FDA denies drugs to terminally ill – Part IV

In a follow-up to my previous discussion (part I, part II, part III), regarding the August 6th court decision which said that terminally ill patients do NOT have the right to access experimental treatments, I was able to attend a policy seminar at the Cato Institute in which panelists Michael Cannon (of Cato), Scott Ballenger (lawyer for the Abigail Alliance who argued the case for the terminally ill), and Ezekiel Emmanuel (bioethicist at NIH who agreed with the court decision) battled it out in front of a decent sized crowd. Forgive me for my tardiness in posting about this (…the seminar was 2 weeks ago) – life has been a bit hectic lately. Here’s a summary of each panelists’ main points, and the impression they left:

Scott Ballenger -He was very articulate and came across as the “nice guy” in the debate. Ballenger argued that terminally ill patients should be able to take a risk (which I agree with), and pointed out that they were fighting for access to drugs that were already in the clinical trial phase – specifically phase 2 and 3 – meaning that since we already let others access drugs at that point (people who qualify for a clinical trial) it should then be considered ethical to let the terminally ill also access these meds.

He then spent a good deal of time discussing the “rational basis test” versus “strict scrutiny”, which if I’m being honest, was a bit more lawyerly and over my head. His point with this (if I’m getting this correct) was that the Abigail case should be strict scrutiny because it involved the fundamental right of life/death. He pointed out a few cases where courts have upheld decisions supporting patient autonomy, such as end of life issues in which a person has the right to die – that is, they can choose not to take food or water, and it may not be forced upon them by medical staff or family. Ballenger also mentioned the right to fight regardless of the means and end, giving rape as an example. A rape victim can fight with whatever means necessary, regardless of whether those means will have any effect (so even if will make no difference in the brutality of the attack, they can still fight). I found this analogy compelling, because his argument is that terminally ill patients should be able to fight with whatever means necessary, regardless of whether the outcome will be positive or negative. Finally, he pointed out that the FDA allows “off-label” prescribing of drugs by doctors, despite the lack of approval (and sometimes hard evidence) to support this use – as an example, prescribing aspirin to ward off heart-attacks is considered an off-label, yet widely accepted, practice. This is useful to consider when we understand that the reason many of these terminally ill patients don’t have access to experimental drugs is because they fail to meet the criteria for enrollment into the clinical trials. A trial may be testing the drug for breast cancer, and you have head and neck cancer and thus don’t qualify. However, maybe your doctor knows the biochemical mechanism by which the drug works and thinks it might have even a tiny chance of making an impact on your cancer – should he be allowed to get that trial drug to you in an off-label fashion? I would argue that he should, and I think Ballenger would agree.

Ezekiel Emmanuel – I disagreed with him on almost every point, didn’t find his examples strongly supported his arguments, and in general he came off as a bitter and angry man. Allow me to explain – the first thing he does when he gets up to speak is complain that up on stage it is 2 against 1 (Cannon and Ballenger vs. Emmanuel), and that despite the fact that he is arguing for the opposite position he is only given 1/3 of the time to speak. Ballenger talked for a long time, so I’ll give Emmanuel a slight break, but Cannon barely got a chance to speak, so I think Emmanuel did in fact get almost half of the time to make his point. Me thinks he doth protest too much, and that really set the tone in my opinion (ah…the importance of first impressions). First he pointed out that the FDA certifies drugs based on their safety and efficacy, and said that the Abigail Alliance doesn’t care whether the drugs are effective or not. This seemed to amaze him (actually, almost every quasi libertarian viewpoint seemed so astonishing to him). Personally, I don’t think the FDA should restrict drugs based on efficacy. Why should they decide what’s considered effective? To a terminally ill patient, a few extra weeks of life might be effective, but if the government disagrees they’re not getting the drug. Moving on…Emmanuel thought that the Abigail Alliance asking for drug access after Phase 1 trials makes it less a principled argument and more a regulatory issue. I kind of agree with this, but I think the AA had to be realistic about what they could accomplish.

Emmanuel also argued that just because we have a right doesn’t mean we can exercise it by any means. Well…yeah…but I think a lot of rational people say that a fundamental right can be exercised by any means so long as it does not infringe upon the rights of another. I don’t think allowing terminally ill to fight for their life infringes upon the rights of others, but Emmanuel thinks that it is not in the best interests of cancer patients and society. He argued that by extending access to experimental drugs to patients not enrolled in clinical trials, you make it more difficult for researchers to meet their enrollment quota, and may deter patients from seeking drugs that are proven to be effective. His paternalistic view was that “sometimes your job as a doctor is to say ‘no’ to someone”…and I guess to him it doesn’t matter whether that someone is addict who needs an oxycontin prescription versus a dying patient who wants a chance to fight. This is where some of his examples didn’t strongly support the claims in my opinion. For instance, he gave an example of a bone marrow transplant study for patients with metastatic breast cancer that took place in the 1980’s. The randomized-controlled study took 4 years to get 1000 people enrolled, while 40,000 got it off trial. The study later showed that the drug wasn’t effective. Call me cynical, but if I’m dying of metastatic breast cancer, I don’t give a damn how long it takes a researcher to meet their quota. They still got people enrolled! And they had metastatic cancer for crying out loud. They were going to die without an intervention – would you enroll in a clinical trial and risk being given a placebo? I wouldn’t. His arguments would hold more water if researchers couldn’t get people enrolled because of access outside of the trial, but in each example he gave that wasn’t the case. His argument was that it took too long (usually 3-4 years) to get people enrolled. Boo hoo. Has he forgotten that these are people with families, and not statistics? (did I mention he left a bad impression on me?)

Michael Cannon – He didn’t get much time to talk, but still made a few good points. He said that “all laws must be both necessary and proper” and that this court decision fails on both counts. It robs patients of their right to self-determination and dignity at best, and at worst it prevents their lives from being extended. Cannon pointed out research he had done on economists and the FDA, and found that most/all economists that have written about the issue came to the conclusion that the FDA requires too much testing and commits more type II than type I errors – that is, more people are harmed by the failure of the FDA to approve useful drugs than by their approval of harmful drugs.

Follow-up points included discussions on giving patients incentives to enroll in clinical trials. If you’re going to risk being given a placebo, than you should be compensated for that risk. Both Cannon and Emmanuel agreed on this point, but Emmanuel thought there was no data to support that it increases enrollment. In my opinion, all that says is that they aren’t paying enough for the risk to take the drug versus the risk of taking a placebo. (it’s basic economics, no?) Both also agreed that the FDA has a really tough job, and that there is no magic moment at which a drug is definitively safe and effective. I think the difference is that Cannon doesn’t think the FDA will ever be able to do the best job, so the decisions should rest with patients (a libertarian utopia), while Emmanuel probably thinks that the FDA can be constantly tweaked and regulations reworked until a happy point is found (his utopia). In case it hasn’t been clear up to this point, I most definitely agree with Cannon and not Emmanuel. I don’t think the FDA can ever be sufficiently tweaked. As long as they are the sole group that determines whether you or I have access to drugs, they will never be able to meet our individual needs. I think it’s important that drugs be safe so people aren’t unnecessarily harmed, and I think it’s important that drugs be effective so people aren’t taken advantage of, but I don’t think the decision of safety/efficacy can be properly done through an agency like the FDA.

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7 Comments

  1. October 28, 2007 at 8:00 am

    The opponents of the Abigail Alliance need to get their information correct and they need to talk with the Abigail Alliance to learn what the Abigail Alliance has been saying, what is in the ACCESS Act, and what is going on in the real world. This is fairly easy to sum up.
    First, every drug for cancer and other serious life-threatening illness that the Abigail Alliance has pushed for earlier access to in our six year history is now approved by the FDA. The number is now 16 drugs. With earlier access tens of thousands of lives could have been save or extended.
    Our opponent’s clinical trial enrollment argument is incorrect. As the Abigail Alliance has been saying for a long time and as written in the ACCESS Act, patients would have to first try and enroll in a clinical trial, before being able to qualify for earlier access. As the Critical Path initiative has been putting forth for years and the FDA is so slow in moving forward on, the FDA needs to use more modern scientific tools and more modern statistical tools. This would further eliminate the need for senseless placebos.
    For over six years the Abigail Alliance has been hearing from patients who are in consultation with their doctors and hearing from doctors seeking access to promising investigational drugs, because these patients could not get into clinical trials. They have not been seeking just any drug. They have been seeking the very drugs we have been pushing for earlier access to, because they too were seeing the positive data.
    Frank Burroughs
    President, Abigail Alliance for Better Access to Developmental Drugs

  2. Lily said,

    October 31, 2007 at 4:08 pm

    Thanks for the comments! I agree that the opponents generally have little idea of what they’re arguing against – they probably haven’t read the ACCESS Act and instead assume it’s pushing for major deregulation of the FDA rather than trying to get access to a few drugs that have shown promise. It’s unfortunate, when you consider that people’s lives are at risk as we deny them the opportunity to fight their disease. I think the Abigail Alliance is doing great work, though, and the message is spreading, so perhaps in a few years it will be a very different story (at least this is my hope).

  3. donna weetch said,

    May 10, 2008 at 3:31 am

    my dad is termialy ill were right on hrs maybe of loosing him,he has suffered really badly and we have had to watch him suffering,he begged everyone to help him kill himself but alas because of the law we are unable to help him and this has killed the family,he didnt want to be in nappies and he didnt want to have a cathata in and he wanted to be able to have the choice….easter my dad was so bad he was in alot of pain for three days and three nights i called the doctor were he came out and said to me he couldnt give him anything for the pain so i replyed dogs cant talk put a vet would put them down if they think its in pain, i could of quite easy strangled that doctor for leaving dad that way. in end they put dad on morphine patches now on a driver of morphine…why is our goverment so crual to patients and there family….i think termialy ill people should have that right its not humain to do this, do they know what they go though NOOOOOOOOOOO

  4. donna weetch said,

    May 10, 2008 at 6:07 pm

    my dad died to day at 3.58 it was vey sad but a relief to know his not suffering any more

  5. Lily said,

    May 25, 2008 at 10:56 pm

    Donna,
    I’m so sorry for the loss of your father – particularly that he had to suffer as much as he did towards the end. I agree that terminally ill patients should have the right to die as they see fit. Maybe if more people could hear your story about your dad they might think differently about the situation. Thank you for sharing.

  6. donna weetch said,

    July 4, 2008 at 3:42 am

    thankyou lily i wish i knew were to start really i do,i know it wont ever bring my dad home to me but if i could do something to fight the rights for terminaly ill people ,then maybe i would feel there would be a reason for me to go on living my dad was my whole world,im not to well my self and after having three heart attacks and living with a mussle disorder i feel preety helpless most of the time i need to do something any ideas from anyone would be grateful,thankyou again for your kind words love donna x

  7. January 11, 2010 at 5:38 am

    Super-Duper site! I am loving it!! Will come back again – taking your feeds too now, Thanks.

    I’m Out! 🙂


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