During the pandemic, I had a saying about antivax tropes and disinformation: Everything old is new again. That saying applies even more in the age of “make America healthy again” (MAHA), the loose coalition of “health freedom” activists, antivaxxers (yes, MAHA is inseparable from antivax, no matter how much its proponents claim otherwise), and advocates of “complementary and alternative medicine” (CAM, also known “integrative medicine”) and outright alternative medicine quackery. The main difference now, of course, is that, unlike the case in past decades, MAHA is in charge of the government. The result is that we have a Director of the National Institutes of Health who wants to convert the NIH into the “research arm of MAHA,” a Secretary of Health and Human Services working on dismantling federal vaccination infrastructure, and a Food and Drug Administration being pushed in one direction to approve unproven peptide medications while demanding “saline placebo”-controlled randomized clinical trials for vaccines, whether such trials are ethical or not.In writing about it all, I’ve been meaning to revisit a strain of “health freedom” that I’ve been writing about for well over a decade, the “right-to-try” movement, which now fits comfortably under the MAHA umbrella.
“Right-to-try,” as you might recall, involves letting terminally ill patients access experimental therapeutics that have passed phase I clinical trials indicating gross safety (i.e., no major adverse events observed in a small number of patients). I’ve explained in depth why these laws are a bad idea, and why their purpose was never quite as advertised (to offer these therapeutics to patients with no other options) but in reality to serve as a wedge to start to whittle away at the FDA’s power to regulate pharmaceuticals. (More on that later, as I haven’t discussed it in a long time.)
First, however, I want to take a look at what the State of Montana—yes, Montana!—has done with respect to “right-to-try,” which is essentially to take it far beyond what any state (or the federal government when it passed the right-to-try 2017) has tried to do, starting with two stories published late last week in the MIT Technology Review, one reasonable and one highly irresponsible. The reasonable one is entitled Montana’s plan to become an experimental medical hub just pushed forward, while the irresponsible one is framed in the usual terms of how this will supposedly help desperate patients and entitled Montana’s new “right to try” law can’t come soon enough for some. Basically, the final rules have been drafted to implement SB 535, which amended Montana’s “right to try” bill originally passed in 2015. Here’s how the first article describes the new program in Montana:
As of this week in Montana, any biotech company with an experimental drug has a clear path to selling it to consumers. Companies whose drugs have been through preliminary testing—sometimes in as few as 10 healthy people—can pay $12,500 to apply to a newly established review board for approval. Once its treatment is rubber-stamped, the company can set the price of the drug and sell it via experimental treatment clinics, the first of which is likely to be up and running around the end of this year.
Montana’s latest right-to-try legislation is unique. While other jurisdictions with similar laws limit access to drugs to people with terminal illness, in Montana access is theoretically available to anyone who gives informed consent and can pay. That includes people desperate for treatments for rare diseases. It also includes those who are interested in longevity and want to try out drugs pitched as preventive therapies.
I trust that, just from this, readers can see why this is a horrible idea. To understand more deeply, let’s recap the origins of “right-to-try” and how it went from a movement to pass laws in various states to a federal law. More importantly, let’s look at how Montana is likely a harbinger of what advocates of “Right-to-Try 2.0” (RTT 2.0) want for the whole country, although Montana goes beyond even this.
Right-to-Try: Created by the Goldwater Institute to weaken the FDA
Come with me to a (seemingly) much more innocent time, before the shift in the antivax movement’s political center of gravity had turned sharply and definitively to the right, before the pandemic and the tsunami of health misinformation, anti-public health activism, and quackery overwhelmed us, and before the term “MAHA” was even an action potential in one of RFK Jr.’s addled neurons. The year was 2014, and Jann Bellamy and I both noticed the incipient “right-to-try” movement promoting “right-to-try” laws. Jann described the Illusions of “right-to-try” laws, and and I pointed out that the ethos behind right-to-try was not unlike the Dallas Buyers’ Club, noting that it was a great movie but a terrible basis for drug regulation policy, not to mention terrible for patients. It was not for nothing that I regularly refer to right-to-try as a cruel sham. Nothing in the more than 12 years since then has changed my mind about it, but even before MAHA the concept was so seductive that many states passed right-to-try laws, and ultimately, after Donald Trump assumed the Presidency for the first time, a federal right-to-try law was passed. Despite its advocates claims, a year after it passed, right-to-try was a failure, and it was still a failure in 2024, when during his campaign to become President a second time Donald Trump falsely claimed that right-to-try had saved thousands of lives.
But what is “right-to-try,” or, if you will, Right-to-Try 1.0? The first thing you need to know about it is that the über-libertarian Goldwater Institute was the prime mover promoting state right-to-try laws back during the initial push for these laws between 2013-2016 or so. Indeed, the nearly all of the first wave of “right-to-try” bills introduced into state legislatures were been based on model legislation designed by the Goldwater Institute, which states like Colorado modified so that the language fit within its existing statutes. But what did right-to-try laws claim to offer? I’ll list key provisions from the first wave of right-to-try, basically paraphrasing them from the Goldwater Institute’s legislative template:
- Eligible patients. An eligible patient has a terminal disease; has in consultation with a physician, considered all other treatment options currently approved by the FDA; has been given a prescription or recommendation by a physician for an investigational drug, biological product, or device; and has given informed consent in writing for the use of the investigational drug, biological product, or device.
- Terminal Disease. Terminal disease is defined as an “advanced stage of a disease with an unfavorable prognosis and no known cure.”
- Investigational Drug, Biological Product, or Device. This is defined as a “drug, biological product or device which has successfully completed Phase One of clinical trials, but has not been approved for general use by the Food and Drug Administration.”
- Availability. A manufacturer of an investigational drug, biological product, or device has the option of making its investigational drug, biological product, or device available to eligible patients under this act. Nothing in this act shall be interpreted to require that a manufacturer make an investigational drug, biological product, or device available.
- Costs. Manufacturers are permitted to provide an investigational drug, biological product, or device to eligible patients without receiving compensation but may require eligible patients to pay the costs associated with the manufacture of the investigational drug, biological product, or device.
- Insurance. Insurance companies and government health care programs are not required to provide coverage for the cost of any investigational drug, biological product, or device, although they may choose to do so if desired.
- Professional licensing. No medical licensing board shall revoke a license, fail to renew a license, or take any other action against a license solely based on a medical professional’s recommendation, prescription, or treatment with an investigational drug, biological product, or device.
Again, most of the laws passed a decade ago were based on this template, with some tweaks here and there depending on the specific state. I’m going to harp on one thing here, because the Montana definition of “investigational” is the same as the Goldwater template, namely that any drug that has passed phase I trials is fair game for right-to-try. As I like to point out ad nauseam, phase I trials are small “first in human” trials in which a new investigational drug is given to volunteers at different doses and the volunteers screened for adverse events and any effect on the disease or pathology targeted by the drug. Phase I trials typically enroll only at most a few dozen subjects, and sometimes it’s few as ten. A drug that has passed phase I trials has not, by any stretch of the imagination, been demonstrated to be “safe.” It has been demonstrated not to have any serious adverse events that are common enough to show up in such a small test population and thus been deemed “safe enough,” if you will, to proceed to phase II clinical trials. Also, phase I trials do not demonstrate efficacy. They are not designed to do that. It’s nice if there is evidence of an effect on the disease being targeted, but the trial is designed mainly to make sure that there aren’t any common or unexpected adverse events that render the drug more dangerous than the condition being treated. That’s it. It was irresponsible enough of the original right-to-try to allow such drugs to be administered as described above, but it’s even worse to do what the new Montana right-to-try will do and basically let pharmaceutical and biotech companies market such drugs to desperate patients, as long as they pay an application fee.
The other thing about phase I drugs is that the percentage of these drugs that make it through phase II and phase III clinical trials to go on to be FDA-approved is quite low. Indeed, only 5% of all cancer drugs that enter clinical testing are ultimately approved for patient use. Among drugs that make it past phase I, only 30% go on to phase III. There is a steep winnowing process for candidate drugs as they go through the three phases of the FDA-required drug testing process, such that only somewhere between 15-30% make it through the process to be approved, even as conceded by the State of Montana. In other words, the chances that an investigational drug might help a desperate patient if it passed phase I are probably much lower than one in ten, but the chances of adverse events that cause harm are certainly much, much higher, and that’s leaving aside the financial toxicity.
I bring that up because the original right-to-try is that it betrayed its roots coming from a libertarian think tank. Patients are basically on their own in terms of the cost; that is, unless a highly benevolent drug company is willing to pay. Insurance doesn’t have to cover it, and in some laws that I discussed back in the day it was unclear whether insurance would be required to pay for additional medical care from serious adverse events caused by use of an experimental therapeutic under right-to-try. (Indeed, the version of the law introduced in Michigan included a provision that allowed drug companies to go after a deceased patient’s estate to recover costs.) There is no liability for companies or doctors, nor are there any penalties for doctors who irresponsibly promote right-to-try. Yet the laws keep passing because to the average person they seem so “reasonable,” and, after all, what sort of hard-hearted bastard do you have to be to deny a terminally ill patient the chance to try an experimental drug that might help them, even if the chance that it will do more good than harm is small?
But where did right-to-try come from? It’s even worse than having come from a libertarian think tank. It also turns out that the for-profit hospital chain Cancer Treatment Centers of America (CTCA), played an integral role in the genesis of right-to-try:
In September 2012, a group of executives from the Cancer Treatment Centers of America reached out to the Goldwater Institute, a small, libertarian think tank named for the former senator and presidential candidate.
Goldwater — which has in past years accepted support from major conservative organizations like the Charles Koch Foundation and Donors Capital Fund — had taken on a wide-ranging libertarian agenda that included activism on campus free speech and school choice. The for-profit hospital chain CTCA also has its own ties to the Koch family.
CTCA and, ultimately, the Goldwater experts believed the FDA’s existing expanded access program — through which the agency approves some 99 percent of requests from dying patients who can’t get into clinical trials but want to try experimental treatments — was too cumbersome.
It was time to do something about it, and a consultant with CTCA, Chuck Warren, coined the term “right to try,” recalled Starlee Coleman, a Goldwater senior adviser.
CTCA and the Goldwater Institute decided to start with the states, and, unforunately, the rest is history. I also note that we’ve written about CTCA before. It’s a chain of for-profit cancer hospitals that market “integrative medicine” quackery along with conventional medicine. So, yes, right-to-try was the product of a hospital chain that markets itself by selling quackery alongside standard medical care, quackery like “naturopathic oncology“, doing questionable genomic testing, and inflating its survival statistics through the cherry picking of patients. Demonstrating how shadowy the whole arrangement was, few people knew CTCA was behind the Goldwater Institute’s push for right-to-try. Even I didn’t know it until the federal law passed.
Ever since right-to-try conquered the states and a version of it was passed into federal law in 2018, its advocates have been chafing at even the minimal restrictions that exist under the law, the first of them being the requirement that the patient have a “terminal illness.” The Montana law represents the ultimate expression of this dissatisfaction, as you will see.
Montana: Paving the way to fleece desperate patients legally
Let’s go back and look at the Montana law as described in the MIT article. Once again, there is no requirement that the patient have a terminal illness, or even a serious illness. As the article points out, the program is open to “people desperate for treatments for rare disease” and “those who are interested in longevity and want to try out drugs pitched as preventive therapies.” That latter category, of course, will be a boon to quacks everywhere, who can presumably just run a quickie phase I trial, declare their supplement or product “safe,” and then profit. Going back to the MIT Technology Report story, which relates the origin of this program, which is the result of SB 535, which was designed to expand the original Montana right-to-try bill beyond terminally ill patients:
“Montana first passed a right-to-try law in 2015. In 2023, with the support of state senator Ken Bogner, the state expanded the law to include all patients, not those just with terminal disease. Last year, Bogner told MIT Technology Review that his vision was to focus “more on preventative medicine” rather than “just treating diseases once they show up.”
Bogner says he had “started working on a bill” that would become the 2023 law when the Alliance for Longevity Initiatives (A4LI), a nonprofit “dedicated to advancing legislation and policies aimed at increasing healthy human lifespan,” got in touch. A4LI connected Bogner with others who helped draft the bill and testified in support of it.
Once that law was in place, the tech entrepreneur and longevity enthusiast Niklas Anzinger got involved. Anzinger has been working to establish a jurisdiction to fast-track the search for drugs that might deliver radical life extension. He is based in Próspera—a private city and “special economic zone” in Roatán, Honduras, which is already home to a separate clinic that sells experimental stem-cell and gene therapies. Anzinger founded a community there called Infinita City; he has also founded an investment company and ”
Silly me for thinking that this law is an excuse to allow longevity quacks to sell their wares legally! Let’s just say that Montana is just an expansion of Anzinger’s empire now:
As the founder of Infinita VC, a venture capital firm based in the charter city of Próspera in Honduras..He leverages special economic zones and enables legislation to unlock “stranded technologies” — innovations held back by traditional regulatory frameworks.
Operating within Prospera, Infinita is building a biotech-focused district that demonstrates how competitive governance can foster development. He pursues a decentralized strategy with 3–5 physical hubs, starting with Prospera and expanding to innovative US states like Montana under its SB 535 right-to-try framework.
And:
Niklas sees parallels between the longevity and crypto communities, including a shared ethos of questioning centralized systems and viewing institutions like the FDA as bottlenecks to progress. He believes the transition to more privatized and decentralized systems is already underway, with governments competing for talent and investment, and sees projects like Prospera as key steps in creating a “Cambrian explosion” of new jurisdictions and network states.
Oh, goody. Because I want my drug development to be like cryptocurrency! And also “experimental” gene therapies and stem cell therapies being developed with minimal oversight because Anzinger “questions centralized systems” and views the FDA as a “bottleneck” to progress.
But, wait! Montana tells us the law and rules have provisions to protect patients:
The state’s Department of Health and Human Services recently finalized rules to implement the law. The rules stipulate that patient consumers provide fully informed consent and that each application be reviewed by a board that includes a Montana-certified doctor, expert scientists, and an ethicist. Supporters of the law stress that they want the process to be responsible. “It will be done in a very rigorous way, with qualified medical professionals and appropriate oversight,” says Matt Kaeberlein, a scientist on the first board, which was formed independently of the state health department.
But other experts are worried about the potential for harm in selling unproven treatments to people without oversight from the US Food and Drug Administration. “I would be concerned,” says Aaron Kesselheim, a professor of medicine at Harvard Medical School with expertise in health policy and drug regulation.
So a board will review each application? That makes me feel so much better. In fairness, Kaeberlein is a legitimate longevity researcher and faculty at the University of Washington. So why did he agree to be on the Montana Experimental Treatment Review Board, a private entity set up to review applications for the Montana right to try program? (As an aside, I like how the METRB website notes drily, (“The Board’s review is a process-level safety check within the SB535 experimental treatment framework, is not a guarantee of safety or outcome for any patient, and creates no physician-patient relationship and no reliance for any patient.”) According to him:
Dr. Kaeberlein has long argued that the current development and approval process for new therapies is too slow and expensive. At the same time, he has frequently criticized the “Wild West” approach to longevity medicine.
“What interests me about Montana’s approach is that it attempts to create a middle ground,” he said. “Rather than pushing patients toward medical tourism or unregulated clinics, it seeks to establish a framework with independent review, physician oversight, informed consent, and systematic data collection. Whether that framework ultimately succeeds remains to be seen, but I believe it’s worth trying to make it as rigorous and scientifically credible as possible. That’s why I agreed to participate.”
Would it be wrong of me to retort, “Oh, you sweet summer child”? Or maybe not. According to the MIT Technology review article, Kaeberlein is a Kaeberlein” “has a prominent media presence, has long raised his own concerns about access to other unproven treatments, including peptides and stem-cell therapies.”
In fairness, the current board looks mostly unobjectionable from a scientific standpoint, and without going in depth into each member’s history I’d have a hard time saying more. I do know, however, that Flanigan is a libertarian who has written a book entitled Pharmaceutical Freedom.
Here’s the full panel:
Here’s the thing. It’s just a step. It will be very interesting to see what happens the first time this panel rejects a right-to-try application. My prediction is that, given the involvement of longevity aficionados at the genesis of this project makes me highly suspicious of just how much this is about granting patients access to promising drugs and how much it is about bypassing the FDA’s regulatory authority to allow unethical companies to profit selling early stage experimental therapeutics to patients, cash (or credit card) on the barrelhead. The law basically allows clinics to be set up for this purpose, and it is even mentioned in some of the stories that it is expected that the first of such clinics will open by the end of this year.
Yes, right now the panel doesn’t look like quacks and currently is the only review panel in the state, but the MIT Technology review article notes that “Anzinger says that other groups are free to establish their own” review panels,” and the only requirements for such panels that I could find is that they consist of at least four members, including at least one Montana-licensed physician, at least one researcher with expertise in clinical outcome data, and at least one ethicist. You know how quacks like to form their own institutional review boards? What’s in the law to prevent them from doing the same thing in creating right-to-try review boards?
Moreover, Kaeberlein points to the difference between current existing expanded access programs administered by the FDA in this way, as if it were a good thing:
There are some key differences between expanded access, which allows seriously ill people to apply for access to experimental drugs that might not have been through any human trials, and Montana’s approach.
And:
Kaeberlein also highlights another key difference, which is cost. Companies that make their treatments available through expanded access are only able to charge for the costs of making, transporting, and monitoring the drug, and they must justify the eventual price to the FDA. In Montana, they can charge whatever price they want. Stanley of WinSanTor says he plans to sell his drugs “at cost.” But Ceres’s Joudinaud says that he’d be more interested in selling his at a market price. When asked what that might be, he hinted that the prices of new drugs for rare diseases can be high. In recent years, the median price of such drugs was $218,872.
“Instead of simply creating a legal pathway for patients, it also creates a business model that companies may actually be willing to use,” says Kaeberlein.
No wonder certain companies are salivating at the prospect of profit:
But the moment when people start spending money on these treatments is already fast approaching. While Montana’s first ETRB prepares to review its first applications, clinics that hope to be part of the program are busy addressing the requirements laid out in the state’s new rules. Treatment rooms are being outfitted. Medical directors are being hired. And experimental treatments should be reaching patients in the coming months.
To my eye, even with the “safeguard” of the METRB, this program goes far beyond right-to-try in terms of a predatory libertarian model that locks out anyone who isn’t wealthy enough to pay for an unproven drug that probably won’t work. I predict that it won’t be long before we start seeing fundraising campaigns on social media from desperate patients wanting money to go to Montana to obtain unproven experimental drugs, the same way such campaigns by patients of cancer quack Stanislaw Burzynski made the news and went viral on social media 10-15 years ago. Am I being unfair? No doubt Kaeberlein would say so. I’m sure that if he sees this post he will be quite taken aback and feel that I’m being unfair. Let’s just say that this ain’t my first rodeo, and I see the way that quacks could easily take advantage of this. Heck, maybe Stanislaw Burzynski will move his clinic to Montana. Or maybe not. He’s an octogenarian now, and such a move would be very difficult. It must be tempting, though. Montana’s right-to-try environment is custom made for a quack like Burzynski to flourish, particularly given that he’s weaponized clinical trials to maintain his ability to keep selling his product.
Ironically, the only thing that might slow this down in Montana is that a lot of companies are afraid of what effect a disaster happening under right-to-try (e.g., the death of a patient using their product) would have on their reputation and relationship with the FDA, which they still count on to approve their drugs for sale in the US. That alone will be enough for the larger, more reputable companies to be wary of this program, but smaller companies might see the profit potential as too tempting compared to what it will take for them to get their candidate drugs through the FDA approval process.
Montana, “Right-to-Try 2.0,” and beyond
The spin that Montana has placed on “right-to-try” isn’t all. The Goldwater Institute, not satisfied with the original right-to-try, has promulgated “Right-to-Try 2.0,” which appears custom-designed to enable just about anything.
Rapid medical innovations have made it possible to take an individual’s genetic information and create a treatment for that individual person. More patients, especially those with rare and ultra-rare illnesses, will pursue these treatments when they have exhausted other options. Unfortunately, the FDA’s current regulatory scheme is not designed to handle these kinds of individual treatments, and that will keep life-saving medication out of the hands of patients unless reforms are adopted.
Under the FDA’s outdated system, new treatments are subject to a clinical trial, which is designed to learn something about a treatment that will be generally applicable to all the patients with the same disease. The current clinical trial evaluation is based on treatments for large population, not an individual patient which were not a reality when the system was created more than a half-century ago. The end result is that an individualized treatment is still subjected to the same clinical trial process as a single treatment that is intended for hundreds or thousands of patients. But that’s not how these new individualized treatments work.
For example, a therapeutic cancer vaccine can be designed based on an individual’s diseased cells or unique genetic mutations, allowing it to stimulate the patient’s immune response and target cancerous tumors. But that unique treatment only works for that patient. The FDA’s existing clinical trial system cannot work in cases like this because its safety and testing protocols aren’t matched to this kind of singular treatment.
One wonders, then, how, for example, CART-T cell therapy was ever approved. This is a form of therapy in which a patient’s own white blood cells are used to generate patient-specific therapies. There are problems, of course, developing individualized therapies and testing them for FDA approval, but the Goldwater Institute writes as though it were the first organization ever to address the problem. The FDA has been researching the “N-of-1” problem for years and has developed frameworks to approve therapies of this type without large randomized controlled clinical trials. A deeper discussion of this is beyond the scope of this post, although perhaps I should undertake one in the future. In the meantime, there are groups like the N-of-1 Collaborative, and in 2024 a framework was proposed to guide researchers regarding the evidence base needed to approve such treatments targeting an individual genetic variant. Meanwhile, the FDA has issued draft guidance on another framework for drug approval based on plausible mechanisms. I’m not saying that the problem has been solved. What I am saying is it’s a lie to claim that FDA doesn’t take the unique challenges that individualized therapies present into account and that Right-to-Try 2.0 is not a solution, but rather an opening to all manner of quacks to do “individualized” treatments without worrying about pesky things like FDA approval
Just look at what Goldwater proposes regarding eligibility. The drugs/treatments:
- must be investigational
- must be individualized to the specific patient, and
- must be based on analysis of the patient’s genomic sequence, human chromosomes, deoxyribonucleic acid, ribonucleic acid, genes, gene products (such as enzymes and other types of proteins), or metabolites.
While the patient must:
- Be diagnosed with a life-threatening or severely debilitating illness;
- Has considered approved treatment options;
- Has a recommendation for an investigative individualized treatment from their physician; and
- Gives written informed consent regarding the risks associated with taking the investigational treatment.
My skeptical antennae started twitching wildly as I read the use of the word “individualized” over and over again. After all, what is the quackery known as “functional medicine” but “individualized medicine” on steroids with lack of oversight? Basically, this Right-to-Try 2.0 looks like a back door to let functional medicine doctors do whatever they like.
As much as I rail against the antivax component of MAHA, the “health freedom” component is arguably more dangerous to science-based medicine, and at the vanguard of “health freedom” is right-to-try. While some of the anti-regulation libertarians who falsely believe that the FDA is killing people through bureaucratic slowness and inefficiency and think that the “free market” can guarantee drug safety and efficacy might really believe that right-to-try is about protecting patients, I argue that it is more about taking as much power away from the FDA as possible and taking us back to the 19th century in terms of drug science, safety, and efficacy. Worse, now that the current administration is in power, right-to-try advocates might just succeed. At the very minimum, as far as drug regulation goes Montana is looking to be a great place to set up a Hallwang-style clinic in which conventional medicine is mixed with unproven experimental therapeutics and alternative medicine.