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):
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 DRUGINFO@fda.hhs.gov.
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.
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.
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.
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