Aug 10, 2018
Roberta Keller, MD dicusses the importance of clinical trials and clinical research in general, but particularly in newborns. Dr. Keller is a neonatologist from UCSF’s Benioff Children’s Hospital in San Francisco.
I'm Dr. Roberta Keller from the University of California, San Francisco, Benioff Children’s Hospital in San Francisco. I'm a neonatologist, and my clinical and research focus is on newborns and children with cardiopulmonary disorders.
Today I'm going to talk a little bit about the importance of clinical trials and clinical research in general, but particularly in newborns, as that's where most of my experience has been.
I've participated and helped lead a number of trials trying to better understand the respiratory outcomes in extremely preterm babies with underlying respiratory disease or either minimal or severe, and interventional trials as well in trying to prevent a condition that we call bronchopulmonary dysplasia, as well as later respiratory disease in former preterm newborns. In addition, I've participated in trials in trying to improve the outcomes of fetuses and newborns with congenital diaphragmatic hernia, which is a birth defect which affects the development of the lung and the lung vascular band.
I think a lot of times, people wonder why they should consider having their child participate in a trial. Honestly, I can't imagine anything more difficult than being faced with having a sick newborn and having to make the decision about whether or not to participate in a randomized study, but even an observational study where we just collect information over time.
First, I'd like to assure everyone that there are federal standards, and even international standards, for how we conduct research and protect the privacy of our research subjects, how we approach people for consent for trials, and how we go about evaluating and reporting and sharing potential adverse events or negative events associated with the trials. I think as investigators, we all take those responsibilities very, very seriously, and of course, our institutions take those responsibilities very seriously.
The second thing I'd like to say is that truly all of the advances we've made in medicine that have helped us care for children with these conditions have been made through doing research. Through research, we've really been able to learn that there are some real interventions that can help children who were not surviving before survive, or among those who do survive, really improve their status and help them grow up to be healthier children who can do the things they like to do, and enjoy time with their families, and build new relationships, and grow as people.
Given that background of those two things, I take my responsibility as a clinical investigator and a clinical trialist very, very seriously. When I speak with parents about potentially participating in a clinical trial, of course I always like to best understand how they think about their child's condition at the time and what they understand might be the longer-term effects of their child's condition. Then I talk about what the potential benefits are of the intervention we are studying. Of course, they're only potential benefits. None of us can ever know if an investigational medication, and we call it investigational drug, is going to be beneficial for a particular condition. We always call that investigational drug, even if it's a drug that is approved by the FDA or another body to treat a different condition. We always consider it investigational for the condition of the child that we are trying to treat at the time, or it may be approved in a different population.
I try to address as best I can what the potential benefits are, what the risks are that we know, what the potential risks are that we don't know, and try to reassure people that we will be monitoring everything very carefully, and collecting the information and sharing the information as we can in a way that will help them understand if they want to continue in the trial or if they feel that they can't contribute any further.
I personally am very, very appreciative of anybody who chooses to participate in research. I really hope that they are able to recognize how important this is for the ongoing treatment of children who share the condition that their child has.
A randomized controlled trial often has some sort of a placebo or a sham or a non-interventional arm. Particularly in the setting of a blinded study, we don't know who is randomized to that arm versus randomized to the interventional arm. I think as investigators, we're absolutely required to minimize any risk associated with giving a placebo drug or any other kind of intervention where we're needing to maintain blinding to the practitioners.
I think that both placebo and blinding are hard to understand. Really, the idea behind blinding meaning that the people who are caring for the child, including the family, and the investigators who are conducting the trial don't know which arm of the trial, whether the child is getting an intervention or a placebo or a sham, they don't know which arm of the trial the child is in through the conduct of the trial. The reason for that is because there may be ways that we can influence how the information about that child is collected and how that information about that child is understood. That really harms the integrity of the trial and the ability to make important conclusions from the trial in a way that we could, again, go on to help children going forward. I think that is a really challenging thing to explain to people, but again as investigators, we are committed and obligated to minimizing any risk associated with a lack of intervention, just as we are committed and obligated to minimizing any risk associated with an intervention.
I think most families, as they should, put their child's welfare and health above everything else. There's obviously a large leap of faith in trusting that we as investigators have done what we need to do to minimize risk and maximize potential benefit, and we're also helped in that by our institutional review boards who oversee any patient-oriented research. But we're absolutely obligated, but it absolutely requires a leap of faith for families to accept that. I think that is an important motivation, important consideration, but I also see that families very much feel that, to the extent they can, that they want to contribute to improvement in care of children going forward.
I'm Dr. Roberta Keller, and I'm aware that I'm rare.
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