An efficient market depends on the consumer being both informed and rational in his choices. This obviously requires familiarity with the performance of the product (success rate, side effects, etc.) as well as an understanding of how the drug works. This is a bit much to ask of your average consumer, considering all of the education and training required to make a doctor or a pharmacologist. It would be a textbook case of a highly inefficient market with a great degree of misallocation of resources. In other words, patients would go broke as they died while pursuing ineffective treatments.
Is a government agency like the FDA the best solution to this problem? I can’t really say. But I think they’re a valid solution. The problem comes when they move away from that role in improving patient information to actually dictating which medicines a patient can and cannot use and for what they can use them.
The ridiculously long clinical trial process is designed to determine how effective a given drug is as well as what, if any, the major side effects are. Based on this the FDA then makes a rather arbitrary decision as to whether or not the costs outweigh the benefits. Strangely enough, moneymakers like antidepressants, ADHD drugs and the like are allowed considerably higher thresholds than treatments for things like lupus, multiple sclerorosis, and other debilitating diseases.
The clinical trial itself is a good thing: It improves patient information. FDA approval is not. What the FDA deems “unacceptable risk” might be far different from what an individual patient may think. Tysabri, a treatment to prevent relapse in Multiple Sclerosis, is a perfect example of this. Three of the 1200 clinical trial participants developed a rather rare brain infection and subsequently died. These are not odds that the FDA likes. However, MS is quite a debilitating disease, and a patient might feel completely differently from a bunch of healthy people in white coats sitting far away from the pain, the debility, and the hopelessness.
Off-Label Drug Use
Another peculiarity of the FDA approval process is that a drug isn’t merely approved as “safe”; it’s approved as “safe and effective”. Which is a horse of an entirely different color. The FDA doesn’t approve drugs, but rather approves the use of a given drug in the treatment of certain conditions.
In other words, if your new wonder drug has a second potential use, guess what? Another trial, another 5-10 years before people at large can actually use it. Even though it’s already been deemed safe.
This is particularly problematic given the nature of the human body. Take Viagra for instance. Bob Dole may use it for one thing, but it has great potential in treating pulmonary hypertension and other cardiovascular problems involving constriction of your blood vessels. Although there are other medications out there, they often take a “bigger hammer” approach and in many cases are incompatible with either the patient’s condition or their other drugs. Viagra, on the other hand, is quite a bit more gentle in its effect.
It’s an interesting thing about medicine in that the core of our knowledge hasn’t changed. I use the same textbooks in physiology and pharmacology as my mother did 30 years ago. They’ve been updated in the ensuing decades, but I bet I could get the same letter grade using her ancient version as mine.
A drug is approved for treatment of one illness because it has a certain effect on the body. If this effect is known to be useful in alleviating other maladies, then why must I be hindered in using it in order to treat my patients, simply because of the FDA’s shortsightedness?
The FDA’s biggest sin is micromanagement. As an overarching organization, it can help both patient and doctor make informed treatment decisions by bringing to the fore otherwise unobtainable data on drug performance (and more importantly harmful side effects). As such, it can liberate an imperfect market and allow better allocation of medical and economic resources.
However, when the FDA decides to make the decision for doctor and patient it not only limits their freedom, it compromises the patient’s health and the doctor’s ability to change lives. At least once in my medical career, I will literally hold my patient’s life in my hands. I will stand between him and an untimely death. The idea that some suits in Washington can tell me what I can and cannot do in such a situation is absurd.
Every patient is different; the etiology of their disease, its progression, severity, and how it impacts the patient’s life qualitatively. For one man with a relatively mild case of Multiple Sclerosis, a 1 in 400 chance of dying may not be worth the risk. For a woman who finds herself in a wheelchair, losing sight, losing dexterity, losing her self, she may choose differently. Perhaps to her a year of relative freedom is worth more than five years of increasing debility. Who has the right to take that choice away from her?