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Author: Richard Jaffe

Unwanted advice for the anti-vaxxer Cali. SB 277 lawsuit reboot

Unwanted advice for the anti-vaxxer Cali. SB 277 lawsuit reboot

Here’s what I’m getting from the Bolen Report’s prediction/foreshadowing of the new SB277 challenge, and so here is some more probably unwanted advice:

500 Plaintiffs:

Really? HELLO: you’re fighting an asymmetrical battle. The government has a lot more financial and personnel power than you guys. Moreover, if what you say is true, namely that big pharma is behind the worldwide vaccine conspiracy, pharma has unlimited resources. You’re going to try to manage 500, or even dozens of plaintiffs? That doesn’t make any sense. What’s the upside? You don’t think the judge will know that there are a lot of you vaccine concerned out there? Get real and keep it manageable. Use just a couple plaintiffs to cover your bases, standing wise (a few affected individuals and a couple groups for associational standing), and that’s it.

Multiple Lawsuits:

Really? Filed by the same lawyers? Same or similar legal theories? You’re just making more unnecessary work for yourselves. The same or overlapping defendants will just file consolidation motions in each of the cases, or xerox the dismissal papers and submit them to all the courts. Filing a bunch of similar lawsuits will do a couple things which you shouldn’t want. It will burn a few months and waste money. Plus, you’ll really ingratiate yourselves with the judiciary, because there’s nothing judges like better than wasteful duplicate litigation which clogs their dockets.

Don’t overstate your case by bad-mouthing all vaccines that were ever given since the beginning of time

I get that the hard core anti-vaxxers think that no vaccine has ever helped a single human being or prevented anyone from getting any disease ever, and that everyone involved in the horrific vaccine genocide should be prosecuted, but for God’s sake don’t put that or any other crazy stuff in the complaint. Those Luddite arguments might play well to the hard core anti-vaxxer element, but you guys who are preparing the papers are professionals. There’s no sense writing stuff which will make the judge and the media think the clients and their lawyers are complete wackos.

Don’t make it personal

There is a tendency in cases like this to identify the boogeyman. The prior lawsuit and the two current pending lawsuits did that, focusing on the pediatrician/legislator and some Santa Barbara government official. It’s already been done and it won’t help your case to personally attack people who advocated or worked for a law passed by the legislature. That is how public health policy is set. Nor will it help your case by attacking government officials who want to make sure that the intent of the law is effectuated. That is the job of such officials. Calling them names and vilifying them will surely make the hard-core anti-vaxxers feel good, but it won’t help you case. If anything, it will hurt it.

Acknowledge the deep legal hole you’re in and move forward from there

All of the extreme anti-vaxxers, and a good percentage of the vaccine-concerned are living in an alternative legal universe. In the universe I and all reasonable and informed people live in, for over one hundred years, starting with the Supreme Court’s decision in Jacobson, every single legal decision has upheld the government’s right to mandate vaccination and has rejected a purported individual’s personal belief based asserted right to avoid vaccination based on an proposed extension of a constitutional right. All of these legal challenges fail because as is is oft-stated, the Constitution isn’t a suicide pact.The personal beliefs of the few will never outweigh the public health of the many.

That means that the only conceivable chance to have a judge think about letting the case proceed –rather than grant the state’s motion to dismiss – is to focus on public safety. Specifically, the public safety of kids, particularly young children, and specifically the vaccine schedule and maybe the dangers of the most popular adjuvants. You guys need to drill down, think small and get into the weeds; pick the most unassailable scientific concerns out there and go with that, rather than big picture things like cover-ups, conspiracies and boogeymen.

Your immediate goal

I would respectfully suggest that the purported goal of having some authoritative judge make some grand declaration about an individual’s rights superceding the right of the public to public health is completely unrealistic and will never ever, ever happen. Even a public health argument is a long shot, because such decisions are usually left to the legislative and executive branches. However, if you present a rational and interesting argument, you might just get a judge to let the case go forward, which should be the primary immediate goal of the new lawsuit, as opposed to filing a facially defective complaint –like the prior dismissed lawsuit, or the wacko papers filed in the two pending lawsuits.

In short, try something different this time; be smart; don’t file papers that the extreme anti-vaxxers will love, but the judges and press will think are crazy. That’s been done and done.

Rick Jaffe, Esq.

www.rickjaffe.com
rickjaffeesquire@gmail.com

Game Changing the Stem Cell Debate

Game Changing the Stem Cell Debate

It’s been almost a month since the FDA’s two day hearing on the draft guidance documents on autologous stem cells. Here is the short version of what happened:

The stem cell research industry, meaning the basic scientists and academic physicians employed at big institutions, and their acronym organizations like the draft guidances. They agree with the FDA that non-homologous, (roughly meaning therapeutic as opposed to replacement) autologous (i.e. my) stem cells (or any kind of autologous tissue or “HCT/P’s”) should only be available in clinical trials until the FDA grants marketing approval for some use. (What happens after approval, in terms of off-label use, is anyone’s guess).

Those institutional players cited four cases of harm to patients. Three were at one clinic where a non-physician injected stem cells into the eyes of the patients which resulted in loss of vision. The other case was someone who took stem cell tourism to the extreme, continent-hopping and injecting every manner of autologous and allogenic stem cells for his degenerative condition.

Testifying on the other side were clinicians providing non-homologous, autologous stem cell treatments to patients. These clinicians have treated or spoke about the tens of thousands of patients who have received various forms of therapeutic HCT/P treatment under the soon-to-be extinct practice of medicine exception (21 CFR 1271.15).

And then there were the HCT/P patients, many with life threatening, incurable or life altering conditions. They all testified in support of Americans having access to their own HCT/P’s in the U.S. It was heart wrenching and uplifting to hear their stories. I couldn’t begin to do justice to their plight and the power of their words, so I won’t try.

But, if there’s one thing I’ve learned about dealing the FDA on access to treatment issues, it’s that the government doesn’t really care about actual patients or even future patients, due to its deep concern about PUBLIC HEALTH and THE SAFETY OF THE PUBLIC, which are sort of like Platonic ideals, and which have only the faintest connection to actual patients and reality.

Powerful as it was, I don’t think the testimony of these patients will change the FDA’s mind on the core issue of a person’s access to his/her own body parts.

There were over six thousand public comments submitted to the FDA after the hearing. However, that’s orders of magnitude too small to make an impact on the FDA.

So after pondering the hearings, I feel like what Roy Scheider felt and expressed when he finally saw JAWS up close: “We’re gonna need a bigger boat.”

Between the media, which has been harpooning all these unregulated stem cell clinics, and the stem cell research industry’s greenlighting the draft guidance documents, we’re gonna need a bigger boat.

The bigger boat has to be a vehicle to reach a lot more people, like many tens of millions. Social media itself can’t do it.

To me, the obvious solution is a documentary. I’ve recently seen Vaxxed. These guys are now on a bus tour all across America. The documentary and the tour is having an impact, and that’s in spite of the fact that Andy Wakefield is a very controversial fellow who has been excoriated in the media.

I was involved in a couple of the documentaries about Dr. Stanislaw Burzynski. He’s also a very controversial fellow and gets a great deal of negative press. Nonetheless, the director/producer made a deal with the cable companies which resulted in the Burzynski documentary being available to something like 40 million cable viewers. Now those are the kind of numbers I’m talking about!

I think the right kind of documentary focusing on the patients and not having a polarizing protagonist could avoid some of the special challenges facing the producers of the two aforementioned documentaries. If done right, it could spark the interest of tens of millions who themselves or their family members are dealing with serious unresolvable medical conditions.

I have to believe that in the stem cell patient community there are media insiders, power players, and even celebs who support access to these treatments and would be willing to help. I also have the feeling that the money to make it happen would show-up.

The stem cell clinical community needs a game changer.

You know what they say: “Go big or go home.”

Rick Jaffe, Esq.
www.rickjaffe.com
rickjaffeesquire@gmail.com

More about the FDA stem cell hearings at:
http://rickjaffeesq.com/category/stemcells/

WHAT’S WRONG WITH THE U.S. HEALTHCARE SYSTEM AND WHAT IT WOULD TAKE TO FIX IT

WHAT’S WRONG WITH THE U.S. HEALTHCARE SYSTEM AND WHAT IT WOULD TAKE TO FIX IT

I spend most of my professional time in the weeds of health care cases, so I thought it might be a nice change of pace to put down some of my observations and general critiques of the system based on my 30 years in the health care arena and as a consumer of health care services.

(I originally posted this back in April, but so much has changed since then, I decided to update the problems, (including the FDA’s recent efforts to take away the most popular form of breast reconstruction surgery from mastectomy patients, add some solutions, and say what it might take to have the solutions implemented.)

Let’s start with a few common macro facts/critiques:

1. The US has the most expensive health care system on the planet, per capita.

2. But by almost all recognized health care benchmarks, American health care has worse health care benchmarks than most other industrialized countries.

3. On the other hand, all types of expensive new technologies are available to Americans with good insurance or who can self-pay, and speaking of new health technologies,

4. Most drug and device innovations in diseases and chronic conditions come from US companies and are tested at least in part in clinical trials in the US, though that is changing somewhat because,

5. The US is the toughest and most expensive place in the world to get drugs approved. But in the last decade or two, access to investigational drugs in the US has opened-up alittle, compared to many western industrialized countries. However, access to investigational drugs is still woefully inadequate (in my opinion). As a frame of reference, each year the FDA allows a thousand or two patients to receive investigational drugs outside of clinical trials. This is a miniscule amount compared to the number of people who want or might benefit from investigational drugs.

The seemingly unavoidable conclusion is that we spend way too much on health care compared to what we get in terms of societal or population level health benefits. But how can that be? We have so much medical innovation coming from the US and we surely have the finest physicians and medical facilities (just ask any US mainstream physician). How can this abundance not translate into the world’s best medical care based on recognized health care benchmarks?

The answer is no doubt complicated, but I suspect that part of our underperforming stems from the nature of statistics and the difference between our health care system and the rest of the industrial world. Almost all other industrialized countries provide health care as a basic benefit to its residents; they have what many here pejoratively call, “socialized medicine.”

In this country, we have two kinds of health care consumers, those with health insurance and those without. Major and moderate medical interventions are far beyond what most people can afford to pay out-of-pocket. Without insurance, people either don’t get all of the health care they need, or get it through ridiculously expensive and inefficient means like ER facilities, and the costs for this inefficient care are ultimately borne by taxpayers or health insureds. That’s no way to run a health care system.

The large number of un or underinsured has to drag down these macro health benchmarks. If so, then a better comparison might be comparing US residents who have good health insurance to the rest of the industrialized world, and ignore the un and underinsureds (which is basically what our Government has done until the advent of Obamacare). I suspect the gap would close significantly but not completely. So beyond the fact that we chose (and continue to choose) to let a portion of our residents live without adequate health care, there are likely other factors which cause us to pay too much for too little.

Maybe another cause of the problem is that we need more health care than people in other countries. Why would that be the case? One answer might be lifestyle and diet. Michael Pollen calls us the “people of corn.” Maybe our diet which is predominately corn based carbs, processed foods and corn fed protein is causing us to need more health care because people in other places just eat better (or less). Being a recent low carb convert/missionary, I suspect this to be the case. But that’s ultimately on us, as consumers. Once the collective mindset recognizes the dangers of the standard American diet, our collective health should improve which would cut our per capita health care costs.

Ok, I’m just fantasizing. It is more likely that we will realize some of the dire predictions about the adverse health effects of excessive carbs and sugar on baby boomers, which will even more dramatically raise health care costs. (See David Perlmutter’s Grain Brain, the books by Gary Taubes, and other books about the dangers of carbs and sugar and what may happen to us baby boomers).
But it’s not completely satisfying just to blame ourselves, let’s look at some of the usual suspects to see if we can lighten the load and stick it to the Man.

Health Insurers

(Full disclosure, I hate health insurance companies. I spent much of my career fighting them, so I am admittedly biased.)
You know what a health insurance company calls a payment to a provider for a critical or necessary health care service to an insured who pays premiums? A “loss.” That says it all.

There is an obvious structural conflict in having any financial intermediary pay for or decide what medical services the insurer should pay for. Insurance companies are in the business of making money (even the so-called “not-for-profits” like the Blues who are as bad as or worse than the “for profits”). The more “losses” they have, the less money they make and the less bonuses for their overpaid, immoral executives. (I told you I was biased) And it’s not significantly different when the carrier is an administrator of a company funded plan. Plan administrators work for the company, and their only allegiance is to the company/client.
Bottom lining it: When you have businesses which have an incentive to pay out as little as possible, so they can make a fair or unfair return on their investment, watch out.

The alternative is a government single-payer system. That is how health care is paid for in all or virtually all other industrialized countries. However we do have government pay heath care in the United States for the old, the poor, veterans and government workers. Results of these programs are complicated and mixed.

I spent a number of years in Israel which has a government single payer plan for all residents. However, they also have a private pay program for supplemental or additional care for anyone who has the funds and desire to retain private physicians or obtain treatments not approved or paid for by the government payer. I think that is the best of all possible worlds. And we already have part of that system here since most unconventional/complementary/integrative care is not insurance reimbursable, and hence is only practically available to the middle and upper-class.

The main criticism of single payer is exemplified by the fact that Canadians wait a long time for non-immediate life threatening operations, and some expensive, cutting edge technologies are not available in government pay England and other such places. But the rich English, Canadians and other foreigners suffering under single pay systems come here for such therapies or to go under the knife quicker, they do medical tourism in other places like South American and Asia. Even if we had single payer here, there is always going to be a demand for new technologies and uncovered services. Where there is a demand, the market will find a way to meet the demand, regardless of how many payers there are for standard, covered care.

Regardless of whether you buy into single-payer, I think all reasonable people acknowledge that the current private pay insurance system we have is a part of the problem. So any comprehensive solution has to involve a fairly radical change from what we now have. And anyone who claims that Health Savings Accounts and/or erasing state lines to promote competition is going to solve the problem is delusional or an ignoramus, or both.

Relatedly, there is one thing I begrudgingly give to those insurance bastards; if the system is going to force carriers to take the preexisting sick and really sick, mandatory enrollment for healthy people is a necessity. The numbers don’t add up any other way. I remember when this was a Republican thing, part of the “personal responsibility” mantra. Somehow, mandatory participation has been transformed into an oppressive government/freedom of choice issue. People supposedly have the God or Constitutional given right to choose not to purchase health insurance. That works fine until the freedom lover shows up at the ER without the money to pay for the needed care, at which point the freedom lover becomes a health care socialist, deadbeat or goes bankrupt. But the result is the same; freedom lovers end up not paying for their care. The rest of us foot the bill. However, reforming health insurance even via single payer will not solve our problems because costs are out-of-control and are not connected to regular market forces.

Hospitals and Providers

After receiving a hospital bill, most people are stunned and realize that the system is broken. The charges for hospital services and testing is staggering, and I would argue unsustainable. One of the biggest problems with Obamacare is that it did not address the cost of services, let alone impose any cost containment measures on facilities, providers or products. It seems to me that any real solution to health care has to involve some kind of price controls/tax/bulk negotiations or some other way to limit the ever escalating cost of medical services for basic necessary medical services.

Many integrative practitioners operate on a cash basis and I don’t see that any such cost containment measures would directly affect them, for the same reason that cosmetic surgeons can charge whatever the market will bear. If you’re cash based, the market, your skill set and your marketing savvy will ultimately determine the true and fair cost and value of your services. However, when the insurance companies rather than the patient pays, the market gets distorted. In that market, without some kind of cost containment mechanism, I think our health care problems are insurmountable.

Apart from lack of cost controls, I think physician mindset and education are major sources of our health care problems. As the CAM (complementary and alternative medicine) mantra goes, we have a disease system, not a health care system, and there is not enough focus on prevention (with some notable exceptions like the anti-smoking and Trans fats campaigns). Money may be behind this as well since medical interventions for diseases and conditions is where the money is, not prevention. While this may be primarily a public health issue, it’s also a physician mindset issue. The best concrete example of this is the lack of medical school training in diet and nutrition. Most CAM practitioners who receive nutrition training at one of the nutrition academies understand how woefully inadequate their medical school training was on the subject.

Finally, I know a lot of CAM practitioners live in the hope/fantasy that all their CAM services will eventually be insurance reimbursable. There are some clever insurance reimbursement ideas which float around from time to time. And every once in a while something good happens, like the American College of Nutrition and its certifying board’s success in getting their nutritionist members qualified for Medicare reimbursement. But on the whole, my opinion is that Medicare and the private pay carriers are never going to knowingly pay for hard-core CAM services like chelation or first-line CAM therapeutics. But there’s no downside to keep hoping and for the community to keep fighting the good fight. By the way, I tell my chiropractor clients and audiences that if they can live without insurance carriers, they’re better off. I feel the same way about CAM physicians. The current and future reality is that the services of CAM practitioners are not for the poor of pocket.

And Let’s Not Forget Big Pharma

In the CAM community, big Pharma is a natural and inevitable target. One issue is of course high drug prices, but it’s complicated because of the length of time and extreme expense it takes to get drugs approved in this country. Still, it was a bad move not allowing the Government to negotiate with Pharma the prices for Medicare drugs. We can thank the Pharma lobbyists and the folks in congress they paid-off. Reversing that enormous Government handout to Pharma is one of the first thing our probable future Dear Leader should do after his coronation, since he’s such a good negotiator (self-described).

Another widespread problem is how Pharma is corrupting scientific research by burying negative research and buying-off physicians. There has been some small corrective action in the last few years. However, savvy physicians shouldn’t accept everything that’s written even in the most prestigious journals, because as some have argued, the mainstream publication system has been corrupted by Pharma money. I also think part of the publication problem is what I referred to in Galileo’s Lawyer as “the church of medical orthodoxy” type thinking, or in Kuhnian terms, the terror of normal science. (http://rickjaffeesq.com/galileos-lawyer/ )

One recent positive is the proliferation of open access, online journals. Their increased popularity among scientists is in part based on the faster turn-around time from draft to publication than mainstream print journals. Open access journals should also lessen the corrupt pharma influence and the anti-CAM stranglehold on the mainstream print journals. There are rumblings and start-ups promising even faster and almost immediate sharing of data, which could change the paradigm in research, the dissemination of results, and expedite access to new technologies.

A final thought: I am hoping there’s a special place in Hell for the Pharma companies and their physician co-conspirators who are getting our kids hooked on ADD drugs so they can be worked up the chain to SSRI’s and be life-long Pharma customer/addicts. I also think some of the teachers may be complicit by too quickly demanding that their high spirited students be drugged. I’d like to see teaches get mandatory training on the dangers of ADD drugs and SSRI’s. May the manufacturers and their pushers get what they deserve.

Provider Shortages for Basic Healthcare

As a consumer, I think there is a shortage of primary care physicians. Witness the uptick in the “concierge” medical model. The idea that you need to pay an annual fee just to have reasonable access to a primary care physician suggests to me that there are not enough of those folks out there.

The other big factor might be that it’s still incredibly hard to get into medical school, and/or there aren’t enough medical schools. It could be that the medical profession likes it that way, to limit competition, and keep the pay higher than it might otherwise be if there were 20% to 30% more medical school graduates each year. So trade protectionism might be a factor.
Interestingly, I’ve seen an increase in the use of physician extenders like NP’s and PA’s in some medical practices and clinic chains. If the model works out, it might help increase access to basic care.

My Brethren, the Lawyers
Of course, let’s not forget the lawyers, and in particular the plaintiff’s malpractice bar. As a result of probably justified complaints from physician groups and others, many legislatures like Texas imposed tort reform. Among other things, these laws limit the pain and suffering component of malpractice awards, which is where the big money is for small and mid-size cases. After the changes took effect in Texas, the plaintiffs’ (and defendants’) malpractice bar was decimated. While there may still be defensive medicine because of feared malpractice cases, tort reform hasn’t slowed down the spiraling costs of health care. So either the lawyers weren’t the problem or tort reform didn’t put enough of them out of business. (I’m guessing most of you subscribe to the latter view.)

And Last But Not Least, the (Over) Regulators

Let me start with a positive. Because of DSHEA, (the 1994 dietary supplement act) we have very good access to anything that can reasonably be called a nutritional supplement.
Now the bad news
The FDA is trying to undo or undermine DSHEA by its new guidance on new dietary ingredients. If the FDA gets it way, any supplement that wasn’t marketed before 1994, in exactly the way or form or formula it was sold pre-1994 will not be able to be marketed without submission of data to the FDA. That will kill the innovative part of dietary supplements.
The other bad news is that because of the regulators and DSHEA there’s not much which companies or even physicians can say about supplements or herbs, in writing at least. The FTC and FDA police the supplement companies, and increasingly, the state medical boards are going after physicians who make “unsubstantiated” or “false and misleading” claims about supplements and their CAM treatments. Parts of the federal law could have been written by Kafka. It is a violation of federal law for the manufacturer/seller and it is a violation of state law for the physician to provide truthful information about the research supporting a supplement, herb or CAM modality unless the research meets the regulators’ threshold of adequate scientific substantiation, a standard which very few supplements or CAM modalities can meet.

I had a case where the FTC went after an herb seller for making the truthful claim that chaparral was used by Native Americans to treat cancer, because of the “implied claim” that it cures cancer. An “implied claim” can be anything the regulators want it to be, and in effect eliminates a supplement manufacturer’s ability to provide any scientific information about virtually all nutritional products.

Physicians must be circumspect and vigilant about what they say about their services and products because the sceptics and quack busters are filing false advertising complaints against CAM physicians based on their web site claims. Nasty stuff; no doctor-patient relationship required. Just a wacko zealot with a computer sitting a thousand miles away with too much time is all it takes to cause grief to a CAM practitioner. Medical boards love these complaints since it’s an easy and cheap way to get practitioners. No medical experts reviewing charts. Just a review of the web site.

The regulators’ position is that they are protecting the public from misinformation. But it seems that much of their efforts are truth inhibiting and are based on an outdated paternalistic view from the days when medical information only came from Marcus Welby, M.D. and Reader’s Digest. So for sure, the regulators are part of the problem, at least for the CAM part of health care.
Of course, the FDA is also in the process of shutting down the hundreds of clinics which offer autologous stem cell transplants to tens of thousands of patients, including post mastectomy patients doing breast reconstruction surgery. This is horrible, and for more details see my previous post, and other posts at www.rickjaffe.com

What’s the solution?

Simple to say, hard to achieve.

Better, more thoughtful and compassionate regulators, congress folk who aren’t controlled by pharma and the insurance carriers and who can focus on people rather than their narrow political agenda.
What’s it going to take to get that? Probably nothing short of a national public outcry. Could be that if enough baby boomers live into their 80’s the system could just break. Between health care and social security, it’s going to be very expensive for our kids when they reach their 50’s and 60’s.
Short to intermediate term prediction: that’s easy, continued stumbling along, with ever increasing costs, decreasing effectiveness, at least at the population and macro level. More people have to spend more money on health care and medical tourism continues to increase.

Or so I see the health care world.

Rick Jaffe, Esq.
rickjaffeesquire@gmail.com

www.rickjaffe.com

THE SCARIEST ATTACK ON PERSONAL FREEDOM YOU’VE NEVER HEARD OF/ THE BIG TAKEWAY: If the FDA gets its way, the most popular post mastectomy breast reconstruction procedure will become illegal, and so will many other life enhancing procedures

THE SCARIEST ATTACK ON PERSONAL FREEDOM YOU’VE NEVER HEARD OF/ THE BIG TAKEWAY: If the FDA gets its way, the most popular post mastectomy breast reconstruction procedure will become illegal, and so will many other life enhancing procedures

Using your own stem cells, tissue and body parts without FDA interference should be a no brainer and a slam dunk, but it isn’t. I mean it’s your own body. How can the federal government interfere with your privacy and autonomy right to use parts of your own body as a treatment? The short answer is they can and if the FDA gets its way, future patients are not going to be able to use their own cells and tissue as tens or hundreds of thousands of patients, including breast reconstruction patients, have been doing for years.  Here is how the FDA is going to stop you from using your own stuff (stem cells and tissue like fat, or what the FDA calls “Human Cells, Tissues and Cellular and Tissue-Based Products” or “HCT/P’s as the federales call it.) But first some short regulatory history

How the FDA thinks of your body parts

Until the 1990’s, removing and reinserting body parts wasn’t regulated by the FDA, other than making sure that it was collected and maintained in a safe and sterile way. But then stem cells started to become a popular research field, and all the hype started about the magical therapeutic power or potential of stem cells. So in the late 1990’s the FDA started making noises about regulating stem cells and other human tissue. That would include both your own stems cells (called in medical and regulatory parlance “autologous”, and someone else’s stem cells or tissue (“allogenic”).

In 2005, the FDA published its final rules concerning HCT/P’s.  (Here is the link to 21 CFR 1271, for the legally curious.  (http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=1271)

The regulations created three different regulatory pathways for HCT/P’s; some were considered “new drugs” which required full FDA approval, which is a hideously expensive and unreasonably long endeavor. (This is sometimes referred to as the 351 pathway); some just required that the facility using the stem cells or other tissue register with the FDA (sometimes referred to as the 361 pathway), and if the stem cells or other tissue was removed and reinserted into the person in the “same surgical procedure,” the FDA didn’t regulate it at all because it was the practice of medicine which supposedly the FDA doesn’t regulate.

The FDA’s 2005 regulations introduced two biologic/analytical binary concepts which it uses as criteria for determining which of the three regulatory pathways applied to a particular use of an HCT/P: Homologous vs. non homologous and minimally manipulated vs. more than minimally manipulated.

To oversimply, a use of an HCT/P is homologous if the use of the material in the donor location is the same as in the recipient location. When the FDA regulations were first put out in 2005, there was some vagueness or flexibility in what constituted the same or homologous use.  The regulations stipulated that if the use was homologous, the use might only require registration under the 361 regulatory pathway (and if a number of other requirements were met). But if the HCT/P use was non-homologous (meaning that the use the cell had in the donor location was different from the intended use in the recipient location), then regulatory magic turned your HCT/P into a 351 new drug with the aforementioned hideously expensive and unreasonably long consequences. Seems crazy, but that was the reg.

Now you usually can’t just take stem cells out of your body; they have to be separated from fat, bone marrow, or blood. That means after you take out the material, the stem cells or other material is separated, using either a chemical agent or a mechanical process (like a centrifuge). Usually there is also other processing needed before the material is reimplanted.

The processing of the material gave rise to the second binary concept, minimal manipulation vs. more than minimal manipulation of the HCT/P.  If the cells are not more than minimally manipulated, the procedure could be done with just 361 facility registration (assuming homologous use and several other technical requirements set out in the 1271.1 (Here’s the link to the regulation if you’re turned-on by that kind of thing.  http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=1271.10 )

But if the cells were more than minimally manipulated, then you are thrown back to the new drug path. Remember, we’re talking about your own stuff. Under the rule, there was some ambiguity as to what was or wasn’t minimal manipulation. Back when the rules first became effective, I had a client who wanted to take out fat, separate the mesenchymal stem cells and reinsert the cells back. To me, it seemed legal under the rules. I called the FDA a couple times to make sure I was right, and everyone at the agency I talked to agreed. But that was then.

Since that time, there has been an explosion of stem cell clinics taking fat from people and reinjecting the stem cells. Worse from the FDA’s point of view, many of these clinics were making all kinds of miraculous cure claims. Problem was there was no proof that the claims were true for most of the diseases or problems claimed to be cured or helped by stem cells. That made the FDA unhappy, which is bad thing. (Trust me, I know how bad it is when the FDA gets unhappy).

Long story short: the FDA decided to clamp down on all of these clinics, and used two of its most effective tactics.

First, it sent cease and desist letters (what the FDA calls a “warning letter”) to a couple of the most visible offenders. (Hit the big guys first to scare-off everyone else). The FDA publishes all warnings letters, and that got the industry’s attention.

Second, in the last year or two, the FDA issued four draft guidance documents “clarifying” the meaning the two key terms homologous/non homologous use and more than minimally manipulated (or not). But the FDA didn’t just clarify these terms; it rewrote the rule to knock-out some of the most popular and effective medical procedures, perhaps most importantly, the most popular post mastectomy breast reconstruction procedure with flap surgery.

No point getting too much in the weeds of how the feds did it (see my last post if the legal weeds is your thing.

http://rickjaffeesq.com/2016/09/13/fda-draft-stem-cell-guidance-documents-exposed-improper-rulemaking-bad-science-heartless-public-policy/

But here is the most egregious example that shows the abject stupidity of what the FDA is trying to do:

Flap reconstructive breast surgery takes fat (usually belly fat) and builds up the breast. Under the guidance documents, the main function of fat is structural. The main function of a breast (in a woman) is lactation.  (Some presenters of the female persuasion who presented at the stem cell public hearing were really annoyed about that characterization).  Lactation is a different function than the function of belly fat (structural). Therefore the use the flap procedure is non-homologous, which means the use has to obtain full FDA approval before it can be used outside of FDA approved clinical trials. Many of the presenters used this as an example of why the draft guidelines are wrong. (more details in my last post: http://rickjaffeesq.com/2016/09/13/fda-draft-stem-c…ss-public-policy/ ‎)

The fact that tens of thousands of women have had this procedure and are walking around feeling better about themselves is irrelevant to the FDA.

I’d like to meet the idiot who came up with this. Better still, I’d like to put him/her/them in a room with a few mastectomy patients who need the flap surgery, and let them explain to the patients why their belly fat and breasts are any of the FDA’s business.

Remember when I said that the FDA told me that docs can take fat out, separate the stem cells and reimplant them without FDA oversight if it was done during the same surgical procedure? Well that’s out the window now. Under the guidance documents, separating the stem cells from the fat constitutes “more than minimal manipulation” of the HCT/P and requires full-on FDA approval because your stem cells are now an unapproved drug and they can’t be reinjected into you without being approved by the FDA.

I get that the FDA is concerned about clinics making unsubstantiated claims. There are also a few well-publicized incidents of harm, most notably some clinic which apparently let a nurse practitioner inject stem cells in the eyeballs of patients. What idiot decided that was a good idea? Still, the remedy to that kind of problem is or could be a combination of civil actions by the patients, criminal prosecution, professional licensure proceedings, or state action to shut the place down. As to claims, The FTC has jurisdiction to deal with false claims issue, as does the state under state consumer deceptive trade practice laws.

The point is that there are a lot of options for dealing with places which do crazy things, harm patients, or make outrageous claims. The solution shouldn’t be that FDA closes down the entire autologous transplant industry because of some exceptional bad examples. Describing this as overkill is an understatement.

So here’s what needs to be done. The FDA has to rescind the draft guidance documents and start over. In the interim, the FDA should get the hell away from my stem cells and other body parts and yours too, at least if we want to take them out and put them back in our own bodies.

What can you do about it? Plenty

The public hearing of the FDA’s stem cell guidance documents ended last Tuesday, September 13, 2016, but the public comment period is open until September 27th.

If you think you might ever need to use your own stem cells or other body parts in the future, or if you know anyone how might need them, or if you think that what the FDA is doing is a bad idea, then write, fax or email the FDA and tell them. Here are some possible points:

  1. Withdraw the four HCT/P guidance documents
  2. Get out of the business of regulating a person’s use of his own body parts
  3. Any opinions  you might have about where they should place their draft guidance documents, or such other opinions you might have on this regulatory exercise, mindful of the rules of polite discourse, based on your discretion and/or temperament.

Maybe if a few hundred thousand people contact these jokers, they might get the message.

 

Where to send your comment?

Here is the information I found about sending comments:

“Submit written/paper submissions as follows: • Mail/Hand delivery/Courier (for written/paper submissions): Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.” Instructions: All submissions received must include the Docket No. FDA-2015-D-3719 for “Draft Guidances Relating to the Regulation of Human Cells, Tissues, and Cellular and Tissue-Based Products; Rescheduling of Public Hearing; Request for Comments.”

If you want to go the email route: do one or both:

CBERPublicEvents@fda.hhs.gov

Or go to Federal eRulemaking Portal:  http://www.regulations.gov.

FYI: here is the sub heading from the eRulemaking Portal:

Make a difference. Submit your comments and let your voice be heard.

That’s good advice.

Here is the FDA Federal Register Notice about the Hearings, where you can read the written comments, and more details on submission of comments.

http://www.fda.gov/downloads/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/UCM497048.pdf

If you want more details about the issue or love the weeds, here is a link to all my recent posts on this topic. They’re in reverse chronological order, so start with the last one “Are stem cells over?”

http://rickjaffeesq.com/category/stemcells/

 

Tally ho!

 

Rick Jaffe

www.rickjaffe.com

 

(To Clinton Miller, I hope we can do you proud.)

 

 

Press Release re Dr. Bob Sears case with Ca. Medical Board

Press Release re Dr. Bob Sears case with Ca. Medical Board

“I represent Dr. Bob Sears in the California Medical Board’s case against him for writing a medical exemption from vaccination.

We take the board’s accusation seriously. But this case is very clear: this child had two unusual and severe vaccine reactions and his situation warranted a medical exemption. To continue vaccination could have put the child at risk of further harm.

All physicians have an ethical duty to do no harm to a patient. This is no less true when a child suffers serious side effects from any medical intervention.

We anticipate this case will do much to further public education on the importance of recognizing severe vaccine reactions and providing informed consent for medical care. ”

Rick Jaffe

 

FDA Draft Stem Cell Guidance Documents Exposed as Improper Rulemaking, Bad Science and Heartless Public Policy

FDA Draft Stem Cell Guidance Documents Exposed as Improper Rulemaking, Bad Science and Heartless Public Policy

Today was a good day for people who want continued access stem cells outside of clinic trials, and also for people who want the FDA to allow faster access to this promising technology.

There was a wide spectrum of opinions. Some stem cell companies involved in clinical trials wanted the non-clinical trials clinics shut down. But at least there were representatives from some of these “unproven” clinics and interest groups who made some important points about the rights of patients and how the needs of patients are not being met by the current clinical trials model as it applies to stem cells. I heard a number like 250,000 people are not getting the stem cell treatments they need because of clogged research and regulatory hold-ups. There were numerous calls from very serious, highly credentialed people for the FDA loosen its death grip (my term) restricting access to these therapies, and the thrust of most of these presenters was that these draft guidance documents make thinks much worse.

 

The guidance document are really bad and deny access for many

And that was the big takeaway for me; that the guidelines were much, much worse than even I thought. I understood that the guidelines would make illegal the  21 CFR 1271.15 exempt same surgical procedures provided by many of the 600 plus unregulated stem cell clinics.

But what I didn’t understand until Monday’s hearing is that the FDA intends to radically change the rules so that, for example, the most popular form of breast reconstruction surgery post mastectomy (flap something) would become illegal under the new guidelines. Many other popular and widely successful procedures in other areas like orthopedics would be eliminated (outside of clinical trials). We’re not talking unboarded docs with no relevant experience who take a weekend course and starts shooting people up with stem cells. We’re talking about big-time breast reconstructive surgeons, highly regarded orthopedists and other highly skilled and specialized physicians who have successfully worked with tens of thousands of patients. If the FDA gets its way, according to these folks, Poof! These best practices transplant procedures are gone.

Fortunately, there were some very smart professionals making presentations, including an extremely knowledgeable law professor from Boston College, Mary Ann Chirba. She and several other people with regulatory expertise made the case that this whole guidance exercise was an illegitimate attempt to pass new rules without complying with the rulemaking requirements under federal law.  Works for me!

They and others honed in on the radical revisions to the two key preexisting terms/concepts used by the FDA to work its illegal magic: homologous use and more than minimal manipulation.

 

What’s a “main function?”

It was also pointed out that the guidance documents invented a new concept not existing in the statute or rule, namely the “main function” of a cell or HCT/P which is used as a way of forcing stem cell procedures from just registration under 362 into the IND/NDA drug approval path. It was argued persuasively by several regulatory experts that the creation of this new concept and its resulting transfer of many heretofore legal uses of stem cells into illegal new drug products turns the guidance documents into rulemaking without following federal administrative rulemaking procedures.

 

The FDA doesn’t understand what fat does

Another extremely cogent criticism made by a variety of people including Professor Chirba, other regulators and by both of the two top presenting stem cell researchers, Arnold Caplan and Keith March had to do with the FDA’s view of fat. According to the guidance documents, fat just has a structural function. But these presenters and especially March and Caplan showed that the FDA’s view was biologically unsound.  Fat has definite, known and extremely important non-structural uses, starting with energy storage and continuing to assistance in the healing function. The FDA’s unscientific, unsubstantiated restriction on fat allows it to find most of the important uses of fat and fat stem cells illegal as either non-homologous or as a more than minimally manipulated product. The FDA was absolutely and repeatedly pummeled on this point by my count, at least a half dozen very, smart experts.  I don’t see how even the FDA, which has a very particular agenda, is going to be able to hold on to its limitations/restrictions on fat/adipose tissue.

 

The Big Guys say regulations are holding back progress

The two big-time researchers (Caplan and March) also made the point that the regulatory climate is holding back research. Caplan said that some bone marrow pioneers had observed that if they had the regulatory environment back then as what exists today, bone marrow transplants might never have taken off. Ouch!

Interestingly, Peter Rubin, the plastic surgeon who last Thursday presented the inspiring cases of reconstruction work from fat transfers, presented again. This time he was more critical of the FDA and stated that many of the most successful reconstructive plastic surgery procedures, including breast reconstruction would become illegal under the draft guidance documents. He and many other excoriated the draft homologous document which classifies fat tissue for breast reconstruction as non-homologous because the primary purpose of the breast is lactation. Several of the female presenters had some polite but pointed words to the FDA about that.  Most of the day’s presenters agreed that regulation/regulatory expense was delaying bringing this technology to patients.

 

The 3 Billion Dollar Player Weighs-in

The biggest dollar player was the California Stem Cell Institute which has a 3 billion dollar budget and 12 research centers. Its director spoke, and his message was clear, concise and right on the money (and with 3 billion, it should be). The FDA has to loosen-up its grip and find an intermediate path between unregulated stem cell clinics and full-on clinical trials, because there is a desperate unsatisfied need and that need will be satisfied  – just as water flowing down a hill will find a path –  with or without the FDA’s help. He was very persuasive. Reminds me of an old TV ad: “When EF Hutton talks, people listen.”

Interestingly, no one picked up on what I though was the most egregious over reach in the draft guidelines, namely that the FDA guidelines silently incorporated or read the homologous and more than minimally manipulated requirements from 361 registration facilities (1270.10) into the exemption for same surgical procedure places (1271.15). Under the actual rule (1271.15) same day surgical procedures can do non-homologous and more than minimally manipulation. At least those two terms are not in that rule. Legal Method 101 instructs that if terms are in 1271.10 but not in 1271.15, then the 1271.10 terms and restrictions cannot be read into 1271.15 which is what the FDA is doing based on its interpretation of “‘such’ HCT/P’s.” (Maybe too technical.  I’ll have more to say about that another time.)

 

Maybe there is a viable lawsuit

Something else I realized as a result of a couple of the astute presentations. I said in the last post that you can’t sue on a guidance document because it’s just the agency’s “current thinking.” However, if a guidance document is really disguised rulemaking without meeting the rule changing requirements, then maybe there is a lawsuit. Many presenters were clear about the fact that these guidance documents are disguised rule changes, so I’m now more optimistic about the chances of a legal challenge.

 

People are Mad and are going to do something about it

And speaking of possible legal challenges, while all of the presenters were very professional, very cordial, ostensibly courteous and complimentary to the FDA panel members on the dais, I sensed that quite a few, many in fact, were pretty upset by what the FDA is trying to do with the draft guidance documents.

So here is my prediction/wish/what I hope to make happen.  There won’t be one lawsuit filed if the draft guidelines go into effect. There will many lawsuits. I don’t think these folks are going to go quietly. My sense is that the big players, sophisticated players, like Rubin, the fellow who started a society and has 5800 members, the guy with dozens of clinics, they have seen too many good results to give up their most effective tools. All these guys either run or are closely connected to prestigious professional societies and  I predict that many of them are going to try to stop these guidance documents, in court or in Congress.

I hope for everyone’s sake the FDA really listened today, because people are mad as hell and they’re not going to take it. They want better and quicker access to this new technology, and my hope is they will get it.

Rick Jaffe, Esq.

www.rickjaffe.com

 

 

 

More on The FDA’s Stem Cell Public “Workshop”: Stem Cell Clinical Trials aren’t the Answer for Everyone

More on The FDA’s Stem Cell Public “Workshop”: Stem Cell Clinical Trials aren’t the Answer for Everyone

Not unexpectedly, the organizing theme of the FDA’s Stem Cell workshop  was that patients should only be able to receive their own stem cells for non-homologous use in FDA approved clinical trials until FDA marketing approval (i.e., until a New Drug Application is granted for some non-homologous use).

I’ve been involved in legal/policy issues relating to clinical trials for a long time. I understand that clinical trials is the standard of care for patients when FDA approved treatments (on or off-label) are not available for whatever reason.  Still, I’m here to tell you that from the perspective of patients with life-threatening/incurable diseases, clinical trials aren’t always the best answer for them. Here is why I think so.

  1. The Purpose of Clinical Studies is to Test Drugs, not to cure patients

 

The fundamental and indeed the stated purpose of a clinical trial is to test the safety and efficacy of a therapeutic intervention, not to cure a specific patient of a specific medical condition.

One of the presenters mentioned some empirical data indicating that there is a disconnect between this purpose and the patients’ understanding of the meaning and purpose of clinical studies. My take-away from what he was saying was that many people mistakenly believe that the primary purpose of a clinical trial is to get the patient better. That misunderstanding is consistent with my experience over the course of several decades interacting with patients on clinical trials.

That the purpose of clinical trials is to test interventions, not to cure patients has specific practical consequences for patients which sometimes mean that patients do not get optimal care for the good of the study.

 

  1. Some Phase 1 study patients may not get enough of the drug/intervention

In phase 1 or toxicity studies, patients are specifically told that the purpose of their participation is to study a drug’s toxicity, not to test the efficacy of the drug, and while the investigators hope the patient will obtain some benefit, that is not the goal of the patient’s receiving the drug.

Early patients in some phase 1 studies receive relatively low doses of the drug, based on what the investigators believe to be the therapeutic dose. Doses often get escalated in later subjects in a phase 1 study. I believe this is common in toxicity studies. It’s kind of like you shouldn’t buy a car that was made right before a holiday weekend or on a Monday. Sometimes, it’s not ideal to be one of the first study participants in a phase 1 study, because you may not get what the investigators expect to be an therapeutic dose.

 

  1. How are you helped by a placebo?

Some studies are placebo controlled. Obviously there is no therapeutic intent for those patients. In cancer and other life threatening diseases, placebo controls are no longer employed, and in some placebo controlled studies, placebo recipients are sometimes offered the study drug later or much later.  Still, if a patient is in a placebo controlled study. There’s a 50% chance the patient won’t receive the study drug (at least initially).

 

  1. In Phase 3 studies, you still might not get the study drug

You still might only have a 33-50% chance of obtaining the study drug in a phase 3 trials,

Most phase 3 studies compare the study intervention with FDA approved standard of care therapies. Patients are randomly selected into the different arms of the study, arms being the different groups that receive the study drug or the standard of care therapy. Some studies involve more than one standard of care control arm/option. If there are two different control arms, there is only a 33% chance of receiving the study drug and a 66% chance of receiving a therapeutic option which probably hasn’t worked too well.

 

  1. Most drugs in clinical trial drugs are not ultimately approved

It is true as reported at the Workshop that less than 5% of therapies entering clinical trials obtain NDA approval, but 1. There could be reasons other than efficacy why that happens, 2. Some agents just don’t work on a high enough percentage of patients to justify NDA approval but they do work on some, and if you’re one of those lucky people, you’re a happy camper. So I don’t see the fact that there is a low approval rate of study drugs as strongly supporting the banning of stem cells outside of clinical trials just because of that fact.

 

  1. What happens after the clinical trial is over for you?

Therapeutic interventions in clinical trials are usually given over a relatively short period of time and often there is some surrogate endpoint or target which is less than a complete cure. In cancer it’s called a response.  Let’s say you get a response, or the target improvement in pulmonary function or whatever the parameter the drug is intending to influence. You’re a responder but not cured. Can you still get the Intervention if you need it?

In drug studies, there is a serious issue of continued access to study drugs after the termination of the study. Drug companies are not required to provide study drugs after the conclusion of the participant’s time in the study. There is a movement afoot to change that. I don’t know whether that is an issue in autologous stem cell clinical trials, but it could or would be if the guidance documents become final because it would then be illegal (supposedly) for the person to have access to his/her stem cells after the study.

 

  1. The Biggest Problem with Requiring Clinical Trials for All Autologous Stem Cell Transplants

Here is the big one. The underlying assumption of the FDA’s and the Workshop’s position –that autologous stem cell transplants should only be available in clinical trials – is that any patient who wants an autologous stem cell transplant can enter a clinical study. That seems unlikely, but that’s just my gut feeling. I’m not familiar enough with the stem cell clinical trials to know whether there is a large unmet demand, but in many diseases like cancer, a relatively small percentage of patients enroll or can enroll in clinical trials. In cancer, I think it’s something like 3 or 4%, and I’ve seen numbers like 40% of cancer patients would enter a clinical trial if they could. There are many reasons why some patients aren’t eligible for clinical trials, tied to a variety of factors. Some are too sick for the protocol entry criteria. Some may have had a prior disqualifying treatment (like a previous clinical trial). But my supposition is that there are many more patients out there why would participate in clinical trials involving stem cells,  but can’t for one reason or another.

Assume that to be the case.

What are the policy implications and practical consequences?

On a policy level, there is going to be an arguably significant number of patients who have no therapeutic options. Of course the immediate response to that is what good is an unproven, possibly dangerous option? While it’s a fair question, it’s a better question for foreign interventions than a therapy derived from the person’s own body, because as stated, there are fewer safety concerns.

And, respectfully, I think it’s a too facile response by academics not facing no treatment options for a life-threatening condition. Let these guys who are so quick to cut-off treatment options come back after they have walked in the shoes of these terminal patients and their families.

What about the practical effect of the FDA’s plan to make illegal same day autologous stem cell procedures?

That’s easy and no crystal ball is needed. The cat’s already out of the bag, the cow has left the barn. Patients want the ability to use their own stem cells to treat a wide variety of medical conditions. Former Governor Rick Perry and Bart Starr believe in the therapy, and I dare say tens or hundreds of thousands of others do, and would try it in a heartbeat in there was no other reasonable alternative, whether or not there is an existing clinical trial for which they could qualify. If you’re the Governor of Texas, you can have someone shoot you up, consequences be dammed. Others will have to find other options.

It seems obvious that the effect of the FDA’s intended action will be to drive more people into stem cell tourism and to places which have less substantive and facility regulations than in the U.S. That’s not necessarily a good thing.

I hear one highly-regarded stem cell transplanter might suggest an expansion of the facility registration requirements (contained in 21 CFR 1271.10) to same day transplant facilities (exempt from that requirement under 1271.15). It would certainly enhance the safety profile of these clinics by providing some federal regulatory oversight. It’s a good and creative idea, but it would require a revision to the current regulations.

I have to believe there are far less draconian solutions to the legitimate safety, training and false or unsubstantiated claims concerns which worry the FDA and the institutional players. But maybe it’s time for some creative thinking, rather than a knee-jerk reaction to eliminate what seems to be much needed treatment options for many patients.

To my presenter friends and colleagues, looking forward to hearing what you have to say, (via the web anyway).

Best,

Rick Jaffe, Esq.

www.rickjaffe.com

 

 

 

 

The most important question at the FDA Stem Workshop was the one not asked

The most important question at the FDA Stem Workshop was the one not asked

I didn’t hear the first couple of presentations in the FDA’s Stem Cell workshop. However, I did listen from mid-morning to Dr. Irv Weisman’s closing.  Yesterday’s post was my take on the highlights.  http://rickjaffeesq.com/2016/09/08/really-tough-day-stem-cell-advocates/

Upon reflection, what is disturbing to me is what I didn’t hear, namely a discussion or even a mention of what I think should have been the most important question in the autologous stem cell public policy debate: whether autologous stem cell transplants should be treated differently by the FDA from other types of therapeutic interventions because the material comes from the person’s own body.

This question is both very simple and quite complex. It is complex because it involves not only scientific issues, but also public policy, legal jurisdictional issues, and even federal constitutional issues. And yet, I heard nothing about these issues from the FDA’s blue ribbon panel of thought leaders despite the extremely impressive academic credentials, accomplishments and experience of the workshop participants.

The question is also complicated because it’s only by virtue of the FDA’s as yet untested, convoluted and counterintuitive interpretation of its rules that it has arguable jurisdiction over most of the stem cell clinics it intends to put out of business with its draft guidance documents. (We’ll get into the legal weeds of statutory interpretation of the applicable rules later.)

The entire workshop’s discussion including the ethics presentations was predicated on the tacit premise that autologous stem cell transplants are just like any other therapeutic intervention. As I think about it, that tacit assumption led to a bizarre disconnect in the presentations.

Almost all of the presenters excoriated the use of stem cells therapeutics outside of clinical trials. Yet the most upbeat and heartwarming presentation, to me, was from the plastic surgeon, Peter Rubin who brought forth examples of fat transplants given to wounded warriors and breast cancer survivors. I don’t recall him saying that every one of his transplant patients were treated in clinical trials. I believe that many people receive autologous stem cells or fat HCT/P transplants for homologous use without FDA approval, including for reconstructive work such as he is doing.

Using a person’s body as the transplant material avoids many of the safety issues which occur from a foreign source, whether the source be another person’s stem cells, a chemical or an extract from a plant (like digitalis or vincristine,). Most non-self-interventions likely have L/D (lethal dose) toxicity limits and attendant serious safety concerns in terms of dosage and side effects. Not so with autologous HCT/P transplants.

Sure, in any transplant there are contamination issues, but that applies to any autologous (or allogenic) HCT/P or blood product transplant and thus is not unique to “unproven” stem cell transplanters. So if many of the safety concerns of drug therapies aren’t applicable to autologous stem cell transplants, then it all comes down to efficacy, and whether and how much proof is necessary for a person to be able to use material that comes from that person’s body.

Although none of the luminaries raised the issue yesterday, I think it’s worth asking why aren’t people allowed to use a technology or service which isolates and removes a body part (HCT/P’s)   and reintroduces all or a part of it back into their own body without federal government oversight.  Same day autologous transplants are basically medical procedures.  Normally the FDA is not in the business of regulating medical procedures because it’s the “practice of medicine” which normally is within the jurisdiction of the state medical boards.

I don’t think it’s crazy to ask why should the federal govenrment be involved in medical procedures regulated by state law, and I’m not alone in thinking that autologous HCT/P transplants should have different rules, that the new drug rules do not or should not apply, and that these procedures should be policed by the state medical boards.

In fact, it was the FDA’s position as well, until the proliferation of these “unproven” stem cell clinics. (I discussed this in my first post on the guidance documents http://rickjaffeesq.com/2016/04/21/stemcells/)

I think the presenters yesterday forgot the actual reason the FDA is doing this whole public exercise: to sell its reinterpretation of its own rules. It’s not clear to me that the courts are going to go along with it. Here’s why.

 

It’s always a good idea to start with the law

 

The primary specific source of stem cell regulation is 21 CFR 1271.

In short, stem cells or more generally HCT/P’s (Human Cell and Tissue and Cellular and Tissue Based Products) are categorized or regulated in three ways:

  1. Solely under 1271 (basically tissue facility registration)
  2. As new drugs requiring full IND/NDA approval and registration, or
  3. Not regulated by the FDA

21 CFR 1271.10 sets out the requirement for registration but not new drug approval, and applies if, among other things, the product is not more than minimally manipulated and is for a homologous use.

Here is this rule:  http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=1271.10

 

What the FDA can’t regulate (at least until now)

 

21 CFR 1271.15 provides that the FDA doesn’t regulate the use of HCT/P’s if the extraction and reinsertion of the material is done in the “same surgical procedure.” Here is the exact language:

“(b) You are not required to comply with the requirements of this part if you are an establishment that removes HCT/P’s from an individual and implants such HCT/P’s into the same individual during the same surgical procedure.”

Under the plain meaning of this rule, all, most, many or some of what the “unproven” stem cell transplanters are doing is or was perfectly legal.

I have personal knowledge about this because around ten years ago, I contacted the FDA several times and asked them about this precise rule, on behalf of a client who wanted to do same-day autologous transplants and sought a legal opinion from me. I called the FDA several times, because sometimes you get different answers from an agency. Each time, I was told that the FDA doesn’t have jurisdiction over same-day autologous cell transplants. And so I gave my opinion clearing the procedure.

But that was before the 600 plus “unproven” stem cell clinics popped-up with all their purported claims of miracle cures. I’ve talked about this in a previous post. http://rickjaffeesq.com/2016/04/21/stemcells/ .

The point is that implicit in the words of 1271.15 –  the reinterpretation of which is the reason we are going through this FDA public show – is that autologous stem cell transplants are different from medical therapeutics involving a substance not originating in a person’s own body, and are regulated differently.

In fact, same-day, autologous stem cell transplants were not regulated by the FDA, until its regulatory interpretive epiphany precipitated by the wild west stem cell business.  That yesterday’s workshop didn’t even address the FDA’s prior position or the public policy and possibly constitution underpinnings of the actual language of the rule which divests the FDA from the business of regulating same-day autologous transplants, is disappointing, but not necessarily surprising, considering that the purpose of the workshop appears to be providing expert cover and justification for the FDA to make the draft guidelines final.

So how does the FDA get away with asserting jurisdiction over procedures which seem beyond its purview?  The answer is in its Adipose Draft Guidance document.

Here is the draft guidance document:

http://www.fda.gov/biologicsbloodvaccines/guidancecomplianceregulatoryinformation/guidances/tissue/ucm427795.htm

It’s a nifty trick: It interprets the phase “implants such HCT/P’s” as meaning that anything the unproven stem cell transplanters do beyond rinsing the material turns the HCT/P’s  into something other than the “such HCT/’s.” i.e. the HCT/P removed. Really!?

Here is the exact language where the magic happens:

“Limited handling such as rinsing and cleansing to  remove debris would allow the HCT/P from adipose tissue to retain the structural function, while other processing steps such as cell isolation, cell expansion, or enzymatic digestion generally would not.  Thus, if such other processing steps are performed that prevent the HCT/P from adipose tissue from remaining “such HCT/P,” the establishment manufacturing the HCT/P from adipose tissue would generally not be considered to meet the exception under 21 CFR 1271.15(b).”

It seems to me that the FDA’s interpretation is trying to backdoor the “more than minimal manipulation” idea contained in 1271.10 into the 1271.15 exemption from regulation and jurisdiction.  But the “more than minimal manipulation” language isn’t in 1270.15. It’s an agency add-in, years after the regulation was passed, and as stated, it was not the FDA’s original position.  The FDA’s textual jurisdictional reinterpretation seems far-fetched, inorganic and a somewhat desperate attempt to create jurisdiction over an activity which no one, including the FDA thought it had. Will it work?

In a fair fight; No, of course it wouldn’t work, but the FDA has the home court advantage on several fronts and these advantages might tip the scales.

First, in a court case, an administrative agency’s interpretation of its own regulations is given deference by the courts. I think the FDA’s position is attackable but you never know how far a court will bend over backwards to defer to an agency.

Second, you can’t sue the FDA on a draft or even a final guidance document because of non-intuitive and arcane non-finality rules of administrative law.

Ditto on warning letters. What that means practically is that if an “unproven” stem cell clinic keeps treating patients after receiving a warning letter, (which is basically an FDA cease and desist from engaging in illegal activity), the company can’t sue. It has to wait to see if the agency takes some kind of enforcement action. And here’s where it gets draconian.

Once there’s a warning letter, the doc or company is on notice that their actions are illegal, and specifically that they are violating what I call the FDA trifecta (introducing into interstate commerce an unapproved new drug, misbranding and adulteration).  Before the notice, or actual knowledge of the violation, we’re talking civil liability and/or at most a criminal misdemeanor, which means probation.

After the warning letter, if there are continuing “violations,” that’s an intentional criminal act which means felony and hard time.  So after a warning letter, the only safe play is to stop transplanting. (That’s what the Regenerative Science guys did after they got the warning letter. They sued, got thrown out of court, got sued by the FDA, fought the suit, but stopped doing stem cell transplants during the course of the litigation in case they lost, which they did.)

Bottom line is that docs and companies are disincentivized by the FDA and the system from challenging FDA action even if the action is or maybe outside of the FDA’s regulatory jurisdiction. But I’m hoping that if these guidelines go through and the FDA starts tossing out warning letters, some company is going to take a stand. I hope they do, and I’d like to be the one that smacks them down. Been there, done that, feels good (See chapters 2, 4 and 10 (on the first stem cell criminal investigation) in my book Galileo’s Lawyer).

So let’s get out of the legal weeds of administrative minutiea. It seems to me that the FDA is trying to exercise powers over same-day transplanters that it does not currently have. Maybe it should have that power, or maybe not. But whatever the ultimate policy answer should be, it needs to be discussed and studied. The luminaries yesterday didn’t do it, and I fear their myopic, one-sided view of the world and their ignoring of why autologous transplants are different and heretofore  beyond the purview of the FDA regulators needs to be discussed. Since it hasn’t been done in this workshop, the FDA needs to arrange a do-over with other more open minded, informed thought leaders.

There’s more to be said beyond these legal and policy points, but the hour is late and tomorrow is another day.

Rick Jaffe, Esq.

www.rickjaffe.com

 

 

 

 

 

 

 

 

 

 

And So it Begins: First Accusation filed against a Vaccine Exemption Writing Doc

And So it Begins: First Accusation filed against a Vaccine Exemption Writing Doc

 

As I’ve told the mostly unwilling-to-listen Vaccine Concerned Community, the real battle, and the one which will have the most direct positive or negative impact on this community is the future actions by the California Medical and Osteopathic Boards against the docs who write unlimited medical exemptions for the kids of the vaccine concerned. Well the future is now.

A few days ago, the Medical Board filed an accusation accusing a well known and highly regarded CAM doctor of gross negligence and inadequate record keeping for writing blanket exemptions. The only good news is that the exemption was written a couple years ago, so it’s not a result of any “conspiracy” between public health officials and the boards stemming from SB 277, but not to worry (or to worry), those accusation are coming too, though probably not for several months at least, and following the administration investigative process.

here is a link to the accusation. It’s a template as to what will likely follow in other cases.

searsaccusation

 

More as things develop.

 

Rick Jaffe, Esq.

 

 

 

A Really Tough Day for Stem Cell Advocates

A Really Tough Day for Stem Cell Advocates

The FDA’s Stem Cell “Workshop” just ended, and if you’re a person who wants continued access to stem cell therapies outside of clinical trials (or a transplanter of “unproven stem cell therapy”), it was a very depressing day. It was way worse than I had feared in my post yesterday.  http://rickjaffeesq.com/2016/09/07/fda-stem-cell-meetings-close-hand-stakeholders-sides-worried-one-side-really-worried/

Sure the two ethicists said that it was wrong and unethical to provide unproven stem cell therapy except in FDA sanctioned clinical trials. But the real punch came from two of the last speakers who discussed some horror stories of the severe harm caused by “unregulated” and “unproven” stem cell transplanters.

Stem Cell Horror Stories

Unregulated Stem Cell Therapy Causes Cancer

A Harvard fellow who co-wrote the recent oft-quoted case study of the stroke patient who traveled the world doing stem cells only to develop a stem cell treatment induced CNS tumor throughout most of his spinal column gave the gory details of the case, including a painstakingly detailed complete histology of the tumor. The only good news (if it can even be called such) is that, if I understood correctly, he concluded that the stem cells causing the tumor were not the patient’s but were the result of an allogenic transplant the patient received.

Unregulated Stem Cell Therapy Causes Blindness

The other presenter, an eye doctor, talked about three patients who received stem cells for macular degenerative disease. The procedures were performed in a South Florida stem clinic by a nurse practitioner, not under the supervision of a licensed physician. In each of the three cases, there was severe harm, requiring emergency surgery with catastrophic negative results to the eye sight of the patients. Of course these might be unusual and exceptional cases, but they will no doubt be used by the FDA to demonstrate the need for exactly the draft guidance documents which it is proposing to stop these medical horror stories from reoccurring.

FDA Implored to Stop the Maiming by Snake Oil Stem Cell Transplanters

Other members of the panel including leaders of disease advocacy/research entities all decried “unproven stem cell therapy” and implored the FDA to put these unethical stem cell purveyors of false hope out of business.

One panel member pointed out that the problem was just not in the U.S. and that there were these kinds of clinics in many parts of the world and because of the internet, it was easy to find out about them. One clinician lamented that when prospective patients call him and he has to tell them that he has no treatment options for them, the patients get mad and argue with him.

 They should all be delicensed!

The day’s moderator was Irv Weisman, who is one of the biggest names in academic stem cell research. In his summary, he suggested that U.S. physicians who perform unregulated stem cell transplants abroad to skirt U.S. regulations be delicensed.

To listen to these guys, it would appear that stem cells given outside of clinical trials have never helped anyone. In one sense that might be true. If all you care about is scientific data from well-designed FDA clinical trials, then they are right.  Anecdotal evidence is not science, and all seemed to agree that all the non FDA trial data which is published is not worthy of consideration because of design flaws.

There is a Consensus

The absolute, universal consensus of today’s workshop was that stem cells should only be available in the U.S. in FDA approved clinical trials, until such trials prove that there is a safe and effective non homologous use for them. And anyone who provides “unproven stem cell therapy” to patients outside of clinical trials is a greedy, unethical charlatan who should lose their medical license…That’s a tough message, but no doubt a welcome one for the FDA since the elimination of non-homologous autologous stem cell therapy outside of clinical trials is the purpose and end result of the four draft guidance documents.

One thing I am clear on: the positions taken today by the people the FDA considers the thought leaders in the field really, really need to be addressed by the stakeholders on the other side next week, head on.

I’m going to give this a good think over the next day or two and maybe make some suggestions to my presenter friends.

Stay tuned.

Rick Jaffe, Esq.