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The Koch Brothers and Vice President Mike Pence back a final push to pass the cruel sham known as “right-to-try”

So-called “right-to-try” laws have passed in 38 states. A cruel sham whose real purpose has nothing to do with helping terminally ill patients and everything to do with the libertarian war on FDA regulation, these laws claim to allow terminally ill patients to bypass the FDA and obtain access to experimental drugs that have passed phase I testing. They do nothing of the sort, which is why right-to-try advocates have “gone federal.” A right-to-try bill has passed the Senate, and Vice President Mike Pence and Koch Brothers-backed groups are lobbying hard to pass it in the House. There is still hope to stop it.

Three weeks into 2018, I once again find myself discussing an issue that I’ve been discussing for nearly four years now. I’m referring, of course, to a law known as “right-to-try.” Such laws, all based on a legislative template published by the libertarian Goldwater Institute, purport to allow terminally ill patients to bypass the FDA and access experimental drugs that have passed only phase I testing. As anyone with a basic knowledge of clinical trials knows, this is an incredibly low bar for safety, and phase I trials are not even designed to show efficacy. When the right-to-try movement made its first big splash on the national scene back in 2014, which was when several states beginning with Colorado passed the first right-to-try laws, both Jann Bellamy and I pointed out how these laws, seemingly inspired by the movie Dallas Buyers Club, were an illusion, a sham, a misguided and actively patient-hostile “solution” (why will be explained below) to a problem that right-to-try advocates greatly exaggerated in order to promote these laws. Indeed, in 2014 some libertarian advocates even latched on to the fear of Ebola as a scare tactic to advocate for right-to-try, while others (like Nick Gillespie and Ron Bailey) accused the FDA of killing millions through slow approval of new drugs. I kid you not. Basically, right-to-try advocates succeeded in portraying opposition to right-to-try as callous and betraying a lack of compassion for terminally ill patients as well as an assault on freedom itself, all mixed, of course, with a heaping helping of anti-government rhetoric directed at the FDA. Basically, the reason so many states passed these laws so quickly was through a combination of ignorance among the general public of how drugs are approved and a campaign to demonize opponents, such that opposition to right-to-try was perceived among politicians as akin to opposition to mom, apple pie, and the American flag.

Eventually, as these laws metastasized, passing in more and more states (38 as of October!), advocates promoted a federal version of the law. The first attempt, the Compassionate Freedom of Choice Act, went nowhere in 2014. So did the second attempt in 2016. Of course, such a law could not have passed then because it almost certainly would have been vetoed by the previous President, but the point is that it didn’t even get out of committee both times. Then came President Donald Trump and a push for deregulation. Let’s just say that the environment in Washington had…changed. For one thing, Vice President Mike Pence is a supporter of right-to-try. In any event, The Trickett Wendler Right to Try Act was reintroduced in 2017. When even in a Republican Senate the bill appeared to be going nowhere, its sponsor, Senator Ron Johnson, threatened to hold the renewal of the bill authorizing the FDA to collect user fees from companies seeking FDA approval for their drugs hostage if right-to-try language was not added to it. Ultimately, to mollify him, Senate leadership agreed to hold a vote on Sen. Johnson’s bill, and, as almost always happens when such a bill comes up for a vote, it passed easily.

That was the situation in August, when last I wrote about right-to-try. To be honest, I was surprised that the House hadn’t passed the bill and President Trump hadn’t signed it into law yet. Unfortunately, earlier this week I learned that one last push is being made to pass the bill. Before I discuss that, let’s take a look at why right-to-try laws are so problematic and harmful to patients.

The problem with right-to-try (state version)

As I mentioned before, the push to pass right-to-try legislation at the state level is basically a product of forces that want very much to weaken the FDA’s power to approve and regulate drugs, with each law following rather closely, with minor variations, the legislative template originally published by a libertarian think tank, the Goldwater Institute. Such legislation includes several major shared features. First is the requirement that the disease the patient has be terminal, usually defined as having a life expectancy of less than six months, although the model legislation is more vague, requiring an “advanced disease,” defined as “progressive disease or medical or surgical condition that entails significant functional impairment, that is not considered by a treating physician to be reversible even with administration of current federal drug administration approved and available treatments, and that, without life-sustaining procedures, will soon result in death.” Various states define this condition in somewhat different ways, some with some room for interpretation.

One of the most problematic passages, if not the most problematic passage, is the one where the term “investigational drug, biologic product, or device” is defined:

“Investigational drug, biological product, or device” means a drug, biological product, or device that has successfully completed phase 1 of a clinical trial but has not yet been approved for general use by the United States food and drug administration [sic] and remains under investigation in a United States food and drug administration-approved clinical trial.

Every right-to-try bill or law I’ve ever read uses the above definition or relatively minor variations. Anyone who knows anything about drug development knows that having completed a phase 1 trial is a dangerously low bar to clear to allow more widespread use of a drug. Basically phase 1 trials are small trials, usually consisting of less than 30 subjects, that look for major toxicities and adverse events. That is not enough to determine safety, nor is it intended to. It is utterly impossible for such a small clinical trial to determine the safety of a drug. 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,” the intent also being to find the best dose to use in subsequent Phase II and Phase III trials. 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 3.

Right-to-try laws are also profoundly unjust, as nearly all of them limit who can access experimental drugs by wealth. The reason is that all of right-to-try laws have a provision that says that health insurance companies do not have to pay for such treatment and nearly all of them also say that the company providing the experimental therapeutic under right-to-try can charge the patient for it. A terminally ill patient could easily go bankrupt before he dies, and many couldn’t access experimental therapeutics through such laws in any event because they simply don’t have the money or the fundraising wherewithal to do so. In at least one state I know of, the right-to-try law allows a drug company to go after a deceased patient’s estate to collect. It goes beyond that, though. If a patient uses a right-to-try drug and suffers complications, many of these laws essentially state that the insurance company doesn’t have to pay for care resulting from that complication, and most such laws state that patients undergoing right-to-try therapies lose their coverage for hospice.

Right-to-try laws are patient-hostile in other ways, too, as Jann Bellamy and I have described many times. The most egregious example of which is how they strip patient protections away from patients who access them. One way to see this is by comparing what happens when a patient accesses an experimental therapeutic under the FDA expanded access program to what happens when another patient accesses one under a right-to-try law. Under FDA expanded access, patients retain full protections under federal and state laws. They can sue for malpractice if there is any, and their care is still monitored by an institutional review board (IRB), with any adverse events recorded and considered by the FDA. Moreover, the FDA approves nearly all such requests (99%). In contrast, under right-to-try, there is no IRB oversight. It’s all between the company and the patient, a libertarian paradise!

Right-to-try laws also limit what patients can do in the event of malpractice or negligence. All of them broadly immunize physicians advising or administering right-to-try medications or using right-to-try devices against malpractice suits or actions against their medical license by the state medical board related to their participation in right-to-try. All of them also immunize companies providing experimental therapeutics under right-to-try from liability. All of them contain provisions stating that state employees can’t interfere with a patient seeking right-to-try, which could be interpreted to mean that a doctor at an academic medical center at a state university couldn’t counsel a patient not to seek right-to-try without running afoul of the law. As Jann notes, even if state authorities believe, for example, that an elderly person is being exploited for financial gain by a physician, presumably this provision would prohibit their acting.

I frequently refer to state right-to-try laws placebo laws because they basically do nothing but make advocates and legislators feel as though they did something good for terminally ill patients. After all, the FDA controls drug approval, and its power overrides state law. For that reason, few would be the drug companies willing to risk their FDA approval by providing experimental drugs to patients without the FDA’s permission. Of course, the Goldwater Institute is not run by idiots. Its leadership knows this. The purpose of state-level right-to-try laws was never really to help terminally ill patients. It was to put pressure on lawmakers to pass a federal right-to-try law as a first big step towards weakening the FDA.

H.R. 878: Right to Try Act of 2017

H.R. 878: Right to Try Act of 2017 is simpler than most state-level laws and is currently very similar to the bill passed by the Senate in August (S. 204: Trickett Wendler Right to Try Act of 2017). It has two main provisions. First:

Notwithstanding the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.), the Controlled Substances Act (21 U.S.C. 801 et seq.), and any other provision of Federal law, the Federal Government shall not take any action to prohibit or restrict—

(1)the production, manufacture, distribution, prescribing, or dispensing of an experimental drug, biological product, or device that—

(A)is intended to treat a patient who has been diagnosed with a terminal illness; and

(B)is authorized by, and in accordance with, State law; and

(2)the possession or use of an experimental drug, biological product, or device—

(A)that is described in subparagraphs (A) and (B) of paragraph (1); and

(B)for which the patient has received a certification from a physician, who is in good standing with the physician’s certifying organization or board, that the patient has exhausted, or otherwise does not meet qualifying criteria to receive, any other available treatment options.

In essence, this part of the law would basically tell FDA and other regulatory agencies not to interfere with the use of experimental drugs by terminally ill patients under state right-to-try laws. That’s no surprise, although the language does seem a bit broader than I would have expected—but, as they say, I am not a lawyer. For purposes of this bill, experimental drugs are defined the same way as in state right-to-try laws, any drug that’s passed phase I clinical trials.

Now here’s the truly pernicious, stupid part of the bill:

(b)No liability or use of outcomes

(1)No liability. Notwithstanding any other provision of law, no liability shall lie against a producer, manufacturer, distributor, prescriber, dispenser, possessor, or user of an experimental drug, biological product, or device for the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that is in compliance, with subsection (a).

(2)No use of outcomes. Notwithstanding any other provision of law, the outcome of any production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that was done in compliance with subsection (a) shall not be used by a Federal agency reviewing the experimental drug, biological product, or device to delay or otherwise adversely impact review or approval of such experimental drug, biological product, or device.

The part about liability is self-explanatory. It basically immunizes manufacturers, distributers, doctors, and patients against federal liability for using and possessing experimental drugs, just as state right-to-try laws immunize them against state liability. No surprise there.

The real surprise is the part barring the FDA and other regulatory agencies from using reported outcomes from patients using experimental drugs under right-to-try. That means that if a patient uses an experimental drug under right-to-try and suffers a serious adverse reaction, the FDA cannot consider that reaction in its deliberations over approval of the drug. This clause is obviously intended to address the concern of drug and device manufacturers that if they provide an experimental therapeutic to a patient under right-to-try and the patient suffers complications due to the treatment, the approval of its product might well be jeopardized. This, of course, is not an unreasonable concern on the part of manufacturers, given that it can cost as much as $1 billion to bring a drug or device to market, and by the time the product has passed phase I clinical trials a great deal of that cost has already been invested in development. However, this clause goes way, way too far. Basically, it means that even if a patient death is clearly due to use of an experimental drug under right-to-try, that death cannot be considered by the FDA in deciding whether to approve the drug.

Think of it this way. Let’s say dozens of patients die from using an experimental drug under right-to-try. This bill, if passed, would bar the FDA from even considering those deaths during its deliberations regarding whether to approve the drug for marketing or not. This is not a good thing for patients. It is profoundly anti-patient. Basically, the Trickett Wendler Right to Try Act of 2017 would not help terminally patients. It would endanger them. Existing Expanded Access (sometimes also called Compassionate Use) programs by the FDA are, as I pointed out above, no longer so onerous and requests under them are almost always approved. Right-to-try laws only strip away patient protections without providing any potentially useful new rights to terminally ill patients.

As I like to do whenever I write about right-to-try, here I will refer readers to the excellent bullet point summary of the problems with right-to-try published by the New York University School of Medicine Working Group on Compassionate Use and Pre-Approval Process.

The Koch brothers make a last push to pass right-to-try

About a week ago, I saw this Tweet:

The story from the Tweet reports:

Koch brothers-backed groups are launching a campaign urging Congress to pass legislation allowing terminally ill patients to request access to experimental drugs the Food and Drug Administration (FDA) hasn’t approved.

Nearly 40 states have this law, known as “Right to Try,” already on their books. But Freedom Partners, in partnership with Americans for Prosperity — two groups funded in part by billionaire brothers Charles and David Koch — say federal legislation is needed to assuage patient fears that the federal government will override state laws.

The new push, launched Monday, consists of a lobbying effort, 30- and six-second digital ads, a social media effort and more.

On Twitter, we can see Americans for Prosperity is pushing the message and the ads:

Here’s the main ad being used by Americans for Prosperity and Freedom Partners to promote right-to-try:

The ad is nauseating in the extreme, full of the usual exaggerated and misleading talking points and appeals to emotion commonly used by right-to-try advocates. So is the propaganda appearing on the Freedom Partners website. Indeed, its arguments are disingenuous and misleading in the extreme:

Though the FDA boasts of granting over 99% of expanded access requests it receives, it only received 1,757 requests for expanded access in total last year, minuscule in comparison to the 596,000 Americans who died of cancer in 2015, the 155,000 who died of chronic lower respiratory diseases, or the 111,000 who died of Alzheimer’s. This number is troubling in light of the nearly 26,000 patients receiving experimental treatments in France in 2016 under a similar program, meaning nearly fifteen times as many patients receiving experimental treatments in a country one-fifth the size. Underlying this disparity is the burden placed on patients by the FDA, discouraging them from even submitting a formal application.

In an article for The Goldwater Institute, Carla Mann Woods, formerly a medical device industry executive, and now a board member of the Alfred E. Mann Institute for Biomedical Engineering at the University of Southern California, put it this way: “In this era of both scientific revolution and information where anyone can find anything on the Internet, ask yourself this: Can you actually believe that only 1,200 dying Americans want to live badly enough to find a legitimately applicable, unapproved therapy and ask to get it?”

No one is arguing that we shouldn’t do better in expanding access to experimental drugs. However, comparing compassionate use approvals to the total number of patients dying of cancer is a false metric given that the vast majority of terminally ill patients wouldn’t be likely to qualify for most experimental drugs anyway, either based on disease type, burden, or comorbid conditions. As for the example of France, I can’t help but note the the French National Agency for Medicines and Health Safety report cited. I was puzzled at first, as I saw little reference to what Freedom Partners was talking about, but it would appear that it was talking about a Temporary Authorization for Use (TAU):

A Temporary Authorisation for Use is an exceptional, special procedure which, since 1994, has given numerous patients, for whom there is no available alternative treatment, access to medicines that do not have a MA in France. They may be named-patient Temporary Authorisations for Use (TAUn), i.e. granted for a specific named patient, or concern a group of patients (cohort Temporary Authorisation for Use, TAUc).

Since 2012, ANSM has been developing a new policy aimed at expanding the use of cohort TAUs in order to foster fair, closely monitored access to innovative treatments for patients whose treatment options have been exhausted.

In fact, this is a false and deceptive comparison. Access to unlicensed drugs in France under a TAU is a very different beast compared to access to unlicensed drugs through right-to-try in that the French government still has to approve the use of unlicensed medicines. In France, a drug requires a marketing authorization (MA) and price and reimbursement agreements. Indeed:

The French early access programme is one of the few European compassionate-use programmes to allow cohorts of patients. In fact, two types of ATU status exist. The first one, known as nominative ATU, is available on a named-patient basis under the responsibility of the prescribing physician. The second one, cohort ATU, is intended to apply to a group of patients and is established at the request of the pharmaceutical company for specific indications [8], Hospital pharmacies are exclusively in charge of supplying ATUs to in- or outpatients. As with other drugs, hospital pharmacies purchase ATUs by directly negotiating with manufacturers. Pharmaceutical companies have the freedom to determine ATU pricing. The National Health Insurance (NHI) is responsible for the cost associated with these treatments. For outpatients, NHI fully reimburses ATU expenditures to hospitals on the presentation of bills. For ATUs delivered to hospitalized patients, additional funding, known as ‘MIGAC’ (Missions d’intérêt Général et d’Aide à la Contractualisation), is available to hospitals on top of the diagnosis-related group (DRG) funding. Several blockbusters have been available in France through the ATU programme prior to getting an MA. Examples include: bevacizumab (Avastin®), erlotinib (Tarceva®), lenalidomide (Revlimid®), ranibizumab (Fucentis®) and sorafenib (Nexavar®).

If granted, the programme guarantees patient access before MA and also during the time of agreement on reimbursement and price.

The contrast between the French early access program and right-to-try could not be more clear. TAU is not a right-to-try law. In the French program, government regulatory agencies are heavily involved. (Indeed, in the very report referenced by Freedom Partners, it points out that there must be good evidence that the drug is likely to work before a TAU will be issued and details how the government must monitor usage.) In contrast, under right-to-try, the FDA is explicitly cut out of the decision-making process and can’t even consider results seen in patients using right-to-try in determining whether to approve an experimental drug. In the French program, the government pays for the experimental drug. Under right-to-try, the patient is completely on his or her own. You get the idea. The Freedom Partners’ propaganda is so deceptive as to be virtually a lie. In fact, the existence of the French TAU program puts the lie to the claim that right-to-try is needed to expand access to experimental drugs. The French have done it without sacrificing regulatory oversight through a right-to-try law. We could too if we so desired.

In retrospect, the involvement of the Koch brothers makes a lot of sense. I had always suspected that there had to be much more behind the right-to-try effort than just the Goldwater Institute, which, while influential, is not nearly large enough to have mounted such an effort in so many states over such a short period of time on its own. I can’t help but remember back when right-to-try passed almost in secret in Michigan, the Goldwater Institute was able to fly in patient advocates from all over the country. It was a suitably impressive effort. The involvement of the Koch brothers reinforces more than ever my conclusion that right-to-try is not (and never was) about helping terminally ill patients. It’s always been about weakening the FDA’s ability to regulate drugs.

An excellent editorial by Steven Joffe and Holly Fernandez Lynch in the latest New England Journal of Medicine makes this very point, the first time I’ve seen it so explicitly argued in a large mainstream medical journal. Although they think that the potential harm to patients and clinical trials may be overstated by opponents, they recognize the real purpose of right-to-try as the “foot-in-the-door” to further deregulation that would weaken the FDA’s health mission:

Nevertheless, its passage would remain important. Indeed, the serious consideration the legislation is being given reflects a broader effort to weaken medical product regulation. The Goldwater Institute has launched a campaign in the name of free speech to allow manufacturers to share information with physicians and others about off-label uses of approved drugs, which has resulted in at least one state law to that effect. The FDA has traditionally tightly regulated this practice, but recent federal court cases have threatened its authority in the name of protecting truthful commercial speech. The FDA’s new commissioner, Scott Gottlieb, appears poised to move the agency toward a more permissive stance on off-label communication.

If a federal right-to-try bill becomes law, the long-standing principle that broad access to medical products should await evidence of safety and efficacy will contend with the principle that patients with life-threatening diseases should have unfettered access to agents that have passed a bare minimum of safety testing. This logic could ultimately extend to patients with serious chronic illnesses and, conceptually at least, to all patients who find their approved treatment options unsatisfactory.

This is what I’ve been arguing for nearly four years now, that right-to-try is only part of a broader push to weaken the FDA and, if passed, that the next step would be to push to make right-to-try apply to more than just terminally ill patients. Ultimately, the push would be to make it apply to everyone. It’s good to see this argument explicitly stated in the pages of a journal as highly read and respected as the NEJM.

What now?

Until now, the likelihood of passing right-to-try in the House was considered rather low. Indeed, in October, FDA Commissioner Scott Gottlieb testified and requested changes to the bill, noting, “If enacted without changes, sponsors and other persons providing eligible investigational drugs to eligible patients under the specified conditions of the bill would not be subject to a number of sections of the FD&C Act and FDA regulations related to clinical trials, premarket approval, and labeling. The current language in S. 204 may therefore preclude FDA from taking enforcement actions based on those provisions.” He also stated, “The perception is that this legislation will pressure companies to supply more expanded access drugs but that remains to be seen,” noting in his written testimony:

It is important to note that access to investigational products requires the active cooperation of the treating physician, industry, and FDA in order to be successful. The most common obstacle to access to the investigational product is the willingness or ability of companies to provide it. Neither FDA nor physicians or patients can compel a company to make a product available – and companies may decline requests for a variety of reasons. For example, they may have produced only a limited quantity of the product (companies ramp up manufacturing after marketing approval), have minimal resources to administer expanded access requests, or have concerns that granting requests for expanded access may exacerbate the challenge of recruiting clinical trial participants – and delay product development.

In other words, the FDA could oversee more expanded access requests, but companies are reluctant to take part.

There’s still hope of stopping right-to-try, however. One hopeful sign is that Senator Greg Walden, Chairman of the House Energy and Commerce Committee, is opposed to the bill, and his is the committee that has to vote to send the bill to the floor for a vote. Unfortunately, he is under great pressure from the Koch-backed Freedom Partners, Vice President Mike Pence, and the House leadership to send the bill to the floor for a vote. Indeed, on Thursday, Pence met with FDA Commissioner Gottlieb and then Tweeted about how he had urged him to help persuade the House to pass right-to-try:

Another is that the version passed by the House would have to be basically identical to the version passed by the Senate last year to avoid a conference committee and two new votes, one in the House and one in the Senate. If the House version is changed sufficiently, which is not unlikely, then those votes will have to occur, and there will be a chance to block the bill again. Indeed, House Energy and Commerce Committee Chairman Greg Walden of Oregon told STAT News Thursday that the Senate-passed “right-to-try” bill would have to be changed significantly before he would bring it up in his panel. This suggests that there is still time to marshall opposition, although it’s not clear how much time there is. With Congress recently so busy dealing with brinksmanship over the federal budget that led to the government shutdown over the weekend that I sincerely doubt that a vote will come up until after that is dealt with. At least, I hope that’s the case.

One of the clearest problems that let right-to-try become the behemoth that it is now is the lack of resistance to it. I’ve complained loudly about how none of the major medical organizations expressed opposition early on, probably because they didn’t want to be painted as the bad guys. Indeed, the American Society of Clinical Oncology, to its shame, said nothing official about right-to-try until its statement of opposition last April, three years after the first laws were passing. More of that will be required, and it will be important to let legislators know that there is actual opposition to this bill among the public by contacting our Representatives.

Right-to-try is a cruel sham by the Goldwater Institute using terminally ill patients as cannon fodder in its interminable war against government regulation that’s been a miserable failure thus far. Here is where it needs to be stopped, because if it isn’t it will only be the first step, or, as Joffe and Fernandez Lynch put it:

Are we prepared to abandon the FDA’s gatekeeping role in favor of unfettered patient autonomy and market forces, risking precisely the problems that prompted Congress to grant the FDA its present authority? The agency has made substantial progress in balancing the needs of desperate patients and the principle that all patients deserve evidence that the benefits of medical products justify their risks. We upset that balance, and diminish the FDA’s public health mission, at our peril.

It can’t be stated too often. The goal of right-to-try is not to help terminally ill patients. It’s to diminish and weaken the FDA. That’s why this latest push to pass federal right-to-try legislation needs to be stopped.

By Orac

Orac is the nom de blog of a humble surgeon/scientist who has an ego just big enough to delude himself that someone, somewhere might actually give a rodent's posterior about his copious verbal meanderings, but just barely small enough to admit to himself that few probably will. That surgeon is otherwise known as David Gorski.

That this particular surgeon has chosen his nom de blog based on a rather cranky and arrogant computer shaped like a clear box of blinking lights that he originally encountered when he became a fan of a 35 year old British SF television show whose special effects were renowned for their BBC/Doctor Who-style low budget look, but whose stories nonetheless resulted in some of the best, most innovative science fiction ever televised, should tell you nearly all that you need to know about Orac. (That, and the length of the preceding sentence.)

DISCLAIMER:: The various written meanderings here are the opinions of Orac and Orac alone, written on his own time. They should never be construed as representing the opinions of any other person or entity, especially Orac's cancer center, department of surgery, medical school, or university. Also note that Orac is nonpartisan; he is more than willing to criticize the statements of anyone, regardless of of political leanings, if that anyone advocates pseudoscience or quackery. Finally, medical commentary is not to be construed in any way as medical advice.

To contact Orac: [email protected].com

27 replies on “The Koch Brothers and Vice President Mike Pence back a final push to pass the cruel sham known as “right-to-try””

So the proposal is basically a return to the days of the medicine show snake oil salesmen but only for the wealthy?

People in health regulatory organizations in other countries must be pounding their heads on their desks. I strikes me that the inability of the FDA to consider any adverse effects in these cases will make other countries much less willing to trust the FDA data in considering drug approvals.

Would this be a small incentive for major phara companies to shift drug development to other jurisdictions? Or just to require the companies to report any such uses of the drugs, leading to the somewhat absurd situation where the EU or China would have more information on the drug than the USA where it was developed?

My question would be, what is the end game? Even if they get 100 people desperate and rich enough to pay $1million to access a drug in phase 1 trails, that is a tiny drop in the bucket for the overall cost of drug production, $1000million. They can say the FDA can’t use the results, positive or negative, to decide if the drug gets approval, but that won’t stop the drug being judged in the press / internet.

I see a lot of downside for both parties in this, and I’m not seeing a real upside. Drug companies get a few more paying test subjects. Terminal people get a last ditch treatment. If the treatment works, it’s not really beneficial to the drug company – No real financial benefit, nor trial benefit from it for the company. Depending on the fees charged, the physician may come out further ahead financially, ignoring reputation effects could make that a real Pyrrhic victory. The patient may benefit, but if the drug makes things worse or fails entirely, they and their family are financially and emotionally screwed.

So where is the upside to this? I refuse to believe that the Koch brothers are stupid, so they must have a tangible goal in mind. Is it getting a wedge in the door to allow future bypassing entirely of the FDA?

Terminal people get a last-ditch treatment.

I would add that many terminal patients’ participation in clinical trials is also motivated by altruism. They realise that they may or may not benefit personally—knowing it’s a long shot—but can contribute to the accumulation of knowledge, advancing science to the benefit of others.

Excluding data derived from RTT is proper, as patients seeking it often fall outside exclusion or inclusion criteria of a given clinical trial, so could muddy up the picture if present. But diverting some patients into RTT and away from clinical trials means less enrollment, and longer time/higher cost spent investigating (and approving, when successful) candidate drugs. Cancer advocates should therefore oppose RTT, if not already convinced to do so on the basis of the externalisation of costs and risks to the patient and potential for abuse in promoting scam treatments like neoplastons.

There’s no upside from my POV, either.

Instead of thinking of a $1 million lump sum, think of $10k per dose.

At that price, a lot of desperate middle-class families could pay for a number of doses by wiping out their savings, putting a second mortgage on their home (at ugly interest rates), taking out loans against their 401k, and signing over the benefits to the ill person’s life insurance.

And in the likely event that the treatment doesn’t work (either because it wouldn’t have, or because the money runs out), some scummy investment group gets the life insurance $$.

Sometimes I think that Libertarians offend me even more than regular garden-variety right wing nuts. What went wrong when they were learning to think?

The same thing that went wrong with the Marxists of the left. They have no appreciation for the cost to a society’s humanity if there were ever a serious attempt to implement the program. But there is a reason you see very few Marxists[1] anymore: the evidence of what happens in Communist-style systems is too great to ignore. There has never been an even partially successful attempt to impose a libertarian system, so those people have never been forced to confront the holes in their logic. Libertarians are also frequently naive: they assume, without having seriously considered the matter, that they would rise to the top in such a system, rather than being turned into objectivist long pig jerky.

That’s before you consider how few genuine libertarians there are. Most people who claim to be libertarian turn out, when the chips are down, to be authoritarian. At least garden-variety right wing nut jobs tend to be more honest on this point.

[1]I refer here to Karl Marx. I am much more sympathetic to Marxists of the Groucho variety (some of whom call themselves Marxist-Lennonists).

Marxism does not require a Soviet-style dictatorship. That is all. Please also note the asymmetry in access to ears of the powerful.

I’m not a Communist, but at some point we’re going to have to acknowledge that we can produce without the need for most people working. The alternative to socialism is mass poverty; we can’t create ‘jobs’ when there is no new profit to be gained. There’s a lot of work needing to be done, but it’s not profitable, it’s service.

Libertarians are also frequently naive: they assume, without having seriously considered the matter, that they would rise to the top in such a system, rather than being turned into objectivist long pig jerky.

A very apt assessment. Adam West on “The Friendly Atheist” did a chapter by chapter deconstruction of Atlas Shrugged. It took him something like two years. I imagine a lot of people who like the book see themselves as one of the heroic Strikers, ground down by the stupidity and corruption of those around them.

…Emma Goldman essay on how there was no true Communism in Russia…

Can anyone say “No True Scotsman”?

Marxism does not require a Soviet-style dictatorship. That is all.

And yet I don’t know of any Communist countries that didn’t wind up as a human rights abusing dictatorship.
The big problem with both Communism and Libertarianism is that neither takes into account human fallibility. Libertarianism is worse as it believes the Free Market will solve everything. I didn’t agree with everything Professor Dutch wrote in his attack on Libertarianism but several of his criticisms about what the Free Market can’t do are spot on.

Can anyone say “No True Scotsman”?

Well that’s glib and flippant. I don’t know if you actually read the article, but all her points are valid. The Soviet Union wasn’t Communist at all. As I already wrote, it was an authoritarian state capitalist regime, and it was the model for so-called Communist regimes of the 20th century, so yeah, they were, uh, similar.

I’m a little bit worn out at the moment from transcribing interviews with young working/lower class people from Belfast (Jesus, they’re half unintelligible), but try me later tonight after a beer or maybe tomorrow morning after I get out of therapy.

@JP:

Well that’s glib and flippant.

Fair comment.

I don’t know if you actually read the article, but all her points are valid. The Soviet Union wasn’t Communist at all.

To be honest, I didn’t at first. I tried, and gave up in Chapter II.
My points, which I see I failed to make clear, are:
• Communism can’t work except maybe on a very small scale, and so;
• Comments about “true Communism” are disingenuous. They are identical to the defenses of quack treatments like the Gershon Protocol where subjects can’t possibly fulfill their onerous requirements and so the Protocol is not to blame despite being impossible.
Ideologies may be interesting as theoretical exercises, but any system that fails to take into account human frailties and fallibility (like Communism) is doomed to fail if applied in the real world.

Comments about “true Communism” are disingenuous. They are identical to the defenses of quack treatments like the Gershon Protocol where subjects can’t possibly fulfill their onerous requirements and so the Protocol is not to blame despite being impossible.

I’m not being disingenuous, and I certainly resent being compared to Gerson.

Capitalism is an ideology too, you know. 42 people own as much wealth as over 3 billion people. We’ve just about created an ecological dystopia for those who will live long enough to see it. Donald Trump is President and we have been run by a bunch of oligarchs and their puppets for a long time now. I could go on.

Capitalism is no more the natural order than feudalism or the divine right of kings were.

It’s an ideology that fails to take into account human frailties.

I wasn’t accusing you specifically of being disingenuous, JP. I was being general.
I was comparing Communism to Gerson in the sense that both demand the impossible from their adherents/disciples. It’s the same reason theocracies don’t work. People are just too fallible.
And yes, I totally agree that things have gone insane under Capitalism. The 42 richest people combined owning as much as the 3 billion poorest combined IS obscene and unsustainable.
Yesterday, I saw something that appalled me. It was a comparative graph of poverty rates among children in the major Western Democracies. In second place was the UK. In first place was the US with a Child Poverty Rate 5 times that of the UK. 5 TIMES! I’m also keenly aware that the current setup in the US perpetuates those inequalities and I agree that this must be addressed.
I know a great deal about living under an ideology. I’m a South African, born in 1976. At the time, South Africa was governed by apartheid, a hateful ideology that held that whites were the best race and Christianity (or rather, a warped version of it) was the greatest religion. Apartheid was also sexist and homophobic. And because of that I reject all ideologies as systems of governance, whether they be unfettered Capitalism, Communism, any type of theocracy, or Libertarianism.

The thing is that everybody has an ideology. Some people are just conscious of and honest about it. Most people are like fish in water, internalizing neoliberalism without even being able to name it. It’s still an ideology.

I’m not ashamed, personally, to be called a Red or a commie. It’s rather a badge of honor.

“You ain’t been doing nothing if you ain’t been called a Red,” as the song goes.

‘Say, do you think it would help this cancer patient to give him a motor-oil enema and fire him from a cannon into a bucket of live shrimp?’

‘Well, there haven’t been any published studies on that; it would be cruel NOT to give it a try….”

Orac writes,

The goal of right-to-try is not to help terminally ill patients. It’s to diminish and weaken the FDA.

MJD says,

What if a pregnant woman legally pursues the right-to-try for self preservation.

Does the unborn child have any rights or protection?

If the answer to this question is no, the hippocratic oath may be compromised.

MJD is certainly a joke. Blocked from liberally discussing his nonsense here by our host, he resorts to childish JAQing off.

His mother must not have paid enough attention to him as a child.

sigh He’s our resident semi-troll who doesn’t swear at anyone and knows how to just skirt the rules (he’s gotten some limitations on what he’s allowed to post after extensive antics) so our gracious host hasn’t banned him.

He’s also generally not this flagrantly awful.

(MJD, this is an actual situation actual women face, and your deeply facetious approach is really cruel. Please go sit quietly and think about why your question is both blatantly stupid and nasty.)

JustaTech,

MJD, this is an actual situation actual women face, and your deeply facetious approach is really cruel.

MJD says,

Of course this may happen and this may be one of the questions that needs to be addressed if/when right-to-try is implemented.

If I’ve upset anyone with the questions construction, my deepest apology.

Let face it, if one desires a feel-good blogging experience Respectful Insolence is definitely not the place.

Hi Orac – this is an interesting post, and it really made me think. I am a bit of a fence-sitter on the Right to Try, not the least because my life was saved by an extraordinarily administered drug. I agree that the Goldwater model formula is missing a lot of parts, but perhaps Right to Try isn’t completely a lost cause? I explore some of these points in my response, which cites you extensively – here: http://chrisvoncsefalvay.com/righttotry
Once again, thank you for a thought-inspiring and well-written article.

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