People dying of terminal illnesses now have the right to take experimental drugs that are not yet approved by the the FDA, a federal court of appeals ruled last month (as reported in June's Nature Medicine). While on the one hand, these drugs may bring some hope to those whose illness has been thus far unresponsive to therapy, on the other it may hasten their demise by exposing them to a range of untested side effects while not even conclusively treating their illness. The recent ruling is the result of a lawsuit filed against the FDA by the advocacy group Abigail Alliance, whose namesake died in 2001 of cancer while trying to enroll in a clinical trial for Erbitux. This drug was subsequently approved by the FDA.
The lawsuit was originally brought in 2004, but was dismissed by a DC court. The appeal process eventually gained them a sympathetic ear, and the case was sent back to the district court where it met approval. The FDA's next move has not yet been announced, but it can request that the Supreme Court or the appeals court revisit the case. Some drawbacks to the ruling that have been raised: it could potentially give drug companies complete control over who gets the drug pre-approval, and may hinder people from joining clinical trials which are essential to the approval process. (More below the fold)
"In the real world, this could create the ultimate nightmare," says Peter Hutt, a partner at the law firm Covington and
Burling, and a former head counsel for the FDA. "It might almost eliminate placebo trials. And if a phase 2 shows efficacy, no one would show up to phase 3."
This seems a callous way to look at it, as the mission of the FDA is to improve public health and to pass on the advances of medical technology to the general population. While an important, no, essential, part of this is the safety certification and efficacy process (yes it works, and yes its safe), this must be suspended for those who have no other options. The opposing doctors (and the FDA, essentially) are taking the "do no harm" mantra to a further degree than necessary, insofar that "doing no harm" doesn't always mean taking no risk. In some cases, the denial of care that might provide some risk of harm is itself unethical when the patient has nothing to lose.
And, for the detractors, why not include the "compassionate care" recipients in your clinical trials? If truly you are worried about a paucity of volunteers in your studies, make it mandatory for experimental drug recipients. Although I seriously doubt there will be much lack of volunteers for free healthcare and drugs, when then alternative will be expensive, non-subsidized, and non-covered drugs.