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Welcome to Right to Try

Update: Brainstorm has now abandoned their Right-to-Try approach. Who’s next?

That didn’t take long. That didn’t take long at all. The federal “Right to Try” bill was just signed the other week, and we already have a company that’s willing – no, eager – to try it out. I will now cruelly caricature some of the the bill’s advocates for a moment. They’re imagining cures, live-saving medications, finally freed from the sticky masses of red tape and lethal inertia that is keeping them from desperate patients. Desperate patients who are free actors, informed citizens and consumers, and certainly capable of making medical decisions on their own behalf and spending their money and time exactly as they see fit to spend it.

Actually, that may not be as much of a caricature as I thought, especially the last part. And I would be lying if I said that I didn’t have some sympathy with the people-can-make-their-own-decisions argument. On that ever-present other hand, though, it’s one thing to say that patients should be able to choose among a wide variety of therapies, some of them unproven, with a knowledge of the risks that they are taking. Objections can be raised (for example, that’s it’s very difficult, if not impossible, to be fully aware of those risks with drugs that are still in development). But even stipulating the above, it’s quite another thing to not just open up patients to the whole pool of possible treatments, but to outright encourage people to pour mud and slop into it in hopes that some patients will choose the slop. Which is what I fear we may have done.

I say this because a small company (Brainstorm Cell Therapeutics) says that they would like to make their investigational cell therapy for ALS available under Right to Try provisions. According to that Bloomberg piece, they’re looking at charging in the low hundreds of thousands of dollars (which, to be sure, is probably what their treatment would go for were it ever to actually get regulatory approval and reach the market). No insurance company will pay for this, as you would imagine – they’re only interested in paying for things that have been shown to work. So those informed citizens and consumers that I mentioned above are now free to give these folks six figures for a treatment whose statistical evidence for efficacy is not at all impressive and whose risks are still mostly being discovered. How many will? Bloomberg:

If it decides to proceed, Brainstorm — a company with no drug on the market yet and no revenue — would introduce a profit motive into an effort many expected to be altruistic, adding more controversy to an already contentious debate. Small drugmakers where much of the innovation in medicine originates can’t afford to provide their compounds for free, and terminally ill patients with no other options may be eager to pay for access. There would be little protection for patients already grappling with a tumultuous time in their lives, adding financial risk to the known medical gamble.

Who expected altruism? Not me. In fact, I expect worse than this. Brainstorm, at least, appears to be a company that is actually trying to develop a therapy. They’re not getting very far, but they’ve generated some data by their own efforts. I’m sure that they hoped for better numbers, but that’s drug development, and it’s especially drug development with as unexplored a technique as neuronal cell therapy against as tough a disease as ALS. My guess is that they’re having trouble raising more money by conventional means – i.e., among competent investors who have reviewed their slide deck – so they’re going this route instead, for both money and publicity. This won’t help their clinical efforts at all, honestly – my guess is that many of the patients showing up are well outside the cutoffs for inclusion in a trial, and indeed, the Right to Try law specifies that they have to be.

Can’t the prospective customers be as competent as those investors? In theory, yes. But in practice, said investors spend all their time looking at biomedical opportunities and evaluating them, and have (one hopes) achieved a level of expertise that will take some time and effort to recapitulate. It can be done, though. But the other big factor is that these investors have only their money in the game, not their health and their lives. It is very difficult to make rational homo economicus decisions when you’re evaluating something that could – might – maybe – do something about the disease that is visibly and inexorably killing you or your close relative. This is the factor that, more than anything else, makes buying health care different from any other purchase that we make.

Running behind it, though, is another: the fact that health care varies far more than most other goods that we have available. Want a new car? Some of them are more expensive than others, have different amenities and options, or can do slightly different things. But they’re all new cars: you can buy one in the reasonable assurance that it will not start shedding important parts half a block down the lot, or that (in spite of your best efforts) that it will simply refuse, for reasons unknown, to take you from point A to point B but will dump you instead at point C while you beat your hands helplessly against the steering wheel. Cars don’t do that.

But drugs do. And investigational drugs do that even more than most. That’s what the investigation is for, to figure out if they (1) can take you to point B at all and (2) to see how many times they do other stuff to you along the way. We call those “efficacy” and “safety” in the biz, and we spend huge piles of our own money trying to establish those two. But hey, you can bypass that and take therapies for which neither of those have quite been proven, and Brainstorm will charge you hundreds of thousands of dollars for the privilege. Let’s see who steps right up.

And let’s see if my nastier, more cynical Derek-Against-Humanity prediction comes true: that the next wave will not be just people who have seized on this plan opportunistically, but who have targeted it right from the start. Who will pick out terrible diseases for which there are no therapies at all, the better to insure a supply of completely desperate patients and families, and provide them with utterly useless therapies at stiff, stiff prices. Utterly harmless therapies, though – don’t want to get sued while you’re raking in the bucks. I’m talking polysaccharide therapy for pancreatic cancer, antioxidant phytonutrients for Alzheimer’s: in other words, corn starch and grape juice. What the hell. The customers are going to die, anyway. Why should they die with their money as well?

48 comments on “Welcome to Right to Try”

  1. Rche54 says:

    This characterization of NurOwn and Brainstorm is a bit disingenuous. It is the only current phase III ALS trial, its advancement to phase III was advocated for by Merit Cudkowicz (see the Stat article on this subject, linked in name), one of the leading ALS neurologists in the world, and the design of its phase III trial attempts to build on the benefits that were seen in the weeks after dosing in PhII trials by implementing repeated doses. This transient benefit is in line with the known short lifespan of transplanted mesenchymal stem cells in some cases. Also, since when is a lack of significant efficacy in Phase II a sign that a treatment is doomed to failure? BrainStorm has the funding to complete their phase III trial in progress and ultimately their fate will be decided by those results. I think this is one of the better possible applications of the RTT mechanism; however, I am also fearful of applications like the hypothetical scenarios you outlined.

    1. Thoryke says:

      Well, given the number of times _success_ in Phase II fails to lead to success in Phase III, I don’t usually hold out a lot of hope for the ‘meh’ candidates from Phase II…

      1. Rche54 says:

        “Meh” is all a matter of perspective. The recently approved ALS drug Radicava failed a phase II trial, before identification of the appropriate patient population (<10%) and subsequent successes in phase II and III. Should this drug have been available for patients not eligible for trials prior to approval? This is important as the length of the phase III trial for this drug was longer than the average life expectancy for ALS patients.
        If your criterion for use in "right to try" or compassionate use is demonstration of significant efficacy then these programs cease to have any purpose. If it's showing some signal for efficacy in appropriately controlled and analyzed phase II trials then NurOwn is a perfectly valid candidate for this pathway.

        1. b says:

          Meaning that >90% would’ve had to shell out huge amounts of money for something that didn’t work (for them)? I think that’s where the objections lie.

          1. Rche54 says:

            The 90% of ALS patients for whom Radicava is not indicated are still free to shell out $$ from out of pocket for an approved but probably ineffective therapy. Many patients do this. I don’t think many would argue that patients should be prevented from this.
            At what stage in drug development does this transaction become unethical?

          2. b says:

            I assume that those patients are well-informed by their doctors that they are not a candidate for the treatment and choose to do so anyway, and I’m perfectly fine with that. I would say that makes a huge difference in the ethical nature of the transaction. The treatment has at least been proven to be safe and efficacious for somebody with a similar condition to yours, just not you, and you know it’s very likely a waste of money going in.

  2. Brett says:

    Not cynical enough. IIRC doesn’t the Right-to-Try bill give the companies offering the treatments a shield against liability?

      1. Will the courts really hold that a commercial supplier is not liable for adverse events and/or non-efficacy?
        We’ll see.

  3. Charlie Kilian says:

    And of course, when the cynical scenario inevitably comes to pass, there will be a lot of people whose takeaway lesson will be, “Scientists don’t even know anything and are just a bunch of scammers who want your money.”

    I fear what happens after that.

    1. anoni says:

      This.

      We have drugs that fail in trials, which is terrible, but what trials were designed to catch. We have drugs fail in a Right-to-Try setting it becomes even more about the profit motive and can only hurt pharma’s already terrible public perception problem.

    2. zero says:

      It looks a lot like “Politicians are all selfish liars anyway, so I may as well vote for the one that looks/sounds/prays/acts like me” and “Journalists are all paid spin agents anyway, so I may as well get my news from Facebook, angry blogs and the pretty ladies on f*x”.

      Erosion of public trust in science (and even trust in a baseline reality with provable facts) is doing enormous damage today. Right to try is destined to make it worse.

  4. as says:

    I work in the rare disease space (not ALS specifically) and interestingly enough, many of the patient advocacy foundations/organizations are against the Right to Try legislation, believing that it will do more harm than good.

  5. Alan Miller says:

    Fact Check: The Brainstorm Phase 3 Double -Blind Placebo Controlled ALS trial is well funded:

    The California Institute of Regenerative Medicine provided a 16 million dollar grant.

    https://www.oaoa.com/news/business/article_f45236a8-78f0-5bc1-935a-b3f9319fce25.html

    https://alsnewstoday.com/2017/07/24/brainstorm-wins-16-million-dollar-california-grant-for-trial-of-its-als-stem-cell-therapy-nurown/

    1. Me says:

      Fact-check: 16 million dollars for a phase III cell therapy trial in a disease that requires such long trials does not constitute ‘well-funded’.

  6. RogerBW says:

    When this was first mooted, I proposed a simple modification:

    A company can make its un-approved drugs as available as it likes… free.

    If it doesn’t want to, fair enough.

    Instant scammer filter, and a company that is really confident of approval and wants to whip up later demand can still treat it as a marketing exercise.

    1. me says:

      Come on. We all know this is the right to SELL act.

    2. loupgarous says:

      Agreed. In fact, not every clinical trial outside RTT gives therapy away. I was in the clinical trials for PRRT (lutetium-177 octreotate for neuroendocrine cancer) and Gadolinium-68 octreotide PET/CT for the same indication, and Medicare/my Medigap insurance was billed for a large number of chargeables by the study center. I didn’t mind because therapy resulted in an almost three-year respite from disease progression, and now both PRRT and Ga-68 octreotate imaging are FDA-approved and hopefully will be available much closer to my home.

      Would I have raised (say) $280,000 for a four-treatment course of PRRT if success rates had been as equivocal as Brainstorm’s ALS therapy in other patients?

      Iin the hypothetical case where PRRT was where Brainstorm’s ALS therapy is in terms of outcomes, I might pay for it myself if I could provide for my wife after my death as well. But I can’t afford to pay for it out-of-pocket, and won’t ask anyone else to. Right to Try wouldn’t be tempting under those circumstances, even if I could pay for it myself.

      Let’s say that PRRT only worked as well as Brainstorm’s ALS therapy. 4/36 patients getting the study treatment responding to therapy long-term, if I’m reading Adam Feuerstein’s article correctly, isn’t as good as previous standard of care for my cancer. Even if the numbers were (again, hypothetically) much better than one out of nine at >=12 weeks but poor enough that PRRT was only available under Right to Try, I hope I wouldn’t be panicked enough to consider it.

      Patients much younger than me would (and probably should) look at it differently.

    3. Design Monkey says:

      Not necessarily a good filter. One can offer fake drug for free, but ask for donations and f*ck off the ones, who do not donate generously enough. Works especially well, if the fake drug itself costs about nothing, and is accompanied by making Shrek kitty eyes and saying ” we would like to give it for free, but we haveee terrible expenses making it, would you pleeez consider a donaaaatiooon….”

      1. loupgarous says:

        Federal Right to Try act limits compensation of manufacturers to direct costs of making the investigational drug available. I’m not sure how FDA’s regulations determine that, but it seems to indicate that the manufacturer would have to account for those costs to FDA. But the Morgan Lewis law firm lists exactly how that would work among the unanswered questions in Federal Right to Try in a report which seems aimed at would-be RTT drug providers (manufacturers and health care facilities).

        1. Design Monkey says:

          Doesn’t change donations squeezing methods not one bit.

  7. cynical1 says:

    Okay, I wonder if a new career will be spawned out of this debacle. Who better would be able to evaluate the likelihood of therapeutic benefit in the absence of data other than a drug discovery scientist? My point is that people that are in the profession of many of the readers of this blog could make some good coin having patients pay them to evaluate whether these Right to Try medicines are snake oil or just might be of benefit. In my humble opinion, we usually don’t have as much problem picking out the drugs that are doomed from the start. (Derek even makes money shorting stock doing that.) But to someone without that knowledge, it’s a lot more difficult. (Side note – for all his genius, Stephen Hawking didn’t switch his career to drug discovery after he was diagnosed with ALS, either. And he lived a very long time with the disease.)

    What are the ethical implications of charging for a service to evaluate these potential miracle cures? How is it different than what many physicians do?

    1. loupgarous says:

      That’s really cynical.

      Hopefully, we’d see the various National Institutes of Health report on efficacy for Right to Try drugs (though this administration might not allocate the money). Is that disinterested enough for you?

      If Big Journalism had been doing its job on the Right to Try story (instead of shouting “Look! Moose and Squirrel over there, next to Boris Badenov!” for a year and a half), the Trump administration would be pressured enough on the ethical implications of Right to Try to include funding for the NIH to evaluate RTT therapies and give patients useful information on their cost-benefit ratio. The way to really hurt an administration is to concentrate on where they’re really screwing up, and RTT’s a prime example.

      1. cynical1 says:

        Actually, since there isn’t data, how would the NIH evaluate them other than the strength of the hypothesis on which they are based. You know, like a drug for Alzheimers targeting beta amyloid.

        “The way to really hurt an administration is to concentrate on where they’re really screwing up, and RTT’s a prime example.” ???? Okay, I just concentrated on all the good points of our current administration. The egg timer hasn’t gone off yet.

        1. loupgarous says:

          Sunlight’s the best disinfectant, but the press coverage on Right to Try basically gave the Trump administration a pass on very troubling implications of the law. Even a law firm that seems to be trying to hustle business from RTT drug manufacturers and healthcare facilities listed how the Right to Try act strips protection from patients:

          “Eligible investigational drugs that are provided to eligible patients are exempt from requirements for FDA approval, INDs and most of the corresponding IND regulations, certain labeling requirements including adequate directions for use, and FDA’s regulations on informed consent and institutional review board (IRB) approval.”

          I don’t recall reading or viewing any of that in coverage of Right to Try.

        2. loupgarous says:

          cynical1: “Actually, since there isn’t data, how would the NIH evaluate them other than the strength of the hypothesis on which they are based. You know, like a drug for Alzheimers targeting beta amyloid.”

          Good example. Strength of the hypothesis on which they’re based would be a prime datum on which to base those evaluations. And in the case of the β-amyloid hypothesis, they’re well-situated to review the literature, including results of clinical trials. But Federal RTT requires at least a Phase I trial, so some toxicity data have to exist (although this is the weakest link in the Right to Try law, because of the number of severe adverse events which went unnoticed until drugs were approved and marketed, while Right to Try drug patients are, essentially, Phase II toxicity volunteers whose experiences won’t be required to be reported in an NDA).
          The NIH have, in many cases, people who are qualified to evaluate investigators’ reasoning in presenting a new investigational drug for a given indication. If they don’t, they have telephones and Internet accounts and can contact qualified people to evaluate that reasoning.

      2. BigSky says:

        “the Trump administration would be pressured enough on the ethical implications…..”
        Huh?

    2. HFM says:

      Actually, my company offers such a service as part of its health benefits. If you’re diagnosed with cancer, you can get your tumor sequenced for free, and then they have scientists that will advise you on your options. Including the options that are currently in clinical trials.

      Fortunately, I’ve never had the need for this service. But I think it’s legit.

      I think many of us have been the designated “person who speaks medical-ese” when there’s a serious health issue in the family. Simply having an advocate who understands what’s going on can be a blessing. And for something like cancer, where best practices are in flux and personalized therapy is very much in its infancy…heck, I’ve done cancer genomics myself, and I’d still sign up to have extra sets of eyes looking over my results.

  8. Anon says:

    “What the hell. The customers are going to die, anyway. Why should they die with their money as well?

    To be fair, what’s the difference vs most approved Pharma medicines? Besides maybe dying 3-6 months later than you would have anyway but still without any money left?

    1. Ian Malone says:

      At least that’s an informed decision you get to make.

    2. loupgarous says:

      The direct-to-patient TV ads for Opdivo put it just about that baldly – “more life… ” and in a flatter, more rapid voice saying “more months… “. And for a young-to-middle aged patient, those added months are important. But you’re right – one of the success stories for modern cancer therapy is even advertised as offering its patients months of added life.

    3. JIA says:

      Hi Anon,
      You may just be trolling, or feeling particularly cynical today, but I wanted to respond anyway.

      While not all new drugs provide large benefits, the trend over time is clear: patients with many serious or deadly diseases are living longer, better lives now due to advances in medical care, which includes (but is not exclusively due to) newly developed and approved drugs. Some of these offer far more than “3-6 months” of life.

      As just one example, please take a look at the data for survival rates in multiple myeloma (link in my handle). The 5 year survival rate has DOUBLED since 1975, due in part to a series of effective new drugs including lenalidomide, bortezomib, daratumumab, and others. These drugs extend lives by years, not months.

      There are plenty of reasons to be cynical about the pharma industry, but let’s not ignore the tremendous progress that is being made and the benefits that pharma has delivered to human health.

      1. zero says:

        3-6 months is an average. For people who don’t respond to treatment, the net gain is around zero. For people who do respond, the net gain is years and sometimes decades. The trick is deciding which treatment to give a particular patient and then monitoring to see if they should be switched to something else.

        Like you said, real outcomes are getting better and better.

  9. David E. Young, MD says:

    There are foundations that help patient’s pay for drugs. Here in the Palm Springs area, there is the Desert Cancer Foundation, an entity that provides money for those who can’t afford co-pays on expensive drugs. Rumor has it that they were once sued, demanding that they help pay for an expensive “right to try drug.” This can get ugly quite fast if foundations are required (through threat of lawsuit) to pay for “right to try” drugs.

    1. A private foundation can’t decide what it wants to do with its own money? What was the outcome of that suit? Would be a dangerous precedent indeed.

      1. Duncan Bayne says:

        That ship sailed a good many years ago. Consider that for starters, around 40% of your money is _taken_ from you to be spent however the takers choose. Of the remainder, consider how many rules and regulations there are around property, investment, savings, business, etc.

        1. zero says:

          So you’re one of those who expects the benefits of modern civilization but is offended by paying for it.

          Many of those rules and regulations protect you (and all of us) from abuse by people who are smarter, wealthier and/or more powerful than you are. You’re welcome.

          Roads, schools, police, fire, EMS, telecom, drinkable water, breathable air, a national electric grid, GPS, the internet, a democratic country with free markets that speak English (instead of a communist country speaking russian or chinese)… You’re welcome.

  10. Shalon Wood says:

    Who expected altruism? I think Gene Wilder said it best in Blazing Saddles: “You’ve got to remember that these are just simple farmers. These are people of the land. The common clay of the new West. You know… morons.”

    As horrible as it is to say, and I really can’t believe I’m saying because it _is_ horrible, I think that perhaps the best possible outcome would be for one of the first few RTT drugs to kill a large number of people who take it, causing a backlash against RTT and getting it repealed. Long term I think fewer people would die if that was the case.

    1. loupgarous says:

      Yep. It’s horrible, all right. You’re entertaining the thought that many patients who have no other choices but trying an investigational drug or accepting death from their illness ought to die – so that a law can be overturned. You’re calling them expendable based on your assumptions about the patients’ intelligence.

      Congratulations, you just joined the Gregor Mengele Übermenschen Verein.

  11. JonB says:

    I don’t share Derek’s faith in the wisdom and expertise of the FDA.

    They may have a very deep understanding of study design in terms of statistical issues but they have often have terrible clinical instincts regarding risk/benefit of therapeutics, substituting an aggregate population judgment of risk for the individual risk benefit. God help you if you submit an IND to a review section who isn’t up to speed with actual clinical practice…..Sometimes I have to wait 1 or 2 days to open a response letter because I know the contents are going to be completely divorced from clinical reality. It’s just so painful to read these things.

    The point of RTT is to develop an alternative credentialing system which will evolve through free market forces. Competition is a good thing. The FDA will get better and the RTT protocols will get better in response to market forces. Is it really necessary to “spend huge piles of money to establish safety and efficacy” or is there a genius like Bezos or Walton or Jobs who is not limited by years of torture in the Pharma-NIH-FDA complex who can invent a simpler and more flexible regulatory structure?

    Would be curious to have someone do a qualitative survey of practicing physicians and ask them about the quality of the information they get from the FDA. I think Derek would be surprised about how cynical the grunts on the line are regarding this regulatory apparatus he is defending vehemently

    .

    1. AVS-600 says:

      How is right-to-try going to help establish an indepedent credentialing company? The data from clinical trials exists independently of the small pool of people who will be able to afford the drug without help from their insurance, so any outside credentials would either be using the clinical trial data anyway (in which case RTT is irrelevant) or be incredibly statistically underpowered (in which case the FDA is going to be more trustworthy).

      1. loupgarous says:

        Expanded Access looks better and better, you’re right. The more you look into the fine print of Federal Right to Try, if the patient would have had any recourse against the manufacturer, or if unfavorable experience short of a clinical hold for severe AEs could be reported, the bill’s authors “corrected” that.

  12. Make America Red Again! says:

    We need universal health care to fund the right to try act. Increase taxes on the middle class to at least another 30% and the rich by about 50%. Property taxes should also be collected at the federal level in addition to the the state. Municipal income taxes in every county should be implemented. I want sales tax to up to 15% and severe regulations on pharma and wall Street. We need a socialistic revolution!

    1. Scott says:

      I really hope you’re being sarcastic.

      I’m stuck with nationalized health care (the Veteran’s Administration). Why stuck? Because no private-party health plan will cover anything that the VA will, and I took enough damage while in the service that the VA covers everything but dental.

      If you really do want nationalized health care, let’s get the VA funded to the point that people aren’t dying while waiting to see a specialist. Then we will have a clue how much nationalizing the entire US health care system will cost.

  13. MaGA says:

    Serious question does a “drug” have to be in clinical trials to count as right to try? What if I make a company and develop glucose as a cure for autism and its in preclinical development can I sell it?

    1. Make America Red Again says:

      Hey Retard, the drug needs to pass some kind of safety testing to even be considered an alternative option for right to try. Seriously, I have data showing that rotenone impairs cancer growth and mouse models – do you want to take some too? I figured not, you imbecile!

    2. Derek Lowe says:

      The text of the law says that its provisions only apply to drugs whose IND application has been granted.

  14. AQR says:

    Is it a requirement of the Right to Try law that the organization that provides the drug keep and publish data on the patient and outcome of the therapy? If not, there is little hope of learning anything about whether this law helps or harms peoples’ health.

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