Dr. Ellen Mowry & Dr Chris Luzzio

Parallel to the clinical trial. The patient will have access to the-, his or her original neurologist. In some cases, it could be the same person that’s actually responsible for the clinical trial at the medical center. So, there are two ways that it could actually go. A patient could just participate in the clinical trial and just report back to the clinical trials officer how they’re doing.

At the same time, they can still have their treating neurologist if that’s a separate person. But that person can’t be giving them new MS drugs, for example, and any medical management that might occur. I would have to know about it, or the principal investigator of the clinical trial would have to know about it. But in our medical center, for example, I’m often the treating physician and the-, and the principal investigator.

Dr. Chris Luzzio: To bring-, to determine whether or not a drug is efficacious and safe for human use it has to go through various phases of experimentation. First, it will go through an animal phase to determine, for example, does it have any efficacy in the-, in the animal models?

Dr. Ellen Mowry: The phases relate to, you know, first in human for early phase kinds of studies, where you’re testing out a new intervention that maybe hasn’t been tested in humans at all, or in humans, with the condition of interest. And then, the next phase, phase two, is really looking in a small group of individuals. Typically, in a randomized fashion, whether there’s any signal for or hope, basically, that yeah, this treatment might really have some potential for benefit, but this group is too small, maybe not representative enough.

Dr. Chris Luzzio: So, in the earlier phases, we determine the doses of the medication that was safe for humans. We determine whether or not it’s it has some efficacy, and is there a justification for a much larger clinical trial? And most all of my patients participate in phase three clinical trials, which are hundreds of patients. And-, by that point it’s becoming clear that the drug probably has some efficacy; we just don’t know how much and-, and that’s what the purpose of the phase three clinical trial is to determine is how much efficacy it has to justify going to market.

Study groups, in the context of trials, really refer to different groups within the population you’re evaluating receiving different interventions, essentially. And so, you know what those interventions are and how they differ depend on the trial. Sometimes you’re comparing one active intervention, say medication ‘A,’ to the standard of care that already exists, medication ‘B.’ Or you’re comparing two, you know, novel medications, ‘C,’ ‘D’ versus each other or combinations, add-ons, things like that. Sometimes, instead of comparing medication ‘A’ to medication ‘B,’ you’re comparing medication ‘A’ to nothing; to a pill that isn’t expected to have any impact, and that’s what a placebo is.

In those cases, say we’ll do medications ‘Z’ versus medication ‘Y.’ Like, is ‘Z’ better than the standard of care? Or, if it seems safe, what if we just gave everybody medication ‘Y’ and then added ‘Z’ to half of the people. One study group will get extra treatment, you know.

Blinding refers to not allowing some people involved in this study, whether it’s the participants or the people assessing the outcomes of the intervention, or both, from knowing what intervention a given participant got. And blinding is helpful because we all carry forward our biases about an intervention and whether we think it is likely to have an impact on the outcome ….

In clinical trials, we want to minimize that kind of bias ….

It’s important not to attribute the benefit of an intervention to the intervention when in fact, it’s something else. So, binding is really good for that.

I think that that is a failure of our health system, right? That leaves people vulnerable and unable to make, you know, informed decisions about participating that are truly, you know, blind of other things.

I’m grateful that there is something I can do. There is a medication that I can get for them through a clinical trial. Because I’m terrified if they aren’t able to access a medication like, how am I going to be able to help them? But that’s ridiculous, right? That’s like living within the constraints of a really broken system. I think that the more equitable thing would be to say, like, you know, say you’re comparing standard of care to a new medication, right? Like, neutralize that experience like they should be able to get the standard of care or medication, whether or not they’re in the clinical trial. I think it’s reasonable to incentive in the clinical trial that maybe you say like, oh, you don’t have to pay the copay or whatever, but it’s not like you can’t get treated if you’re not in the trial.