger or impressive-sounding case studies of a few isolated incidents, including patients who were not treated by medical doctors and who were treated with embryonic stem cells abroad.
Be on the lookout for assumptions that oversight is only good. Also consider who is making money off government and corporate grants, greater barriers to entry, and who stands to gain if there’s more oversight.
Paul, Will you personally be at the meetings? If so, hope to see you there. Edward.
Great cartoon, Paul! I hope you don’t mind if I plagiarize it? Thanks for putting the credits on it.
Rick Vulliet