Tuesday, December 30, 2008

Awesome Opinion on FDA, Crohn's and Osiris

Written by Gideon J. Sofer- University of California, Berkley student - from WSJ

Right now there are millions of individuals whose lives are directly dependent on the rate at which new drugs come to market. I'm one of them. I'm fighting for my life.

To date, half of my intestine has been removed to manage Crohn's disease. Last year, at age 23, I enrolled in a clinical trial for a treatment that could save my life: an adult stem-cell therapy that helps damaged intestinal tissue regenerate from the relentless inflammation and scarring caused by Crohn's.

The sponsor, Osiris Therapeutics, reported that Crohn's patients in the therapy's Phase II trial all experienced clinical improvement after receiving the cells. A Phase III trial for the treatment is now nearing completion, but Food and Drug Administration (FDA) approval could be years away, despite its FDA "fast track" designation.

In accordance with antiquated FDA policies, the Phase III trial is randomized with three groups of patients, and double-blinded, which means neither the doctors nor patients are told what treatment is being administered. One group received full-strength stem cells, another received half-strength, and a third got a placebo (the proverbial "sugar pill"). It appears I got the placebo.

Foregoing all other treatments, I received the four scheduled infusions, and yet my disease progressed with a vengeance. In a matter of weeks, I became dangerously malnourished. I've since been readmitted to the hospital countless times, as my doctors continue to plead with Osiris for information. But Osiris has refused, citing adherence to FDA protocol.

I am now a lab rat. I have no right to know what happened to me in the study, nor do I have a right to try the promising treatment as my health deteriorates. It doesn't have to be this way.
Under the Fifth Amendment's guarantee that "No person shall be deprived of life, liberty or property without due process of law," a critically ill patient should have access to a potentially lifesaving drug that has been deemed safe for human consumption, if the patient agrees to bear the risks involved. But earlier this year, the Supreme Court refused to hear a case on the issue, denying countless patients their right to pursue life.

Thankfully, some members of Congress have stepped in to ensure our rights as patients. In May, Sen. Sam Brownback (R., Kan.) and Rep. Diane Watson (D., Calif.) introduced the Access, Compassion, Care and Ethics for Seriously Ill Patients Act. If passed, this bipartisan legislation will begin to restore the rights of millions of patients by widening access to promising investigational drugs.

Human clinical research is an intricate scientific and moral process, but it does not justify taking immoral advantage of patients. Tragically, FDA and Osiris think it does.

Typical approval protocols almost always guarantee patients taking the placebo access to the actual drug -- at the very least -- after the study has ended. But in what appears to me a deliberate act of cruelty, Osiris hung its patients out to dry without any recourse, refusing to confirm which patient got what. The FDA has endorsed Osiris's decision by enabling it to proceed with the study.

Withholding a potential cure is just as bad -- if not worse -- than the potential death sentence of a serious illness. If patients like myself have the audacity to put their lives on the line for the betterment of science and those in their predicament, their decision should not only be embraced, it should be rewarded.

Furthermore, trials without ethical recourse can lead to inadequate and incomplete data, compromising the integrity of the study. If trial patients are treated like lab rats, they won't feel obliged to cooperate unconditionally and report accurate data -- something the FDA and the drug industry rely on heavily, but have failed to consider.

Everyone agrees it is a fundamental right for patients to dictate their course of treatment with FDA-approved drugs. So why do the rules evaporate at the most critical moment, when the only life-preserving options are highly promising investigational drugs?

9 comments:

Anonymous said...

I'm surprised Osiris gave you guys true placebos instead of treatment as normal (i.e. what your treatment regimen would have been without the clinical trial). Using treatment as normal comparisons would tell them wehether their stem cell thearapy is more effective and/or efficacious than standard care. They can even compare rate and severity of side-effects. A true placebo design just tells them whether their stem-cell therapy is better than no treatment. There are scientific advantages and disadvantageous to both designs, but ethically I can't see how they can justify a true placebo in this case. That's just my opinion

Anonymous said...

I'm surprised Osiris gave you guys true placebos instead of treatment as normal (i.e. what your treatment regimen would have been without the clinical trial). Using treatment as normal comparisons would tell them wehether their stem cell thearapy is more effective and/or efficacious than standard care. They can even compare rates and severity of side-effects. A true placebo design just tells them whether their stem-cell therapy is better than no treatment. There are scientific advantages and disadvantageous to either design, but ethically I can't see how they can justify a true placebo in this case. That's just my opinion

Anonymous said...

Most people would assume that for serious and terminal diseases, giving placebos to patients in clinical trials would be considered unethical, but the FDA thinks otherwise and often requires that drug companies do it to get their drugs approved. It absolutely does harm and kill people and the FDA not only knows it, they conduct predictive calculations to determine how many patients need to be harmed or die in the control arm to deliver "statistically significant" results for the trial. It is clearly unethical, but the FDA has progressively rationalized its drug development and approval policies over the years to effectively eliminate the application of real ethics from its clinical testing requirements. The trial Mr. Sofer entered allowed enrollment only to patients who had exhausted other approved treatments, which meant they had little choice but to risk randomization to a placebo because their was no other way for them to obtain the potentially effective treatment. In this case, because the drug obviously worked in an earlier non-randomized Phase II trial (everyone responded positively), there was no need for a placebo arm, even by the FDA's twisted logic, but Osiris included it, which (to those who understand the FDA's policies and processes) means FDA wanted it. Why they continued the blinding for placebo patients taken off study after the specified 28 day evaluation period because they didn't respond, is indefensible, and also probably something the FDA wanted. Why? Don't expect the FDA to explain it. Osiris might, but also probably will not. There was no defensible clinical research, medical or regulatory reason for a placebo arm, and absolutely none for continuing to blind the patients after they were taken off the study. So now all the patients who got a placebo in this trial are prevented from enrolling in other trials because knowing your prior treatment history is lmost always an absolute requirement for enrollment. One might think putting a living, breathing human being at risk in this fashion would be something the FDA would want to prevent, but instaed the FDA is the reason for it. So just who is protecting patients in clinical research the answer is actually - no one.

AlisonH said...

There is worse: there's a Parkinson's drug where deathbed patients got up and lived their lives again, and the company making the med they were on got bought out. The new owners decided they couldn't make enough money before the patent ran out and refused to continue to supply the med. To anyone. Even the ones promised in writing that they would continue to receive it.

These were patients who had had brain surgery to implant the shunt whereby the drug could be administered. It is SO outrageous. The Supreme Court said the law unfortunately sided with the drug company.

I write this as a Crohn's patient resistant to steroids and very interested in how your trial goes. I'm with the commenter above; it would have been better science, not just greater compassion, for the ones running your trial to have at least kept you on what you were on. Good luck!

Anonymous said...

Without a placebo group to compare to how would they know how effective the drug is, itself, instead of just an interpretation of treatment (placebo effect)? What if the drug does no better than the placebo? If they found a symptom how would they know if it was caused by the drug, by the solution the drug is in, etc? You have to have something to compare to to make valid conclusions.

I would like to see Osiris give the drug to participants in the study who showed no improvement with the placebo but completed the full study, but with the drug so expensive to produce, how do you suggest they do that?

-C

peanenluv78 said...

i am new to your blog and a first time reader and just wanted to thank you so much for your work! i am 30 years old and was recently diagnosed with crohn's disease after battling years and years of abdominal pain! i found your site to be so helpful! thank you!!

Anonymous said...

It is quite amazing that you posted this today. My Husband and I have been researching this for the last few weeks, with the hopes it would get him of his 24hr TPN and bowel rest. The this problem is, 90 days off his meds, would kill him. yet he is on all meds available. At this point we dont know what to do.
-Monica

Anonymous said...

If one has crohns and needs drugs then one needs the drugs. This is not a disease where one can stop medications to experiment with drug trials. I would never take Prednisone, Imuran or Remicade or Pentasa unless I truly need these drugs.

If I stop I know i jeopardize my health especially if a flare occurs.

I was in the ICU with a partial SBO in November. The drugs I am taking are a necessity and nothing to take away from me if I wish to live a long life.

Osiris needs to give people their meds and not kills crohns patients.

I hope that Osiris comes to market soon. It looks promising and might spare me all the nasty side effects of the dreaded Prednisone.

I do not want them killing or damaging fellow crohns patients during the trials.

Anonymous said...

I think there is some misconception here about the Ositis trial. Patients are permitted to remain on stable doses of their current oral medications, including prednisone, 5ASA's, immunosuppressants, and antibiotics.

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