FDA to ALS Patients: Fuck You

If you like this blog, please pass it on to your friends. Let’s keep the conversation going.

I will spare my readers the long saga of my fight with Biogen Idec about the dex trial and my plans to get a diaphragm pacer. If you want all the background, you can read it in three installments: here, here, and here.

When the company refused to budge, I appealed to the FDA Ombudsman both for help with my situation and to assure that every large trial had an ombudsman to resolve disputes between trial participants and the drug company running the trial.

Great friend to drug companies everywhere.

The FDA has spoken. In short they turned me down flat on both requests — apparently without seriously considering my arguments or the letters that many of you sent on my behalf. Here’s what the FDA said.


Dear Barbara,

Thank you for writing to the Food and Drug Administration (FDA). This is in response to your email to Dr. Janet Woodcock, Dr. Russell Katz, and Ms. Virginia Behr concerning access to dexpramipexole. The issues you raised have been carefully considered, and I’ve been asked to explain our thinking to you.

We understand that Amyotrophic lateral sclerosis (ALS) is a very serious disease which deeply affects patients and their loved ones. We appreciate you sharing your concerns

No thanks to the FDA

about your possible lack of continued access to the study drug, dexpramipexole, if you begin using a diaphragm pacer before Biogen Idec’s EMPOWER trial ends in September.

FDA works with drug developers like Biogen Idec to ensure that patients volunteering to take part in studies are treated fairly and ethically. We understand your argument for being allowed to start use of a diaphragm pacer while staying in the dexpramipexole trial. We also understand Biogen Idec’s argument that making exceptions to the rules of the study would jeopardize the ability of the study to show if dexpramipexole is effective. We wish we had a way to resolve dilemmas like this, but we simply do not. We are acutely aware of the immediate need for effective treatments for ALS, but the only way we know how to learn if a drug is effective is to require sponsors to conduct studies with rules about the types of treatments patients receive, including, of course, rules that assign one group of patients to the experimental drug and a comparison group to placebo for some set period of time.  The rules for these studies must be consistent with established ethical and scientific principles, but, within these standards, the specific elements are generally chosen by the sponsors for reasons that are specific to the particular study.

In response to your request for an ombudsman and independent review of each clinical trial, please be aware that in addition to FDA’s responsibility to ensure that clinical trials are conducted ethically, clinical trial procedures are reviewed by institutional review boards (IRBs) which essentially act as an independent ethics committee for clinical trials. These boards are composed of at least five members who include scientists, doctors, and lay people, and they must approve every clinical trial taking place within their jurisdiction- generally a hospital or clinic.

We wish you the best with your continued treatment.

Best regards,

Catherine Y. Chew, PharmD

Deputy Director, Division of Drug Information

Center for Drug Evaluation and Research

Food and Drug Administration

This communication is consistent with 21CFR10.85(k) and constitutes an informal communication that represents our best judgment at this time but does not constitute an advisory opinion, does not necessarily represent the formal position of the FDA, and does not bind or otherwise obligate or commit the agency to the views expressed.


Essentially, the message is patients have no recourse once the trial design has been approved, no matter what the circumstances are. As I understand it, the IRB’s to which the FDA refers consider issues before the trial begins, not after.

Here’s what I wrote back to Dr. Chew:


Dear Dr.  Chew,

This is an outrageous response.

You take Biogen’s argument hook, line and sinker.

I guess I know now who the FDA works for.

I see nothing here that addresses the issue a subset analysis for participants who have been in the trial at least 12 months.

I’m now in a situation where, with this decision,  I will be forced out of the trial 11 days before it ends.

This decision makes ALS patients hostage to an irrational rule. If I am forced out of the trial by Biogen, I want to bar them from using any data gathered from me during the course of the trial. But I’m betting you will say that I cannot do that.


This would have been a shorter and clearer message from the FDA

Bottom line here is that ALS patients are screwed, first by their disease, and then by the companies and agencies who claim to want to help them. For shame.


© Barbara A. Brenner 2012

This entry was posted in ALS, ALS Treatment, Health Policy and tagged , , , , . Bookmark the permalink.

28 Responses to FDA to ALS Patients: Fuck You

  1. Ellen Siegel says:

    I agree – fuck the drug company.
    I hope the procedure is helpful.

  2. Gen Howe says:

    I agree, fuck the drug company, the trial, and the FDA. The FDA sent you an unbelievably cold, harsh, robotic bureaucratic response. They made it extremely clear that their damned trials and rules and more important than people’s lives. I love that you keep going after the profit-centered zombies.

  3. Kim Klausner says:

    I’m so sorry to hear this. It makes me angry that she didn’t address the point that data about you has already been collected and your “leaving” the study now absolutely does not affect their analysis.
    This is consistent with my dealings with the FDA around tobacco — they are out to protect corporate interests rather than public health.
    Thinking of you often.


  4. MDupree says:

    Thank you Barbara for your efforts on behalf of the entire ALS community.
    I find their response unacceptable.
    The Diaphragm Pacing System may help maintain breathing but does not slow, stop or reverse the disease course so I find this decision despicable. Dex is supposed to protect neurons under stress. I’m no scientist but it seems like a drug trial given orphan status should allow some participants to have the opportunity for a better quality of life while on a trial. You know, like breathing!

  5. Jenny says:

    “I guess no one understand “humanitarian use” protocol that was granted by the FDA for the Diaphragm pacer. If this doesn’t qualify for that, I don’t know what the heck does.

    The reply back from the FDA was unconscionable…guess they don’t really get it that ALS just isn’t a “very serious disease.” I really can’t even believe that anyone who is involved with this disease, can call it just “very serious.” Give me a friccing break. It’s a deadly disease, 100% of the time, there are NO survivors.

    This response sounds like a canned letter to me, just change the name of the disease, and the name of the drug trial…I’m sorry you were treated like an idiot…sheesh.”

  6. Donna Brorby says:

    What’s an address for Dr. Chew, I’d like to give her a piece of my mind. I don’t think she’s reading your blog. db

  7. Fred Gertler says:

    Once again Barbara speaks truth to power! Why can’t drug companies, doctors, medical institutions, etc., remember that their primary responsibility is to do right by their patients! Seems like such a simple idea, but apparently not.

  8. Eve says:

    Shame on the FDA. I do hope they’ll come to their senses and use some common sense soon.

  9. Pan Haskins says:

    I’m so sorry that Dr Chew’s response is so irresponsible and unjustifiable. I sent her the following response (and just for good measure, her address is Catherine. Chew@fda.hhs.gov):

    Dr. Chew,

    Ms. Brenner has shared with me your response to her (which I have copied below) about not being allowed to continue in the Dex trial while getting a diaphragm pacer. I find your response to be appallingly lacking in substance. You write that the FDA works to ensure that patients are treated fairly and ethically. I would like to know what part of this decision you consider to be either fair or ethical.

    The trial by Biogen Idec requires patients to be in the trial for 12 months. Ms. Brenner has fulfilled her side of that bargain, a bargain that is looking more like a bargain with the devil. The only reason the trial is still running is to complete the 12 months for patients that joined the trial later. Biogen Idec has the data from Ms. Brenner that it needs from her for the test; they do not need further data from Ms. Brenner.

    You wrote that you wish you had a way to resolve dilemmas like this. You do. It is not like Ms. Brenner is asking to modify the terms of the trial. She is only asking to be to allowed her to continue in the study while the remainder of data is collected from others.

    An independent evaluation of the ethics and fairness of your decision is clearly needed. Since you don’t otherwise have an independent ombudsman, and the IRB’s job is to act an an independent ethics committee, then it seems that this request should go before them.

    Sincerely ,

    Pan Haskins

    Pan Haskins, CPA, MS (Taxation)
    6778 Paso Robles Drive
    Oakland CA 94611
    v 510.339.3003
    f 510.339.3004

  10. bbzinger says:

    Thanks, Pan. I learned today that the IRB at CPMC can and does review complaints about trials at any time during the trial. So I submitted a complaint about this situation today. I will keep everyone posted.


  11. C says:

    Is this the open-label extension that goes with the trial?

    I sure don’t get why taking a very reasonable action to get an FDA-approved device after all of the prior trial data were gathered fits their exclusion criteria.

    Perhaps they will eliminate patients who don’t opt for the DPS, too, for not being of sound mind?

    Do they have more confidence that a DPS would affect data more than their drug?

    If a person started taking Neudexta for emotional lability be grounds for dismissal? If a person started taking Neudexta off-label for bulbar symptoms, would that be grounds for dismissal?

    So many questions.

    It seems that researchers like to chat up hope and promise, yet they design trials that have no way to adapt if some hope and promise finally deliver (whether inside or outside the trial). The trial designer handles all of the “what ifs” by granting the investigator all the decision power. It’s yet another “don’t you worry your pretty little heads” attitude that people with ALS face constantly. Somebody else will hold your decision cards.

    I believe that people with ALS would add a lot to clinical trial design. Perhaps some people with ALS would help those optimistic and pessimistic “what ifs” be described with some creative solutions. There are options that don’t throw patients under the bus and don’t compromise good science.

    My few cents fwtw.

  12. Phil says:

    I’m sorry to hear of your illness, and response to your “appeal” here.

    Listening to the tone of your email, obviously you are upset – but who are you upset with?

    I believe there are great progresses in ALS research, but greater effort is definitely required. You mentioned Biogen Idec has the power to influence the course of dex trail and ALS as a whole. And I praise you for participating in this trail. You have the power to find a solution for ALS which will benefit other ALS patients, and you have chosen to do so. I also praise scientist, entrepreneurs, regulators and anyone who participated in finding a solution to this. Their intentions, hearts and minds might not be completely on this disease, or this one disease, but they did choose to be a part of this. Without them, we will not even know the existence of this deadly disease.

    That being said, let us continue to be part of the solution for the greater of all ALS patients. I don’t think we will gain anything by attacking anyone personally or attacking any organization. I’m actually very saddened by this blog. We have the power to attack the problem, and not to derail its progress. We have the power to help in other diseases as well – there are many neurodegenerative disorders, and this is only one kind of disease! While we can hope that Biogen Idec could provide us choice to the impossible situation we are in, we can set our minds on others who are in worst situation than us. I know for certain that there will be no progress if no one cares. And, I hope you don’t mind me bragging, because I don’t think you can find another country today that spent more efforts in ALS or any other diseases, than America.

    Reading from some of the responses, I can’t tell if they are canned responses so I’m not going to assume it is – can’t find pattern that matches these replies in google; maybe your IT friends could? However, I could read great efforts (and care) that were put in to response to your inquiries. I don’t think anyone could be heartless in responding to your appeals and the emails that were sent on your behalf. Maybe they do care?

    My hope is that they could find more safe options for us, real soon.

  13. bbzinger says:

    Progress in research is no progress in treatment. I’m angry at Biogen, the FDA and anything and everything that stands in the way of people with ALS making the best decisions for themselves.

  14. Jenny says:

    Dear Phil,
    I don’t want to hijack Barbara’s blog, but….in my opinion,

    I don’t think ALS research is brag worthy until a treatment is found.

  15. JJ says:

    As someone who has performed research in both privately and publicly funded internationally accredited research institutions, I’d like to provide some objective input.

    The most important in research is having a standard, a control to which all data is compared. Ergo, if you don’t have controlled setting, then your data collected is useless, and that is why the FDA has rules in place restricting clinical patients from taking other drugs because you never know how that will affect the outcome of the trial. While I’m not sure why Ms. Brenner is still taking the medicine after the 12 months, I’m assuming that if they’re still taking data, they are using it for the study. If other medication or treatment is taken, the integrity of the data is compromised. While it sounds like the FDA is choosing to better its own research over the patient’s health, realize that the FDA is not a hospital but a regulatory agency.

    Each drug requires a complicated process to synthesize, and it’s not easy, it’s expensive. Realize — it’s the taxpayer’s money that pays for it. So if a patient in a clinical trial starts using another drug/device that could possibly change the environment of the research, pretty much the government & taxpayers are paying for free medicine. Unless that was part of the contract, then the medicine is paid for on the taxpayers’ dollar. Although the research aims to aid many people, and the patients are assisting with that end goal through participation, ultimately, the purpose of the government funding for research, as cruel as that sounds, is not to give free treatment, but work cooperatively with patients to discover new treatments.

    Research takes time, and while technology has advanced rapidly, the unknown is still endless. That is why researchers of levels and fields spend a ton of hours in the lab, whether they are synthesizing new chemicals for treatment or developing methods for drug delivery — and by a ton I mean 12 hours workdays (including weekends) or days on end staying in the lab overnight. Do they get paid as much as doctors? No, they are lucky if they even get one-third of an average doctor’s salary. But they are still doing their part, and just because the work done is behind the curtains doesn’t mean it’s insignificant.

    Yes, healthcare is expensive. It’s no different for anyone else who is sick either. I can sympathize with your situation, Ms. Brenner, but contracts are binding, and I’m sure that one person working in the FDA can’t bend the rules to their will.

    • bbzinger says:

      Spoken like the scientist you are, JJ. I understand research protocols. I worked in health care for many years.

      The notion that researchers work cooperatively with patients to find better treatments is, as this case indicates, unrealistic. If the researchers and the FDA wanted to cooperate with patients, they would at least understand the issues they raise.

      In my life before health care activism, I was a lawyer. I know what makes up a contract. Informed consent documents are not contracts in any traditional sense of the word. They are documents that set out the rules established by the drug company for their trial. Patients can take them or leave them, but that doesn’t make them contracts.

      While I appreciate your sympathy, I don’t think you understand the situation. My getting a diaphragm pacer 2 weeks before this trial ends WILL NOT have an effect on the trials outcome.

      • JJ says:

        1. Healthcare and research = NOT THE SAME. Salary, what you do, schooling… all vary

        2. I don’t know legal terminology…but is it really a legal issue?

        3. Most of my post was for your commenters — trying to inform them of what they don’t know so they can make more educated comments, especially regarding research, which is the basis of your entire post.

        4. I don’t know if getting a pacer will affect the trials outcome, but you don’t either. fact is, no one knows what exactly affects the outcome. I’m no biologist or doctor, but i’m assuming the diaphragm pacer benefits your body in some way, or else you would not be using it. Thus, if your body is changed in any way, then the conditions are different than those of other clinical patients being tested for the same study.

  16. bbzinger says:

    Health activists worth there salt understand research. I suggest if you don’t think I do you google me and my work at Breast Cancer Action.

    I do know that getting a pacer 2 weeks before the trial ends will not affect the trials outcome, and so do the sponsors of the trial. I suggest you look up the trial protocol.

  17. JJ says:

    It’s not that I don’t think you personally understand research, it’s just that it’s highly doubtful you can really understand it unless you have personally spent time performing it as there are many preconceived notions of “research.” Like I said, there were some comments about scientific research that I think were unfairly said, and most of my post was geared towards that audience. I admire your passion for fundraising for the cures of diseases that affect many people — it’s highly commendable. Perhaps instead of directing your anger at Dr. Chew, who might have written you the letter but probably had little say in the actual decision by the FDA, try understanding their point of view. Wish you the best in your treatment.

  18. bbzinger says:


    I don’t know you and you clearly don’t understand me, whatever your goal is in posting. I have no passion for fundraising for cures. Read other posts besides this if you want to understand.

    Thanks for your good wishes.

  19. AMP says:

    bbzinger your situation is unfortunate, but you are a fool to think you can participate in a drug trial while undergoing other forms of treatment. The integrity of a drug trial is jeopardized if research participants are undergoing additional forms of treatment. Surely you signed a contract that said you understood this in preparation for the trial. I understand why you’re upset, but you have no right to throw dirt on the FDA because you didn’t think things through!

  20. AMP says:

    in a nutshell im backing up JJ

  21. Frank says:

    You are stupid! Studies have parameters for a reason.

    • bbzinger says:

      I don’t know you, but I am not stupid. If you read more of this blog you will see that the FDA is also concerned about ALS trial paramenters. If you insist on insulting me with your comments, keep your comments to yourself.

  22. Clearly these people do not have a spouse, child or relative living with ALS. I was involved in the selecting trials for my father to participate in. The Dex trial was one, as well as NP001. My father was accepted into both trials but selected the NP001 because the odds of getting the actual med was greater. My experience is most inexperienced person setting up the appointment have no clue as to requirements of the study. Same studies ie. meds but different locations gave different answers to what was acceptable medications to begin and procedures to have. The Diaphram Pacer is an FDA approved device and should be allowed despite the study. Rilutek was another approved medication – same trial medication mind you, one location allowed you to start another refused if you were not already on it. So based on the ALS specialist of the trial further decisions can be made. My father’s NP001 was an 18 month trial I believe and when he completed the med. after 5-6 months with two follow up interviews he was allowed to do whatever he wanted due to being seven hours from the trial location (and now knowing he wasn’t getting a med. So JJ and the other Bozo you have no clue what exceptions are being made behind closed doors. If you finished the trial med. I would have flipped them the bird and contacted Dr Onders and scheduled the DP surgery. In my daughter ie lay terms… Piss on them you have the disease. The Dex med is crap anyway. I am a FALS family my opinion matters.

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