Right-To-Try laws have been passed in many states across America, paving the way in theory for gravely ill patients to have the right to try unproven treatments of various kinds, and now there is a serious push underway for a national Right-To-Try law.
What is a Right-To-Try law?
Typically, these Right-To-Try laws allow for a patient facing serious illness, with consent of their physician and the business that makes the still investigational therapy, to not have to wait for full vetting of the new treatment. They can take the risk, for example, on a new chemical drug or stem cell-based regenerative medicine therapy before it is proven to work or definitely to be safe. For a patient with a fatal illness such as ALS, it seems reasonable that they should have a right to try different options, but it should be noted that the FDA already has a program for this kind of situation called compassionate use or as the agency calls it, “expanded access”.
On the stem cell front, one of the biggest concerns over Right-To-Try is that it could be exploited by some of the hundreds of stem cell clinics out there. The new stem cell law in Texas, which in an earlier incarnation as a bill I called “Right-To-Profit” for stem cell clinics, has some Right-To-Try elements. Another concern is that some proponents of Right-To-Try don’t want it limited to fatal illnesses.
Overall, the campaign for a federal Right-To-Try law is gaining momentum via a legislative push called the Trickett Wendler Right to Try Act. Yesterday, for instance, Sen. Ron Johnson (R-Wis.) released a statement saying Right-To-Try should be included as part of the FDA reauthorization bill. Johnson is a sponsor of Trickett Wendler and has threatened to hold up FDA funding over this cause.
I asked stem cell scientist Sean Morrison, who is also the Chair of the ISSCR public policy committee, for his take on the push for a federal RTT law, particularly as it relates to stem cells, and here is what he said:
“1. These right to try bills are bad for patients because most of the new therapies patients will be able to access as a result of these bills would be expected to prove unsafe or ineffective once additional clinical testing is done. This is because we know that most therapeutics that seem successful in phase 1 clinical trials prove to be unsafe or ineffective upon additional testing in phase 2 and 3 clinical trials.
2. These bills are often introduced by well-meaning legislators who have very little understanding of medical research or clinical testing. They don’t realize these bills will victimize patients by opening the door to snake oil salesmen to sell unproven therapies to patients. Patients will be forced to make life and death decisions without the benefit of clinical data on whether the therapies are safe or effective.
3. These bills have been introduced nationwide as part of a general assault on FDA regulation. Most people who support these bills don’t realize that the FDA already has a very effective, and efficient, accelerated access program under which patients can get therapeutics prior to FDA approval. Most don’t realize the extent to which the FDA protects Americans from snake oil salesmen trying to make a buck by exploiting the hopes of desperate patients, by selling unproven and often scientifically implausible therapies that are promised to cure all manner of incurable diseases.
4. There are scores of companies in the United States who want to sell unproven and scientifically implausible stem cell therapies to patients, some of whom are among the main supporters of right-to-try legislation.”
It’s better than 50-50 that Right-To-Try will become federal law, but it’s not a done deal and if it does become law it isn’t entirely clear what the practical impact would be since state laws of this kind haven’t to my knowledge led to dramatic changes in terminal patient access to investigational drugs plus many drug manufacturers do not want their experimental treatments utilized in this manner for various reasons. For stem cells specifically, I expect that if there is a federal Right-To-Try law that we’ll start seeing “Right-To-Try” popping up in clinic advertisements, which wouldn’t help matters.
Paul,
Greetings. What is the FDA’s “new RMAT mechanism”? Has the Agency issued an advance notice of these forthcoming new guidelines? Please advise me with a topic specific link available on the FDA’s website or to other publications/blogs.
I view the ‘Right to Try’ debate as a timely discussion of an important medical-scientific ethics issue with a healthy dynamic interplay between powerful institutions and corporations and caring, intelligent and innovative scientists and physicians, who are motivated by a vision to advance the benefits to patients of credible, compassionate and creative innovation; this beneficial innovation is usually initiated independently, by committed creative individuals or small groups. These creative innovators are frequently constrained by both market forces as well as the established institutional ‘diligence paradigm’ with its long standing political-economic motivations of control and maintenance of established authoritarian decision making power; i.e. the reliance upon the ‘Gold Standard’ – the randomized Clinical Trial(s).
Desparate patients, who need an innovative, new treatment to save their lives can be counseled by knowledgeable professionals to explain the risks and benefits of such treatments; these professional counsellors can explain and interpret the state of medical-scientific knowledge, plus answer the patient’s questions about risks and benefits, plus outcomes, and address the concerns of family members and loved ones, and then have the patient sign an Informed Consent Agreement, if they feel comfortable and confident after these deliberations. This is similar to the ‘subject recruitment’ process for obtaining volunteers to participate in experimental Phase II or Phase III studies, which are performed routinely by academic medical centers and the pharmaceutical or biotechnology companies, who fund these clinical experiments.
Obviously, innovative and caring professionals can undertake and sustain the same or similar ethical and legal methods used in the conduct of these Clinical Trials, plus responsible clinicians can provide close and more intimate patient follow up, while maintaining the preservation of their patient’s confidentiality to comply with HIPPA requirements. The cost to patients will be significant, but the benefits of these innovative interventions to their physical health, emotional well being and longevity, will, I submit, equal or exceed the very slow and uncertain clinical benefits of the institutional conduct of randomized Clinical Trials, which are then followed by the review of the tabulated results of these Clinical Trial by both the FDA, as well as the editors of professional journals, who review the papers submitted for publication of the results of those Clinical Trials that produce positive clinical results and statistically validated data.
Paul, could you elaborate on the differences between the FDA’s “compassionate use” and the proposed “right to try” legislation? This article almost makes sound like legislation is being proposed that already exists.
Would it be correct to assume your problem with it is that it would allow clinics to offer some of the procedures that currently only hospitals are allowed to offer? What makes the FDA’s current program better than what this proposed legislation could offer?
Fundamentally, I find the arguments against this to fail to hold up rationally. I feel people should have the right to access these experimental drugs and procedures when facing certain death. I do agree that anything less important than choosing the possibility of life over death, or a procedure that results hereditable changes must be prohibited for now until we understand how to use it more safely. I don’t think it’s a cold thing to say that I know and accept many of these procedures aren’t going to work. Having read your book last week, I imagine you might sight a study suggesting the public would be against this too. I don’t see that. I think the public would be in agreement with you on the issue of designer babies, or allowing superficial changes like eye color, but not compassionate care. Besides, it’s obvious the ability to alter intelligence or even eye color is simply not yet possible in the first place due to our lack of understanding of the human genome.
I think the public is tired of being told what is right and wrong for them in these types of situations. Why not benefit from careful study of the results? Most importantly, why not be hopeful some procedures will work or at least extend someones life? I know I would try anything if I were dying. I would be willing to take the risk with the hopes that something can be learned from it. I would hope my sacrifice might speed the progress of this technology and save lives in the future sooner than it otherwise would.
Hi Jon,
Thanks for weighing in.
With Compassionate Use, although the FDA reportedly almost always approves such requests (http://www.raps.org/Regulatory-Focus/News/2015/02/04/21243/From-100-Hours-to-1-FDA-Dramatically-Simplifies-its-Compassionate-Use-Process/), they are evaluated carefully and there are specific guidelines such as the patient having a fatal illness.
With RTT there is a sense that things would be handled much more loosely and that some strictly-profit driven enterprises would take advantage of patients including some without fatal illnesses. I’m 100% convinced that would happen in the stem cell arena.
On the other side, RTT proponents are often critical of the FDA compassionate use program (http://goldwaterinstitute.org/en/work/topics/healthcare/right-to-try/dead-on-arrival-federal-compassionate-use-leaves-l/).
One thought, more specific to the stem cell area, is that with the new RMAT mechanism that is pretty speedy, FDA hopefully will accelerate therapy development and approval. We’ll all learn more in September when the FDA starts releasing new guidelines about this. Paul
As a human being and especially as an American, I should have the both the right and the opportunity to evaluate any therapy that I believe will help me with a medical condition or my health generally. The FDA or the AMA has the right to define their “standard of care.” And I should have the right to reject either organization’s authoritarian rule regarding my own health. I understand that if a therapy is outside the “standard of care” then it is at my own expense. This is fine. But medicine and medical technology is advancing rapidly, yet the FDA is stuck in the model of trying to protect me from myself….assuming that I really do not know how to best evaluate therapies and products for myself. Their approach to protecting me from the advances of medicine, often for decades, must end. And some of the best therapies never see the light of day because of the “enlightened” medical thinkers at the FDA. This legislation needs to pass. We are the land of the free in many areas, but certainly not in medicine. This legislation would help to level a very un-level playing field.
And to your comment regarding those selling snake oil; I say that a free and open market will expose those who sell products or services that have low value or do harm. The marketplace weeds out the poor performers. And the marketplace is the only authority with the sense to judge.
Love your e-magazine. Keep up the good work.
Steve Wilsher
Gardendale, Alabama
@Steve,
Thanks for your comment.
I can see where you are coming from.
For me as a stem cell researcher I know for sure there are some predatory stem cell clinics out there in our country that are almost 100% for sure going to take advantage of Right-To-Try to simply make money. I’m not sure what we do about that because even as they may be exposed in an open market as you rightly said over time, in the mean time a lot of innocent people probably will pay the price financially and possibly health-wise. It’s a tough situation.
Paul
I really want to understand why you write these pernicious hystrionic things that are no business of a laboratory scientist like yourself
Thomas ichim
Thomas,
I don’t get it.
I don’t see how this post is at all histrionic let alone pernicious.
And if you disagree with the quoted statement of the stem cell scientist Dr. Morrison, why not comment here with your opinion in a constructive way on the issue of national right-to-try and its potential impact on the stem cell field? You could even bring in Medistem Inc.’s opinion since you are in a leadership position there.
Finally, it seems to me it would be beneficial if more laboratory scientists like myself participated regularly in discussions of important policy matters like this.
Cheers, Paul