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“Right to try” laws and Dallas Buyers’ Club: Great movie, terrible for patients and terrible policy

One of my favorite shows right now is True Detective, an HBO show in which two cops pursue a serial killer over the course of over 17 years. Starring Woody Harrelson and Matthew McConaughey, it’s an amazingly creepy show, and McConaughey is amazing at playing his character, Rustin Cohle. I’m sad that the show will be ending tomorrow, but I really do want to see how it ends.

Unfortunately, as much as I like Matthew McConaughey as an actor, he is in part responsible for re-inspiring a movement that has the potential to do profound harm to patients and cancer research. That’s because his other big role over the last year has been in an Oscar-nominated movie, Dallas Buyers Club, where he plays Ron Woodroof, an early AIDS patient who in the 1980s smuggled unapproved pharmaceutical drugs into Texas when he thought he found them effective at alleviating his symptoms, distributing them to fellow sufferers by establishing the “Dallas Buyers Club” while battling the FDA. I haven’t seen the movie, and I really don’t want to, given that, from everything I’ve heard about it, it’s basically the story of a “brave maverick” who bucks the FDA, complete with all the tropes about indifferent bureaucrats who don’t care if these brave patients die. That might not be so bad if it weren’t also riddled with inaccuracies and misinterpretations of the AIDS crisis in the 1980s. Worse, the real Woodruff rejected the one truly promising drug at the time, AZT, as hopelessly toxic and instead smuggled drugs like Peptide T, which never panned out. Basically, what Woodruff appears to have smuggled as part of his activities for the “Dallas Buyers Club” was a mixture of useless supplements, experimental drugs that were never approved, and a handful of experimental drugs that showed promise. Meanwhile, the movie portrays the FDA as the implacable enemy of these sorts of activities, jackbooted thugs not unlike the stereotype promoted by “health freedom” quacks who don’t like the FDA preventing them from selling their quackery. As far as I can tell without actually seeing the movie, the overall message is a typical uplifting story of an underdog who fights the power and in doing so finds redemption.

Of course, Dallas Buyers Club is just a movie, no matter how good a movie it might be and no matter how much Matthew McConaughey might have deserved an Oscar for his performance. Unfortunately, it appears that legislators in several states seem to think that it should serve as a template for health policy. This public policy, which is extremely bad policy and is being considered and promoted in four different states, comes in the form of bills known as “right to try” bills. Basically, “right to try” bills, if passed into law, would grant terminally ill patients the right to have access to experimental therapies. According to these laws, the drugs need only have passed phase I trials. Now, remember, phase I trials do not demonstrate efficacy. They are only designed to test for safety and toxicity, determine the maximum tolerated dose, and provide an estimate for the dose to use in real clinical trials. The concept of “right to try” bills is highly popular, because if you don’t know a lot about medicine and how clinical trials work it sounds like a good idea What could be the possible harm, after all? A lot, it turns out, but I’ll get to that in a moment. First, let’s take a look at the rationale for Arizona’s “right to try” bill, which is being promoted by the Goldwater Institute:

Arizona legislators are poised to green-light legislation in committee Thursday that would pave the way for terminally ill patients to access experimental drugs not yet cleared for market. Known as the “Right to Try” Act, similar bills are currently being considered by lawmakers in Colorado, Louisiana and Missouri, and legislators in California and Massachusetts have expressed interest.

Designed by the Goldwater Institute, the Right to Try Act would enable terminally ill patients who have exhausted all of their available treatment options to access experimental drugs that have been deemed safe but whose efficacy has yet to be determined. Under the current system, even after an investigational drug has passed the Food and Drug Administration’s Phase I (the testing phase during which safety is established), it can take an additional six or more years for the drug to be approved for market–even if clinical trials are yielding promising results.

And while many patients facing terminal illness attempt to get into those clinical trials, the vast majority cannot, because they are too far along in their illnesses or because of other factors. 40% of cancer patients pursue admission into clinical trials, but only 3% succeed. Last year, more than 500,000 Americans died from cancer alone.

According to Christina Corieri, a health care policy analyst at the Goldwater Institute, the tragedy is that many of the drugs terminal patients can’t access today will be saving the lives of future patients just a few years from now.

“The sickest Americans don’t have the luxury of time to wait for these drugs to come to market through the traditional process,” said Corieri. “The Right to Try Act puts the decision about whether to try an experimental treatment back where it belongs: in the hands of patients and their doctors.”

This is, of course, partially true. There is always an inherent conflict between wanting to push for faster approval of drugs in order to treat patients who are dying now and the need for rigorous testing to assure safety, so that more patients aren’t harmed than helped. Patients and their families ask, “What’s the harm?” while advocates like Corieri sell their policy with the assumption that experimental drugs are highly likely to help these patients, or at least not so unlikely as not to be worth trying. Here’s the problem. Just because a drug has passed phase I trials does not mean that it is effective. It does, however, frequently mean that the drugs have significant side effects. Indeed, determining those adverse events is part of the entire reason that we do clinical trials in the first place.

“Right to try” laws versus science

The “right to try” bill currently being considered in Arizona follows the template for such bills that are metastasizing to other states, such as Colorado, so I’ll look at it first. The text of the bill is available online. The major provisions of the bill includes the following definition of an eligible patient:

1. “ELIGIBLE PATIENT” MEANS A PERSON WHO MEETS ALL OF THE FOLLOWING:
(a) HAS A TERMINAL ILLNESS.
(b) HAS CONSIDERED ALL OTHER TREATMENT OPTIONS CURRENTLY APPROVED BY THE UNITED STATES FOOD AND DRUG ADMINISTRATION.
(c) HAS RECEIVED A PRESCRIPTION OR RECOMMENDATION FROM THE PERSON’S PHYSICIAN FOR AN INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE.
(d) HAS GIVEN WRITTEN INFORMED CONSENT FOR THE USE OF THE INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE OR, IF THE PATIENT IS A MINOR OR LACKS THE MENTAL CAPACITY TO PROVIDE INFORMED CONSENT, A PARENT OR LEGAL GUARDIAN HAS GIVEN WRITTEN INFORMED CONSENT ON THE PATIENT’S BEHALF.
(e) HAS DOCUMENTATION FROM THE PERSON’S PHYSICIAN THAT THE PERSON HAS MET THE REQUIREMENTS OF THIS PARAGRAPH.

The bill defines an “investigational” drug as a drug that’s passed phase I trials but is not yet FDA-approved. The bill doesn’t require manufacturers of an investigational drug to make it available, but basically allows them to do so if they so choose. Tellingly, the bill also doesn’t require that the manufacturer provide the drug for free, as it must to patients undergoing clinical trials in order to achieve FDA approval:

A. A MANUFACTURER OF AN INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE MAY MAKE AVAILABLE THE MANUFACTURER’S INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE TO ELIGIBLE PATIENTS PURSUANT TO THIS ARTICLE. THIS ARTICLE DOES NOT REQUIRE THAT A MANUFACTURER MAKE AVAILABLE AN INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE TO AN ELIGIBLE PATIENT.

B. A MANUFACTURER MAY:
1. PROVIDE AN INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE TO AN ELIGIBLE PATIENT WITHOUT RECEIVING COMPENSATION.
2. REQUIRE AN ELIGIBLE PATIENT TO PAY THE COSTS OF OR ASSOCIATED WITH THE MANUFACTURE OF THE INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE.
C. THIS ARTICLE DOES NOT REQUIRE A HEALTH CARE INSURER TO PROVIDE COVERAGE FOR THE COST OF ANY INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE. A HEALTH CARE INSURER MAY PROVIDE COVERAGE FOR AN INVESTIGATIONAL DRUG, BIOLOGICAL PRODUCT OR DEVICE.

And, to top it all off, the bill prohibits the state medical board (or any other state regulatory board) from going after the license of any physician or health care practitioner who recommends and/or administers such investigational agents to patients while making any state official or employee who attempts to block access of an eligible investigational drug to an eligible terminally ill patient potentially guilty of a class 1 misdemeanor. If the bill passes both chambers in Arizona, it would next go to the voters in November. My guess is that if the bill makes it onto the ballot this fall, it will likely pass. I can picture the ads now. They’ll feature cute, terminally ill children and brave adults battling fatal diseases invoking their right to choose what goes into their body and begging voters to “give them a chance to live” and asking, “What’s the harm?” Against such images it will be hard for science-based medicine to prevail.

Currently, similar bills are wending their ways through the legislatures of multiple states, including Colorado (of course!), Missouri (where legislators recently listened to emotional testimony and the bill is sponsored by a legislator, Jim Neely, whose daughter suffers from stage IV colon cancer), and Louisiana, while states as disparate as Utah, Oklahoma, Massachusetts, and California are showing early interest in such laws. The bills under consideration are basically all based on the Goldwater Institute’s template (indeed, the Arizona bill is almost verbatim the same), which is based on this policy report available on the Goldwater Institute’s website, deceptively entitled Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment.

The report itself is loaded with emotionally charged language about the FDA and terminally ill patients and highly dubious statements. For example, it’s hard not to notice that every experimental drug is apparently “potentially life-saving,” at least the ones that made it through phase I trials. There’s so much loaded language, coupled with so many dubious assertions, that I can only hit the “high” points, such as they are. For instance, the Goldwater Institute bemoans the expansion of FDA authority in the 1960s by the Kefauver-Harris Amendments that required that the FDA not just require demonstration of safety but of efficacy as well before approving new drugs. This expansion of FDA power was in reaction to the thalidomide debacle, leading the Goldwater Institute to make the rather bizarre (OK, very bizarre) argument that because the issue with thalidomide was a safety problem, not an efficacy problem and because thalidomide was never approved in the US (mainly due to the FDA, let’s not forget), the expansion of FDA power in response to the thalidomide debacle was “unwarranted.”

Another highly dubious argument follows:

Phase I involves administering the investigational drug to a small group of 20 to 80 volunteers to test for toxicity and immediately observable side effects.30 The major emphasis of Phase I testing is safety. Over 60 percent of investigational drugs in Phase I testing are deemed safe enough to move on to Phase II.

Seriously? Phase I trials can be as few as 20 patients. That is not enough to determine safety, nor is it intended to. Phase I trials are designed primarily to identify major side effects and to use a process known as dose escalation to determine what is commonly referred to as the “maximum tolerated dose.” It is utterly impossible for such a small clinical trial to determine the safety of a drug, and even the Goldwater Institute inadvertently undermines its own argument. Note how the report says that over 60% of investigational drugs pass phase I testing and are determined to be “safe enough to move on to phase II.” That’s the standard: No unexpected major adverse events and a side effect profile that isn’t grossly more unsafe than the disease itself. Phase II and Phase III trials are needed to confirm safety. That’s why the premature diffusion of unapproved drugs has the potential to increase morbidity from adverse events and even hasten death. One example is amonifide for treating breast cancer. The drug made it through phase I trials, but serious life-threatening hematologic toxicity emerged during phase II trials.

Think of phase I trials as a screening test looking for the most obvious toxicities, with phase II and III studies confirming them. Indeed, even phase III trials can’t always adequately demonstrate that a drug is safe; it’s not uncommon for less common adverse effects not to show up until post-marketing surveillance, when much larger numbers of patients receive the drug. Moreover, only 5% of all cancer drugs that enter clinical testing are ultimately approved for patient use. Among drugs tested in phase II trials, only 30% go on to phase III.

The Goldwater Institute also doesn’t like the current expanded access programs, not because it doesn’t like expanded access programs, obviously. (After all, what are “right to try” laws other than much more liberal expanded access programs?) No, what the Goldwater Institute doesn’t like is the current system because of all those nasty regulations. Under current law, the Food and Drug Administration Modernization Act (FDAMA) of 1997, single patient INDs (we’re discussed these before in the context of Stanislaw Burzynski patients), which are basically compassionate use exemptions, can only be granted if:

  • The patient’s physician determines the patient has no comparable or satisfactory alternative therapy;
  • the FDA determines there is sufficient evidence of safety and effectiveness to support the use of the investigational drug;
  • the FDA determines that provision of the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; and
  • the sponsor or clinical investigator submits information sufficient to satisfy the IND requirements.
  • the sponsor of the investigational drug must also be willing to supply the drug to the patient.

If all of these conditions are met, then the treating physician or drug sponsor submits an IND application, a proposed treatment plan, and a commitment to obtain informed consent from the patient and permission from the Institutional Review Board (IRB). No one denies that the process could be more streamlined. The report lists some of the burdens of the IND process and bemoans the FDA’s veto power over single patient INDs. What’s particularly disturbing about what the Goldwater Institute advocates, though, is its attack on the FDA’s requirement for a full IRB review of single patient IND applications as being a bad thing that keeps patients from accessing drugs. The language sounds as though it could have been written by Stanislaw Burzynski himself. Particularly Orwellian is the part of the report that demands that legislators must “act to protect patients.” I don’t know about you, but I’m not sure how, basically, stripping all restrictions on letting patients have access to drugs that have passed only phase I trials “protects patients,” but then I’m not a libertarian.

The Goldwater Institute justifies its proposal through still more dubious assertions. For instance, the report starts out with patient anecdotes in which patients with terminal cancers exhausted all conventional therapies and then applied for experimental therapies, which they either couldn’t get or had to wait too long to get. In each case, the stories are told in such a way as to suggest that “if only” these patients had had access to experimental drugs sooner they might have survived. This is, of course, nonsense. All these patients had stage IV cancer. Barring the incredibly unlikely possibility that an experimental drug is a miracle cure for advanced cancer even better than what the quacks claim in Tijuana for their treatments, these unfortunate patients would have died anyway. True, if the drug were effective, they might have gotten a few more weeks or months, but they would have died. Using such stories is nothing more than tugging at the reader’s heartstrings. It is not a good scientific argument.

Actually, there isn’t a single good scientific argument in the entire report. There’s a survey of orthopedic surgeons cited in which a majority of them have said they thought that the slow FDA approval process had hurt patients. This is well-nigh meaningless for a discussion like this one because (1) most orthopedic surgeons don’t take care of cancer patients (orthopedic oncology is a subspecialty, and, fortunately, orthopedic malignancies are rare) and (2) orthopedic surgeons are much bigger into medical devices than drugs. Moreover this survey is bundled in a table with other with results of other surveys that range from 1995 to 2007. Finally, the reference cited is from the Competitive Enterprise Institute, a free market “non-profit public policy organization dedicated to advancing the principles of limited government, free enterprise, and individual liberty.” I will admit that I’m a bit disappointed that between 68% and 73% of physicians would support a proposal to change FDA law so that unapproved drugs or medical devices could be made available to physicians as long as they carried a warning label about their unapproved status. Apparently, the physicians polled didn’t realize what a boon to pharmaceutical companies this would be! Why bother with those pesky and expensive phase II and phase III trials if you could market your unproven drugs or medical devices direct to doctors to use on patients after nothing more than a favorable phase I?

Unfortunately, the “right to try” movement is not limited just to the US. A reader made me aware of a similar bill being promoted in the UK right now. Known as the Medical Innovation Bill, this legislation was introduced into the House of Lords by Lord Maurice Saatchi, whose wife Josephine Hart died of cancer in 2011. While it’s easy to feel sympathy for Lord Saatchi, that doesn’t change how misguided the Medical Innovation Bill is. The bill is similar to “right to try” laws in that its ostensible purpose is to allow patients with terminal diseases to undergo treatments that are experimental or unorthodox. It’s different in the sort of tack it takes. First, Lord Saatchi seems obsessively focused on shielding doctors who use unconventional treatments from liability for doing so, even though malpractice suits are not nearly as big a concern for physicians in the UK as they are in the US. (One UK physician described them to me as being virtually “not an issue” for most docs in the UK.) That’s one difference.

Another difference is that Lord Saatchi’s bill doesn’t so much allow the use of experimental drugs outside of a clinical trial as to claim both to support “innovation” and to “expose the maverick.” Supporting the bill, Lord Saatchi claims that the “NHS predicts it will face a bill of up to £24 billion for legal cases in the coming years, and the figure is rising,” but doesn’t say how many years or how much of that is due to doctors being sued for using unapproved treatments for patients who are dying. My guess, but I don’t have the data, is that that fraction is very, very small. Even in the US it’s pretty rare for dying patients (or their families) to sue doctors who try unconventional therapies on them. The other aspect of the bill is this:

Doctors will not be protected by the Saatchi Bill unless they go through a rigorous and specific process to ensure that the attempted innovation is the right course of action for the patient.

As the draft Bill states in paragraph 2 (3a) a doctor wishing to try a new treatment – for example in the case where standard treatments aren’t working – must consult a body of senior and relevant medical experts and get their consent.

The doctor must also record their opinion, including and dissenting voices.

The decision of the panel of experts must then be presented to the patient – including any contrary opinions if there are any – and the patient must of course also agree to go through with the innovative treatment.

Finally, the note of the opinions must then be attached to the patient’s consent form as a permanent record.

Only then will the doctor be legally covered by the Bill.

The Bill imposes a much higher standard of consent than other health legislation.

If that’s truly the case, one wonders why the alternative medicine community has rallied behind this bill. If, as Lord Saatchi claims, this bill would tighten up considerably regulations over “brave maverick doctors,” why does one of the biggest organizations promoting quackery in the world, the Alliance for Natural Health, so strongly support it, the way it has supported, for example, Stanislaw Burzynski, arguably one of the most famous “brave maverick doctors” in the world? My guess is that it all hinges on the definition of “medical experts” in the bill. After all, for “integrative medicine,” who will be the “experts”? Other “integrative medicine” docs, of course! Meanwhile, in the comments we see stories like this from Dr W. Witvliet, who linked to this website on his name (I’ll let the website speak for itself but cite his comment):

my patient, was threatened with loss of his NHS contract just as I was if I refused to stop advising patients that arterial disease is reversible even though I had the photographic evidence from the retinal arteries to prove it.
The new science which I named CardioRetinometry not only eliminates

coronary heart disease and heart bypass operations but also reduces the incidence of cancer and diabetes.

It is so suppressed by the NHS that they were forced to get me struck off the register on a technical misconduct charge, for defending public health, despite my having 200 written testimonials and no lay complaint against me.

Quacks seem very enthusiastic about this bill. I wonder why. I also can’t help but note that supporters of Stanislaw Burzynski are enthusiastic supporters of “right to try” laws in the U.S. Indeed, Steve and Melissa Heil, whose daughter has a brain tumor and is being treated by Stanislaw Burzynski, were prominent among the patients and patients’ families testifying in favor of the Missouri “right to try” bill. Again, I wonder why. Parents like the Heils, I can understand. They honestly, albeit almost certainly mistakenly, believe that antineoplastons are the only thing keeping their daughter alive. Other than with family members who watched loved ones die and have been mistakenly led to believe that “right to try” bills might have saved their loved ones, I find it hard to be charitable with most supporters of “right to try” bills, as they appear to be mostly ideologically motivated, like the Goldwater Institute, or motivated by support for quackery, like the ANH.

I will likely have to look into Lord Saatchi’s bill in more detail, but it sure does smell, as one Twitter user described “right to try” laws, like a “Trojan duck,” a wonderful term that I will have to appropriate forthwith for legislation that appears reasonable on the surface but upon closer examination will promote the wider use of quackery. It is also based on the same magical thinking that underlies “right to try” laws.

The magical thinking behind “right to try” laws…

Given how bad an idea “right to try” laws are, here’s the funny thing: They almost certainly won’t do what they are designed to do. The reason is simple. They are nothing but state laws, and the FDA controls drug approval. Arizona can say as much as it wants that patients can get any investigational drug they want, but what the state of Arizona says is meaningless as long as the FDA says that you have to have a single-patient IND approved for patients to have access to an investigational agent outside the auspices of a clinical trial being undertaken to win approval for the drug. Because the FDA exercises a lot of its power through the federal government’s power to regulate interstate commerce, perhaps the only way I can imagine for a state to get around this would be if the pharmaceutical company is in the same state as the patient. If the company ships an investigational drug across state lines in violation of federal law, it’s screwed. Of course, even this might not be enough, because a lot of states have a “mini-FDA” act, which bans the use of drugs that haven’t been FDA-approved. Indeed, the only reason Stanislaw Burzynski managed to get away with administering antineoplastons to patients early in his career is because at the time Texas didn’t have a mini-FDA act, and Burzynski was very careful never to ship antineoplastons across state lines.

Even more pertinent, drug companies need their drugs to be FDA approved to recoup their investment in drug development and make a profit, because without FDA approval they can’t sell the drug. The Arizona “right to try” law requires that the “investigational drug” (1) have passed phase I trials and (2) still be in the clinical trial process. There’s only one reason for a drug to be still in clinical trials after phase I trials, and that’s because the drug company still wants FDA approval for its drug; i.e., that it hasn’t abandoned development. That means the investigational drug is still under FDA regulation. A single patient IND would still be required. Pharmaceutical companies providing drugs to patients in “right to try” states without proper INDs would be violating FDA regulations and would thus endanger their chances of ultimate FDA approval. In other words, these “right to try” laws are nothing but feel-good placebos. They have no real effect because reputable pharmaceutical companies will not cross the FDA by providing an investigational drug to patients without a proper IND. That means that the only companies that might take advantage of “right to try” laws would be disreputable companies like Stanislaw Burzynski’s institute. Like Burzynski, “right to try” laws offer nothing to cancer patients but false hope. In fact, I’m hard-pressed to think of anyone whom these laws would benefit other than Burzynski, which makes me wonder if his minions have anything to do with promoting them.

Indeed, making essentially untested drugs more widely available, as these “right to try” laws propose to do, will be far more likely to harm the individual patient than to help him. Advocates of these bills ask, “What’s the harm?” I’ll tell you: If there’s anything worse than dying of a terminal illness, it’s dying of a terminal illness and suffering unnecessary complications or pain for no benefit and having to pay for the medications causing the complications yourself. Remember, these laws allow the pharmaceutical companies to charge for their investigational medications. They don’t all necessarily require the company to provide the drug for free, as current law does in the case of single patient INDs. For example, as discussed before, the Arizona law doesn’t require drug companies to provide the investigational agent for free; the Colorado bill is being amended to have that requirement by its cosponsor Janak Joshi.

Worse, Dallas Buyers Club-style “right to try” laws risk undermining our entire clinical trial enterprise, which is a major part of the scientific basis for evidence-based medicine. After all, if early-stage experimental drugs were made widely available outside of clinical trials and taken for a wide variety of cancers, the signal-to-noise ratio would become very low. It would become very difficult to tell which drugs were working and for which cancers (and which were not), particularly since it would be reasonable to expect that such a policy would result in enrollments in clinical trials plummeting. And what would be the potential payoff for the shredding of patient protections proposed in these bills, even if the FDA didn’t put the kibosh on widely providing experimental drugs early in clinical testing (and, make no mistake, only having passed a phase I trial is early in clinical testing)? Very little at best, if even any at all. In reality, the likelihood of saving the lives of even a handful cancer patients by giving them access to early-stage investigational agents is quite low and hard to justify on a moral and practical basis, given the high likelihood of potential harm or premature death to so many other patients through the damage or destruction of a system that has been built up at such cost over several decades.

The entire justification for “right to try” laws also seems to rest on a misperception that there are “miracle drugs” out there that we will have to wait years for because the FDA is too slow to approve them. However, if there really were such a “miracle drug” that was amazingly effective compared to anything we have now, a large randomized phase III trial would not be necessary to detect its efficacy. Indeed, its efficacy could show up in even a small phase I trial or, at the latest, in phase II trials. There’d be examples of clinical trial subjects demonstrating amazing tumor shrinkage or even outright cures. In reality, we don’t see these things in phase I trials, because there are no miracle drugs, at least not yet (if there ever will be). Dallas Buyers Club-style “right to try” bills rest on a fantasy, and it’s a fantasy of false hope. Indeed, these bills serve an ideological purpose rooted in libertarian politics far more than they serve patients. It’s not coincidental that virtually the only sign of opposition to the Missouri “right to try” bill was from lobbyists representing the hospice community, who warned lawmakers that such legislation raises false hopes for patients when further treatment is likely futile.

The same is clearly true for Lord Saatchi’s Medical Innovation Bill, which seems to rest on an underlying assumption that there is a cornucopia of “unconventional” treatments out there waiting that could save thousands of patients with terminal cases of cancer and other diseases, if only doctors were allowed to use them more widely. Never mind that pesky scientific and clinical trial process that’s usually needed to demonstrate whether a treatment works or not. Given Lord Saatchi’s experience watching his wife die of cancer, one can understand his sentiments. However, I will point out that I watched my mother-in-law die what was undoubtedly an equally unpleasant death from metastatic breast cancer, and I know that no treatment, conventional or “unconventional,” could have saved her life. And, yes, I saw to it that she was evaluated by one of the best, if not the best, phase I oncologist in the world, an oncologist who is currently part of a Stand Up To Cancer Dream Team of researchers. My wife and I learned the pain and frustration of being informed that that she wasn’t eligible for any phase I trials. It is painful for me to admit this publicly, but I think it is important for you (and Lord Saatchi, if he sees this) to know that I have a personal understanding of the pain of watching a loved one die of cancer after having been told there’s nothing more that can be done for her. I still conclude that “right to try” bills and Lord Saatchi’s Medical Innovation Bill are profoundly misguided and based on magical thinking that I myself was tempted by.

Conclusion

These bills seem new, but they’re just the latest wrinkle on an old story. We’ve been down this road before, just without an Oscar-nominated movie to add glitz to the campaign. For example, the Abigail Alliance has been lobbying for similar “right to try” laws for a decade now. Libertarians love these bad ideas because, you know, the market cures all and people should be allowed to sell and buy pretty much anything they want. So does the press because of the human interest stories coupled with the “little guy” battling the FDA. Even though expanded access programs could definitely use some tweaking, never forget that the reason the laws we have exist is to protect the public against drugs that don’t work or are too toxic and, just as importantly, from companies that would sell such drugs with no evidence of efficacy or safety and from “investigators” like Stanislaw Burzynski.

Supporters often point to the example of Gleevec (imatinib) for chronic myelogenous leukemia (CML), and, indeed, Gleevec did show spectacular results in its first phase I trial. What advocates gloss over is that results like what were seen in three early stage phase I/II trials for Gleevec in CML are incredibly rare. In fact, before Gleevec, such results were virtually unprecedented:

Two years later, this chemical, which was called ST1571 and eventually renamed Gleevec, entered its first clinical trial: a small phase I trial involving just 31 patients. Remarkably, all 31 individuals experienced complete remission; in other words, their blood counts returned to normal. In some of these patients, there was also cytogenetic remission, meaning that the Philadelphia chromosome was no longer found in their blood cells. As Druker noted, “That was virtually unheard of in a phase I clinical trial. Usually in a phase I clinical trial, if you see a 20% response rate, that’s remarkable. We had a drug that was extremely well-tolerated and had a 100% response rate. It was absolutely incredible to see this unfold” (Taubes, 2003). Subsequent clinical trials produced results just as astonishing.

Also, Gleevec was an unusual drug from a mechanistic standpoint as well because CML depends upon a single mutation resulting in a single aberrant protein whose targeting can completely reverse the cancer:

In a way, Gleevec is an exceptional case, and the same success is not likely to be achieved with other cancers any time soon. Unlike most other cancers, which are caused by a multitude of complex interacting genetic and environmental factors and therefore have many targets, CML is caused by a single aberrant protein related to a consistent chromosomal translocation. Scientists were thus able to focus all of their efforts on this single target. Nonetheless, the Gleevec story is no less an excellent and, some would say, beautiful example of how knowledge of the biological functioning of a cell can lead to life-saving medical treatment.

If there’s a drug that’s shown as incredible a result in an early phase I trial as Gleevec, I am unaware of it. Again, that’s the point. Gleevecs of the world are rare, but Dallas Buyers Club “right to try” legislation rests on the assumption that they are much more common than they actually are and that thousands of patients with terminal cancer would be saved if only the FDA weren’t such prigs about insisting on evidence of efficacy and safety. It’s a fantasy, not reality.

Don’t forget: Dallas Buyers Club is just a movie, nothing more. It might be a really great movie (given its Oscar nominations and McConaughey’s winning Best Actor, it almost certainly is), but it’s not fact. It’s a fictionalized account of man’s battle with AIDS that is designed to entertain and uplift more than to teach about history and science. As such, it’s a terrible model for health policy. Widespread adoption of Dallas Buyers Club-inspired “right to try” laws is far more likely to harm rather than help patients with cancer and terminal diseases.

Posted in: Cancer, Clinical Trials, Politics and Regulation, Public Health

Leave a Comment (60) ↓

60 thoughts on ““Right to try” laws and Dallas Buyers’ Club: Great movie, terrible for patients and terrible policy

  1. mho says:

    Is the release from liability for indefinite? Are there any provisions for how long can a “terminally ill patient” can continue on these drugs if a miracle occurs?

    Nevermind, I can see in the laws that the answer is no.

  2. mho says:

    dang,I hit send too soon.
    my first question should be, Is the release from liability for an indefinite period of time?

  3. weing says:

    I think these laws should be prefaced with “You have the right to be stupid and pointless”. Will these laws apply to peyote, coca leaves, opium, psilocybin?

  4. Stephanie says:

    I agree with this post whole heartedly, but I just don’t think the AIDS epidemic in its early stages is a good example to illustrate use of a bad policy. The HIV/AIDs community had to organize and advocate for itself for more research, development, and treatment by pushing through this ‘right to try’ policy. I don’t think we would be wise to perpetuate it for reasons you outline, but it is perfectly understandable how an ostracized and abandoned community demanded the opportunity to test drugs themselves out of sheer desperation to survive. The drugs themselves never turned out to be any better, but what led to finding the right drugs and combinations was most certainly (and still is) a result of the intense pressure from activists put on the authorities and pharma, and the eventual partnership with laypeople who educated themselves and became experts on their own health. Some of the same key players today in international HIV/AIDs funding, research, and development are some of those original patient activists. I can’t imagine we would ever have a repeated scenario such as this, but there was no incentive then to test these drugs any quicker. The actions of these patients were effective catalysts.

    1. Andrey Pavlov says:

      @stephanie:

      I didn’t read Dr. Gorski as condemning what actually happened back then, merely that for right now it is a bad platform on which to base laws. As you pointed out it was an act of desperation in what was truly a desperate situation. That just doesn’t quite apply these days. Also, that doesn’t negate the “creative license” taken with the movie itself, which may make for a good movie but also engenders a false impression of “evil” FDA and clinical trials.

      I actually haven’t seen the movie (yet, it is in my queue) but a friend of mine did when it came out. She is very literally a crunchy vegan hippie type (who is a pilates instructor and professional dancer), but I love her to death because she is also reasonable and very fun to be around. She asked me about the movie and her impression was that it was a true story about how the evil FDA tried to harm people and prevent them from seeking the care they wanted. She came away with the impression of an oppressive authoritarian government cracking down in raids and otherwise stifling individual freedoms of those desperate people. The reason I like her is because even though she felt that way (and to some degree still believes it) she has hung around me to be skeptical of it and asked if it was really true. I reckon many others wouldn’t be so skeptical and assume that it was a close approximation of the truth, which is simply false.

    2. WilliamLawrenceUtridge says:

      If you read some of the online criticisms of the movie, you’ll see a lot of people pointing out the large number of historical errors it contains. I hate to cite the Huffington Post, but:

      Researchers conflicted by the ethics of the trials that they were conducting called to leak findings to us in advance of their publication.

      It’s accuracy on a number of points is strongly questionable, and I dislike how passively it portrays the gay community, awaiting their straight saviour (who was actually probably bisexual) to sell them the drugs they wanted.

      The FDA made a practice of looking the other way for people buying drugs for personal use, and Woodroof actually sued the hospital for the right to participate in an AZT trial. And let’s not forget the imprtant fact that he was wrong, AZT is and was a lifesaving drug (that was poorly dosed). The film focusses on a very, very narrow slice of history, a slice that allows it to portray the FDA at its worst before accomodations were made to expedite the drug approval process and ignores the political context of the day. As the Salon article says – it wasn’t imported unapproved drugs that put a stop to the AIDS crisis (not that even HAART has done so, but at least it lengthened life expectancies dramatically).

      The film goes out of its way to make the FDA look draconian if not outright murderous of homosexuals and their saviour, who started out looking for ways to save his own life, and eventually turned it into a lucrative financial enterprise.

  5. Frederick says:

    I saw the movie yesterday, as much as as like the actors, it could not like the way they distorded the truth, trying to make clinical trials look evil, and what not. it is not that bad, the movies seem to try to look neutral, but it fails. It is also hard because the director , Jean-marc vallée is a Quebecois, like me. He did not write it at least.
    The make the Doctor who supervised the Trials look Evil ( when in real life what the done saved thousands of live) and the Outcast doctor in mexico, who sell cheap, not proven and nonworking stuff, as the hero. But he was a crook. yeah the company did profit ( and a tons) on a product that most research were done with public fund, But none of that critic appear in the movie and it is the real critic the movies should have talk about. But AZt worked, and still is. As for right to try law, it is ridiculous, I don’t have anything to add, it was all well summer up Here and by orac.

    off topic :
    As far as Director from Quebec doing movies in the US goes, I think Denis Villeneuve is better, Prisoner was a lot better than DBC and i can not wait to see Ennemy.

    1. WilliamLawrenceUtridge says:

      I love how the people selling the ineffective drugs that probably led directly to deaths are treated as the heroes, and the fact that the drug they opposed is actually safe and effective in appropriat doses, is completely glossed over except for a single sentence at the end. “Oh, by the way – AZT was approved by the FDA and Peptide T was worthless”. Yeah, that makes up for two hours of “DRUGS ARE EEEEVIL”. Totally guys. Totally.

      1. David Gorski says:

        Indeed. The real Dallas Buyers Club probably didn’t really directly medically help a single patient and clearly indirectly harmed patients through Woodruff’s telling AIDS patients that AZT was poison that they shouldn’t take. AIDS patients who listened to Woodruff about AZT missed out on the single drug existing at the time that had a shot at prolonging their lives and died sooner than they needed to. Yet, Woodruff and the doctor in Mexico providing largely useless treatments are portrayed as heroes, while the scientists doing clinical trials to try to find drugs that would help AIDS patients are portrayed as hidebound, greedy, and obstructing AIDS patients from getting real treatment.

  6. mousethatroared says:

    I haven’t seen the movie, but from what I’ve heard (here and other places) the storyline seems kinda disappointing, from a creative perspective. It’s like they took an event that would have been interesting and intellectually challenging in it’s own right…that could have really had people questioning all sides – patient, FDA, the public, politicians – and forced it into a pre-conceived narrative.

    Oh well, I guess I will have to see it before I can really say that though.

  7. Marion says:

    I never know with these “naturalists” whether they are gung-ho anti-drug like the DEA or whether they are gung-ho pro-drug-choice like the legalize-pot people. Clearly, they just arbitrarily pick & choose according to their own political whims.

  8. Ryan says:

    Eh… the historical accuracy of this movie in question aside, I find the article unconvincing. I mean, the article points out that the drugs might be dangerous with low efficacy, but that’s kind of the point. These people are on their way to their grave. Sorry if this sounds a bit creepy, but they’re probably the best group to test out potentially dangerous drugs out on. I’m actually really surprised this isn’t already how things work. People might try some woo, sure, I guess. But I doubt that will be the primary beneficiary of these laws. I think it will be primarily used by companies who’ve sunk a couple billion dollars developing a new drug (I just looked that up – 4 or 5 BILLION dollars is how much it costs to develop a new medicine) who can now skip over some portion of animal testing. This means the drugs can come out quicker and more cheaply and make medicine a little bit more flexible. Anything that costs billions of dollars is going to be bound to be cumbersome.

    So, if these bills are because people have difficulty distinguishing reality from Hollywood, that isn’t in itself a good thing. But the law might wind up doing more good than bad.

    1. WilliamLawrenceUtridge says:

      Several points Ryan:

      - such trials are undertaken (phase I trials are often in patients with active disease, depending on drug and disease)
      - these sorts of laws will, if enacted, grind research to a halt (or at least delay it significantly) as it will be harder to recruit patients, nigh-impossible to analyze the data, and if Big Pharma can sell directly to the desperate, remove much of the incentive to undertake drug development and testing
      - while the health and well-being of desperately ill patients is something to be respected, there is something also to be said for trying to help future patients
      - as discussed in the article, most protodrugs simply don’t work because human biochemistry is hard
      - how you calculate how much a drug costs is actually a complicated question, mostly because human biology is complicated and you have to go through thousands of potential compounds before an active one is identified

      But the law might wind up doing more good than bad.

      There seems to be this perception that “good drugs” are just waiting to be found if only Big Pharma weren’t so lazy and greedy. But “good drugs” are hard to find because they are hard to find. They have to have a metabolic effect. They have to survive digestion, injection, transportation, processing by the liver, excretion by the kidneys, passage through the blood-brain barrier, storage, and more. All while being non-toxic in a body that duplicates its receptors repeatedly because evolution is a lazy, “good enough” process. Imagine trying to find a hotel room with a key that is labelled with the number “20,603″, but the rooms themselves are not sequential and the numbers have to be spelled out with only three pieces of uncooked macaroni, because it was cheaper than buying the actual numbers and the staff knows their way around.

      As noted in the article (or possibly the other article on this topic), these laws are pretty unlikely to help people because there aren’t many effective drugs just lying around and participation in these trials, and they encourage people away from things like living your last months in peace with well-controlled pain, doing things like spending time with family and taking that trip to Italy you always wanted.

      1. agitato says:

        “Imagine trying to find a hotel room with a key that is labelled with the number “20,603″, but the rooms themselves are not sequential and the numbers have to be spelled out with only three pieces of uncooked macaroni, because it was cheaper than buying the actual numbers and the staff knows their way around.”
        Clever analogy. Was there a reason you chose the number 20,603?

        1. WilliamLawrenceUtridge says:

          That’s my bench, in ounces.

          1. agitato says:

            Wow. Strong like (Belgian Blue) bull.

            1. WilliamLawrenceUtridge says:

              Bulls have a surprisingly poor bench, you have to surgically attach opposable thumbs and they hate it when you put them on their backs. I prefer to think of myself as a gorilla. But sadly, with more body hair.

    2. mho says:

      “These people are on their way to their grave.”
      Actually, everyone is on the way to the grave.
      Cancer patients, in an effort to minimize some of their fears about how close they are to death, joke, oh well, I could walk out the door and get hit by a bus.
      Should the terminally ill be thrown under the bus? I don’t think so. . .

  9. RE: Is comparing the terminal patient’s “right to try” to the unscrupulous physician’s “right to prey” relevant, nowadays!?

    In the above magnum opus, I thought that David Gorski has had presented a very dubious case of defending the SBM/FDA issues, against the upcoming Arizona’s proposed “right to try” bill for the terminally-ill patients — by confusing and interpreting the issue as “that the only companies that might take advantage of “right to try” laws would be disreputable companies like Stanislaw Burzynski’s institute. Like Burzynski, “right to try” laws offer nothing to cancer patients but false hope. In fact, I’m hard-pressed to think of anyone whom these laws would benefit other than Burzynski, which makes me wonder if his minions have anything to do with promoting them.

    Fear not: the “right to try” is a “terminal patient advocacy” law (if passed); whereas Burzynski has had been practicing his “antineoplaston quackery” and deceiving patients under the guise and privilege of his “physician-patient confidentiality” laws since the 1970s, while bypassing the normal IND-FDA laws totally, big time — until recently!?

    In fact: the “right to try” laws will not only help any competitive, responsible, companies widen the bottleneck of their FDA-regulated channeling of their IND research and development programs; but also help accelerate their future clinical trials and RCTs (if necessary) alike, in times to come!? — Won’t that be a good thing for all healthcare concerns (SBM; FDA; critics; patients; pharmas; biotechs; hospitals; etc)!?

    Best wishes, Mong 3/11/14usct2:57p; practical science-philosophy critic; author “Decoding Scientism” and “Consciousness & the Subconscious” (works in progress since July 2007), Gods, Genes, Conscience (iUniverse; 2006) and Gods, Genes, Conscience: Global Dialogues Now (blogging avidly since 2006).

    1. David Gorski says:

      Fear not: the “right to try” is a “terminal patient advocacy” law (if passed); whereas Burzynski has had been practicing his “antineoplaston quackery” and deceiving patients under the guise and privilege of his “physician-patient confidentiality” laws since the 1970s, while bypassing the normal IND-FDA laws totally, big time — until recently!?

      Oh, really? If that’s true, then why is it that Burzynski patients are being urged to write their legislators in support of such laws, while legitimate pharmaceutical companies remain very cool about them? Why is it that Steve and Melissa Heil, whose daughter has a brain tumor and is being treated by Stanislaw Burzynski, were prominent among the patients and patients’ families testifying in favor of the Missouri “right to try” bill?

      No, these bills are being sold as “terminal patient advocacy” laws, but they are not. True, some of their advocates intend them that way and think these bills, if passed into law, would help terminally ill patients, but the way the bills are written makes it clear that the primary motivation is to try to bypass regulations that protect patients. The reasons vary. In the case of the Goldwater Institute, it’s clearly because of libertarian ideology more than anything else. The entire text of the Goldwater Institute’s justification for the Arizona bill is dripping with contempt for government regulation in general and the FDA in particular; I doubt the Goldwater Institute really gives a rodent’s posterior about terminally ill patients here. In the case of terminal patients, it’s largely because they’ve been sold a bill of goods, in which the potential benefits of these “right to try” bills are exaggerated beyond any reasonable estimation of their likelihood of helping terminally ill patients.

      No, these bills are, as I’ve called the, Trojan ducks. They look reasonable on the surface, but they are designed to provide a means for the unscrupulous to bypass the FDA. It’s not a coincidence that pharmaceutical companies didn’t ask for them and don’t support them, while quacks enthusiastically do.

    2. windriven says:

      “In fact: the “right to try” laws will not only help any competitive, responsible, companies widen the bottleneck of their FDA-regulated channeling of their IND research and development programs; but also help accelerate their future clinical trials and RCTs (if necessary) alike, in times to come!? ”

      Horse hockey. Vanishingly few legitimate pharmaceutical or biotech companies will be eager to sell into a “right to try” or compassionate use market. There are too many opportunities for uncontrolled use to go awry with potentially devastating downstream consequences for the agent in question.

      1. WilliamLawrenceUtridge says:

        I am not a business person, so I wonder about the effects of these amendments on Big Pharma’s bottom line. Would they make more money selling half-tested drugs to desperate patients, and would the bad publicity be enough to keep them away? Would it quash true innovation and research because it is too easy to pass the low bar of phase I trials then stop? Would it put more drugs in the hands of publicly-funded researchers who could then run trials free of pharma influence? Would it drive people into mostly phase I trials, or out of them completely? What are the long-term implications, and who will try to game what for sport and profit?

        Of course, naturally the quacks will use it to shill their nonsense. But then again, they’re already doing so with DSHEA.

  10. windriven says:

    ” the market cures all and people should be allowed to sell and buy pretty much anything they want.”

    That, I think, misstates the libertarian position which would hold that people should largely be able to buy whatever they wish but that sellers would be prohibited from selling using deceptive or fraudulent tactics. And this presumes that the seller is a willing seller.

    1. WilliamLawrenceUtridge says:

      Probably depends on the libertarian, some would doubtless feel that they can do adequate research to inform and thus protect themselves (much like Woodroofe’s fictionalized character did) and object to any restraints on commerce.

      Me? Give me some nice socialized medicine any day. Trust me, it’s great. The United States is doing itself a huge disservice stopping at mere health insurance reform.

      1. windriven says:

        William,
        I love the idea of a single payer system I hope we adopt one here – but I’m not holding my breath.

        In a perfect world I might argue that a free market solution would be more efficient – but there is nothing … no thing whatsover … that is free about the healthcare marketplace. And there is never going to be.

        So, if we can’t have a free market solution then there is no point pretending; go for a single payer and work to make it efficient. Maybe we should hire the Danes to run US healthcare ;-)

        —-

        But again I digress – libertarianism demands the rule of law including hard restraints on dishonest trade. What you are suggesting looks more like laissez-faire and that is something I would never advocate.

  11. CommonSenseBoulder says:

    I am guessing that most people here are pro-choice and should grasp that people have some right to control their own bodies.. for better or worse. I’d suggest that there should be a high barrier to straying from that concept to give government control over choices that should be up to the individual. I’ll note that on the issue of treatment choices I am firmly in the scientific medicine camp and opposed to the nonsensical “alternative” treatments you folks debunk. The cause will be won however through educating the public, not through trying to use the legal system to impose our approach.

    It doesn’t make sense to push government to infringe on rights in ways that can come back to haunt you if those you hate are ever in control. Despite lack of scientific basis for alternative treatments, their providers tend to have political clout in part because not enough of the public is scientifically literate. Consider a thought experiment (even if it is fortunately never likely to happen), what if one day alternative medical providers had control of government policies and dictated the FDA approve only remedies they approve of and outlawed many treatments we’d approve of?

    Even if they don’t go that far and merely have some ant-science types in the FDA, try exhibiting some empathy and placing yourself in the position of someone who is well informed and wishes to try a treatment but aren’t allowed to because of some anti-science politician that tried to make it harder for pharmaceuticals to be approved.

    Part of the problem today is that the FDA is badly run, in part since it is a monopoly, so it takes a long time even for good treatments to be approved. You folks have the fantasy we can get a competent science based medicine FDA and government if we just wish hard enough and try to convince them using reason. Economists who study how governments work in reality (rather than naive fantasies of how we wish they did) suggest that is unlikely. (e.g. see regulatory capture theory, which won George Stigler his nobel prize, which argues special interests tend to control regulation by default (which slows down approval of new drugs, to benefit existing drugs). Public choice theory which won James Buchanan his nobel and which he described as “politics without the romance” also argues that government benefits special interests (including alternative med types) not necessarily the general interest). Unfortunately studies show most people are economically illiterate, and many who push sound medical science push junk economics and dismiss rational economics out of hand in your desire to believe in a fantasy model of a government that acts in our interest. Jefferson noted we hadn’t found angels in the form of kings to govern us, and today we haven’t found angels in the form of politicians and bureaucrats.

    Competing private safety approval agencies would speed up the process of drug approval, with perhaps options like providing insurance backing up their assertion of safety to provide them with added incentive to get things right.

    If someone is dying and wishes to try a treatment you don’t approve of, what right have you to prevent someone from putting whatever they wish into their body? Aren’t they more likely to get solid medical advice if they can choose to do so legally with a doctors advise rather than being forced to go underground and no risk talking to a competent doctor? It may be that you have science on your side, and that the bulk of treatments people wish to try are nonsense, but obviously there is a chance some new treatment will be found that will help.

    People seem to forget that this is supposed to be a free country, no matter how much you would prefer to force everyone to do things the way you wish as benevolent dictator. You can argue “but more people will be harmed by granting people freedom”, but that is ignoring that individuals are supposed to have rights, including the right to make stupid decisions, and the right to save their own lives. Jewish members of the ACLU defended the right of neo-Nazis to march since they grasped that even those you despise should be granted rights. What if some group were in power that despised you and wished to take away your rights, like the right to use pharmaceuticals you approve of?

    1. windriven says:

      “The cause will be won however through educating the public, not through trying to use the legal system to impose our approach.”

      Hear, hear!

    2. WilliamLawrenceUtridge says:

      I am guessing that most people here are pro-choice and should grasp that people have some right to control their own bodies.

      You can’t really compare drug approval to abortion though – abortion has recognized effects, known efficacy and is extremely safe. Drug approval, as evidenced by “right to try” amendments, is characterized by uncertainty of safety and efficacy, not to mention outcome. The oversight over abortion that ensures safety is what renders it a viable choice, superior to the unregulated option. “Right to try” laws would remove such oversight.

      Competing private safety approval agencies would speed up the process of drug approval, with perhaps options like providing insurance backing up their assertion of safety to provide them with added incentive to get things right.

      Private governance and oversight is no panacea, witness Standard & Poor, and Moody’s atrocious handling of credit default swaps on sub-prime mortgages leading to the 2008 financial meltdown. Collusion would probably be a not-insubstantial concern.

      1. CommonSenseBoulder says:

        re: “Private governance and oversight is no panacea, witness Standard & Poor, and ”

        The securities rating agencies are *not* remotely close to free market entities. They are a government propped oligopoly with a backwards business model that never would have survived in a free market. The government requires the use of one of a small number of agencies by banks for instance, and sets things like capital requirements for banks based on them, and others wind up using them since they figured they were better than nothing (which proved to be questionable). In the world of individual lending banks choose the most accurate credit report they can find. If it operated like the securities rating agencies, you would instead get to find the best credit report someone would compete to sell you, and your bank would be given government incentive to use it.

        Often what people misguidedly think is “market failure” is in reality bad regulations, and they simply don’t know enough about the topic. For instance the FDIC (instead of private deposit insurance) didn’t risk weight its premiums until after the S&L crisis, and only then added minimal risk weighting, so it created perverse incentives for banks to take more risks. In contrast private deposit insurance would have deterred risk taking by appropriately risk weighting premiums, and set them at a level that covered the costs.

        re: “abortion has recognized effects, known efficacy and is extremely safe.”

        You seem to be implying that if abortion were risky you wouldn’t allow it? Shouldn’t the risk be up to the woman to decide? People engage in risky activities like riding motorcycles, playing football, boxing, etc.

        Why should other people get to decide what risks someone takes rather than leaving it up to them to decide for themselves, and merely advising them on what makes sense?

        If you can decide what others can put in their bodies, why can’t religious right types outlaw people putting other things in their bodies (like a woman not being allowed to put a man not her husband in… or a man not allowed to put another man in..)? Why should you have the right to dictate the private actions of an individual in their own home away from others? Let me guess, because you assume you are right, and they are wrong. They think they are right, so from a standpoint of politics you both seem to make the same argument,that you wish to control others for what you think is their own good.

        The answer should be to prevent anyone from taking away people’s right to do with their own bodies what they wish. I am a non-believer, I’d disagree with the judgements of the religious right and agree with science based medicine types regarding medical treatments, but I don’t see what right I have to impost my views on others.

  12. CommonSenseBoulder says:

    re: “Because the FDA exercises a lot of its power through the federal government’s power to regulate interstate commerce”

    People should ask themselves why a constitutional amendment was needed for alcohol prohibition, yet magically the FDA these days gets away with banning whatever it feels like. The answer is that the constitution’s meaning has been distorted over time to usurp authority the federal government wasn’t intended to have, and was understood not to have for most of this country’s history. . Many people today seem unaware that the commerce clause was part of the constitution to prevent trade barriers from being enacted between states. The connotation at the time of “regulate” was to “make regular”, and the connotation of “commerce” was trade. It has been unfortunately distorted to allow micromanagement of the production of goods that are traded, which is outside of the act of “commerce”, i.e. trade itself.

    When the constitution was passed there was a great deal of debate over it since the states were wary of giving away more power to a central government. There is little chance they ever would have approved a constitution they thought would allow the level of federal micromanagement of the country that that exists today. The Constitution should be amended to grant the federal government new powers, not ignored as it has been. The idea was to leave most power in the hands of the state&local governments who could compete and learn from each other to see what approaches work best.

    Unfortunately many people try to suggest we have a “living constitution” and should just go with the flow. Pragmatically we may need to live with it, but we can still argue to try to change that approach over time. The problem with letting the courts get away with granting the federal government powers you like is that they may also grant it powers you don’t like, such as perhaps letting a future religious right controlled government impose their views, censorship be imposed, more spying on the public, and various other infringements of rights. A “living constitution” is a dead constitution since if it can mean anything, it means almost nothing and can’t be viewed as a safeguard for the rights you do value. The power you wish the government to have to infringe on rights to do things you like could be misused someday by a government composed of people who wish to do things you don’t like.

    1. David Gorski says:

      People should ask themselves why a constitutional amendment was needed for alcohol prohibition, yet magically the FDA these days gets away with banning whatever it feels like.

      Uh, no. There are laws regulating what the FDA can and cannot do and regulations based on those laws regarding the process for “banning something.” That process is quite long and requires a lot of investigation and justification, so much so that cranks like Burzynski kept operating for decades despite many violations of FDA regulations. In fact, arguably in the case of supplements, the laws hamstring the FDA and keep it from being able to protect the public.

      1. CommonSenseBoulder says:

        re: “Uh, no. There are laws regulating what the FDA can and cannot do”

        You seem to have trouble grasping that there is a difference between a Constitutional amendment for a ban, such as alcohol prohibition was, and laws.

        re: “That process is quite long and requires a lot of investigation and justification”

        Regulatory capture theory suggests that industry will try to protect existing drugs from competition by new companies with new drugs, which motivates them to see the approval process be long. In addition the FDA tends to be bashed when people get sick due to an inappropriate approval, but not due to people dying due to lack of availability of a drug. That leads drugs to be kept off the market too long, which leads to a need to allow people to bypass that slow process.

        The problem is drug approval is a monopoly when done by government, but there is no need for that to be the case. Private safety labels could compete to earn the trust of doctors and insurers (the FDA could be privatized to become the first), perhaps providing some insurance to back up claims of safety so they lose money if they get it wrong. Competition will lead to faster approval times, and monetary incentive to get things right will promote accuracy to balance speed. Labels would bash each others faults, and insurers and doctors would decide which to use.

  13. CommonSenseBoulder says:

    Just as many alternative medicine types dismiss strawman versions of science they don’t really understand (often mocking science which seems counter-intuitive to them), many who don’t understand economics and business dismiss simplistic strawman versions of concepts they don’t understand. People that bash junk science sometimes seem to fail to consider that they may be pushing emotionally appealing junk economics, refusing to grasp that real economics can be counter-intuitive.

    re: “Libertarians love these bad ideas because, you know, the market cures all. ”

    Economically illiterate people often push bad policies because they refuse to learn that government doesn’t cure all. Despite the strawman above, educated libertarians grasp that neither markets nor government cure all.

    This nationally prominent GMU economics professor (who gets published in places like the Wall Street Journal) has a blog post referring to the problem of junk economics, including on the part of some high profile economists that are comparable to what a Dr. Oz would be in the medical world:

    http://cafehayek.com/2014/03/witch-doctory.html
    “Witch Doctory…
    To assume or to suggest (as do many economists) that governments operate more ‘perfectly’ than do markets is no more scientific than to assume or to suggest that, if your child is seriously ill and medical doctors cannot guarantee that they will cure him or her, a local witch doctor can be trusted to supply the desired cure….

    The theme of Jim’s talk was that it is not only intellectually sloppy or lazy but, in fact, deeply unscholarly and unscientific for economists today to ignore public-choice analyses of political decision-making. …
    Stated differently, Jim presented powerful evidence from several current economics textbooks that an embarrassingly large number of such texts – many written by the world’s most acclaimed economists, such as Paul Krugman – are surprisingly naive and unscientific. The authors of these texts pretend to write about reality, but they instead write about a fantasy world. Far too many economists, such as Krugman – because they either ignore or are ignorant of public choice – simply assume that government somehow is not affected by the many imperfections that these economists readily find in markets. As Jim said, this continuing ignorance of public choice is embarrassing to those economists who do think seriously and realistically about their discipline and about the reality that that discipline aims to illuminate.”

    1. What is your background. If you have not read and practiced Travell/Simons, Rachlin, Devin J. Starlanyl, Pybus, Gokavi, Chaitow, Baldry, Wyburn-Mason. or Gunn … shut the hell up.

      1. CommonSenseBoulder says:

        You posted a list of names completely unrelated to my post that you replied to, they have nothing to do with the issue of economics and public policy that I addressed. It isn’t clear what the point of your post is, perhaps merely a mindless troll.

        1. MadisonMD says:

          Well, CSB–have to give you credit– you nailed that one.

    2. windriven says:

      @CSB

      “To assume or to suggest (as do many economists) that governments operate more ‘perfectly’ than do markets is no more scientific”

      The problem – and in many ways I agree with you – is that markets, pure markets in any event, are really hard to come by. Economists tend to ignore harsh realities including the potent reality that political influence and money are almost perfectly fungible and that political influence is frequently used to distort markets and shape regulatory oversight.

      This isn’t, of course, a new problem. Theodore Roosevelt fought it over a century ago … with decidedly mixed results.

      The rock-ribbed Austrian view of economics also loses sight of the fact that economics is a tool rather than an end in itself, a servant rather than a master. One might design a society that was perfectly tuned to maximize the generation of wealth. But that society might not be particularly attractive from a host of other perspectives.

  14. WilliamLawrenceUtridge says:

    yet magically the FDA these days gets away with banning whatever it feels like.

    Um…really? I’m pretty sure the FDA has to do a pretty thorough risk analysis based on the scientific literature to revoke the approval of a drug. I mean, it’s not like we’re in dartboard territory here, they’re basing decisions on empirical evidence. Arbitrary decisions is what characterizes the exceptions to FDA oversight, vis. supplements, herbs, and alternative medicine in general.

    The answer is that the constitution’s meaning has been distorted over time to usurp authority the federal government wasn’t intended to have, and was understood not to have for most of this country’s history.

    Yeah, but the Constitution was also enacted in a time where medicine wasn’t real medicine, and the right to bear arms didn’t have to cover fully-automatic assault weapons with grenade launchers that can be operated by a child. I would probably be one of those people to consider it a “living document” since a political system that doesn’t change with changing realities doesn’t seem particularly great. Then again, I don’t live in the US so the whole argument is rather academic to me.

    1. CommonSenseBoulder says:

      re: “Um…really? ”

      Yes really. The issue was the fact that alcohol prohibition required a constitutional amendment to grant the federal government the power to ban it. Yet the FDA (and other government entities) ban things all the time without a constitutional amendment.

      re: “to consider it a “living document” since a political system that doesn’t change with changing realities doesn’t seem particularly great”

      The issue is to have it consciously changed via the amendment process where the changes can be publicly debated, instead of having the government merely usurp power and get away with it through appointing courts over time that go along with it (the fox guarding the chicken coup, judicial independence exists to some extent but they are still appointed by the same government they are supposed to watch over).

      re: “Yeah, but the Constitution was also enacted in a time where medicine wasn’t real medicine, and”

      and they included the ability to amend the constitution to allow people to update it if circumstances warranted. Alternatively tasks can be performed by state&local governments, who can always voluntarily cooperate if they wish to share costs.

    2. windriven says:

      William, I’ve spent a lifetime working under FDA regulation. Most FDA personnel in my experience are dedicated and ethical. But there have been times and people where there have been substantial deviations. I am barred from discussing details of an instance where I was directly involved but reviewers who were clearly going far beyond regulations and in some cases were fueling exploration of personal interests at regulated companies’ quite considerable expense were … reassigned. It solved the problem for a group of companies. But there was no real discipline and certainly no one got fired.

      “Yeah, but the Constitution was also enacted in a time where medicine wasn’t real medicine, ”

      And we have a perfectly good and frequently used system to address changing times: Constitutional amendments. The first 10 Amendments were adopted not long after the Constitution itself. Since then the Constitution has been amended with some regularity and the list now numbers 27.

      The US system is designed with tension between the Executive (like FDA), the Legislative, and the Judicial branches. In the last 40 years we have been in a period of significant strengthening of the Executive Branch as well as a fairly activist Judicial. Partisan rivalry that has risen to the level of blood feud has largely prevented the Legislative from asserting its prerogatives and reining in the Executive. But the time will inevitably come. Or the system will collapse. Imagine, if you will, the NSA revelations having emerged 40 years ago during Nixon or Carter. It would have marked a Constitutional crisis. Today, only wringing of hands and some mild gnashing of teeth. I had thought that the Bush leap into Iraq would have turned the tide and led to a resurgent Legislative but while Legislative mouths are commodious, the brains and testicles are rather smaller.

      My but do I digress…

      So you noted: ” I would probably be one of those people to consider it a “living document” since a political system that doesn’t change with changing realities doesn’t seem particularly great.” And it is a living document. And there is a well-established method for changing it. Unfortunately, that requires a bit more effort than many of those who speak of it as a “living document” are prepared to invest. For many of them a living document means: the Constitution should mean what I want it to mean.

    3. MadisonMD says:

      yet magically the FDA these days gets away with banning whatever it feels like

      @CSB and windriven:
      You propose that anyone should be able to sell any drug for any purpose without proof of efficacy or safety?

      Revoke the Pure food and Drug Act and going back to this?

      Really?

      1. windriven says:

        @Madison

        “You propose that anyone should be able to sell any drug for any purpose without proof of efficacy or safety?”

        There are limits as I have noted in other comments on this subject. Antibiotics, for instance, where improper use poses a threat to the general public rather than to the individual. And it is always unethical and should be illegal to use false or deceptive information to sell a product or service.

        But that is very different from an adult choosing what chances to take with his or her health – especially when in extremis. I will tell you immediately that taking a flying stab at a rolling donut would not be my way. But I have no reluctance to let the guy next door make his own choices.

      2. windriven says:

        @Madison

        BTW – Toadstool Millionaires is now available in its entirety of the SfSBM wiki. It you aren’t yet a member I hope you’ll join.

        1. MadisonMD says:

          I joined, of course– but have perused it already on quackwatch.

          1. windriven says:

            Madison, I was just using that as a reason to trot SfSBM out for the readers again – and to stir a little interest on the wiki. The wiki conversion of the entire range of Quackwatch sites isn’t yet complete but it has come a long way.

      3. CommonSenseBoulder says:

        re: ” You propose that anyone should be able to sell any drug for any purpose without proof of efficacy or safety? ”

        Most products on Amazon and in local big box stores that you’ll see have no requirement to be proven effective for whatever purpose they are being sold for. That assessment is done via things like consumer reports and consumer reviews and other assessments.

        Most safety concerns aren’t handled privately because people assume it is the responsibility of the government to keep us safe. Competition produces better results than monopoly production of any good or service, and the same is true of evaluation of efficacy&safety. In the case of something like efficacy and safety of a medicine , if the government weren’t involved then private certification labels (perhaps the FDA could be privatized to be the first) would compete to earn the trust of doctors and insurers, and compete based on providing more timely certifications in addition to accuracy. They could provide things like insurance to back up their claim of safety so they are punished if they get things wrong and have more incentive than the FDA to get things right.

        In the case of things like food safety, despite most viewing that as governments role so people don’t bother with private approaches, there are starting to arise private global standards due to the low quality of standards in many countries, to help keep people safe, because if you make your customers sick it is bad for business.

        1. windriven says:

          “Most products on Amazon and in local big box stores that you’ll see have no requirement to be proven effective for whatever purpose they are being sold for. ”

          There is however a requirement, if not a program of prior restraint, for safety. FTC will ruin your whole day if you sell dangerous products whether they are inhalable infant toys or exploding leaf blowers.

        2. Chris says:

          Please provide a viable non-regulatory method that would have adequately dealt with the following:

          swill milk, with add plaster, water, flour etc.

          Elixir Sulfanilamide, with added diethylene glycol

          thalidomide

          the 1901 smallpox vaccine with added Clostridium tetani in St. Louis, MO

          fenfluramine/phentermine, also known as fen-phen

          Just curious. Especially how “market forces” work when several consumers end up having to bury a child. Some reading I have done:

          The Poisoner’s Handbook by Deborah Blum

          Natural Causes: Death, Lies and Politics in America’s Vitamin and Herbal Supplement by Dan Hurley

          Protecting America’s Health: The FDA, Business, and One Hundred Years of Regulation by Philip J. Hilts

          1. windriven says:

            Chris, I don’t want to get too deeply into a fight I don’t really want to wage. But the issue isn’t zero regulation, it is where the line should be drawn. It is never ethical to sell products that are clearly dangerous or to sell them using deceptive or fraudulent inducements. Selling anything containing anti-freeze for human consumption would be illegal in any system that is seriously debated.

            It really boils down to the issue of agency and the line beyond which one should be required to cede one’s agency to some social authority. We are not talking about children and we are not talking about diminished capacity and we are not talking about situations that present a clear danger to others as would be the case, for instance, of unfettered access to antibiotics.

            Speaking only for myself, I come at this from the position that individual agency should be preserved in every case where it does not infringe unduly on the rights and prerogatives of others. CSB seems, and I hate to speak for him/her, to be more interested in the economics of various regulatory schemas than in the philosophical issues.

            1. Chris says:

              “Selling anything containing anti-freeze for human consumption would be illegal in any system that is seriously debated. ”

              Except it was not illegal before the 1938 Food, Drug, and Cosmetic Act. It was the events from Massingill Company’s Elixir Sulfanilamide that killed a hundred kids. The company’s owner stated: “We have been supplying a legitimate professional demand and not once could have foreseen the unlooked-for results. I do not feel that there was any responsibility on our part.”

              It is discussed in detail in the first and third books I listed. The smallpox vaccine that prompted legislation after the 1901 deaths in St. Louis is discussed in Pox: An American History by Michael Willrich.

              There are people in the world that the public needs to be protected from through regulation.

              1. windriven says:

                “There are people in the world that the public needs to be protected from through regulation.”

                I have no argument with that. To be fair, that is in no way inconsistent with the position I’ve staked for myself.

              2. Chris says:

                I will confess to not keeping up with the conversation due to spending most of the day in a rare day of March sunshine. So there will be lapses (plus a huge pile of rose trimmings to be cleared from the lawn, especially from the Cecile Brunner, also known as the “porch eating rase”).

                I believe the premise that is being promoted by a movie is to let certain companies sell their products to desperate patients without any regulatory, or even scientific oversight. The cinematic narrative claims that was a good thing, but reality showed an opposite effect. Ever hear of a dude called “Burzynski”?

                While I understand the frustration caused by dealing with a bureaucracy, I want to see a suggestion of something better offered. And from what I remember by reading the last two books in my list… the same company that actually harassed an FDA employee’s supervisors to approve thalidomide without the requested data has in last few decades worked to reduce its powers while Reagan was president. They really wanted it to be standard practice that the doctors prescribing the meds report side effects instead of going through safety trials:
                “Dear Drug Company symptoms were not relieved. The child died. ”

                Who wants to write that report?

                If anything, I want CSB to propose a better method. One that does not require deaths of loved ones to go into effect. Plus if he does not like how the FDA works, he should do a John Salamone and fix it from the inside. Not write screeds to a couple of blogs.

                A simple search for “FDA community input” brings up this:
                http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/ucm298136.htm

                I hope Mr. Common Sense from Boulder is all over that.

              3. MadisonMD says:

                CSB seems to be proposing an Underwriters Laboratory type of voluntary safety testing organization. I don’t think that that would be as effective since safety/efficacy of drugs is much more subtle than that of electrical items. And moreover, it would be far more dependent on the industry it is regulating. Finally, nothing would stop copycat regulatory agencies from popping up with far fewer standards.

                I already have patients bringing into clinic non-FDA approved supplements without ever noticing the disclaimer about no FDA oversight.

  15. Josh says:

    It’s pretty careless to write that somebody currently being treated for stave IV colon cancer is dead. Jim Neely’s daughter is alive. Is this the first place that this false piece of information has been presented? Let me know if you’ve seen it elsewhere, because I want to make sure that the mistake is corrected before his daughter or family reads it.

    “Of course, Dallas Buyers Club is just a movie, no matter how good a movie it might be and no matter how much Matthew McConaughey might have deserved an Oscar for his performance. Unfortunately, it appears that legislators in several states seem to think that it should serve as a template for health policy.”

    Several reporters have asked legislators touting Right to Try about DBC. None of them have even seen it. If you’ve seen any report to the contrary, please correct me.

    If you take the time to watch the movie, you may understand that the access provided through buyers’ club is, in no way, a template for Right to Try. DBC drugs were not through phase I trials. DBC drugs were not prescribed by physicians. There was absolutely no regulation.

    Right to Try actually leaves the FDA with a lot of authority over the drugs that can be made available. If the FDA halts a trial for any reason, physicians would not be allowed to prescribe the corresponding drug to terminal patients, even if it has completed phase I trials. Fortunately, that means that Burzynki is explicitly excluded from this legislation.

    1. David Gorski says:

      Jim Neely’s daughter is alive. Is this the first place that this false piece of information has been presented?

      This has been corrected. Thanks for the heads-up.

      If you take the time to watch the movie, you may understand that the access provided through buyers’ club is, in no way, a template for Right to Try. DBC drugs were not through phase I trials. DBC drugs were not prescribed by physicians. There was absolutely no regulation.

      Yet Dallas Buyers Club is frequently used as a label for these sorts of bills. That should tell you something, regardless of the intent of the bill’s authors. In any case, as I’ve pointed out near the end of my post, these sorts of bills are not new ideas. The Abigail Alliance has been pushing them for a long time. What’s happened is that lately their advocates have taken advantage of the popularity of Dallas Buyers Club.

      Right to Try actually leaves the FDA with a lot of authority over the drugs that can be made available. If the FDA halts a trial for any reason, physicians would not be allowed to prescribe the corresponding drug to terminal patients, even if it has completed phase I trials. Fortunately, that means that Burzynki is explicitly excluded from this legislation.

      The Arizona bill only says “remains under investigation in a clinical trial.” One could very easily argue that this does apply to Burzynski’s antineoplastons, as the FDA hasn’t completely halted Burzynski’s clinical trials. It’s only put a partial clinical hold on them. His trials are still ongoing with existing subjects, and, although it is unlikely, the hold could be taken off at any time. The law as written is too vague in that Burzynski patients could easily argue that clinical trials are still ongoing (they’re just closed to accrual right now), and they might win that argument. Also, one notes (actually, I did note in the post) that Steve and Melissa Heil, whose daughter has a brain tumor and has been treated by Stanislaw Burzynski in the past, were prominent among the patients and patients’ families testifying in favor of the Missouri right to try bill. Why is that, if they don’t think that this law would help them get their daughter back on antineoplastons? That’s what they want, to get her back on antineoplastons.

      Also, part of my argument is that these laws are basically completely useless because the FDA has final authority. The state can’t override that, and the only imaginable way that a reputable drug company might be willing to give investigational agents to such patients is if the pharmaceutical company is in the same state as the patient. Even then, if the drug is still under development (i.e., there’s a clinical trial), the pharmaceutical company is not going to buck the FDA and give the drug out without first getting a single patient IND. It’s just not. So these laws would only give the illusion of doing something. They won’t actually do anything—except cause no end of confusion and angst among families, researchers, regulatory bodies, and pharmaceutical companies, all for very little, if any, benefit to the people who, it is claimed, would benefit from them.

  16. NB says:

    I’m a stage IV metastatic breast cancer patient with one treatment option left to me. I am far from uneducated or unintelligent. I know full well what occurs in clinical trials and I’m likely to die in great pain, suffering, so if I want to take my chances and risk side effects, I should have that option. I’m in Canada and I don’t. Unless you’re in our shoes, you really have NO IDEA. It’s very easy to spout platitudes and actually suggest that terminal patients are too stupid or drug addled to make an informed decision but we aren’t. Your belief that we are perpetuates discrimination and, I for one, am offended.

    1. David Gorski says:

      I’m terribly sorry to hear that.

      I happen to be somewhat familiar with the kinds of drugs that are in the pipeline for breast cancer, mainly because breast cancer is my specialty, and there are no drugs there right now that I’m aware of that are likely to be so much better than existing drugs for metastatic breast cancer as to be worth the risk outside of a clinical trial. Unfortunately.

      One would also point out that I have not said anything about terminal patients being too stupid or drug addled to make an informed decision. That is your perception, not what I said. Where you see that I don’t know. What I do see are ideologues taking advantage of the understandable human desire not to die and to try almost anything to avoid death, wrapping it up in high-sounding rhetoric, when in reality the goal is to decrease regulation on drug companies and weaken protections against the selling of harmful or unproven drugs. It’s not a coincidence that the main impetus for this latest round of promotion of “right to try”-style laws isn’t so much patient groups as it is from libertarian groups like the Goldwater Institute.

    2. MadisonMD says:

      @NB:
      Also sorry to hear about your illness. Not sure what the rules are in Canada, but it is possible to legally get investigational drugs in the U.S. already under Expanded Access. This program balances safety with access to investigational drugs that are promising, and appropriate medical oversight. It also provides feedback to the drug sponsor about any serious adverse event that occurs (So that unexpected or rare drug risks are detected and communicated to other doctors and patients).

      I hope your caregivers will care for you adequately so that you will not have great pain and suffering. It is not required that you suffer.

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