Not So Funny Things Happened on the Way to a Diaphragm Pacer: When “Informed” is Not “Consent” and Related Adventures of an ALS Activist

Regular readers of this blog have read a lot about my adventures trying to get Biogen Idec, the makers of dexipramipexole (dex), to let me get a diaphragm pacer without being dropped out of the dex trial in which I’ve been participating for the last 15 months. When the company proved intransigent, I turned my efforts and readers’ considerable energies to the trying to get the FDA to “do the right thing.” This blog post updates those efforts, and, for me, brings to close, at least for now, this chapter of my ALS activism.

What the FDA Says Now: A Chance to Make Changes?

A number of people at the FDA actually read my blog, and I guess the title of the last one (FDA to ALS Patients: Fuck You), as well as the comments posted to it, got their attention. On September 6, I got the following message from the Deputy Director of the Division of Drug Information for FDA’s Center for Drug Evaluation and Research (CDER):

Dear Barbara,

The FDA would like to assure you that we are taking your concerns seriously.                  The issues that you have raised have been under discussion internally, and the                     Agency is considering your arguments about how to design and conduct trials in                  ALS that put the patient first.

Additionally, we would like to let you know that the Agency is in the process of                planning a public meeting that will address your general concerns as well as the                 more specific concerns you and others have raised, such as an independent                         patient ombudsman. Once a meeting date has been set, I would be happy to               provide you with the details.

             The passion for ALS treatment that you and the readers of your blog have is                    obvious from your posts and the comments you receive. Please know that                      patients’ perspectives are an integral part of the drug approval  process, and     we value and appreciate these comments.

              I understand that the scheduled date for your pacer implant surgery is quickly                   approaching. I want to extend our best wishes to you for a successful surgery.              Please email me afterwards to let me know how you are doing.                                       Best regards,

Catherine Y. Chew, PharmD

Deputy Director, Division of Drug Information

Center for Drug Evaluation and Research

Food and Drug Administration

While this doesn’t help in my current circumstances (see below: Treatment Delayed is Treatment Denied), the prospect of a public meeting at which people affected by ALS will have an opportunity to influence the future structure of ALS trials is heartening.

I hope everyone concerned about these issues — and everyone should be — will contact the FDA to be sure they get notice of when this meeting will take place. Normally, the FDA accepts written comments as well as in-person testimony, and I have asked if that will be the case with this meeting. Write to

Activism can work. Try it. You’ll see.

The IRB Should Be Called the CYA

In the earlier response from the FDA, which I described as “fuck you,” it was suggested that I approach the IRB (note: that stands for “Institutional Review Board,” not “Independent Review Board”) at CPMC, where the Forbes Norris clinic is based. I did so.

This is a covered ass.

The basic position of the IRB was that, since the research folks had followed all the informed consent procedures, they did not need to take action. The ethical issue of holding me — and others with ALS — hostage to irrational rules was not considered.

Interestingly, CPMC has a medical ethicist on staff, but that person said the ethical issue in this setting was for the IRB to address. Evidently, the IRB didn’t think so.

The response from the IRB, and my contact with other ALS folks about this topic, led me back to look at the informed consent I signed. There was not one word in it about the diaphragm pacer. The rules of the trial changed in the middle. I explained this to the IRB, and told them that, while I know I had signed one or two consent forms after the first one, no one had mentioned to me whether subsequent forms precluded a diaphragm pacer.

The IRB thought this might be problem, so they asked the ALS research team for documentation of what I supposedly knew. (Nothing like asking the folks who may have dropped the ball whether they did or not, eh?) After reviewing what the ALS research team provided, the IRB again turned me down. They acknowledged that I had never signed a consent form precluding the pacer, but since I had acknowledged receiving a email from Forbes Norris informing me of the pacer limitation, I had no basis to complain.

Before I get to my rant on “informed consent,” I should say this about my email communication with Forbes Norris about the diaphragm pacer. I did get an email from the research coordinator saying that a pacer would bounce me from the trial. In response, I told the coordinator that I would spare her my views of the ruling since she had already heard my rant about how irrational that was. Consent?

Informed” v. “Consent”

I deeply appreciate all the people who stood up some years ago to say that people in trials need to understand for what they are signing up. There was a time when people got no information. (A terrific book about one aspect of this is The Immortal Life of Henrietta Lacks by Rebecca Skloot.) Unfortunately, the noble effort to change that reality has turned into another sort of nightmare for patients.

Is there really such a thing anymore?

Informed consent documents for trials, if the Biogen forms are any indication, now run 20 to 30 pages long. While they included information about the trial and the risks and benefits, they also include things like the follow up schedule, the information sharing  rules, and a myriad of other things. The notion that being informed amounts to consenting to all the things these forms contain is folly.

And consider the patient’s option. S/he can sign the consent form as written, or not join the trial. There is no give and take in the process of “informed consent.” So is it really consent?

Add to these problems what happens when drug company sponsor like Biogen Idec  decides to change the trial rules after the trial has started. Patients are then “re-consented,” asked to sign a new altered consent form. Again, they can sign or be dropped from the trial. The drug company holds all the cards.

And, evidently, the company can play those cards without even getting a signed  consent document. An email stating the change in trial rules evidently binds the patients.

It is a misnomer to call this process “informed consent.” The law has name for it: unilateral contract.

Treatment Delayed is Treatment Denied — No Diaphragm  Pacer for Me

While all this effort to try to stay in the dex trial if I got a diaphragm pacer was going on, I was trying to get set up to get a pacer implanted.

Here are a few dates to consider as you read about this. The pacer was approved by the FDA in late September, 2011. On October 11, 2011, I let the clinic know I was interested in getting a pacer.

I had been in discussions with my neurologist at Forbes Norris since early April about getting the tests I need to establish my eligibility. I had respiratory tests at the clinic in February, 2012, which I passed. On April 30,  2012, after arguing with my neurologist about which other tests to get, I had a SNIFF test (a flouroscopic view of my diaphragm). I had  to argue with my neurologist to get that test.

I heard nothing about pacers from the clinic again until early June, 2012, when I was asked the clinic folks if I was willing to be the first person at Forbes Norris to get a pacer. I said yes.

I assumed — incorrectly — that things  would move along with scheduling the surgery.

The only hold up I heard about  was in June,  2012. MediCare approval had not yet been confirmed. For some reason, the clinic was unwilling to start scheduling the surgery until coverage was confirmed, even though it involved several doctors with busy schedules. MediCare coverage was obtained in late July, 2012. At that point, the earliest available date for surgery was September 12.

ALS, as the neurologists certainly know, is a progressive disease that effects respiratory capacity. Since there is  no effective treatment, the respiratory numbers go only in one direction: down. Yet the doctors at Forbes Norris did nothing that I know of to expedite getting me a diaphragm pacer while my respiratory numbers were good enough to be eligible.

By the time I got to the clinic on September 10 and had my respiration checked again,  my function had dropped below the eligible criteria. So, I cannot get the pacer.

In ALS, as in many diseases, treatment delayed is treatment denied. The people at Forbes Norris know this. I would love to see them act like it mattered.

© Barbara A, Brenner 2012

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18 Responses to Not So Funny Things Happened on the Way to a Diaphragm Pacer: When “Informed” is Not “Consent” and Related Adventures of an ALS Activist

  1. Liz Hendrickson says:

    Barbara: This is outrageous and heartbreaking. I’m so sorry to read this. My experience the past 10 years with other family and friends dealing with different medical conditions has been very similar and I”m grateful for all your effort in articulating these issues. Mainly, I’m shaking my head and crying with frustration at how we are treated with such disrespect by “professionals” and by our own government. Liz Hendrickson

  2. Donna Brorby says:

    Did you find that picture of a covered ass, or did you knit the cover onto the ass?

    Great articulation of the issues, as usual. You are a brilliant example of activism having an impact. But it’s frustrating that the impact isn’t more faster. db

  3. C says:

    My few cents on the “ignore and delay and it will go away” institutional strategy for dealing with ALS issues…

    I look forward to the public meeting. Thanks, Barbara.

  4. You had me at “fuck you.” (xoxox, W.)

  5. Ted Schrecker says:

    Barbara has it absolutely right with the reference to hostage-taking, but is too polite to follow the line of argument to its logical conclusion: ethics review processes that allow it, and the supposed ethical principles on which they rely, are nothing more than forms of racketeering if they permit the practices that Barbara describes. I am drifting toward that view of the research ethics enterprise as a whole.

  6. Elizabeth Plapinger says:

    [I am so upset. But as I am beginning to learn from Barbara and Susie, I can be both deeply sad and also angry. So, below are the beginnings of my comments to the FDA and to Forbes Norris. With love, liz]

    It is mind-boggling to me, naive as I may be, that the folks at the Forbes Norris clinic moved in this slow, linear way when triage and simultaneous action were required. Why don’t they know this? Why did they move in this way? These are your advocates? What is this about?

    As for the FDA, I will hope for change and will draft my comments. Certainly, it is time for this rigid vestige of unilateral control by the scientists to be discarded. In the exchange of emails over Barbara’s request, it seemed to me that the company and researchers simply didn’t want to have their tidy regime disrupted by reasonable and rather modest requests for individual accommodation, such as Barbara’s. Well, where life and death are at issue, the justification of administrative convenience is simply unacceptable. And it is completely unnecessary. As my many years in conflict resolution have taught me, we can trade on differences and address our competing positions with honesty and compassion. We are so very able, when we try, to accommodate competing interests. Indeed, the complexity of our world requires that we do so. This lopsided and old paradigm that privileges the researcher needs to disappear. It is unnecessary and toxic. We can do better.

  7. Robert Cronish says:

    I happened upon this blog quite by happenstance while searching for something else. I don’t know that much about ALS, and sincerely sympathize with Barbara and others who suffer with this or other degenerative illnesses. Yet, it seems to me that Barbara, and others who’ve commented are confusing treatment and research.

    Without knowing much about the research protocol design, I DO know that the purpose of research is to discover and identify meaningful therapeutics, and not necessarily to provide “treatment” to the research subject.

    An informed consent is a vital part of research, because the research subject needs to know what will happen, what their responsibilities are, and what the responsibilities of the people doing the research are. To complain that it’s too long seems somewhat antithetical. One of the primary purposes of informed consent is to make clear that what the subject (“research subjects” are different from “patients” and that’s why they are referred to that way) is taking part in RESEARCH. The there are parameters necessary to determine whether, and how effective an intervention is. Or isn’t.

    Again, without knowing the particulars, I suspect that the sponsor of the research needs to know how effective the drug is at, among other things, stemming the decline in respiratory function, or improving it. If another intervention – in this case – the pacer is going to have an effect on that measurement (and isn’t that why she wants it?), then whether the drug does or not won’t be possible to measure.

    Biomedical research is really done to help future patients. One HOPES that there will be direct benefit for research subjects. But they must endure the fact that parameters need to be adhered to know if the drug really works, and how well, for how long.

    Confounding treatments only make it impossible to determine. And if it looks like the drug is working because of the pacer, and FDA approves it, future patients won’t reap the benefit, because it was never the drug that caused the improvement.

    Future patients will curse the FDA just as vehemently for putting a useless drug on the market, giving them false hopes, and costing the healthcare system a hell of a lot of money.

    Understandably, patients with serious illnesses WANT treatment. But they want, and need treatments that work.

    I think it’s not that there is no such thing as “informed” consent. I think it’s that Barbara didn’t read and absorb the document, and understand that she was part of a research study, and exactly why it’s called “research.”

    It’s not nice, but it’s necessary to do the research, and to adhere strictly to the requirements of the protocol, so that all people in the study are treated the same way, and the results can be compared in a meaningful way. That’s the only way of getting worthwhile drugs to the market that people know what to expect from, are worth their trade-offs in safety, and hopefully worth their economic cost.


    • cathy speck says:

      Thank you Robert for your calm, methodical comments , suggestions and questions. I found your message to be clarifying, and enlightening.
      When I first read Barbara’s experience, i felt my adrenaline pumping and my chest puffing up, and my hands turned to fists ready to fight, not type. Well, punching a keyboard is not an effective way to communicate, so I calmed down and kept reading instead of replying in the heat of my fury.
      And then I saw your message., and it all made sense. I could see the issues from many perspectives, and i think you objectively nailed it: it is NOT treatment;it is RESEARCH. It’s not pretty, but it’s pretty simple.
      My family is being studied at Nortwestern Univ by the Siddiques. We are a SOD1 family with a rare mutation on eon 5 E133A. Maybe because I’ve lost so many family members I’m kind of a “tough gal”–no nonsense when it comes right down to it. We ahve to be our biggest advocates, and the means making sure we know the risks we are taking. And pretty much any time we’re part of research, the bottom line is yes, we’re lab animals and we don’t get to choose which wheel to roll in. There is no cure. That’s it. Play the game–know the rules. If we choose to help, we know things might not go well.
      This is after all, the most horrific and crulest disease. There is no cure. Yet.

  8. bbzinger says:


    Youi clearly don’t know me, and have not read other blog posts. Before speculating about what I read or didn’t or what I’m arguing for, I suggest you dig deeper into this blog.


    • cathy speck says:

      Barbara, I just now read your comment to Robert, so I bet your reply to my letter would be similar. I will take your advice and read more blog posts to get to know you and your position better. You are obviously intelligent, articulate and highly educated. Your writing style is excellent, and your points are well-presented. I’ll be back after I “prime myself.”

  9. c says:

    Am Academy of Neurology just tweeted this link for neurologists with ethical problems with patients
    I wonder if they ever considered that a patient might have an ethical problem with a neurologist.

  10. j says:

    Barabara, check with Dr. Raymond Onder’s team at Case Western University in Ohio -contact info in Synapse website. They are the first and major center to implant the pacer and may do patients who have lower respiratory function than the rest of the ceneters

  11. C says:

    And there is yet another opportunity to comment to the FDA regarding ALS… This time it’s a public comments period as they identify diseases that would provide them with valuable patient-centered input. ALS isn’t on their list, yet it fits their criteria (perhaps too well?). Please speak up during the public comments period on the value of ideas from people who are dealing with the ticking ALS clock, the lack of treatments, and a system that is stuck in the mud. Thanks.

    Here is more info —

  12. Shirley Schmelzle says:

    Outstanding work, Barbara. For those of us living in the paradigm of ‘Don’t look forward to a better day tomorrow, baby it’s down hill all the way’
    are angry and frustrated about delays and bureauocrats seemingly making progress.

    I posted my low opinion of the progress of the National ALS Registry, but was told, ‘Wait! Good things are ahead.’

    I was one of those startled to learn that the plug was pulled on the Ceftriaxone trial that I had been on for almost 2 years. I find it dispicable that we have to wait and wait to learn if we had the drug or placebo.

    We are a group bonded by one heinous villain and are not willing to wait around like sheep heading to slaughter although it sometimes feels like we are.

    Please take a look at http://www.sunflowertodefeatals and let’s all unite as one visible entity in this battle.

    Fuck ALS.

  13. We must join together and fight for our lives! The FDA should be on our side! We have patiently waited too long, there are drugs out there now that need to be tests on PALS now. We need to demand more from FDA, researcher and pharm.Show your support for the FDA putting ALS on priority list for the next 5 yrs, meeting Oct 25 in Maryland, come to meeting or show your support outside meeting! Speak now or,wait another 5 yrs!

  14. C says:

    More on the FDA public comments period regarding the patient-focused drug development disease areas —

    I believe that this is a serious opportunity to get people with ALS at the table for direct patient input into patient-focused drug development.

    ALSA issued some statements yesterday on their facebook wall that I interpret as meaning that they represent PALS with the FDA and that these patient-focused drug development disease groups are not important to PALS.

    I disagree. I think that we have an opportunity to make a cogent case for ALS to be one of the 20 disease areas that the FDA will work with in developing the patient-focus for drug development. Why would we not want people with ALS directly at the table?

    I find it ironic that ALSA is not encouraging patient input into a patient-focus initiative. Why not add some direct patient voices to what the FDA hears from ALSA? People with ALS aren’t easy cats to herd, but perhaps that’s exactly what the FDA needs to get out of its rut.

    My few cents. Thanks for your consideration and action.

  15. Als are just tossed to the side. As in barber as situation the forbes norris centering California waited to long contact barbera thereforee she lost the chance to get the pacer. Why did it take do long for them to contact her when she passed all the test and qualified for the pacer. This unacceptable and the FDA should look into this matter. This is inhumane . Pattients should not be treated this way . Al’s patients are treated this way all the time. Everything is a waiting game and then these patients get no treat mental. Al’s is a deadly disease, and there are treatments out there that need to get dipersed to the patients sooner. The FDA has to change the protocol of when the drugs need to be given to the patients. These pals neen to be treated now they are dying . Please , fda ,change the laws and give the pals a chance to prolong their life.this is do unfair. Let’s treat pals now!!!!

  16. C says:

    For your consideration…

    If anything, the two largest ALS organizations proved at the microphone that they don’t get the concept of patient-focused discussions with the FDA. Thanks for anyone who might submit comments to the FDA to get people with ALS seats at the patient-focused drug development table.

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