What is the “proper” amount of freedom of choice for patients in medicine?
What if the treatments in question are experimental and come with their own baggage of associated risks, personal costs, and potential costs to society?
More broadly, do patients have a fundamental right to medical choice?
These questions seem particularly appropriate today on a number of fronts including Right To Try laws and vaccines as well as emerging stem cell and other biomedical technologies. The recent measles outbreaks including the one sparked at Disneyland are warning signs of what harm is awaiting us if the choice to opt out of vaccinations continues to be available to the general public. The California Senate today passed the mandatory vaccination bill. Assembly lawmakers are now weighing the bill.
As we discussed at our recent UCD Stem Cell Ethics Symposium, that phrase “a fundamental right” is very powerful. A potential benefit of medical freedom would be the power to make choices that may benefit oneself or one’s loved ones that otherwise would not be available if solely decided on a collective, governmental level.
For example, there may be a new experimental drug that a doctor and patient together decide is worth the risk to try, but the FDA has not approved it or even given the green light for a clinical trial on it. In principle, that drug might help the patient or it might hurt or even kill the patient. Advocates of medical freedom believe patients should broadly be allowed to evaluate and take such risks with their own personal physician if they so choose. They see the FDA as an obstacle to their freedom.
As someone who has faced a very serious form of prostate cancer, I get the sense that being in a serious medical situation can change one’s views. Everything feels different when you are faced with a potentially lethal medical diagnosis yourself or for a loved one. Sometimes it’s not that simple though.
As individuals our freedom and desire for choices can conflict with a greater good. For example, as a biomedical scientist and parent I do not believe that childhood vaccination should be optional. While there is no compelling evidence that vaccination is harmful beyond for that tiniest fraction of those who have severe reactions or pre-existing immune diseases, there is profound evidence–I would go so far as to say proof–of powerful benefits to both individuals and society as a whole of vaccination.
Some parents feel that the best choice is not to have their kids vaccinated. Should they be allowed that choice? I don’t think so in the sense of the unvaccinated kids still being allowed to go to school where they can interact with and potentially harm other children via infectious diseases. Not being vaccinated also poses risks as an adult for other workers in the workplace.
While some might say that mandatory vaccination is “anti-freedom” and that there are costs to such governmental mandates, from my view the benefits dramatically outweigh those risks. But some argue a libertarian view that there is a price for that in loss of free choice.
Another example of a hotly debated area today that shares some of the same issues is emerging stem cell technology. Some say that patients should be free to entirely make their own choices as to whether to get an experimental, potentially risky stem cell intervention. The argument is also in conjunction often made that patients are in the best position with their doctors to make such decisions. “Get the government out of my doctor’s office”, might be the clarion call for those folks.
These kinds of “freedom-based medical decisions”, as we might call them, have possible societal costs even if they do not involve highly contagious infectious diseases. If companies sell unapproved stem cell treatments, then patients buying those treatments are at the same time as they think they are trying to help themselves, also are undermining the authority of the FDA more generally and in that way indirectly putting future potential patients at risk. As much as I think the community that reads this blog can agree that the FDA is imperfect and could benefit from reforms, the FDA performs a very difficult, complex and crucial mission, without which we’d face medical chaos leading to great harm.
It seems appropriate at this point to also point out that many stem cell clinics also sell these interventions to children. As such, if you believe that people should be allowed to get any stem cell therapy they want, keep in mind that there are possible broader consequences to you making that choice beyond undermining the FDA mission. One might say, for instance, that you are supporting a sketchy company that is putting other people at risk. Some of those other potential patients may not be in the same position as you to make decisions about risks. They may be kids or they may be less educated than you. By giving this stem cell business your money you are enabling them to put others into risky situations.
As much as some people might advocate for specific stem cell clinics selling unapproved stem cell treatments, I’d say that within this community most of us also can point to some clinics and doctors (and fake doctors) that are truly frightening and dangerous. Even if reasonable people can disagree on broader issues regarding the appropriate level of governmental regulation and freedom of choice, relentlessly attacking the FDA for the cause of near universal freedom of medical choice for investigational stem cell treatments poses the risk of giving the wildly dangerous clinics a freer hand. Sometimes what we believe is best for ourselves cannot be viewed only in a bubble.
The bottom line is that freedom of medical choice for an individual is far more complicated than it might seem and it is easy to oversimplify it. The reality is that our medical freedom and decisions have impact on others. I don’t see that we in the America, for example, have a fundamental right to medical choice based on The Constitution. Our medical rights and ability to make health-care related choices change over time too.
Should there be a constitutional amendment for a right to freedom of medical choice?What’s the best goal for these rights (or lack thereof) even if not a constitutional right for the immediate future? How much freedom in this area is best in the longer term?
This morning, David Gorski, MD, did a much better job than I ever could of explaining why right to choose legislation and the political motives behind it:
https://www.sciencebasedmedicine.org/the-con-game-that-is-right-to-try-legislation-continues-to-metastasize/
Dear Professor Knoepfler,
I would like to know, what do you think about these therapies?
http://www.stemcelldoctors.org/tag/such-as-tendons
http://www.prweb.com/releases/stem-cell-therapy/arizona-pain-specialists/prweb12616624.htm
https://www.preferredpaincenter.com/stemcellinjections.html
Thank you in advance
Do the right to try laws include cellular and gene therapy after phase I clinical trials ? IMO they do not ….. The laws in most states are written for drugs and biologics and the patients must be terminal ……What good is false hope? ….Cell repair and rescue is done at a cellular level an done toxic drug can not compete
with the working of thousand of functions of the right stem cells…..
We sit here with the Tufts dog trials curing dogs with human MSC …..yes human cell in dogs…Lanza was studying tolerance in these dogs……Andy Hoffman let the results slip out …… from a long line of auto immune disease at 100%but others b/c of the biology of MSC and the potency of Ocata’s hMSc have not recognized this as superior by the cell surface markers and gene expression that affect microenvironment…… the work that is being done ……Yet no papers are written ….Drugs can not do this ….Biologics can not do this….
This cellular treatment will take years form IND and phase with a drug process that is obsolete, to say the least…..How immoral and unethical is that?……The 450 or more MSC clinical trials continue with inferior cells ………Yet no one is paying attention to Lanza two MSC papers ( you can read them at Ocata.com)…..
Freedom of the patient is limited by the federal government process look at what Paul and Catherine are saying …Paul’s quote “As someone who has faced a very serious form of prostate cancer, I get the sense that being in a serious medical situation can change one’s views. Everything feels different when you are faced with a potentially lethal medical diagnosis yourself or for a loved one. Sometimes it’s not that simple though.”
Paul,
I was particularly drawn to this comment by you..
.”As much as I think the community that reads this blog can agree that the FDA is imperfect and could benefit from reforms, the FDA performs a very difficult, complex and crucial mission, without which we’d face medical chaos leading to great harm.”
You seem to be walking a tightrope of appeasement towards the FDA on one side and appeasement toward your readership.on the other. Commendable on your social adeptness but slightly disengenuous as to taking a more proactive stance.
Many here may know that the FDA is as much a quagmire of indecsisivness as it is effective medical protector of the masses. Beholdent to corporate and political pressures, make The FDA a rather gunky regulator and retards its ability to be an effective and faithful public servant. Getting reforms to aid patients in more immediate need is, many times, a time-consumming and daunting task…at least until the next presidential election…and then starts the process all over again. Uugg….
I listen and sympathize with Ms. Danielson. She has felt the FDA ‘gunkiness’ first hand and seems to say, in a more polite way, enough is enough…wake up..walk in her shoes..let the pendulum swing with your heart and not just your head.
Written with the pitfalls addressed that you mention, Paul, a Right to Try law can be a beacon to guide sane and sound solutions for both regulators and patients.
Thanks Brian! 🙂
I wouldn’t say that the movement is being “manipulated.” I know exactly who sponsored the Right to Try laws, and who wrote the model legislation. So do all the other patient advocates I know. It was all funded by the Goldwater Institute, and I know exactly who they are and what their beliefs are. They are conservative libertarians and proud of it (which isn’t the hardest thing to guess if you know anything about the original founder, Barry Goldwater!) However, Goldwater was a very old school conservative, and there were quite a few things associated with modern conservatives that he had absolutely no interest in. And without going into details that would probably get me into trouble, I believe that’s why the Institute is willing to fund this bill. It’s hard to imagine a scenario in which they don’t know that Right to Try laws will have a profound effect on drugs derived from stem cell research.
I may not personally agree with all of the politics of this organization, but here is the central point. They were the ones who stepped up to the plate on this. They were it. Nobody else had any interest. There were plenty of organizations with political beliefs much more attractive to me who had all the time and opportunity in the world to support a patients’ movement. And maybe they would have helped to craft Right to Try laws that don’t have many of the problems that are legitimately pointed out. But they did not do it. My sister ran for senate on the Pacific Green Party ticket last year, and I *still* can’t get that group the least bit interested in Right to Try.
We cannot always pick our ideal allies. But we know who they are, and we are not being manipulated. As Otto von Bismarck said, politics is not a science, but an art, and it’s the art of the possible. Not the ideal. The possible. And for better or worse, Right to Try is not about science, but politics. It’s not happening in an ivory tower. That’s the situation which exists, and it’s the one which has to be dealt with.
Well… hopefully, this post will end up being approved, because I’ve really tried to stick to either facts or opinion that is clearly identified as such. These are issues that need to be talked about, and dialogues that need to happen,
Interesting post, Paul. And I’d like to thank Catherine for a very informative and thoughtful comment.
There are situations where compassionate use is appropriate, and so-called orphan or extremely rare diseases is one of them, and I am thinking of diseases that are so rare that it would be difficult or impossible to get together enough patients for a proper trial. I did not mean to imply that compassionate use is never appropriate.
However, I think the current right to try movement is painting the issue with much too broad a brush, and it is being done in a way that can be quite damaging. I also think that the movement is being manipulated by political factions for their own reasons, so there is reason to be cautious here.
I also suspect that, in most cases, doctors are not being dismissive (although there are bad apples as there are in any profession), but they are either being appropriately cautious or they are not familiar enough with unusual conditions to be of help. In the latter case, there is a way to get around that. Find a specialist who can handle the situation. I really do not think we should be upsetting the cart for cases like that.
I have every reason to have a very personal interest in the Right to Try laws. Four years ago, I was diagnosed with a disease no doctor had ever seen before. All they could be sure of was that it behaved like age-related macular degeneration or Stargardt’s macular dystrophy, and that it would end in blindness. There was a drug in development that has now passed Phase I testing with flying colors, and my doctor told me that it would help me– if it weren’t many years away from approval. I had a remission that nobody could explain either, and I’m still in remission. But I have not been cured.
And when I first found out about Right to Try, I was not a fan. I saw all of the problems and limitations, and there are a lot of them. For one thing, if I had another flareup, the laws still wouldn’t help people like me, because they are limited to patients with an immediate terminal diagnosis. For another, they are not applicable to anyone right now because of the constitutional clause of federal supremacy. The list of problems goes on. After a lot of research, study, and talking to other patients, though, I have to say that I now support Right to Try.
The reason is this: the only way to support a theoretical Right to Try in the face of all the limitations is if these laws will provide pressure for change at a higher level, and if there is just no other realistic way for this pressure to occur. An excellent article in the Yale Law Review Journal pretty much laid it out.
http://www.yalelawjournal.org/forum/right-to-try-unapproved-drugs
The only way *this* is justifiable is if there are be huge obstacles blocking effective treatments for patients in ways that are simply not being addressed. This really isn’t the case for traditional drug therapies, but for cellular therapies, it is. Stem cell clinics are terrible and should be outlawed, but there are good reasons why people are going to them.
I don’t blame the FDA for everything in the way that some do, but it is a fact that other countries are handling the issue of these novel therapies very well, and we are not. The European Medicines Agency, for example, has the Adaptive Pathways Initiative, which provides for the early approval of drugs for restricted patient populations based on small initial clinical trials. Approval is then expanded progressively with input from health assessment bodies. The EU has certainly not somehow thrown out all regulation. But it’s effective regulation.
The RPE drug that could cure me (and the 180 million other people in the world with all types of macular degeneration) already has better results than most drugs for any purpose that are out on the market right now, and the results have twice been published in a peer-reviewed medical journal (the Lancet.) That’s not even mentioning the supporting results from South Korea, which were also published in a peer-reviewed medical journal. This drug should have been given breakthrough designation by the FDA long ago, and it still hasn’t.
There’s also the issue of the FDA’s lack of transparency and evasiveness when it comes to the issue of who actually gets access to the compassionate/extended access program. The Right to Try bill recently passed the Oregon House, and I went to the legislature page to read the supporting and opposing material for it. There was a good article in the New England Journal of Medicine: Practical, Legal, and Ethical Issues In Expanded Access to Experimental Drugs.
https://olis.leg.state.or.us/liz/2015R1/Downloads/CommitteeMeetingDocument/43602
The claim was made that all of the work involved with filing documentation for expanded access for a patient would only take “eight hours” of a physician’s time. Well, the problem is that the FDA itself says that the actual figure is *one hundred hours*.
http://www.raps.org/Regulatory-Focus/News/2015/02/04/21243/From-100-Hours-to-1-FDA-Dramatically-Simplifies-its-Compassionate-Use-Process/
I went to the original FDA document in the federal register from 2009 (can’t find the link right now, but I have it,) and read through it. Not very fascinating reading, but it’s pretty important. The FDA did indeed claim that only eight hours would be required, and there were lots of facts and figures to back that claim up. So what happened. How did eight hours expand to one hundred? How can anyone think that this provides anything remotely in the realm of decent access for the average patient when HMO’s require doctors to schedule appointments every fifteen minutes? There has been absolutely no transparency here, and the FDA did it to themselves.
I’m working on quite a few articles about Right to Try, and this clearly could go on for many pages, so I’ll cut it off here. But I would really ask people to think deeply about all sides of this issue. No matter which way you look at it, Right to Try is not a simple thing. But it exists for a reason, and it’s largely that patients have been brushed aside and not listened to.
Patients do have a right to medical choice, but as with all freedoms, there should be limits. Also, it is the doctors’ jobs to help their patients to both understand their situation and to help them understand the reasoning behind what is going on.
I happen to be a good example. Five years ago, I was being evaluated for a clinical trial for high risk prostate cancer. The work up revealed an incidental renal cell carcinoma. The correct course of action was to drop the clinical trial, which might have significantly delayed the treatment of the renal cell carcinoma, and go ahead with the surgeries. For me that was a no brainer, but I think it would not have been for many people. They would have to be educated so that they understand how to arrive at the best choice.
In addition to that, there are risks in giving patients unfettered access to experimental therapies. First, they might cause more harm than good, even, perhaps, no good at all. After all, they are experimental. Also, you might have difficulty recruiting patients into clinical trials if they could just go off on their own and get any experimental drug on a compassionate use basis just for the asking. The regulation of the drug approval process is there not to just protect the patients, but also to protect the process itself, to make sure that it effective. Undermining that process, would be, in my opinion, quite dangerous.
As for the health freedom advocates, I think many of them have either not thought clearly through what they are advocating, or they are using health freedom as a proxy to promote some agenda, whether it be anti-vaccine, pro-some form of quackery, or some sort of political agenda.
We do need some science-literate people in government to protect our systems, which seem to work fairly well, although they are not perfect. Sadly, there seems to be fewer and fewer of them