Late last week I posted here a message I sent I had sent to the CEO of Biogen Idec, the company running the dex drug trial I’m in. I encouraged people to write to the CEO, Dr. George Scangos, about the company’s plan to drop from the trial anyone who gets a diaphragm pacer before the trial goes to open label in September of this year. That post is the one immediately below this one, called “Let’s Broaden ALS Research to Help Patients.”
Dr. Scangos has not replied to my message. But he did send a canned reply to the many people who emailed him on my behalf.
Based on that reply to others, I have sent another — more pointed — message to Dr. Scangos. That message is here. Also here is a copy of the doctor’s canned reply.
It’s not too late to email Dr. Scangos and tell him what you think. I hope you will. If you’ve already emailed and were unhappy with his response, my message here might give you ideas of what more to say to him. His email address is: email@example.com, unless he changes it to avoid these messages.
This is not just about me, or just about dex. It is about all health research and whose interests it serves: when is it important to let patients decide what is in their best interest without being forced to make impossible choices? When being forced to make those choices makes no sense either in science or ethics.
© Barbara A. Brenner 2012
My response to Dr. Scangos’s messages about my request.
July 22, 2012
Dr. George A. Scangos
RE: Continued participation in the dex trial
Dear Dr. Scangos:
As you no doubt know, I have not received from you the courtesy of a reply to my message to you of July 19, 2012. But people who contacted you on my behalf– some of whom I don’t even know — have forwarded to me the response you sent to them. I write to address what you say in that response in hopes of getting a prompt resolution to this very important matter.
I hope that, at some point, we can discuss ways to better design drug trials for the benefit of ALS patients, but what we need to resolve now is why Biogen Idec’s policies are inconsistent with the rules set up for this trial. You say in your message, which I have copied below, that each patient has to be followed for a minimum of a year. As I pointed out in my first message to you, I have been in the trial for 14 months. Under these circumstances, I fail to see — and you do not pinpoint — the exact violation of the trial protocol that my obtaining a diaphragm pacer at this stage would represent.
I am not asking for an exception to your protocol. I am asking that you implement policies consistent with it for the benefit of patients with ALS. To do otherwise is to hold ALS patients hostage to your trial. That is inhumane.
I fail to see — and nothing you say addresses — what inhibits you from doing a subgroup analysis of patients in the trial for more than a year who undertake other treatments thereafter, or cutting off analysis of their results after a year.
I understand that you need to protect the comparison between the placebo group and the treatment group. What I don’t understand is how, given the one year participation requirement of the trial, a diaphragm pacer in patients who have been in the trial more than a year would jeopardize that comparison.
This is not 1950. You have the capacity to do a staggered analysis of the data from this trial based on when patients entered it and one year has passed. As I understand it, that is what your protocol requires.
I am entitled to and expect a response to this message.
Barbara A. Brenner
Dr. Scangos’s message about my request
I have received 18 emails requesting that Biogen Idec allow Barbara Brenner to stay on the trial while she receives a diaphragm pacer, and they are still coming. I am touched by the letters and it is obvious that Barbara has a great number of people who care deeply about her. Our mission at Biogen Idec is to bring better treatments to people with ALS and with other neurodegenerative disorders. We have physicians working for us who still treat patients with ALS, and we have employees with MS, ALS, and other degenerative diseases. I, and all the employees of our company are keenly aware how devastating the disease can be, and that these diseases affect not only the patient, but their family, friends, and colleagues. It is the desperate need for better care for these patients that motivates us to work the long hours that we do.
We are conducting the phase 3 trial of Dexpramipexole under an agreement with the FDA. The trial is intended to demonstrate whether or not Dexpramipexole has the ability to reduce the rate of disability progression and/or prolong the lives of patients with ALS. Half of the patients are receiving drug, and half are receiving placebo. Neither the company nor the treating physicians know whether a patient is receiving drug or placebo, and we won’t know until the end of the trial when it is unblinded. Each patient is being followed for a minimum of one year. The last patient was enrolled in the trial last September and therefore the trial will be completed this September, after the 1 year period is reached by all patients. One of the requirements to ensure that a valid comparison is made between patients treated with the drug and those on placebo is to ensure that they adhere to the same treatment protocols. Therefore we have rules that a patient must meet to be included in the trial. If we make exceptions to these rules, and patients begin to receive different treatments, then the validity of the comparison between drug-treated and placebo-treated patients could be jeopardized. If that comparison is jeopardized, then our ability to determine whether or not the drug actually works also could be jeopardized, and therefore its availability to all patients with ALS could be substantially delayed. Therefore, we are unable to grant these requests because to do so jeopardizes the validity of the trial, which could prolong the time until the drug is available to all patients.
I want to make sure that you understand that the drug has not been proven to work. The drug had encouraging data in a phase 2 trial, but there are many examples of drugs that had such data and then failed to demonstrate a benefit in larger phase 3 trials. The purpose of doing the phase 3 trial is to determine if the drug does indeed provide a benefit to patients with ALS. After the last patient finishes their 1 year treatment period, it will take some time to unblind and analyze the data. We should know whether or not the drug provided a benefit later this year. It is possible that the drug will not work. In that case, we will stop the trial and move on to our other projects designed to find a treatment for ALS. If the drug does appear to provide a benefit, we will work with the FDA to make the drug available to some patients even before it is approved. We understand the desperate need for treatment and the importance of time for patients with ALS, and are doing everything we can to bring a treatment forward as quickly as we can. I am deeply sorry, but we simply can not, however, allow deviations that could jeopardize the validity of the trial.
You should know that we remain committed to find a cure for this disease over the long term. We just last week initiated a collaboration with Duke University and the HudsonAlpha Institute to sequence the genomes of 500-1,000 patients with ALS to understand the genetic causes of the disease. Our own researchers are working hard and working with top academicians around the country to find better treatments, and ultimately a cure for this horrible disease.
On a personal note, I am deeply sorry that we can not comply with your request. Please know that my thoughts are with you and that we are doing everything that we can.
George Scangos, Ph.D.
Chief Executive Officer
133 Boston Post Road
Weston, MA 02493
seems the doc does not have your e-mail…..
Dear Ms. Siegel,
I have tried to respond to everyone’s email – more than 30 of them. Ms. Brenner’s email did not reach me, I do not know her email, and have no way of responding. If you have her address, please forward it and I will respond.
George Scangos, Ph.D.
Chief Executive Officer
133 Boston Post Road
Weston, MA 02493
Date: Monday, July 23, 2012 6:41 AM
Subject: follow up
I thank you for your response.
I see that you have not yet responded to Ms Brenner – I am not sure why. Certainly you do not want to be rude to her or cause more stress on her. She makes a point that she has been on this trial for over one year. So I still do not understand why she cannot obtain a pacer. She seems to have met the criteria.
Don’t be cruel – respond to her directly.
Thank you again,
Thanks, Ellen. I just emailed hm — for the 3rd time — at the same address you have and others have been using. I guess I’ll have my partner call him later if I don’t get a response. I directed him to the blog.
and the summary of Dr. Scangos’s letter sounds like this in my mind when I play it back in my memory…”No, just because…no. The clinical trial is everything. Your individual efforts to save your own life are nothing. blah blah blah my validity”
I find these sentences of Dr. Scangos’s letter particularly disturbing…
“I have received 18 emails requesting that Biogen Idec allow Barbara Brenner to stay on the trial while she receives a diaphragm pacer, and they are still coming.”
“I want to make sure that you understand that the drug has not been proven to work.”
“If the drug does appear to provide a benefit, we will work with the FDA to make the drug available to some patients even before it is approved.“
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