This brief piece concerns an awful incident that occurred during a recent safety trial of a new medication, by a private firm in France. The drug itself was developed by a Portugeuse pharmaceutical house. It’s of added interest because the medication was designed to affect the breakdown of endocannabinoids in the brain, and was intended for possible use with patients suffering from chronic pain, anxiety, and cannabis withdrawal.
Yes, Virginia, Big Pharma is already preparing for an increase in persons who need help withdrawing from marijuana.
Anyway, in this instance, things went badly wrong. The first six subjects wound up in the hospital– four with significant neurological symptoms, and one who was brain dead. Here’s the story.
Such outcomes are admittedly rare. Still, they do occur, with some regularity. Ten years ago, Phase 1 testing of a new med resulted in lasting damage to the immune systems of a number of subjects. An extensive investigation followed, resulting in recommendations for changes in future test procedures. Not much comfort to the victims, but such things seldom are.
I should clarify that Phase 1 research involves a small group of subjects receiving the drug for the first time under close monitoring and supervision. The aim is to evaluate safety, establish dose ranges, and identify possible side effects. Assuming that goes well, testing moves on to Phase 2, with a larger group. Phase 3 significantly expands the subject group and compares the new med’s efficacy with other products on the market. Phase 4 studies– some of you may have participated in these through your doctor or local clinic– occur after the drug has been on the market, and are intended to gather information on its use with various subpopulations, as well as the effects of longer-term use.
The Phase 1 process in particular is cloaked in secrecy. Subjects are paid– in this recent case, about $2000 for two weeks in residence. I’m confident all signed releases signifying they understood the risks involved. I’m not as confident that they really did.
In a way, drug trial catastrophes are like airline disasters, in that the aura of secrecy extends well beyond the incident itself. Investigators scrutinize the procedures involved in great detail, and often identify contributing factors rather than a single cause. It’s one thing to establish that a required procedure was not correctly followed; it’s much more difficult to prove that this error caused the harm.
I couldn’t help but notice that the Big Pharma firm referred to Phase 1 subjects as ‘heroes’. Did they think of themselves that way? Did they sign up in the hope of advancing science, or finding relief for their own symptoms? I sincerely hope they weren’t just there for the cash. That’s a terrible return.
If you’re interested in the issues around safety testing of new medications, here’s a followup analysis from another source.