The Right to Try

Right to Try legislation permits patients fighting a terminal illness to get access to not-yet-FDA-approved drugs. Thirty-one states have passed Right to Try legislation with massive shows of support but so far these laws are untested by the courts so it’s not clear whether they are anything but expressive. The massive support for Right to Try laws, however, suggests that there is demand for a better FDA as Bartley Madden writes:

Freedom is a powerful rallying call and 31 states have now passed Right To Try legislation with sky-high approval ratings by citizens.

…[But] the states do not have the legal authority to circumvent the FDA. Moreover, drug developers have a major disincentive to participate because, to survive, drug developers need to secure FDA approvals for their new drugs. And circumventing the FDA by providing not-yet-approved drugs to terminally-ill patients could easily slow or prevent FDA approvals.

…A better solution is Free To Choose Medicine (FTCM). It would solve the dilemma facing politicians who are pulled in one direction by citizens’ demands for more freedom and in the opposite direction by FDA proponents with demands for a highly-controlled process. A clear, brief explanation of FTCM is available on the Internet in the PowerPoint presentation, “Free To Choose Medicine and Right To Try.” It explains how we will all benefit from more freedom of choice.

First, the Free To Choose track (separate from the FDA’s conventional clinical testing track) enables patients and their doctors to make informed decisions about the use of FDA-approved drugs or not-yet-FDA-approved drugs. Patients, under the guidance of their doctors, would learn about initial safety results and up-to-date treatment results of FTCM drugs. FTCM drugs for a wide range of illnesses (not just terminal illnesses addressed by Right To Try) would be available up to seven years before conventional FDA approval.

Second, FTCM legislation would provide for government oversight of an open-access, Internet-accessible database. It provides up-to-date information for patients and doctors about a FTCM’s drug’s potential benefits and risks before they choose to use it. This is a self-adjusting system wherein more patients use FTCM drugs that work well and vice versa.

The open-access database would contain treatment results of FTCM patients including their genetic makeup and relevant biomarkers. This database (not part of Right To Try legislation) would reveal subpopulations of patients who do extremely well or poorly with the new drug. Pinpointing such groups of patients is a huge benefit to, not only patients, but to biopharmaceutical researchers working on new breakthroughs in medicine.

Third, FTCM federal legislation needs to provide a new type of drug approval – Observational Approval – based on treatment results for real-world patients who receive the FTCM drugs. This would motivate drug developers to participate as well as expedite insurance reimbursement for patients.

Naturally, I agree with Bart on the need for FDA reform.

Comments

The FDA kills more people than it saves by preventing life-saving medication, before even accounting for how much it raises prices for everyone. The FDA is a net destroyer of human life and quality-of-life.

Now, before I go full Randroid (abolish ALL the regulations!), I've been thinking about how to do effective regulation. I think I've hit upon a good principle for regulators, the skin-in-the-game principle. In real life I test foundations, structural materials, etc. I do not design them, I do not build them, I am only a third party inspector to verify things are as they were designed. If something goes wrong, I have skin in the game - I will get my pants sued off of me if I were negligent in my duties. In fact, the point of my company is largely a legal construct - to assume liability and to be a scapegoat if something goes wrong, particularly if it is foundational/structural.

This principle could be applied to all regulators, I believe. Sell whatever drug you want. Put arsenic in pill form and sell it if you want, and caveat emptor. People consumed them willingly in the Victorian age. And, in such a case, the manufacturer and possibly merchants are responsible for whatever side effects it produces. HOWEVER, if the manufacturer goes through the effort of acquiring an FDA Seal of Approval, the FDA now has skin in the game - the FDA assumes a measure of liability and provides a certain amount of protection for the seller. 50% of legal costs or something.

If the FDA puts too high of a bar for getting approval, no one will pursue FDA certification. If the FDA puts too low a bar on approval, they get sued out of existence. Somewhere in between you'll have companies making a strategic decision to pursue FDA certification because they want that to help sell their products, and customers will know that it's a mark of quality. Both manufacturers and consumers will win.

Good idea? Bad idea?

Liability? I watched yesterday one of those NatGeo shows on accidents. In one case the engineers liable for the accident got out just with minor charges because they followed "all the maintenance regulation". So, would the would be better if there were no regulations and these guys got life sentences? Something similar may be happening with drug approval. The FDA is bad, but if developers had full liability they could be out of business at the first mistake.

Mind sharing what the accident was in your anecdote? Engineers can't be responsible for unpredictable acts of nature. The best an engineer can do is design for what is predictable. Requiring anything else would be exorbitantly costly, and nothing would get built. An engineer can only be liable for the standards to which they've designed, which is what the owner also agreed to when they hired the engineer.

A bit late, the German ICE crash of 1998. The train operator received an informal communication from other train operator of fatigue problems before the crash. But, this communication was experience, not regulation.

In the beginning, before the FDA, patients took and doctors prescribed a random array of cures for a random array of diagnoses. In those days, the only claim a patient had to consider was one from a doctor, to a patient, saying that his way was best. Perhaps that felt good to the patient, if the doctor was compelling enough. Maybe it comforted the patient, regardless of the outcome.

The problem with returning to those bad old days is that you give up large scale data analysis. You have as many treatment regimes as there are doctors, and as many competing claims for "who's the best."

Is that emotionally attractive to you? If you imagine yourself ill, would you rather take what some random doctor or drug company *says* is effective? Or do you want some better data, perhaps even a randomized trial behind it?

Alex comes at this as politics and economics, but I'm sorry, it is science. You can bang your head against the wall all day, but freedom to choose does not produce better medicine. It produces a greater confusion of completing and untested approaches.

If you change the FDA we'll go back to the Stone Age!!

You liberal bleating is becoming hyperbolic.

I do believe your fact-based, reasoned response has changed my mind on this one, Al.

I usually let Alex's one man campaign against scientific medicine blow by, but I decided to take a moment today because it really is a trade-off between individual choice and progress, choice and health.

Alex frames this as about "terminal illness." In the vast majority of cases those are cancers and heart disease with understood best-chance treatments. Maybe the survival odds are low, but they are still best.

When you are free to choose something different, trendy, cult-driven, you are not choosing the best. I mean, look at the saga of Charlie Sheen. He quit HIV treatment, went to Mexico, to avoid the FDA, and (can this be true?) took an arthritic goat milk treatment. He got much worse, until he returned to the US and FDA approved treatment.

Alex is for the Charlie Sheen model of personal choice and treatment.

Anti-science? Science works by experimentation. Blocking the right to try limits experimentation.

Look, you can still have the FDA and highly controlled tests AT THE SAME TIME as having the right to try. These are not mutually exclusive. They are, in fact, mutually beneficial to each other - you can start focusing on promising results faster from the controls and science standpoint, and have freedom to pursue your own business transactions from the consumer standpoint.

That's a funny comment to put after Charlie Sheen. Here is a guy who is not a medical expert, making a highly emotional decision, seduced by a fringe doctor with a dubious cure.

Yay science.

It's kind of funny putting Charlie Sheen as the poster boy of Right to Try. You know that rich people have more options and ability to travel than normal people, right? And that Right to Try or not, rich people CAN and DO this stuff. I'd use the millions of people who cannot take intravenous aspirin (approved in the EU, but not US), or various heart medications, or arthritis medication, or diabetes medications would be much better poster boys. Except they're not eligible, because they aren't terminally ill. So I'll use an 8-year old girl dying of leukemia as my poster girl instead. Better to condemn her to death than to let anyone possibly scam her parents (as though that does not ALREADY happen)!

So back to science, Anon. I'll repeat myself since you failed to rebut it the first time:

Anti-science? Science works by experimentation. Blocking the right to try limits experimentation.

Look, you can still have the FDA and highly controlled tests AT THE SAME TIME as having the right to try. These are not mutually exclusive. They are, in fact, mutually beneficial to each other – you can start focusing on promising results faster from the controls and science standpoint, and have freedom to pursue your own business transactions from the consumer standpoint.

@Sam, there are existing pathways that are extremely low resistance that allow individuals and their physicians the ability to INCREASE experimentation by seamlessly adding people to existing trials as one-off patients. The idea that Right to Try increases the scope the legit clinical trials is ridiculous. Those trials exist and nonetheless people can be added to them very easily--and if there is a true emergency and a therapy a patient wants to try right now literally all it takes is a PHONE CALL and they have authorization from FDA.

"Alex is for the Charlie Sheen model of personal choice and treatment" [SNIP]

'Tiger's Blood Tabarrok'!

Jan, if that is true, then the Right to Try does not add much - but I do not see where it subtracts, either (beyond being More Laws On The Books, of which I'm not a fan). I remain very unsold on the Scammer argument for the harm in this law.

I have participated once in a medical trial for some extra cash. 4 years ago a company was trying to get FDA approval for intravenous aspirin, which I was informed was already in use in Europe. If that is how slow the gears turn, I am skeptical that FDA authorization is merely a phone call away. Skeptical, but without any further knowledge, so I'll defer that it is indeed as you claim. STILL, assuming this is all true and there are no further negatives, all it takes is for the FDA to stop being as responsive for this law to become useful again... and FDA response time is an administrative issue wholly out of the voter's jurisdiction.

The risk is that you open the door up too wide to where people start throwing away savings on charlatans. I generally lean towards having the government do less to protect folks from themselves, but medical stuff is a clear exception for me. People don't make rational decisions about chronic treatments let alone terminal diseases.

I don't think it would be a disaster, but people are already overtreated in this country, with end-of-life care being one of the worst offenders. I don't think we need to go further down that road.

Also, I'm assuming this is only talking about care purchased with private dollars, but the moment you go down that road, you'll get people who want medicare/medicaid to cover this stuff, too, and when the Government is paying the bills, the push towards expensive, useless treatments will really start in earnest.

"When you are free to choose something different, trendy, cult-driven, you are not choosing the best."

How do you know what I'm choosing?

The world actually works like it's presented in econ 101. And that's why people continue on to PhDs and most of the issues remain debated heavily. Because econ 101 logic is 100% correct.

Anon is probably right, no matter that your first and second year texts probably did not have a word for these extraordinarily complex situation where there are more than two goods, two people, two time periods, etc.

FDA regulations are not science. They may--or may not--facilitate science like scientists who gorge at the public trough, but this is fundamentally about politics.

Why does one "gorge at the public trough" when the public supports the research but "engages in R&D" when it's a private company? Don't you think that the research scientists who want to "gorge" will be found in the private sector and those who are committed to public benefit will be more likely to work in the public sector?

Public sector research scientists do not get to "gorge". Very often, PhD-holding research scientists in the public sector make less than a junior accountant with a couple/few years experience. Outside of war and spying industries, public sector scientists are most certainly not "gorging".

I agree that there is too much politics in the FDA though.

"before the FDA, patients took and doctors prescribed a random array of cures for a random array of diagnoses" This is just patently false. "The problem with returning to those bad old days is that you give up large scale data analysis. " As is this. It's certainly possible to perform large scale data analysis in the absence of the FDA. Both you and Tabarrok paint this absurdly black-and-white picture where your way is nothing but sunshine and roses, and the other way is calamity and death.

When I read accounts of how older treatments were developed the doctors seem more like butchers to me. There is plenty of 'well the patient was going to die anyway' to mask that these people were just chopping into patient's bodies with no actual plan or analysis of results. Got breast cancer? Let me cut most of your shoulder blade out and a few ribs for good measure see how that works, Caveat Emptor, blah blah blah liability.

Do we really have to go back to people popping arsenic pill and tapeworm eggs before we appreciate the FDA?

Why do libertarian types thing they won't suffer from the same asymmetric information that all the rest of these sick people have to deal with?

Lots of blanket statements here and literally no evidence to back it up.

I'm not sure where I said I wanted to do away with the FDA or regulation. My position is that if a regulator says something is safe, they should assume some liability if they lie. As it is, regulators are just another hoop to jump through - they don't have any real skin in the game.

And if someone wants to gamble their lives on something that doesn't have an FDA seal of approval, THEY CAN AND DO ALREADY DO THAT. I'd rather have more reputable gambles available. FFS, isn't this part of the reason why abortion is legal? People are going to do it anyway, let's have it out in the open?

You've just made a great argument for anarcho-capitalism.

I like this bit:

"First, the Free To Choose track (separate from the FDA’s conventional clinical testing track) enables patients and their doctors to make informed decisions about the use of FDA-approved drugs or not-yet-FDA-approved drugs. Patients, under the guidance of their doctors, would learn about initial safety results and up-to-date treatment results of FTCM drugs" [SNIP]

I bet half the readers of this blog wouldn't be able to parse the statistics and contra indications of these alleged 'wonder pharmaceuticals' in their addled, confused states which the evil Pharma Regulator is withholding from general use.

And their physicians wouldn't know either.

UPSHOT: Another Tabarrok Nursery Tale.

==> "...the dilemma facing politicians who are pulled in one direction by citizens’ demands for more freedom and in the opposite direction by FDA proponents with demands for a highly-controlled process. "

... and just who are these "FDA proponents" that somehow have equal footing with the "citizens" who politicians are sworn to represent ?

If politicians honestly represented their constituents and followed the Constitution ...there would be no dilemma, no loss of medical freedom, nor an FDA.

The preceding has been fully approved by the snake oil PAC, and is guaranteed to be accurate, or double your money back.

'Right to Try legislation permits patients fighting a terminal illness to get access to not-yet-FDA-approved drugs.'

Finally, amygdalin will have the chance to save all those desperate enough to buy it, without those selling it needing to worry about being jailed. Particularly if one buys laetrile, a modified form first patented in 1961, and kept off the market for decades by the FDA, unavailable in the U.S. for those with terminal cancer. https://en.wikipedia.org/wiki/Amygdalin

And shouldn't the Bartley J. Madden Chair in Economics at the Mercatus Center note that fact when citing Bartley Madden?

I didn't want to deny you the pleasure.

Thanks - but I just noticed that there is a (recently added, compared to a previously saved version of the post) link to your GMU home page, where that position, which has nothing to do with any Commonwealth of Virginia taxpayer funded institution of higher learning, is listed as your first accomplishment, even before noting you are a member of the economics faculty at George Mason University.

Bartley J. Madden must be a truly inspirational figure, much along the lines of how Prof. Buchanan inspired Prof. Cowen. It is a shame, based on previous experience, that at best only a select few readers would be able to look at links to Mr. Madden's interests in public policy.

But if it makes you feel better, this must have only been a slip up, right? You really have done so much better recently putting your Mercatus Center accomplishment front and center when it comes to posts relating to such concerns.

Did one of these guys flunk you out of a class or something? What's with the special hate for the university and especially the Mercatus Centre?

He was fired from GMU. Had nothing to do with Tyler or Alex, however, they are just paying the price.

Should be called, Right to Die.

Actually, they should call it "Right to Lie" legislation. See my post below on why this problem is 100% manufactured.

I noticed that California, which is a "right to die" state is not a "right to try" state. What sorted of twisted logic leads to someone believing that people should have the right to take drugs that they believe will kill them but not drugs that they believe will keep them alive.

It makes sense if you believe "right to try" is really "right to scam people on their death bed."

In your own formulation it is "that they believe will" and not "what will" keep them alive. A key difference that you implicitly acknowledge.

You write as though FDA approvals assures that something is safe and effective while non-FDA approved is a scam. The reality is that it is just one of many data points of varying value in guessing whether a medical intervention will be beneficial. I understand that you are comfortable giving them the ability to make the decision for you, but I'm not. I suggest that we compromise. We'll both continue to support and fund the FDA, but allow people to opt out of being restricted by them. You can opt to not be allowed to buy medical care that they don't approve and I can opt to be allowed to make my own decisions. Seems fair, even generous, to me.

As a functional truth, people are free. Charlie Sheen, and many poorer people, do run to Mexico for dubious cures.

Tell me, what dubious cures have come out of Mexico, as effective and now mainstream? Tiger Blood?

This guy says it better than I ever could.

http://scienceblogs.com/insolence/2015/05/25/the-cruel-sham-that-is-right-to-try-continues-to-spread/

That was pretty weak. Patients might lose insurance coverage for certain services in the event of complications? Not exactly the island of Dr. Moreau. And besides that, two paragraphs earlier the guy is telling us these laws won't benefit most people because only wealthy patients will be able to afford experimental drugs in the first place. Awful lot of fuss the guy is making over a law that might potentially deprive dying rich people of their money.

That's a strange spin to put on it. I wonder if anybody else has the same take on that article. I think it is a very well-reasoned takedown of why these "right to try" laws are very harmful to patients.

I thought it was a good read. Very informative.

I was not impressed. His points were

A) The policy comes from the Goldwater Institute. So? Who cares where it came from, focus on the merits of the policy itself.

B) This is state-based, so it doesn't work because federal law squashes it. So? The federal law being in the way does not mean that it's a bad policy.

C) The consumer assumes all liabilities when taking a risky drug. So? I mean, so? Really, what's the problem with that? Have the consumer sign a waiver acknowledging that medicine isn't an exact science.

D) False hope. So? Is that worse than no hope?

Mark, I bet you could say it way better than that article. It was not very persuasive, and meandering to boot.

Is medical insurance required to pay for this experimental treatment?

A legitimate pharmaceutical company would have to be out of their minds to supply a drug for this market - never mind what the FDA says.

This isn't a new market. This is an existing market involving drugs that are likely already in the approval process, have not yet received full approval, but show very promising results based on existing tests.

A legitimate pharmaceutical company wouldn't need the FDA to protect its business against competition.

Yes, drugs like antineoplastons.

http://scienceblogs.com/insolence/2014/04/16/sad-news-a-burzynski-success-story-is-gone/

That there are all sorts of efficacious drugs in the pipeline, stymied only by FDA regulation, is a fallacy. Most drugs fail in Phase II/III. Drugs that make it through Phase III have a >80% chance of FDA approval. Thus, the reason that only 8-10% of compounds make it through this process is because they are either not safe or not efficacious, not because of FDA red tape. Increasing patient access to drugs outside of controlled clinical trial settings would only make it more difficult to determine those drugs that are of clinical value.

Moreover, true breakthrough therapies tend to be approved quickly. A few recent exapamples: 1) imatinib for CML went from first clinical trials to FDA approval in 3 years 2) sofosbuvir for HCV had a similar 3-4 year timeframe 3) novel immunotherapy has had an even shorter developmental timeline (2-3 years with some of the new PD1 inhibitors). The challenge for drug development is on the science side of things: finding these targets and compounds to target them. It is not with the FDA.

See more: http://blogs.sciencemag.org/pipeline/archives/2014/06/10/right_to_try_here_we_go

Generally agree. Some say the reason is that companies don't put enough money back into research, while others have asserted that regulation of clinical trials is too stringer. I have a some sympathy for the latter view--academic IRB's are often too risk averse. But I think most of the seeming slowdown in innovation of meaningful therapies that benefit large populations is simply that the low-hanging fruit has been picked. It is a lot harder to innovate now. The basic science, the kind of stuff industry doesn't fund, really needs a boost in investment if we want to see more drugs come online in the next few decades.

They could do lower quality study of a larger number of drugs, presumably a larger number of which they could market, if more got approved. Whether that involves actually delivering any improved outcomes is another question ...

"Right to try" legislation in search of a problem that the Goldwater Institute is pushing due to an ideological opposition to the FDA's existence.

In fact the FDA already has a very easy and quick method for patients to access trial drugs in such circumstances. In 2014, the agency rejected just 9 of nearly 2000 requests like this. In general, it approves 99.5% of requests. And it has only made the process simpler since then.

http://www.raps.org/Regulatory-Focus/News/2015/02/04/21243/From-100-Hours-to-1-FDA-Dramatically-Simplifies-its-Compassionate-Use-Process/

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM432717.pdf

First sentence should read *is a solution in search of a problem*

Why shouldn't two consenting adults be allowed to do whatever they want to do together? If a black and a white want to marry each other, that's fine with me. If two men want to sleep with each other, that's fine with me. If somebody wants to buy bourbon from somebody else, that's fine with me. If one person wants to buy heroin from another person, that's fine with me. If a person wants to buy medical treatment from another person, doctor or not, terminally ill or not, that's fine with me.

You are in a fuzzy state. You might have just had a stroke. You are in an emergency room. Your doctor says you have two choices for treatment. He doesn't tell you that one flys him to Maui every year.

Do you trust naked capitalism to give you the best advice?

What? The doctor says i get a free trip to Maui every year if I take the stroke treatment he recommends? (Blinking adamantly in agreement, not yet having recovered motor control ...)

As long as We, the Taxpayers (and We, the Holders of Mandated Insurance Policies) don't have to pay to clean up the mess caused by people self-medicating I'm all for it.

So once you want to "try", you should be ineligible for Taxpayer-funded treatments.

Alex,

For a few years I've been following your criticisms of the FDA and your calls for reform. The only theme you seem to be concerned with is the principle of autonomy — like here in your support of "Right To Try"/"Free To Choose". However, that is just one part of medical ethics. In both theory and application, ethical systems of medicine must consider several other principles as well (regardless of whether you're approaching it from a deontological or teleological perspective). Perhaps you're aware of the complexities of the ethical questions involved, but that isn't readily apparent in the basic econ arguments you tend favor.

I won't write at length here. But I will point to things like the Burzynski Clinic or Hippocrates Health Institute as repugnant results (i.e. frauds, taking advantage of vulnerable families, persuading people to forego legitimate treatment, all without the possibility of treating a patient) of what happens when you focus solely or too much on autonomy.

Thus far, your posts over the years only highlight the fact that medicine is far too important to leave in the hands of academic economists.

That's funny. I interpreted his criticisms over the years to highlight the fact that medicine is far too important to leave in the hands of government bureaucrats. I don't see him calling for academic economists to assume any role in how individuals should make medical decisions.

There are times when I wonder whether the big pharmaceutical companies aren't proponents of the regulatory barriers to drug approval. The barriers certainly create real anticompetitive hurdles; hurdles that the few mega corporations have less trouble clearing than any startup. Big pharma has plenty of lobbyists, and they were certainly able to influence the Medicare D law in their favor. As things stand now, they are able to charge huge markups on old molecules for new indications while shedding crocodile tears about the mean old FDA.
The laws, rules, and regulations that we have didn't arrive on tablets of stone from Mt. Sinai. Some of them may have been written by cadres working on five year plans, but I think that lobbyists often have a big role in writing them.

Thread is probably dead, but another interesting take;

http://www.nytimes.com/2016/08/09/opinion/cancer-drug-ads-vs-cancer-drug-reality.html

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