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Dec. 18th, 2006 08:06 amThe Right To Trial - Do you have a right to experimental medicine?
I have to admit, this article annoys me on so many levels. While it's important to discuss these issues, it's also important to present both sides in roughly equal fashion. Instead, the first 2/3 of the article is devoted to telling you how one man beat cancer by enrolling in a clinical trial and then his efforts to get his dying daughter into another clinical trial, or at least get her access to the promising drug.
The last few paragraphs are where they point out to you how expanded access in a non-controlled way may actually make it more difficult to determine which drugs are actually giving better performance than treatment standards, how expanded access would cause issue for recruitment to clinical trials, not to mention the fact that some experimental treatments just don't work as expected.
While I am more biased to support the current clinical trial system, I do have experience with the other side of wanting a magic bullet to stop a disease or cure an illness. However, I also point to another problem --- very very few insurance policies agree to pay for any sort of experimental treatment. It's one of the reasons that all clincical trials agree to pay for all costs of care and transportation for enrollees (though sometimes the transportation is within a limited area necessitating some enrollees to move to participate). Who hasn't seen jars asking donations to help so-and-so pay for radiation or a bone marrow transplant not covered by their insurance? Under the provision of expanded access that the article presents, our health care system would once again be further segregated into the have and the have-nots. Of course, in this instance, the have-nots might be somewhat better protected.
As to the issue of access to experimental drug versus placebo, the article is very deceiving. For clinical trials involving life-threatening conditions, the "placebo" is always the standard treatment of choice. It is unethical to give a dying patient sugar water. Therefore, every experimental drug for a life-threatening disease such as cancer must be able to demonstrate that it is either a) significantly more effective at increasing survival rate for a given period (generally depending on whether we are talking stage 2 or 3 trial) or b) as good as the standard treatment but with significantly fewer side effects. Generally, extending "remission" is not a criteria for success unless said remission is more than a year in a stage 3 trial.
The problem with any life-threatening illness or devastatingly chronic condition such as Parkinson's disease is that it causes desperation for the sufferers and the families. Ethically, the medical and research community must attempt to not pander to this desperation or feed unrealistic hopes that may be crushed. It's a fine line. Even for members of my immediate family, I've always told them that I would only recommend they participate in a clinical trial if, and only if, I could find published evidence as to mechanism of the treatment, efficacy of the treatment, and preferably if I knew someone in the trial from whom I could get the low-down. I've been to too many conferences where "promising drugs' that proceeded to stage 2 trials not only failed, but also produced severe side effects that eliminated all quality of life for the patients right up until the end. The number one reason that people drop out of clinical trials is due to the side effects, especially in cancer trials.
I'd argue that based on what we know of recently approved drugs for non-life threatening diseases, such as certain cox-2 inhibitiors, the stringent process used with cancer drugs, even though a higher amount of side-effects is generally allowable provided that life-threatening organ damage is one of them, is a good thing. While getting drugs to treat diseases that hamper one's way of life, like arthritis or crohn's disease, it's far better to know that the drug is safe than to have relief from the disease for a couple of years only to find out later that you've permanently damaged your heart or kidneys.
Medicine is not an exact science. Research scientists aren't perfect. The pharmaceutical industry is driven by profit, but one has to understand that much of their profit is reinvested in the pipeline for future drugs. The prices may seem evil, but what they say is true --- today's drugs pay for tomorrow's miracles. Granted, the shareholders get a stake too, but the cost of the stocks also pays for the drugs.
Let the flaming commence because they aren't going to my inbox.
I have to admit, this article annoys me on so many levels. While it's important to discuss these issues, it's also important to present both sides in roughly equal fashion. Instead, the first 2/3 of the article is devoted to telling you how one man beat cancer by enrolling in a clinical trial and then his efforts to get his dying daughter into another clinical trial, or at least get her access to the promising drug.
The last few paragraphs are where they point out to you how expanded access in a non-controlled way may actually make it more difficult to determine which drugs are actually giving better performance than treatment standards, how expanded access would cause issue for recruitment to clinical trials, not to mention the fact that some experimental treatments just don't work as expected.
While I am more biased to support the current clinical trial system, I do have experience with the other side of wanting a magic bullet to stop a disease or cure an illness. However, I also point to another problem --- very very few insurance policies agree to pay for any sort of experimental treatment. It's one of the reasons that all clincical trials agree to pay for all costs of care and transportation for enrollees (though sometimes the transportation is within a limited area necessitating some enrollees to move to participate). Who hasn't seen jars asking donations to help so-and-so pay for radiation or a bone marrow transplant not covered by their insurance? Under the provision of expanded access that the article presents, our health care system would once again be further segregated into the have and the have-nots. Of course, in this instance, the have-nots might be somewhat better protected.
As to the issue of access to experimental drug versus placebo, the article is very deceiving. For clinical trials involving life-threatening conditions, the "placebo" is always the standard treatment of choice. It is unethical to give a dying patient sugar water. Therefore, every experimental drug for a life-threatening disease such as cancer must be able to demonstrate that it is either a) significantly more effective at increasing survival rate for a given period (generally depending on whether we are talking stage 2 or 3 trial) or b) as good as the standard treatment but with significantly fewer side effects. Generally, extending "remission" is not a criteria for success unless said remission is more than a year in a stage 3 trial.
The problem with any life-threatening illness or devastatingly chronic condition such as Parkinson's disease is that it causes desperation for the sufferers and the families. Ethically, the medical and research community must attempt to not pander to this desperation or feed unrealistic hopes that may be crushed. It's a fine line. Even for members of my immediate family, I've always told them that I would only recommend they participate in a clinical trial if, and only if, I could find published evidence as to mechanism of the treatment, efficacy of the treatment, and preferably if I knew someone in the trial from whom I could get the low-down. I've been to too many conferences where "promising drugs' that proceeded to stage 2 trials not only failed, but also produced severe side effects that eliminated all quality of life for the patients right up until the end. The number one reason that people drop out of clinical trials is due to the side effects, especially in cancer trials.
I'd argue that based on what we know of recently approved drugs for non-life threatening diseases, such as certain cox-2 inhibitiors, the stringent process used with cancer drugs, even though a higher amount of side-effects is generally allowable provided that life-threatening organ damage is one of them, is a good thing. While getting drugs to treat diseases that hamper one's way of life, like arthritis or crohn's disease, it's far better to know that the drug is safe than to have relief from the disease for a couple of years only to find out later that you've permanently damaged your heart or kidneys.
Medicine is not an exact science. Research scientists aren't perfect. The pharmaceutical industry is driven by profit, but one has to understand that much of their profit is reinvested in the pipeline for future drugs. The prices may seem evil, but what they say is true --- today's drugs pay for tomorrow's miracles. Granted, the shareholders get a stake too, but the cost of the stocks also pays for the drugs.
Let the flaming commence because they aren't going to my inbox.
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