A Patient’s Story of an ‘Inhumane’ Clinical Trial

What would you do if you were suddenly diagnosed with a life-threatening condition? What if your predicament was such that your doctors told you that your best hope of staying alive was to take your chances on an investigational therapy that has yet to be approved by the Food & Drug Administration? Would you take your chances and enter a clinical trial?

After battling Crohn’s disease for over a decade, I have now exhausted every approved drug. When I was diagnosed in 1996, the first line of therapy was steroids, whose side-effects could prove deadly. But now it’s 2007, and the reality is I can no longer count on one hand the number of investigational therapies being developed for Crohn’s. This mere fact is real reason for hope.

This past February I stumbled upon a drug that could potentially save my life: a stem cell therapy that could help damaged intestinal tissue regenerate and recover from the relentless inflammation and scarring caused by Crohn’s disease. Who would’ve thought modern medicine could get this good?

After spending the summer months self-administering intravenous antibiotics, I finally got the phone call: The stem-cell study I’d been waiting for had cleared my doctor's review board in New York. They faxed me the protocol, and I made plans to leave immediately.

I am now entered in what’s called a randomized double-blind placebo-controlled clinical trial in which only the drug company knows who is receiving the medicine or placebo—patients and their doctors are uninformed. I took a careful look at the 28-page protocol, but before signing my life away, I wanted to know the terms of the study. I prepared a list of questions to ask my doctor’s study coordinator: What are my chances of my getting the actual medicine versus the placebo? If I get the medicine will I still be able to continue taking it after the study? What if I’m in the placebo group and don’t get the drug?

It used to be that drug companies would generally offer patients some form of financial compensation for their participation in a clinical trial. As protocol policy has shifted, those days—more so than not—are history. So what’s the real trade-off for patients myself? If I get the actual medicine, I start to get my life back. Indeed, there are some things money can’t buy.

But while most of the aforementioned questions were answered, it turns out that the drug company failed to explicitly promulgate what will happen to the placebo group. After following up with several coordinators of the study, it came to my attention that many participating institutions had confronted the drug company about this backward flaw in the fine print. But the company still has not budged. At this point, they will not dispense the actual medicine to the control group under any circumstance, even after the study has commenced.

Typical protocols almost always guarantee the control group access to the drug, at the very latest, after the study has commenced. It is otherwise considered inhumane to leave the sub-group of patients behind with no recourse.

As more and more patients like myself fall prey to this dramatic shift in protocol ethics, many find themselves both desperate and defenseless. Patients often find themselves too weak to question the status quo. Many don’t have the energy or resources to demand further inquiry. On any given day, my doctors are occupied with seeing up to 50 patients. Without a personal advocate, most patients find themselves subservient to accept the status quo.

The biggest dilemma facing the majority of critically ill patients entering a clinical study is time, which certainly is not on our side. I found out about this anomaly in the protocol but was powerless, because I have no other choice. I needed intervention as soon as possible, and entering this study was my best hope for survival. While the need to advance medical knowledge by using human beings in research is an intricate moral process, it does not by any means justify taking advantage of desperate patients like myself. Initiating a trial without ethical recourse can lead to inadequate and incomplete data, and can ultimately compromise the integrity of the study. The truth is, nobody in my shoes should ever be faced with such a quandry.

With minimal oversight, this is unfortunately becoming the new normal in present-day medicine. In order for things to change, patients will ultimately have to take fate into their own hands by remaining skeptical, questioning the status quo, demanding inquiry and getting their stories out there.

In the mean time, I’m doing the best I can to step-up to the plate. And if it is true that moral values emerge out of human experience, then it is my greatest hope that my experience will create compelling justification for change.

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