Experimental Drugs for the Terminally Ill - a Debate Rages On

Scenario: a patient is sick with a disease that will end her life within months. A drug developed for that disease is in the process of being researched through clinical trials and will be submitted for approval through the FDA within one or two years.

Should that patient be allowed to try that experimental drug before it is proven to be effective with the hope it will save her life?

That's the question being posed through a bill introduced to Congress called ACCESS: The Access, Compassion, Care and Ethics for Seriously Ill Patients Act (HR 6270). It proposes that terminally ill patients be allowed to try those potentially life saving drugs once the first of the four phases of clinical trials has shown the drug to be effective.

I learned about the bill and the debate through an article published in the Wall Street Journal which tells two stories of people who lost their lives as they waited for the FDA to provide "compassionate" approval, meaning, a long drawn-out, process that means these patients didn't last long enough to let that system work for them. They died as they waited.

The research process for FDA approval of new drugs requires three clinical trials which each must result in increasingly "definite" proof that a drug will do what it's supposed to do, and will be safe, too. (A fourth trial is expected to be run once the drug is approved and marketed, although the jury is out on whether this happens nearly so often as it's supposed to.)

The proposed ACCESS bill bypasses the FDA approval process for the terminally ill patient. If the drug is deemed effective fter the first trial, which may include only a handful of people, a patient deemed terminally ill would be allowed access to it.

At first glance, the answer seems like a relative no-brainer. If someone is dying, and an experimental drug is available, why shouldn't she be able to try it if she wants to?

It turns out, it's not that easy. An article from the New England Journal of Medicine points out some of the potential arguments against such an approach -- the slippery slope it may create.

Here are some of the reasons the professionals are against this easier access:

• It interrupts the clinical trial process, meaning, patients would gain access to the drug instead of participating in a clinical trial which would have a control group involved. No matter whether a terminally ill patient uses the drug and lives or dies, it won't really be known whether the drug was involved in improving her health or making her sicker.
• Allowing drug companies to bypass the FDA approval process would lead to more fraud and illegal drug activity than we already experience.
• The legal ramifications are many. What happens if the drug doesn't work? What happens if the patient develops another problem unrelated to the terminal illness and it can be attributed to the experimental drug? You can see the lawyers right now just salivating over the prospect.
• The manufacturers of the drugs cite supply problems. They aren't far enough along in the process to know how much of the drug to make ready for the market, nor do they know how to price it. And my somewhat cynical opinion here is that providing the drugs early to an individual interrupts their ability to profit sooner rather than later.... but I digress....
• Additional reasons are cited in various publications.

My observation is that reasons for and against, are really the divide between benefit to the individual, vs benefit to mankind. Evidence based medicine is developed in groups. In a group of 100 people, if a new drug was beneficial to 51 of those people, then the "evidence" shows it worked. But for the individual patient, who might be better represented by the other 49 participants, it may or may not work. The evidence is based on the many, the group.