{"id":687,"date":"2012-08-18T21:50:01","date_gmt":"2012-08-18T21:50:01","guid":{"rendered":"http:\/\/barbarabrenner.net\/?p=687"},"modified":"2012-08-18T22:25:42","modified_gmt":"2012-08-18T22:25:42","slug":"fda-to-als-patients-fuck-you","status":"publish","type":"post","link":"https:\/\/barbarabrenner.net\/?p=687","title":{"rendered":"FDA to ALS Patients: Fuck You"},"content":{"rendered":"

If you like this blog, please pass it on to your friends. Let\u2019s keep the conversation going.<\/em><\/p>\n

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<\/a>, here<\/a>, and here<\/a>.<\/p>\n

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.<\/p>\n

\"\"<\/a>

Great friend to drug companies everywhere.<\/p><\/div>\n

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\u2019s what the FDA said.<\/p>\n

****************************************************************************************************<\/p>\n

Dear Barbara,<\/p>\n

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.<\/p>\n

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<\/p>\n

\"\"<\/a>

No thanks to the FDA<\/p><\/div>\n

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.<\/p>\n

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. \u00a0The 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.<\/p>\n

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.<\/p>\n

We wish you the best with your continued treatment.<\/p>\n

Best regards,<\/p>\n

Catherine Y. Chew, PharmD<\/p>\n

Deputy Director, Division of Drug Information<\/p>\n

Center for Drug Evaluation and Research<\/p>\n

Food and Drug Administration<\/p>\n

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.<\/p>\n

****************************************************************************************<\/p>\n

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.<\/p>\n

Here’s what I wrote back to Dr. Chew:<\/p>\n

********************************************************************************************<\/p>\n

Dear Dr. \u00a0Chew,<\/p>\n

This is an outrageous response.<\/p>\n

You take Biogen’s argument hook, line and sinker.<\/p>\n

I guess I know now who the FDA works for.<\/p>\n

I see nothing here that addresses the issue a subset analysis for participants who have been in the trial at least 12 months.<\/p>\n

I’m now in a situation where, with this decision, \u00a0I will be forced out of the trial 11 days before it ends.<\/p>\n

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.<\/p>\n

***************************************************************************<\/p>\n

\"\"<\/a>

This would have been a shorter and clearer message from the FDA<\/p><\/div>\n

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.<\/p>\n

 <\/p>\n

\u00a9 Barbara A. Brenner 2012<\/p>\n