Something horrible happened – a clinical trial failed, causing one unexpected death and seriously injuring 5 people. And instead of dealing with a dysfunctional research system, “experts” are spouting off on their own. The few articles written to date focus on the drug (aka the money), not on what people want to know.
My Initial Thoughts
ClinicalTrialsArena asked me to comment, due to my unique expertise that straddles the patient and research worlds. After researching the topic,* here are my concerns – most of which have not been discussed yet.
This poses a much larger question – why am I the one bringing these up?
Fact 1: Something really bad happened – the first 6 people to get “repeated higher doses” died or were seriously damaged. This is a clear sign of system failure (e.g. approval, protocol, procedures, formulation, PharmcoDynamics).
Fact 2: A phase 1 clinical trial means it is the first time a drug is studied this way in people. 127 people in the trial is far larger than classic phase 1, and most don’t have placebos. Isn’t this more like a phase 2, or the new-fangled phase 1-2 trial? If so, what happened to the rules?
Fact 3: The drug showed activity in laboratory dishes and animals first. We don’t know what testing was done, or for how long. How realistic are the animal models, and how closely do they relate to humans?
Fact 4: Existing articles stress the need for more ‘transparency’ to share the drug’s molecular structure. Patients want honesty first, which in this case may mean, who screwed up?
Fact 5: I’m a big believer (and trail blazer) in presenting trial results, but that’s when we have results. NOT during an ongoing, active clinical trial. Why aren’t we calling for the protocol to be publicly published immediately?
Fact 6: Healthy volunteers joined this trial after reading an ‘informed consent’ form about its procedures and risks, and they were paid well. Why isn’t the consent form (for every trial) publicly available once the trial is approved?
Fact 7: In the U.S., the Institutional Review Board (IRB) would be shut down and investigated before any new trials could be opened.
Fact 8: A thorough investigation on all parts of this system failure is obvious. This, too, should be open and as public as soon as possible, and at all times.
Fact 9: Risk is an inherent part of clinical trials because risk is part of everyday life. Even when rules are followed, bad things can still happen. Patients aren’t stupid, so there is no need to shut down other clinical trials.
A Setback for Other Clinical Trials?
Amazing we even have to ask this question, right? Of course it will!
Only other researchers may think their trial will be ok because, oh let’s see, because that trial was different – yes, that’s it! That trial (take your pick):
- was in a different disease
- was in a different country
- was run by a company I don’t know
- had a different study design
- was a horrible accident that would never happen to me
- add your own excuse
But patients won’t care about any of that. They’ll steer away from danger or discomfort. And let’s face it, anything called a “clinical trial” isn’t comforting. And as far as whether the rule were (or weren’t) followed – that doesn’t matter either. The rules in the case of a clinical trial include the protocol, of course.
I am firmly in the patient/participant camp. Some may call this a bias, but I consider it the only worthwhile endpoint. I know patients want better answers NOW, and I know people in research deal daily with regulations and hubris.
There was clearly a system failure here, and people needlessly paid too high a price. Let’s find out why and make the pieces work together so people won’t worry about needlessly putting their lives on the line.
- Bloomberg Business (1/15/16)
- Herald Scotland (1/15/16)
- The Register-Herald.com (1/17/16)
- Nature.com (1/18/16)
- The Pharmaceutical Journal (1/18/16)
- Scientific American (1/19/16)
- STAT News (1/19/16 & 1/20/16)
All content © 2016 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.