Neurologist Edward Smith, MD, explains how patients and families approach clinical trials, what details are often overlooked, and why setting realistic expectations is an essential part of ethical clinical research.
Transcript
Typically when patients, families come to me regarding a clinical trial, they’ve done quite a bit of homework already. So oftentimes they’re very, well educated on not just the disease, of course, but, but the particular clinical trial and what the, what the drug being tested is. And, you know, the mechanism of action, even.
I think what’s, you know, often lacking, obviously, is, are the details about the trial, particularly the, the, the details of the visits and the actual study burden on the, the patient, the family in terms of duration of the trial, how many on-site study visits, how long those are, what some of those assessments are. Are there invasive assessments beyond just the blood draws, for instance.
So, but I’ll tell you, it’s extremely variable, as you can imagine. Again, some people again are very, have done, done their homework on the trial and have been looking at maybe other potential competing trials. And, and others may have just heard about it through a webinar or, or through a friend.
Over the years, I think that I feel like one of the main things I’ve learned to at least try to do a better job of is, without being a downer, or being pessimistic in any way, is to try to manage expectations within the context of clinical research or clinical trial.
I think sometimes, you know, and we’ve seen this with a number of now treatments that the gene therapy trials, in SMA and other conditions, and other, other trials, some families, some patients come to a trial with the idea that this is a treatment and it’s going to help them. That’s the hope, right?
But, but the reason it’s a trial, and I would just, I’ve just bluntly used the term experiment and say, “I want you to think of this as an experiment. What are we doing? We were testing a hypothesis.”
Not blindly, I mean, you know, clinical trials at least Phase 2, even some even Phase 1 trials will have a large amount of preclinical work, preclinical data supporting them, in, you know, from lab assays and animal models of mice and otherwise, to try to learn more about the safety, the mechanism of action, potential efficacy.
But, you know, at the end of the day, people, people aren’t mice. You know, we’re not monkeys. And in some ways we’re similar. But I mean, in many ways we’re different. And the diseases that are modeled in those animals don’t usually manifest exactly like they do in humans. Right?
So, so it gets very, it can get very complicated. And, and, I, you know, I think that that is probably one of the, one of the biggest aspects of clinical research, clinical trials that I try to explain from the very beginning.
We don’t know. You know, we don’t know that this investigational product, I try not to say treatment, you know, the, this investigational product, investigational drug, whatever it may be. We don’t know that it’s going to be helpful. It may be harmful.
And you, if you join this trial or if your child enters this trial, you know, we may find out things that we did not want to find out. We may find out that there was much more risk there than we thought, and that it’s actually a dangerous treatment.
The other side of that is we would hope that, you know, it’s a safe, it ends up being a safe, beneficial treatment and leads to FDA approval and becomes available in the clinic.
