When I wrote yesterday about the cruel sham that is “right-to-try,” , one criticism (among many) that I made of these misguided, profoundly patient-unfriendly laws was that I have as yet been unable to find a single example of a patient who has managed to obtain access to an experimental therapeutic through such a law, much less been helped by it. So-called “right-to-try” laws, of course, claim to provide a mechanism by which patients with terminal illnesses can obtain access to experimental therapeutics not yet approved by the FDA but still in clinical trials. They are, as I’ve pointed out, a cruel sham, placebo legislation that makes lawmakers feel as though they’ve done something good but do nothing of substance for patients while providing them with false hope. The federal government through the FDA controls drug approval, which means that states can’t compel a drug company to provide a drug to a patient, and most drug companies would not want to risk jeopardizing approval of their drug, which is what could happen if they grant access to an investigational drug under right-to-try and the patient suffers an adverse event. After all, the success rate for drugs that have passed phase 1 (which is all that right-to-try requires) in phase 3 trials is only on the order of 9-12%, meaning that that’s the most optimistic probability that such drugs would benefit a patient. In reality, it’s almost certainly much, much lower.

Basically, the whole right-to-try movement is built on a delusion, namely that there are scads and scads of cures out there that are only being kept from the people by the cautious bureaucracy of the FDA. If the people could just get the FDA out of the way—or so the delusions go—cures would flow to the people. Add to that the libertarian delusion of the Goldwater Institute, upon whose model legislation nearly all of these bills and laws are based, that the free market will take care of safety issues, and it’s possible to see the long game being played, a strategy designed to drastically weaken the FDA’s control over drug approval. Then add to that the way that right-to-try legislation strips virtually every protection away from patients in clinical trials. For instance, no IRB oversight is required in right-to-try. There is nothing in the laws that provide for paying for the experimental therapy being used. Indeed, not only don’t insurance companies have to pay for right-to-try, but they don’t have to pay for any care that results from complications resulting from right-to-try. If a patient chooses right-to-try and it goes on, he’s completely on his own.

All of this brings me to a story I saw after I wrote yesterday’s post. The story is about an unfortunate man named Bob Bardone who last summer got one of the worst pieces of news a human being can get. He found out that he had amyotrophic lateral sclerosis (ALS), and it was estimated that he probably had about two or three years to live. His story is, as is the case for most ALS patients, heartbreaking. ALS, as many of you know, is a relentlessly degenerative neurologic disease in which the patient gradually loses motor control. Ultimately, patients lose the ability to walk, to move, to speak, and, ultimately, to breathe. Indeed, if there is a disease that I fear more than cancer, it’s something like ALS, which produces a nightmarish, unstoppable deterioration. There’s currently only one drug approved for ALS that I’m aware of, riluzole, but it only adds at best months to life expectancy. If ever there were a disease that needs new treatments, it’s ALS.

As you might recall, Missouri was one of the first states to pass right-to-try legislation. As such, and because of his past connection to the state, Mr. Bardone and his wife decided to move back there in order to be closer to family and, not coincidentally, to try to take advantage of right-to-try:

The move to St. Louis happened to bring them hope. They learned Missouri had become the third state to enact “right to try” legislation, which went into effect in August and allows terminally ill patients to try promising drugs yet to be approved by the FDA.

The family had been closely following news about a drug, GM604. Its maker reported that it significantly slowed the progression of ALS in 12 patients over 12 weeks with no dangerous side effects, though some scientists question the findings. The drugmaker, Genervon Biopharmaceuticals, is seeking fast-track approval from the FDA to sell the drug before spending years studying it in larger numbers.

“My dad had hope for the first time since diagnosed with ALS when he found out Missouri was a right-to-try state,” Connors said. “He thought that because he was moving back to Missouri, that he could try this drug.”

Bardone’s neurologist tried to get more information about the drug, and the family made emotional requests to the company. Genervon, however, refused to provide it.

After months of trying, the family is disappointed and frustrated, and Bob Bardone’s health is worsening.

“Can you imagine the hope we felt?” Connors said. “This entire process puts the most emotional time in our lives on even a more emotional roller coaster.”

The article further notes:

But right-to-try laws may be meaningless because companies can refuse to provide their experimental drugs. They may only create a false sense of hope among desperate families.

“It gives people a false impression that somehow because it’s a state law, it mandates this, but really it just gets everyone up in arms,” said Dr. John DiPersio, the deputy director of the Barnes-Jewish Hospital Siteman Cancer Center. “All it’s doing is causing commotion and confusion.”

This is, of course, exactly what I’ve been saying about these laws for over a year now. They are placebo legislation. They do absolutely nothing to help a man like Bob Bardone, but they do give patients like him false hope, only to cruelly take it away.

I can anticipate right now one objection. Why did Genervon Biopharmaceuticals refuse to give Mr. Bardone access to the drug? There are quite a few reasons why companies might not want to do this. This story, for instance, reports that Genervon has been overwhelmed by requests for its drug from ALS patients. Therefore, it chose to concentrate on getting fast track approval from the FDA, which “would give immediate access to all ALS patients, require doctors to prescribe and monitor patients’ progress, allow us to continue gathering data … and the cost would be supported by health insurance.” This is, of course, a perfectly reasonable justification, particularly given that if a patient had an adverse reaction to its drug outside of its clinical trials, something that is more likely if the drug is given outside of the controlled setting of a clinical trial, it could delay or even scuttle approval of the drug by the FDA. Also, some companies are small venture companies with inadequate resources to dedicate to fulfilling right-to-try requests. Indeed, some of these smaller companies might just barely have raised enough capital to make enough drug to do the clinical trials necessary to gain FDA approval. These are, of course, exactly the sorts of companies that we want to encourage, not impede, because they aren’t the big pharma behemoths and tend to be more imaginative and daring.

The Goldwater Institute’s response to these problems with right-to-try shows just how clueless or disingenuous it is. Basically its flacks argue that the FDA should assure companies that outcomes of patients who receive drugs for compassionate use will not negatively affect a drug’s approval. Seriously? That response basically indicates to me that the Goldwater Institute knows that right-to-try is a sham and has no power over the FDA, given that it’s reduced to suggesting that the FDA do something that it knows the FDA cannot do legally or ethically. Besides, the FDA already has a compassionate use program that rarely turns down a request. Also, as I’ve described, even the problem with how onerous the forms are for a physician to fill out is being dealt with. As this story notes, most applications are dealt with in four days, and the FDA has drafted a new form for such requests that, when finalized, should take a physician 45 minutes or less to complete.

There’s an excellent summary of the problems with right-to-try laws that was published on Friday on the Health Affairs Blog that I missed before but won’t now. It’s by David Farber, Preeya Noronha Pinto, Arthur Caplan, and Alison Bateman-House. They are quite blunt, noting that, contrary to the name given these laws, they provide no new rights to patients. They also point out that these laws have created an expectation that terminally ill patients will be able to quickly access life-saving experimental drugs by being exempted from FDA oversight but that that expectation is “quite simply, false.” They also note that legally:

If RTT laws are understood as actually providing a right to direct access to an investigational product without FDA approval or oversight, they would be “preempted” by federal law, meaning the federal laws are so powerful as to effectively nullify their state RTT counterparts. Indeed, several federal courts have already concluded that FDA’s comprehensive regulatory regime governing the manufacturing, approval, labeling, and distribution of drug products preempts state laws designed to legislate in this area. If challenged in court, we anticipate RTT laws will be similarly treated.

And:

It is only a matter of time before courts nullify RTT laws. In the meantime, these laws offer vulnerable patients misplaced hope and do not attempt to minimize the serious health and safety risks inherent in treatment with unproven treatments. State legislatures still evaluating the merits of RTT laws should think twice. Patients facing terminal illnesses have enough issues to navigate without being given false hope through legislation that is ethically, legally, and clinically flawed.

I don’t know if it’s definitely a matter of time before the courts nullify right-to-try laws, but that would seem to be a likely outcome. Of course, as I’ve discussed before, the purpose of right-to-try laws is not what is ostensibly claimed. Rather, it’s a long game in which—or so it is thought—the buildup of pressure through the states will force the FDA to loosen its grip on drug approval. Some of the more “open” libertarians will even argue for the neutering or even outright abolition of the FDA, claiming that the free market will take care of drug safety and efficacy.

I do find one thing hopeful. Over a year ago, when the very first of these laws were wending their way through state legislatures, few, if any, medical groups spoke out against them or openly opposed them. Indeed, as I described in my very own state, when our right-to-try bill came before the legislature, none of the major medical societies or cancer centers openly opposed it, and I was definitely discouraged from doing so. Again, as I’ve said so many times before, opposing right-to-try can easily be painted to be akin to opposing mom, apple pie, and the American flag and having a propensity to drop kick puppies through flaming goal posts. Never mind that right-to-try laws are profoundly anti-patient laws.

That time seems to be coming to an end. After a year, people are starting to realize that, as far as it goes to right-to-try, the emperor has no clothes. The story about Bob Bardone is one indication. I don’t recall seeing such a skeptical story about right-to-try in a long time, if ever, complete with a human interest story that demonstrates quite effectively what a sham these laws are. Then there’s this story about the right-to-try law introduced into the California legislature:

The California bills have drawn opposition from the California Medical Association and from groups representing nurses and oncologists. A group representing pharmaceutical manufacturers has sent a letter warning against skirting the FDA’s established channels.

“We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process, which is not in the best interest of patients and public health,” a letter from Pharmaceutical Research and Manufacturers of America says.

And:

The California Medical Association states that they have significant patient safety concerns with allowing access to unproven drugs outside of the FDA’s clinical trials and compassionate use programs. They further argue that offering unapproved therapies without credible scientific rationale or controlled monitoring could lead to not only endangering terminally ill patients further but potentially exploiting their hopes and circumstances. The Association of Northern California Oncologists cite the difficulty of identifying a terminally-ill patient, the danger a “right to try” policy outside the context of a clinical trial would present to adult clinical trial enrollment and the lack of an informed consent process to protect patients seeking investigational drugs as reasons for establishing their oppose position on the bill. The California Nurses Association/National Nurses United (CNA) maintain that this bill does nothing to address the real barriers to “compassionate use” revealed in a recent article in the NEJM entitled Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs, specifically stating that nothing in this legislation impacts the availability of drugs to terminally ill patients unless the manufacturer of the drugs allow it to be used in advance of FDA approval. The CNA goes on to state instead of taking on the cost of drugs and challenging drug manufacturers that charge excessive prices for all drugs sold in California, this bill reinforces the status quo for investigational drug costs.

These are all good points. However, it’s probably too little, too late, given that the CMA refused to take a stand on this legislation initially. Indeed, there remain editorials trotting out the same Goldwater Institute talking points. Contrary to these dubious arguments, right-to-try laws sell terminally ill patients and legislators a bill of goods. they promise access to life-saving experimental drugs while leaving vulnerable, desperate patients open to false hope, lack of the oversight that patients in clinical trials receive, huge medical bills, and loss of medical coverage for any complications that might result from trying unproven drugs. People seem to be finally waking up to the sham that is right-to-try, but too late.

Comments

  1. #1 zebra
    May 26, 2015

    Someone with something to say:

    http://tinyurl.com/k6zpawf

    As opposed to: “Gosh, some guy somewhere mistakenly thought he could get this experimental drug, and he sure was disappointed when he couldn’t.”

    Beam, mote.

  2. #2 Orac
    May 26, 2015

    Which is, of course a non sequitur. Just because there are problems with research at the University of Minnesota does not mean my criticisms of right-to-try aren’t valid.

  3. #3 Nigel Tufnel
    May 26, 2015

    How not to argue, by Zebra:

    Orac: right to try laws are a cruel sham. State laws cannot trump federal regulations and patients with terminal illnesses are given false hope for cures that never materialize and left on their own to deal with complications from untried drugs that, at best, have a 10% chance of having any positive impact.

    Zebra: Here’s a link that shows that the Institutional Review Board at the University of Minnesota is corrupt and ineffective and suggests that IRBs should be replaced by independent regulatory bodies unconnected with the institutions doing the research. I win!

  4. #4 Eric Lund
    May 26, 2015

    Thanks for the follow-up. It answers my question from yesterday that FDA authority basically overrides these state laws. Of course, the Goldwater Institute might be gearing up to try passing a federal “right to try” statute.

    And while the previous post mentions this, Mr. Bardone’s story makes abundantly clear that we aren’t just talking about Big Pharma. My guess is that companies like Genervon Biopharmaceuticals, which are running on venture capital and therefore have just enough resources to produce just enough product to get through standard clinical trials, would be where most of the action is–“Little Pharma”, if you will. Most of those companies will go belly-up when their experimental drug fails to clear clinical trials. Perhaps a Big Pharma company will buy out a Little Pharma company that makes it through clinical trials.

    Incidentally, I know some people who play the VC game, and they tell me that successful VCs only expect about 10% of their bets to pay off (the payoff from the successes is more than enough to make up for the failures). That’s right in the 9-12% success rate quoted for drugs in clinical trials.

  5. #5 zebra
    May 26, 2015

    @2 Orac,

    See my comment on the previous post.

    All these issues you discuss are the result of the problems that exist in the mainstream.

    You say something like “it may be too late”; yep, you didn’t fix the FDA process and someone is taking advantage of that.

    You are blaming the mosquito because you didn’t fix the window screens. And using it as a distraction from fixing them.

  6. #6 Orac
    May 26, 2015

    @Nigel: Yep. zebra’s argument seems to boil down to this: If one IRB is corrupt then they all must be and therefore being under IRB oversight is no advantage and therefore right-to-try must be OK.

    Of course, problems with IRBs are not the main driving force behind the right-to-try movement. Indeed, I bet that most of the politicians and people supporting such bills are, at most, only vaguely aware of what an IRB is and does, if even that. That’s why zebra’s argument is such a lovely non sequitur.

  7. #7 zebra
    May 26, 2015

    #6 Orac,

    The Strawman is about the most pathetic form of rhetorical fallacy.

    If you want to be a crusader/writer, I refer you the author of the cited NYT article as an example– it takes skill and discipline, and a broad view with a clear sense of priorities.

    Or, there’s always the Dr. Oz paradigm.

  8. #8 Gray Falcon
    May 26, 2015

    zebra, you’d have a better chance at being understood if you didn’t spend your time being deliberately incomprehensible.

  9. #9 MarkN
    May 26, 2015

    With the recent animal model success into developing the EV vaccines, I was particularly discouraged by the push to “fastrack” anything around the FDA safety protocols. ‘See? This is the way to do it…we don’t need due diligence & arm’s length oversight — everyone be “buddy buddy” etc..’

    Ugh. Yes 10+ years to get a worthwhile therapy on the market is brutal, but these safeties and evidence-based protocols are supposed to mean something. Anyone recall the giant push to empower women by offering the morning sickness pill in the 70s? Can you imagine the pressure on the FDA doctor who decided NOT to approve it? Kinda like saying you’d ban Viagra, at the time. Obviously, we would have been dealing with thousands of birth defect cases in this country had such an end-around been used. Tell you what though, that doc had some serious cahonnes.

  10. #10 ann
    May 26, 2015

    The Strawman is about the most pathetic form of rhetorical fallacy.

    I take it that you’re apologizing for this?

    As opposed to: “Gosh, some guy somewhere mistakenly thought he could get this experimental drug, and he sure was disappointed when he couldn’t.”

    That’s very gracious of you. I encourage and applaud it.

  11. #11 Lawrence
    May 26, 2015

    @MarkN – correct, FDA clinical trials and safety protocols exist for that very reason. This push to circumvent those rules – even in cases were we are dealing with terminal patients, undercuts the entire process and could lead to more problems down the road.

    I had heard the companies don’t like these laws, since the single or small use cases are entirely unprofitable & can cause issues with the regular trial process – though in most cases, these new “laws” exempt the companies themselves from any harm that may come from the untested treatment.

    I find it ironic that it is a lot of the same people that claim the FDA isn’t doing enough to maintain the safety of medicine are asking the same agency to back away from safety.

  12. #12 Orac
    May 26, 2015

    This push to circumvent those rules – even in cases were we are dealing with terminal patients, undercuts the entire process and could lead to more problems down the road.

    This is how it begins. Terminal patients are vulnerable, and it’s an emotionally appealing argument to say that they have “nothing to lose” and therefore should be able to try whatever they want or take on whatever risks they want, asking “How could it be any worse?” Of course it can always be worse. You could not just have a terminal disease, but you could also have bankrupted yourself and your family paying out of pocket for a drug that’s unlikely to help you. You could also have suffered complications from that treatment, making your brief remaining time more miserable than it has to be or even shortening it, while losing your health insurance coverage to treat that complication.

    Don’t think that these laws are just designed for the terminally ill. That’s what they say, but they are a wedge to widen the population bypassing FDA approval or compassionate use programs.

  13. #13 Narad
    May 26, 2015

    You say something like “it may be too late”; yep, you didn’t fix the FDA process and someone is taking advantage of that.

    You are blaming the mosquito because you didn’t fix the window screens.

    I take it that the whole preemption part failed to sink in.

    (What kind is a separate question.)

  14. #14 Lawrence
    May 26, 2015

    @Orac – I agree wholeheartedly…the “terminal” cases are merely the front of the emotional appeal to open up compassionate use to anyone who can afford (or not, as the case may be) treatments that haven’t undergone the FDA vetting and testing process.

  15. #15 Todd W.
    http://www.harpocratesspeaks.com
    May 26, 2015

    Orac, thank you for bringing this up again and relating the story of Mr. Bardone. Seems like a good time to point back to my open letter to legislators. Feel free to use it in communication with your state reps.

  16. #16 zebra
    May 26, 2015

    Wikipedia:

    The FDA approves a drug for prescription use, and continues to regulate the pharmaceutical industry’s promotional practices for that drug through the work of the Office of Prescription Drug Promotion (OPDP, formerly the Division for Drug Marketing, Advertisement and Communication (DDMAC).[14] The FDA does not have the legal authority to regulate the practice of the medicine, and the physician may prescribe a drug off-label. Contrary to popular notion, it is legal in the United States and in many other countries to use drugs off-label, including controlled substances such as opiates. Actiq, for example, is commonly prescribed off-label even though it is a Schedule II controlled substance. While it would be legal for a physician to independently decide to prescribe a drug such as Actiq off-label, it is illegal for the company to promote off-label uses to prescribers. In fact, Cephalon, the maker of Actiq, was fined for illegal promotion of the drug in September 2008.[15] Under the Food, Drug, and Cosmetic Act (FDCA) at U.S.C. 21 §§301-97, manufacturers are prohibited from directly marketing a drug for a use other than the FDA-approved indication. However, in December of 2012, the United States Second Circuit Court found that promotion of off-label uses by a company sales representative was considered to be protected speech per the First Amendment.

    So, no clinical trials needed, no scientific evaluation of relative risks to benefits. And insurance may or may not cover it.

    Who needs wedges and slippery slopes?

  17. #17 Lawrence
    May 26, 2015

    @zebra – those are all drugs that already went through FDA approval, as opposed to “compassionate use” or right to try, which have not.

  18. #18 Orac
    May 26, 2015

    A fact that seems to have eluded zebra.

  19. #19 zebra
    May 26, 2015

    Lawrence, Orac,

    Talk about non-sequitur.

    What’s the difference, apart from perhaps some better knowledge about safety/side effects?

  20. #20 Todd W.
    http://www.harpocratesspeaks.com
    May 26, 2015

    Is zebra really arguing a form of tu quoque, albeit pooh-poohing a significant difference?

  21. #21 Lawrence
    May 26, 2015

    It isn’t a non-sequitur (unless you are calling your own statement a non-sequitur – which it pretty much is)….as again, off-label uses are for drugs that have already been proven for safety and effectiveness.

    Very little, if any of that information is available for the types of drugs covered under these bills….

  22. #22 Orac
    May 26, 2015

    At this point I have to wonder if zebra is being deliberately obtuse if he can’t see the difference between right-to-try and off-label prescribing.

  23. #23 JP
    May 26, 2015

    At this point I have to wonder if zebra is being deliberately obtuse if he can’t see the difference between right-to-try and off-label prescribing.

    “Being deliberately obtuse” is a pretty good description of the Zorse’s general modus operandi. Either that, or Z. is simply obtuse.

  24. #24 Helianthus
    May 26, 2015

    @ Zebra

    What’s the difference, apart from perhaps some better knowledge about safety/side effects?

    You will have a hard time convincing us that’s a trivial difference.

  25. #25 Todd W.
    http://www.harpocratesspeaks.com
    May 26, 2015

    @Orac

    At this point I have to wonder if zebra is being deliberately obtuse if he can’t see the difference between right-to-try and off-label prescribing.

    If I recall correctly, zebra once boasted about being deliberately obscure to promote critical thinking, or some such. In reality, all zebra accomplishes is nonsensical arguments and being able to claim we’re arguing against a straw man when people try to make sense of it’s digital spewings.

  26. #26 Orac
    May 26, 2015

    There was a certain other commenter who used the same technique, but I forget who it was.

  27. #27 Narad
    May 26, 2015

    Talk about non-sequitur.

    What’s the difference, apart from perhaps some better knowledge about safety/side effects?

    You really never learn, do you? The field considered is different,* but the discussion in Oullette v. Mills gives the basics of preemption, which I had already brought up in the comments to part 1.

    * Although not unrelated.

  28. #28 shay
    May 26, 2015

    For all the libertarians out there, Genervon’s refusal to use this legislation to sidestep the FDA is a perfect example of market forces at work .

    It’s just not working the way they think it should work.

  29. #29 Narad
    May 26, 2015

    Genervon’s refusal to use this legislation to sidestep the FDA is a perfect example of market forces at work

    They may well have also taken note of the fact that being shielded from a state tort action in Missouri is no great shakes. Or it could be something else.

  30. #30 Todd W.
    http://www.harpocratesspeaks.com
    May 26, 2015

    It’s interesting how history seems to be repeating itself here. About 20-25 years ago, there were complaints that FDA was moving too slowly, holding up the approval of new drugs. So FDA created the fast track approval system. The number of new drug approvals went up, along with an increase in recalls of newly approved drugs (e.g., dexfenfluramine), prompting outcries that FDA was approving drugs too quickly. I can see a similar set of events playing out in the near future. There may be a different set of parties to “blame”, but it’ll likely come about.

  31. #31 Narad
    May 26, 2015

    The Genervon story gets better.

    While patients often say that this drug represents their only hope, [Steve Perrin, president and chief scientific officer of the ALS Therapy Development Institute] has been quick to point out that there are a number of ALS drugs in the clinic. And if the patient campaign were to succeed, patients taking the drug would be excluded from trials for a drug that might actually work.

    FierceBiotech followed up by asking Genervon CEO Winston Ko a simple question: Has the company actually filed a new drug application–which would be necessary for any kind of approval, accelerated or otherwise, that it’s been demanding–and how has the FDA responded? If an NDA was filed, then the agency would have had a chance to see if the application was acceptable for review, or not.

    The company, which is based in Pasadena, CA, and has cited biblical prophecy as an inspiration for its work, did not answer this question in a reply to FierceBiotech Friday afternoon. In an e-mail, Genervon did say it was considering the FDA’s request, then repeated its demand for an immediate approval in order to save the lives of tens of thousands of patients.

  32. #32 Orac
    May 26, 2015

    Oh, dear. I might have to look into Genevon’s drug and clinical trial a little more closely this week…

  33. #33 Denice Walter
    May 26, 2015

    I am formally requesting a higher quality of troll at RI: if this should keep up, we might get lazy- which is not at all efficacious.
    Thank you. Merci.

    OT but are crunchy moms organising to save innocent children from the ravages of GMO crops ((shudder)) ever TRULY OT at RI?
    PLUS it’s late and I’m suffering miserably from allergies…

    For the past few weeks, I keep metaphorically running into ZEN Honeycutt, of Mission Viejo, a mother to 3 with have autism and allergies betwixt them who have been aided immensely by a GMO-free, organic diet.
    OK, she’s associated with BOUGHT!, will be featured at Fearless Parent Radio tomorrow and initiated Mums ‘Cross ‘Murka ( actually, Moms Across America.com).
    She’s on You Tube, met Stephanie Seneff at Autism One, was on Oz’ show and wants to change the world.
    Then, there’s her name. ( She’s not referenced at RI search)

  34. #34 herr doktor bimler
    May 26, 2015

    Therefore, [Genervon] chose to concentrate on getting fast track approval from the FDA

    Just not enough to file an application.

  35. #35 Lawrence
    May 26, 2015

    Very interesting – as it appears that this “right to try” legislation could be viewed by some unscrupulous companies as a means to market and sell untested products without the need for FDA approval……

  36. #36 JustaTech
    May 26, 2015

    Lawrence @35: And viewed by scrupulous companies as a way to get totally screwed.

    If you announce promising results from your Phase I trial in the media, you get swamped by RTT requests.

    If you fulfill the requests you don’t have enough product (or possible research patients) to do a proper phase II.

    If you don’t fulfill the requests then you’re evil and denying people life-saving drugs.

    If you don’t announce your Phase I results in the media, then you’ll never get enough good press to get the VC funding you need to complete the phase II.

    You’re just screwed all the way around.

  37. #37 Narad
    May 26, 2015

    Very interesting – as it appears that this “right to try” legislation could be viewed by some unscrupulous companies as a means to market and sell untested products without the need for FDA approval

    This was touched on at In the Pipeline. Personally, I don’t think it really has a shot except, again, at the intrastate level.

  38. #38 Narad
    May 26, 2015

    ^ To wit.

  39. #39 Todd W.
    http://www.harpocratesspeaks.com
    May 27, 2015

    @Narad

    Personally, I don’t think it really has a shot except, again, at the intrastate level.

    Agreed. Keeping it in-state skirts some Federal regulations that would otherwise apply. But then, they don’t need RTT for that.

  40. #40 Mary M
    May 27, 2015

    This also reminds me of the drama around the experimental Ebola meds. It would have been great to get them out in quantities faster–but you can’t expect small biotechs to have the capacity to do QCed drug production on a large scale suddenly. You just can’t–they don’t have the capacity, and you can’t pretend that somehow they suddenly will.

    This is what irks me about the Pharma haters. They have no grasp of drug development, nor of drug production and distribution. What do they think–just because Derpcola can sell you snake-oil by the buckets that some small biotech can? Or do they really think academic labs would have the ability?

    There are a lot of non-trivial steps in the process of getting legit compounds to people. Even if they haven’t passed all the safety studies.

  41. #41 JustaTech
    May 27, 2015

    Mary M: Exactly! It’s like those chain e-mails about the ‘ingredient’ costs of common drugs. “Cholesterol-be-gone costs 3 cents to make, why are you playing 75 cents?!”

    They never take any kind of QC into account.

    I often give the QC folks at my work crap about being incredibly conservative about everything (related to work), but at the same time I know it’s their job to be cautious.

  42. #42 Kiiri
    May 27, 2015

    Mary M and JustaTech – Yeah, it is interesting to watch people who hate drugs with a blind purple passion start screaming for drugs/vaccines when a new (or scary) pathogen pops up. Then they just can’t figure out why we can’t crank them out by the barrel full. Plus if they aren’t tested even though they will be screaming for them anyway (see Ebola scare) they will scream even louder if it turns out the drug was actually harmful. Oh why didn’t you say anything about it not being tested! It’s a lose lose scenario all around. Having gone through H1N1 in PH I know. We reacted aggressively and tried mitigation with the tools at our disposal early on (while everyone was screaming we do SOMETHING). Then when it turned out not to be as lethal as feared (though if you run the numbers a lot of people did die and in groups who shouldn’t have but not the kind of numbers that freak out the public) it was why did you do all this stuff! Overreaction! Too much money spent! You can’t win.

  43. #43 JustaTech
    May 27, 2015

    Kiiri: Yup. Sadly, while utilitarianism is great for getting things done, it does nothing for your PR. (That’s this week’s topic in my MPH ethics class.)

  44. #44 L Clark
    San Diego
    May 29, 2015

    LIMITATIONS OF RIGHT TO TRY LAWS: If a drug is already on the market for another use, the drug maker is more likely to have the financial resources to offer it for “off-label” expanded use (compassionate use). However, if the therapy is completely new, the drug produces no revenue stream and the company needs FDA approval to sell it and recover R&D costs. Insurance only pays for treatments that are FDA approved so patients would still have to come up with tens of thousands for medical staff, hospital space, lab work, monitoring, etc. even if they can get the drug. States are leading national discourse with initiatives like medical marijuana and gay marriage and forcing the federal government to respond. The Right to Try laws that are passing handily in many states could have the same impact. That is why we support them even though they are not a panacea. They are an important step in the process to change the status quo at the FDA. It’s a matter of life and death for all those with a deadly disease.

    • #45 Orac
      May 29, 2015

      Thank you for admitting that right-to-try laws are a sham, but we knew that already.

      In all empathy and sympathy for patients with terminal illnesses, hooking up with the Goldwater Institute and using its template were not the best ideas because they really don’t know who they’re in bed with. Contrary to its airy pronouncements, the goal of the Goldwater Institute is not primarily to improve access to experimental therapeutics. That’s why it doesn’t really appear to care that much that right-to-try laws are a sham and will not accomplish what they claim. The Goldwater Institute is playing a long game whose end goal is ideology-based, the idea being to drastically weaken or eliminate the FDA to let the magic of the “free market” fix everything, providing (once the FDA is safely out of the way) miracle drugs to patients. Patients are merely pawns being manipulated with the false hope of right-to-try. That’s why the Goldwater Institute can’t tell patients that it knows right-to-try laws won’t do anything to provide better access to experimental therapeutics that can’t already be accomplished under the current system; it needs them to pressure legislators, who also vote for these bills because they don’t understand that they don’t do what they claim to do either.

      Oh, wait. These laws actually do do one thing. They strip protections from patients pursuing investigational agents outside a clinical trial and tell the insurance companies that they don’t have to pay for the investigational agent (which most don’t now anyway) but go beyond that and say that insurance companies don’t have to pay for medical care for any complications that result from the use of an experimental therapeutic under right-to-try.

      Overall, they’re very, very bad laws, throwing patients with terminal illnesses under the bus in the name of the long game of weakening the FDA.

      You know what would really improve the access of patients to experimental therapeutics? Fully fund the FDA. The reason for a lot of the delays in drug approval as well as issues with compassionate use programs is that the FDA is underfunded and understaffed. The pay sucks (as in many other government jobs) so that the FDA has trouble retaining talented and knowledgeable people, who not infrequently leave take jobs in industry. But that would not fit in the the Goldwater Institute’s narrative that there are all these miracle cures out there that would get to the people rapidly if only the evil FDA would get out of the way.

  45. #46 Joseph Hertzlinger
    Planet Earth (for now)
    May 31, 2015

    Should there be a right to try Lyme disease vaccine?

  46. #47 Chris
    May 31, 2015

    You’ll have to ask the people who brought on the lawsuits that caused the company to stop making it.

  47. #48 name
    June 6, 2015

    Really, court has ruled freedom of speech trumps regulatory rhetoric as end run around truth in advertising requirements, so greedy corporations can say anything, make false claims, and generally lie about their product, under the protection of freedom of speech. Constitutional rights and protections were written to apply to citizens, people, not to corporations which are business entities for profit. However they typically have more money to support politicians hence the multitude of problems arising from political support of special corporate interests. Then of course, the sort of people desperate for some miraculous untested cure for terminal illness who sue for millions of dollars when it doesn’t work, and the scumbag lawyers they find to represent them. Nevermind the profitable incentive of drug companies and technology to genetically create and modify diseases and germs and better ways to spread them in order to expand markets for their products (on and off label use).

  48. #49 Johnny
    127.0.0.1
    June 6, 2015

    Nevermind the profitable incentive of drug companies and technology to genetically create and modify diseases and germs and better ways to spread them in order to expand markets for their products

    Ya’ see, at this point is where you went from quasi-reasonable, if somewhat exaggerated, to full conspiracy loon. You got any proof anybody is trying to “genetically create and modify diseases and germs” to make money from causing sickness?

  49. #50 Narad
    June 6, 2015

    Really, court has ruled freedom of speech trumps regulatory rhetoric as end run around truth in advertising requirements, so greedy corporations can say anything, make false claims, and generally lie about their product, under the protection of freedom of speech.

    This is the part where one traditionally points to actual case law.

  50. #51 herr doktor bimler
    June 6, 2015

    so greedy corporations can say anything, make false claims, and generally lie about their product, under the protection of freedom of speech.

    To the same extent as greedy non-corporate individual scammers and grifters with websites.

  51. […] force potentially for ill, as I’ve documented in my posts about the understandable but misguided “right-to-try” movement. After all, what politician can say no to a constituency representing desperately ill people who […]

New comments have been temporarily disabled. Please check back soon.