Do Patients Have a Fundamental Right to Choice?

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.right to choice in medicine

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?

Meeting review of unique stem cell ethics symposium @UCDAVIS

On Thursday here at UC Davis School of Medicine we held our second annual Stem Cell Ethics Symposium. The symposium was organized by Drs. Mark Yarborough and Nanette Joyce as well as me.

In this post I report on key take homes from the meeting and summarize the specific talks.

As far as I know, this is one of the few events in the world where there is such a wide diversity of stakeholders present both as speakers and in the audience to specifically discuss stem cell ethics for a full day. The participants included numerous patients and patient advocates, bioethicists, stem cell researchers, physicians, attorneys, institutional compliance officers, and more.

Stem cell ethics meeting

One of the things that I most appreciated about this meeting is that diverse views were welcomed and we dealt head on with some of the most challenging ethical issues related to stem cells.

I also got to meet in person some people I had only previously known on-line including keynote speaker Tim Caulfied, patient advocate Ted Harada, and advocate and frequent commenter on this blog, Cathy Danielson, which was fantastic. My colleague Leigh Turner was also present. Old friend and Huntington’s Disease advocate, Judy Roberson, was there as well. You can see the picture above of Ted, Judy, and me.

After a great introduction by our new Dean of the School of Medicine, Dr. Julie Freischlag, Nanette Joyce started things off with a very powerful talk about her experiences with her ALS patients. Her patients are facing incredibly intense challenges as they deal with all that ALS brings. Both in her medical specialty and amongst her patients, she noted, there is growing interest in stem cell treatments. Dr. Joyce expressed her concern over the lack of evidence of safety and efficacy, and how elements of hype have entered into the arena of ALS and stem cells. ALS is a very heterogeneous disease with ups and downs as well in individual patients, but life expectancy on average is very short after diagnosis.

One of Dr. Joyce’s patients, Michael, then gave a talk about his experiences with ALS. I found it really striking. Literally every day he goes on-line and checks out resources to search for opportunities for ALS patients such as clinical trials. Michael indicated that as far as he knows he’s not eligible for any trials so far. This was a theme that several people mentioned during the day: just because there are trials out there does not mean that patients with ALS or other conditions can be part of them. Patients are seeking other opportunities.

Next up was Ted Harada, who spoke passionately about his experiences with ALS, the ups and the downs, what is really like to get a stem cell treatment and be in a clinical trial. His talked about his willingness to take risks. He said he’d literally rather die trying something than not try to do something about his ALS. Ted has had two rounds of treatment for ALS as part of the Neuralstem clinical trial and he reported that it has really helped him. One of Ted’s concerns is that he may not be eligible to get a future third treatment from Neuralstem for various technical rules of the clinical trial, which is one reason he is such a strong advocate for Right To Try laws. However, Ted said he feels that most likely few or no patients will be directly treated as a result of Right to Try, but rather that it will open up a dialogue and create other positive outcomes that lead to more patient opportunities.

From both Michael’s and Ted’s talks, i also learned that it can take a very long time (potentially a few years) to definitively diagnosis ALS. Patients facing ALS are likely to have a much higher level of risk tolerance than others might imagine. Understandably they have a strong drive to take action.

I spoke next and focused on the growing challenge that the FDA and the stem cell field face of chains of stem cell clinics that appear to be selling unapproved stem cell drugs without FDA licensing. I talked a bit about the growth in providers offering unapproved stem cell “treatments” at Cell Surgical Network as an example. More broadly, the clinics argue that what they do is beyond the scope of the FDA’s regulatory authority. I discussed new FDA guidances that bear on this issue and which would seem to directly contradict the views of many of the clinics, squarely placing them and their products in the FDA sphere from my perspective. The clinics of course disagree. I also emphasized how the FDA currently is asking for comments on these guidances for the next couple weeks.Tim Caulfield

Tim Caulfield spoke next, giving a wonderful talk. In the stem cell world, hype is not just limited to dubious stem cell clinics. Tim focused on hype in the stem cell field and in particular hype involving scientific publications. He even talked about hype about hype. In the current environment there are strong pressures for scientists to hype their work, including in particular in abstracts. What is the relationship between hype in science articles and in the media? He discussed how widespread pressure to commercialize biomedical research including stem cell efforts sometimes has negative consequences and can contribute to hype.

Alison SorkinAlison Sorkin, Deputy General Counsel for University of Colorado Health, talked about Right To Try from her experiences in the trenches. With the passage of the Right To Try Law in Colorado, Sorkin was there for the intense repercussions. The next working day after the law was passed, patients starting contacting the University of Colorado seeking treatment specifically under the law. Sorkin talked about strict the Colorado law actually is, making it very limited in scope in reality. She also discussed problematic issues with the specifics of the law such as that patients would be responsible for paying for all of their own healthcare for 6 months after treatment under Right To Try as insurers would be exempt from having to provide ANY coverage. There seems to be a growing sense that Right To Try in Colorado may not actually lead to any patients getting non-FDA approved drugs. However, the FDA is paying close attention to Right To Try and one potential consequence of the various Right To Try laws passed in states is that the FDA could be influenced in terms of policies on expanded access (compassionate use).Leigh Turner

Leigh Turner gave a very timely, excellent talk on the FDA, stem cell clinics that do not have FDA licensing or approval, and the potential consequences of this current dynamic. Leigh went into helpful detail on the clinic claims, the types of stem cells they use, and how these relate to the new FDA guidances. He discussed Cell Surgical Network and Stemgenex. It is notable that Leigh gave specific examples and names clinics by name, which I agree is vital to the discussion of the regulatory sphere and the place of stem cell clinics.

Gerhard Bauer, my colleague here at UC Davis and one of my favorite stem cell scientists, gave a wonderful talk on his experiences and perspectives over the decades as a pioneering stem cell and gene therapy clinical researcher as well as earlier as a regulator at the FDA of Austria. Gerhard’s discussion ranged from his experiences in these roles to also his own person experiences as someone who has dealt with doctors and also lost many friends to disease including HIV. I also appreciate how he provided his views from his real world experiences wending his own clinical trials through the processes and how much work it takes just to get an IND. How great it feels to get to that point!

A few additional notes

Mary Ann Chirba, a voice for increased patient autonomy in terms of their cells, was going to be a speaker, but got snowed in at Boston. Richard Garr, Neuralstem CEO, was also scheduled originally to speak, but couldn’t make it. They were missed, but still we had a diverse, dynamic group of perhaps 70-80 attendees.

A big thanks to Julie Bechtel who helped to arrange the event.

UC Davis 2nd Annual Stem Cell Ethics Symposium: Save the Date Feb. 12

We here at UC Davis School of Medicine are gearing up for our 2nd annual Stem Cell Ethics Symposium (see flyer).stem cell ethics

As one of the organizers, I’m really excited about our lineup of speakers including Timothy Caulfield, Leigh Turner, Alison Sorkin, Mary Ann Chirba, and Richard Garr as well as others.

We will be focusing on “Right To Try” laws, FDA regulations, and stem cell tourism.

It should be a cutting edge, spirited meeting and discussion.

All are welcome.

Please RSVP to Julie Bechtel @ 916-734-6181 or [email protected]

Hope to see you there.

Caplan Article on Bogus Stem Cell Research: Some Different Views

When bioethicist Arthur Caplan talks about stem cells, people pay close attention and for good reason.

Art Caplan

Caplan has provided important perspectives on the stem cell field over the years. For example, you can see a guest post on this blog here about human cloning.

However, in my post today I respectfully discuss how I disagree with several parts of this week’s piece by Caplan on why there are allegedly so many ethical problems in the stem cell research field.

Caplan’s article (see screenshot from video at right) is focused on a question articulated by the title:

Why so much Fake, Unduplicable Stem Cell Research?

One might start off the bat by challenging the article’s title and intrinsic question above, since in reality that the vast majority of stem cell research is quite real and replicable.

David Jensen over at California Stem Cell Report, writing about Caplan’s article, pointed out that serious research issues are not unique to the stem cell field as, for example, there have been disastrous issues in the cancer field too:

There is no doubt some spectacular fraud has surfaced in stem cell research. But the problem of replication within stem cell research may not be entirely out of line with problems elsewhere in science. Los Angeles Times columnist Michael Hiltzik wrote last fall about a study by Amgen that examined 53 “landmark papers” in cancer research and blood biology. Only five could be proved valid, a shocking result, according to Amgen. Similar results were turned up byBayer in Germany, Hiltzik said.

On the other hand, there have been some truly terrible stem cell research fiascos of late so let’s focus on Caplan’s reasoning for why the stem cell field has had these problematic events.

Caplan begins by talking about the STAP cell fiasco in Japan involving allegedly faked research reported in Nature that powerful stem cells could be made by simple stressors such as low pH.

Caplan writes that the researcher in question, Haruko Obokata, “confessed that she had made it up.” In fact, quite the opposite is true. Dr. Obokata says that her study is correct overall and that she did not make it up. To my knowledge, she only admitted to careless errors arising innocently from lack of experience that she says do not affect the conclusions of her papers.

Getting back to the central question of why stem cell research sometimes runs into ethical problems Caplan argues that lack of funding is one reason for the problems. I wish there was indeed more stem cell research funding, but I do not believe this is a clear reason for ethical problems in the field. I don’t see this playing a significant role and funding woes certainly aren’t specific to the stem cell field.

A second reason given for trouble is what one might call the “stem cell hero temptation” effect. In other words, breakthroughs in stem cells might gain a researcher the world’s attention  (“being a hero to the world” is how Caplan describes the attraction ) so there may be perceived incentive to fudge or outright fake stuff. From some of the cases we’ve seen in recent years, this reason seems accurate.

An additional Caplan assertion for explaining the ethical issues facing the stem cell field can be boiled down to a lack of people to provide oversight. Caplan writes:

Another major problem in the stem cell field is that the number of people doing research in this area has shrunk…That may mean that there are fewer people to watch one another.

I’d be interested to see if he has any data to back up this claim. In fact, my sense is the opposite about the size of the stem cell field. If anything the number of people working on stem cells seems to continue to grow overall. I do not believe that the stem cell field lacks sufficient people power to adequately review itself.

So if I disagree with two out of three of Caplan’s reasons, why then do I think that there are sometimes ethical challenges in the stem cell field such as the STAP cell problem?

First, let me say again that he’s right about a few unwise research folks chasing international fame at any cost.

However, another issue here is that a heck of a lot more people around the globe are paying attention to the stem cell field. As a result, ethical problems that are also present in other fields of science (e.g. image manipulation, non-reproducible papers, etc) get noticed far more if they are in stem cell papers. To sum it up, there are more eyes on stem cell papers looking for troubles after publication.

As I blogged before, I also believe that in the specific STAP case, the reviewers and probably editors too were unduly positively biased by the addition of some stem cell big wigs to the authors list on the STAP Nature papers. This points to another contributing problem to broader problems in the field: a small number of stem cell bigwigs have way too much power as reviewers. In other words, journal editors rely on too few eyes to review the highest profile manuscripts. Big journals and their editors need to diversify their stem cell reviewer lists and the review process needs to be more about data and less about names.

In the end the stem cell field is likely to continue to run into a few bumps and even land mines as it proceeds. Addressing recurring problems in an open, expeditious manner would be wise. Training in ethics for researchers seems to be in need of a boost. The journal review process also is a logical place to focus. Is it naive to hope that Nature might take the lead on reform of the review process?

Stem cell field stumbles on cloning dialogue

circle the wagonsWe had the big news last week that for the first time ever, human embryo cloning (aka “therapeutic cloning”) worked to produce apparently normal embryonic stem cells.

I posted about it a few times here, here, and here.

I have tried my best to be factual, open minded, and realistic about the issues.

Frankly, the stem cell field as a whole has done mostly a lousy job handling the human cloning dialogue sparked by the publication of the therapeutic cloning paper last week.

It was one of those classic challenges that was also an opportunity. Unfortunately that window of opportunity is closing, while the challenges will remain or intensify even.

Instead of being part of a rational, fact-based discussion of the ethics and policy issues that were raised, there seems to be a lot of wagon circling and group think going on within the stem cell field.

Some have gone so far as to contact me to say, in effect, “Cut it out!” regarding my open discussion of the cloning issues on this blog. They are not happy with me for my bluntness.

I must not have gotten the memo about the memes that good, well-behaved stem cell researchers are supposed to be sticking to, huh?

Openness and transparency on cloning is crucial, but that’s not what the stem cell field has been advocating in the past week.

What went wrong?

First of all, with all due respect I believe the authors of the paper should have included in their discussion section an overview of the ethical and policy implications of their work. Why didn’t this happen? I don’t know. To be clear, I liked the paper and found it fascinating, but it desperately needed more big picture context.

Second, the journal Cell should have included an accompanying preview and editorial also placing this major finding in the appropriate historical context online. Instead, nothing. Maybe something is coming for the print edition?

Third, the stem cell field should as a whole have been open to discussing the very real issues surrounding this complicated topic of therapeutic cloning and by logical extension the issues surrounding reproductive cloning. The two are at least somewhat linked. That’s a fact that we cannot pretend away.

Yes, cloning is a complicated, ethically challenging issue, but the stem cell field needs to be more open and genuine about talking about it or the field will continue to get criticized  by people on the other side. As a field we also do not engender public trust by futzing around the real issues.

Ethics and policy issues related to cloning should be discussed openly by the field and the leaders of the field have a particular responsibility in this area. I hope to see some of them step up to the plate and show true leadership by talking openly about this stuff.