Understanding Clinical Research – PediaCast 538
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Show Notes
Description
- Dr Cynthia Gerhardt and Myeshia Harmon visit the studio as we consider pediatric clinical research. How are clinical trials designed, approved, funded and implemented? How is safety woven into the process, and how are results shared with the medical community and general public? We answer these questions and more!
Topics
- Pediatric Clinical Research
- Clinical Trials
- Translation of Research into Practice
Guests
- Myeshia Harmon
Director of Clinical Research Operations
Hematology, Oncology, Bone Marrow Transplant
Nationwide Children’s Hospital
Links
- Clinical Research at Nationwide Children’s Hospital
- Pediatric Trials Network
- Should My Child Join a Clinical Trial (Healthy Children)
- Doing the Work to Improve Diversity in Pediatric Clinical Trials (AAP)
- Evidence-Based Guidelines for Pediatric Clinical Trials (NIH)
- ResearchMatch
- StudySearch
Transcription
Announcer 1: This is PediaCast.
[Music]
Announcer 2: Welcome to PediaCast, a pediatric podcast for parents. And now, direct from the campus of Nationwide Children's, here is your host, Dr. Mike.
Dr. Mike Patrick: Hello, everyone, and welcome once again to PediaCast. It is a pediatric podcast for moms and dads.
This is Dr. Mike coming to you from the campus of Nationwide Children's Hospital. We are in Columbus, Ohio.
It's episode 538 for May 17th, 2023. We're calling this one "Understanding Clinical Research". I want to welcome everyone to the program.
We have another important topic for you this week, as we consider pediatric clinical research and the role that clinical trials play in advancing pediatric medicine and improving health outcomes for children, teenagers, and families.
0:01:09
And it turns out it's a timely topic because Clinical Trials Day is right around the corner, on May 20th. And that's an opportunity for us to raise awareness about the importance of clinical trials, which is what we are going to do today.
You know we often use the words evidence-based on this podcast, but what exactly does that mean? How do we arrive at evidence? And why should parents and the general public trust us when we say our recommendations and management plans are evidence-based?
Well, that evidence often comes from clinical trials. But again, what exactly is a clinical trial? How are they designed and approved? How do researchers wrestle with ethical considerations? How is safety implemented despite trying something new?
Of course, that something new could be a drug or a procedure or an activity or education and guidance. Whatever that something is, how do we proceed safely when the results are not certain?
0:02:09
Well, we are going to answer these questions and many more as we explore the world of pediatric clinical research.
And I also want to add for many folks, and I will include plenty of medical professionals in this bunch, especially those who primarily practice medicine and are not necessarily involved in conducting clinical research. For many of us, research and clinical trials are a little bit like a black box. A question goes in and an answer comes out.
But what is inside that box? Well, today we're going to open it up and peer inside again as we explore the design of pediatric clinical trials.
Also, how are they funded and approved? What safety features are part of the process? We'll talk about informed consent, participation in trials, and compensation.
And we'll consider the mechanism by which the results of clinical trials are shared with the medical community and how these results impact the way in which health care is delivered.
0:03:07
So lots coming your way as we journey together through the world of pediatric clinical research and clinical trials.
Of course, in our usual PediaCast fashion, we have a couple of terrific guests joining us. Dr. Cynthia Gerhardt is the chief clinical research officer for Nationwide Children's Hospital. And Myeshia Harmon is Director of Clinical Research Operations for Hematology, Oncology and Bone Marrow Transplant at Nationwide Children's Hospital.
Before we get to them, let's run through our usual quick reminders. Don't forget you can find PediaCast wherever podcasts are found. We're in the Apple and Google podcast apps, iHeartRadio Spotify, SoundCloud, Amazon Music and most other podcast apps for iOS and Android.
If you like what you hear, please remember to subscribe to our show so you don't miss an episode. Also, please consider leaving a review wherever you listen to podcasts so that others who come along looking for evidence-based child health and parenting information will know what to expect.
0:04:02
We're also on social media. We love connecting with you there. You'll find us on Facebook, Twitter, LinkedIn and Instagram. Simply search for PediaCast.
And we also have that handy contact page over at pediacast.org, if you would like to suggest a topic for a future episode.
Also, I want to remind you the information presented in PediaCast is for general educational purposes only/ , we do not diagnose medical conditions or formulate treatment plans for specific individuals. If you have a concern about your child's health, be sure to call your healthcare provider. Also, your use of this audio program is subject to the PediaCast Terms of Use Agreement, which you can find at pediacast.org.
So let's take a quick break, we'll get Dr. Cynthia Gerhardt and Myeshia Harmon connected to the studio. And then we will be back to chat about pediatric clinical research. It's coming up right after this.
[Music]
0:05:19
Dr. Mike Patrick: Dr. Cynthia Gerhardt is a clinical psychologist at Nationwide Children's Hospital and a professor of pediatrics at the Ohio State University College of Medicine. She also serves as the Chief Clinical Research Officer for Nationwide Children's.
Myeshia Harmon is Director of Clinical Research Operations for hematology, oncology and bone marrow transplant at Nationwide Children's Hospital. And she's serving as interim director for Clinical Research Services. She has over 15 years of experience in coordinating clinical trials and implementing new and novel research.
Both have a passion for advancing pediatric care and improving outcomes for children, teenagers and families through clinical trials and research. That's what they're here to talk about today, pediatric clinical research.
Let's start with a warm PediaCast welcome for our guests, Dr. Cynthia Gerhardt and Myeshia Harmon. Thank you both so much for visiting with us today.
0:06:08
Dr. Cynthia Gerhardt: Thanks for having us, I'm happy to be here.
Myeshia Harmon: Thank you so much. I'm so happy to be here and share with you guys today.
Dr. Mike Patrick: We are really glad to have both of you with us, two very experienced experts on clinical research. And I think where we want to start is really just a definition. Myeshia, if you could let us know what exactly is clinical research?
Myeshia Harmon: Oh, absolutely. Clinical research is where we have people or data or samples of tissue that are studied to understand health and disease. Clinical research helps us find better ways to detect, diagnose, treat and prevent disease. There's different types of clinical research. You can have observation types of research projects, also clinical trials. And where we are looking to gather information about either a new treatment or how a new treatment may work against an existing treatment.
0:07:00
We're looking to see if there's a primary way of determining if there's a new form or prevention or something, or a new medical device that we can see if it's effective and safe in humans. It's usually designed to learn if the new treatment is more effective or less harmful, if there are side effects. We're looking to test new ways to diagnose diseases early sometimes before there are symptoms.
We're looking to find approaches to prevent a health problem, including people who are healthy but at increased risk of developing a disease. We're looking to improve quality of life sometimes in clinical research. We also study the role of caregivers and support groups.
There's different types. And there are a lot of those types of projects that are happening at Nationwide Children's Hospital.
Dr. Mike Patrick: So when you say that you're testing something, I mean that something could be a medication or a drug, whether it's a new one or you're comparing one drug treatment with another drug treatment. It could be a procedure. It could be an activity, could be education or counseling.
I mean, there's just so many different things that you could be testing. It's not always just medicine, correct?
0:08:09
Myeshia Harmon: Right. So that was the next point I wanted to make, is that there's different things that we can be looking at. And you just described those pretty clearly.
Dr. Mike Patrick: And then, when we talk about clinical trials in pediatrics, it's not exactly the same as research in adult medicine, right? Cynthia, how is it different when we look at pediatric research versus adult research?
Dr. Cynthia Gerhardt: Sure, that's a great question. So pediatric research involves more considerations for safety and ethical implications of our work. So, for example, children are considered a vulnerable population because they can't often developmentally understand or fully understand the purpose or risks and benefits of research participation. So this is why we often have to obtain informed consent or get permission from their parents for them to be involved to participate.
0:09:04
And then, we also allow children who are at least nine or older to be able to assent, to be involved in research. So this means that they also have some say in what their participation involves if they're a bit older.
And in addition, some types of treatments or interventions may have particular risk for younger children that may not be apparent in adults. And so, we need to be really thoughtful about ensuring the safety of the children that participate. And this is why we take extra precautions to ensure studies are carefully reviewed by our Office of Human Research Protections Program and or other regulatory services in the hospital.
And then, we also ensure that all the staff have specialized training to work with these pediatric populations.
Dr. Mike Patrick: Yeah, so important. And there's so many more pieces, parts to a clinical trial in pediatrics versus adult medicine that in the past, a lot of times, especially with drug manufacturers, would be like, "Well, we don't even want to spend the money to get this approved in kids because we got it in adults. And if pediatricians want to use it, it just has to be off label."
0:10:16
But that's changing, right? We're really trying to study things in kids because kids aren't just small adults. And so we want to make sure that things are safe and effective for them and not just assume it is based on adult studies.
Dr. Cynthia Gerhardt: Absolutely. That's a big challenge. We do have a lot of medications or drugs that we just don't know how effective they are in children and adolescents or what the safety issues may be. And they are not little adults. So we do need to study them separately and involve them and find treatments that are unique to them.
Dr. Mike Patrick: And then Myeshia, when we think about the results of clinical trials, it's not always a, "We have this question, we're going to design some sort of a study, we get an answer and then suddenly things change." How do clinical trials kind of play into advances in the practice of medicine?
0:11:10
Myeshia Harmon: So, clinical trials and the advances of medicine, we're looking at the study of health and illness in people. We're looking to see what information we can provide along the way of the trial. We're looking at the new information that changes the risk-benefit profile, including the identification of new risk that might happen, an increase in the magnitude of what is known and what is not known.
We're looking at study procedures that may have been added and modified and new alternative treatments that become available. So those are the things in what we're looking to see how we can advance the field of medicine and the information from these trials used to make new decisions, new treatment recommendations, what may be the standard of care in the future, those are the things that we're looking at and using these trials and clinical research to advance the field.
0:12:06
Dr. Mike Patrick: So sometimes, there are big landmark studies that really change things. And I guess a good example of that would be the study of the COVID-19 vaccines. Like, this was a big trial with lots of people and a big change came about.
But more often, really, changes in medicine come about from looking at several studies, right? Like, you have to look at the whole context and background, what's gone before, how does this add to it? What do these results mean in light of maybe results from other studies?
And so, often it is sort of a complex picture that then we have to put together many pieces of results in order to translate those results into a new practice or something that becomes the new standard of care. It's pretty complex, right? And a lot of thought goes into that.
Myeshia Harmon: Yes, definitely, a lot of thought goes into it. And especially in pediatrics, we're very careful about the trial and what the recommendation is at the end and making sure that we remain compliant and that our data is clean and good so that we can advance the field.
0:13:09
We want to make sure that what we're doing is accurate. And that when we appeal to the FDA or a regulatory body that we do want to add this as a new treatment, we want to make sure that everything that we've done is contributing to advancing the field for our patients.
Dr. Mike Patrick: And sometimes, it may begin as a small study. And then, you want to know, does the same results reproducible with a much larger number of folks? And if you could also speak toward diversity in recruiting folks to be a part of trials, because we really want to represent all of kids, right? Not just one particular subset of children.
Myeshia Harmon: Yes. We definitely want a diverse population that's enrolled in clinical trials because we want to make sure that the medication or the procedure, if it's an observation study, that we're looking at a wide variety of children or patients and teenagers. So that we know how the intervention, whatever it may be, will perform across different types of patients. We want to make sure that we're looking at girls and boys, if that's appropriate.
0:14:18
We're looking at different folks with different ethnicities and how medications may synthesize in their body, because we don't all metabolize things the same way. And as we advance the field, we want to make sure that the medications or interventions or whatever it is that we're looking for, that we can recommend that it be used in multiple populations. Or if there's something where it doesn't work appropriately, that we're able to have that data and have that information and make sure that our physicians and anyone who will be treating our patients is aware of that.
Dr. Mike Patrick: Yeah, absolutely. Cynthia, if you could just describe, and you've done this many, many times designing a clinical trial, what's the thought process and how do you get to the place where you know what you're going to do?
0:15:09
Dr. Cynthia Gerhardt: That's another good question. As you said, sometimes, these trials are resulting from multiple studies that happen beforehand. And when you're working in the lab, obviously, there's often pre-clinical trials or animal studies that happened when you're developing a drug or testing a device. And then, those will evolve into the first in human studies or some of the early pilot data to establish safety and efficacy before it eventually becomes a common treatment or medication that's rolled out.
We often think about bench, to bedside, to beyond or to the community so that we're hopefully advancing public health within the communities around us. And there are lots of considerations in terms of thinking about what do we already know, and then how do we fill those gaps from our early studies to begin to piece together what will or won't work.
0:16:11
And so, it takes a bit of time. And Myeshia can probably speak a bit to the more technical pieces of going about conducting the clinical trials because we have a wonderful infrastructure here with clinical research services that can actually help investigators run their clinical trials at Nationwide Children.
Myeshia Harmon: Thank you, Cindy. As Cindy stated, we have a robust team within clinical research services that includes our feasibility team that is going to look. And once you've come out of the pre-clinical phase of your research, when we're ready to test new medications and devices in humans, we go through a pretty robust review process.
Looking at our patient population at the hospital, do we have enough patients to be able to meet the aims of the study? Is the study feasible to be done not just from a patient population, but also the funding and the budget? So we're looking to make sure that if it's an industry study or an investigator-initiated study, that we have enough resources to support the trial.
0:17:09
And we're reviewing that. And we do a robust review with Legal and our IRB to review to make sure that we're protecting human subjects.
We're reviewing your protocol to make sure that you have the endpoints that you need when you're building your database for data. Are you collecting the appropriate amount of data? We don't want you to ask questions that you're not going to need later, because we want to make sure that your study teams and those that are helping you collect the data are able to give you good data that you can use. You don't want to collect too much, but you also don't want to collect too little. So we're looking at those things.
We're working with Legal to negotiate any contracts you may need with vendors or co-investigators that you may work with other sites. If it's a multi-site study, we're making sure that we have our ducks in a row contractually. Legal is reviewing any contractual documents to make sure we're protecting ourselves as an institution, we're protecting our principal investigators and study staff, that we're making sure that our patients and families will be protected. And that we are looking at the risks of us running a trial and the legalities that come with that.
0:18:17
We're making sure that our consent form is informing our patients and families about the study, what our principal investigator may be involved in? What happens if they get injured while they're participating in a trial? We're looking at all of those things to make sure that we have covered our bases and that we're informing the study team, our principal investigator, and our patients and families accurately about the study.
The IRB is reviewing the science and reviewing the studies to make sure that we continue to protect our human subjects and our patients that participate. We have compliance folks here that look at the study while it's going on, to make sure that the data that we have submitted is accurate and that we're compliant to the protocol, that we're not deviating unnecessarily.
0:19:00
There are instances where you might need to deviate. But all of that is a pretty robust team within clinical research services that not only has a whole team that can help you conduct your trial, but we're also looking at your trial to make sure it's feasible and that you're going to be able to meet your study aims once you've completed your work.
Dr. Mike Patrick: So many important pieces and parts to this whole process that I'm sure most folks don't even recognize as happening behind the scenes, that's really important in terms of making sure that the study is going to answer what you want it to answer and also that you're going to do that in a safe fashion or as safe as possible.
I want to take one step back because the primary audience for our podcast today is parents. And not all parents have a science background. And so I really wanted to talk about clinical research at a very basic level.
And starting with, it sort of begins with a question, right? Maybe it's based on observations that an investigator has seen, maybe it's the result of other studies that have come before it. But you have a question in mind of, like, could this particular medicine work better than the one we're currently using? Or can a particular new medicine work for a particular illness?
0:20:19
And so, once you have that question, then you're going to recruit people to be involved. And often, sort of at the simplest design of a study, is that you're going to divide them into an experimental group and a control group. And the thing that's going to be different between those two groups is whatever it is that you're studying.
And you really do want to try to make sure that all the other factors are as equal between the two groups as possible, so that you can say that whatever our intervention is, is the thing that made a difference because everything else is similar.
So at that very basic level, and then you start to add on all those things, Myeshia, that you were talking about, which are also important. But it all just starts with that simple question in trying to get at an answer, right?
0:21:03
Myeshia Harmon: Definitely. It takes a lot to be able to answer that question. So all of those checks and balances are important, because the last thing we want is for a patient or a family to know that a trial is coming or be anticipating that my child is going to have access to this new and novel therapeutic. And if we don't do all of our checks and balances on the front end, we may have to not open the study or not be able to offer that treatment and send a patient somewhere else, which is that's not what we want to do.
Or it could be a situation where they've started the trial, and then we have to withdraw them from the trial because there was not a proper checks and balance. So we're really working hard to not have that happen. And making sure that we have enough patients, the team understands the study, that we're able to execute the research procedures that have been identified. And that we're able to make sure that our patients and families understand what is going to be needed of them once the trial is available for them to enroll on.
0:22:03
Dr. Mike Patrick: Yeah, and that whole system of support for the principal investigators is just so important, because they may be a fantastic scientist, but when it comes to making sure do we get everything checked off, we don't want this have been for nothing because we didn't mark a box. And of course, we want it to be as safe as possible for kids and families and be able to explain to parents that this is why we feel that this is a safe thing to enroll your child in.
You had mentioned also a couple of times this thing called an IRB. IRB is an institutional review board. Cynthia, can you explain to us what exactly is an IRB? And pretty much, they exist everywhere that clinical research is happening, right?
Dr. Cynthia Gerhardt: They do. So we have an IRB. I think we have a full force that has about 17 different members. Most of them are often scientists or clinicians who care for children and families.
0:23:00
And we also have a few community members who get to weigh in on the kinds of research that we do. And their job is really to ensure that the work we're doing follows all the local, state, federal guidelines that we need to engage in to ensure the safety of the participants in our research. But also to ensure that we're treating families and their children in an ethical way. As I mentioned, children are often seen as a vulnerable population because they're not developmentally able to make decisions, sometimes for themselves. And so, we have a lot of protections that are in place.
And so, the IRB ensures that we're following all the regulations, ensuring that we're getting informed consent and permission from families for them to participate, that they understand the purpose of the study, the risks and the benefits and what their involvement will entail. And so all of this is to ensure that families know that their participation is voluntary and that they understand exactly what the purpose of the project is.
0:24:08
Dr. Mike Patrick: And the IRB piece of this comes after you've designed the whole thing, correct?
Dr. Cynthia Gerhardt: Yes.
Dr. Mike Patrick: So you've done all these checks and balances, you've figured everything out, and then you put it before a board with 17 members, and they get to poke holes in it and say, "Wait, why aren't you doing this?" Or, "Why aren't you involving this group of people?"
And I guess that collective community approach really helps to make sure that we're being ethical in what we're doing, that we're being thoughtful, that we're going to get good results and not harm people in the process.
Dr. Cynthia Gerhardt: Absolutely.
Dr. Mike Patrick: So important. And then, so once, let's say that we've designed a trial that's going to give us some answer. And it's gone before the Institutional Review Board and they have approved it, now we are going to need some money to make things happen.
And Myeshia, you had mentioned it may come from industry, it may come from the hospital. There's lots of places where funding can come from. What are some of those places?
0:25:08
Myeshia Harmon: Absolutely. We can be funded by governmental agencies like the NIH, Department of Defense and the VA. They all provide funding and support clinical research. You can receive funds from a private sector. I mean, we have many instances where there's a family who may have a child that had some sort of disease or health disparity that they want to further research. So they may make a gift to be utilized for clinical research or want it to be used in some way, shape or form to further the field.
We have pharmaceutical companies, like we talked about, that they may have a financial stake in the completion of the trial. They might have a drug that didn't work for something but may work for something else. So they will provide funding to help us run the trial and provide the resources we need to continue to do the work that we need to do to get the trial completed.
0:26:02
Academic medical centers also provide funding. Our hospital has several vehicles where you can apply for funding to be able to support your research. We have the Intramural Funding Program where you can apply and have an idea, and it's reviewed here by that board. And they may provide opportunities for you to receive funding for your clinical research.
Some of the divisions may have funds where they want to support a physician or a study team to do research. There's all sorts of avenues to obtain. You can apply for grants.
It's great. We love to see when someone comes to us with a nice pot of funding for us to be able to do the research the way it is intended, but it doesn't preclude us from doing research. NCH is very good at trying to find ways for us to move the research forward and they support clinical research.
And there's an instance, for instance, in HemOnc, if there's a trial that doesn't have enough funding, some of our investigators can apply for divisional funds to cover said gaps. Because we believe in the science, we believe in the research, and we want to have this opportunity for our patients and families.
0:27:11
Dr. Mike Patrick: Yeah, really important. And I'll also point out that, really, anyone can support Nationwide Children's Hospital and the research that we're doing through the website. And the money that you give specifically for research really is used for research, correct?
Myeshia Harmon: Yes, it really is used for research.
Dr. Mike Patrick: And we'll put a link in the show notes so folks can find that exactly how you donate money. And since Nationwide Children's is a nonprofit organization, it's tax-deductible in whatever ways are lawful, right?
[Laughter]
Dr. Mike Patrick: All right, then once we have funded our study, we've designed it, we've gotten it approved, we've got it funded. Then how do we go about recruiting patients and families to be a part of a trial, Cynthia?
Dr. Cynthia Gerhardt: Well, I think depending on the study, this can happen in a variety of ways. So most of our studies may have particular types of patients or families that we're looking to include. So, that may be because we're focused on a specific disease or particular age of child that we want to include in the study.
0:28:18
So there may be some criteria that we consider when we're looking for participants in studies. And we can recruit from a variety of places. So some of our studies at Nationwide Children's will approach families directly in clinic or when they're in the hospital to invite them to participate in a project.
Other studies may contact families by phone or in the community. And we do actually a lot of research in the community, collaborating with schools, visiting families in their homes to do surveys, as well as inviting them to the hospital to participate in our research.
0:28:59
So oftentimes it's nice to be able to recruit families in person and be able to really fully explain the project. But nowadays, and with COVID as you might imagine, some of that really had to switch to remote recruitment. And so, families can now do a lot of their surveys electronically or be involved in research in a variety of ways.
And there are different programs or websites where you can actually sign up to be a participant in the study. So, StudySearch and ResearchMatch are two web bases or registries that we often use where families can volunteer to participate. So it just depends on the project and the objective of the study.
Dr. Mike Patrick: And we'll put a link in the show notes for ResearchMatch and other places where you can find studies that you might be interested in enrolling yourself or your child in. And we also have some of those at Nationwide Children's Hospital in terms of announcements of what studies are out there and who they're looking for. And we'll put a link to that in the show notes as well, so folks can find it.
0:30:08
Dr. Mike Patrick: I think that from a very practical standpoint, it makes sense for parents to say, oh yeah, the clinical trial is important. We're going to advance pediatric medicine that's going to help all kids. But, at the back of your mind is always, is this something that's safe for my child to participate in? Especially as their parent, you're going to be asking that question.
And so we know that nothing is 100% risk-free. And with a clinical trial, there may be some adverse effect that we don't know yet. Or it could be that we're withholding the standard treatment and doing some new thing, but the standard one would have worked better. There's always that chance.
And so, how do you go about kind of walking parents down that benefit-versus-risk road? And I guess to some degree, it depends on the family's individual risk tolerance. Some people accept more risk than others. And so, that can be a tricky conversation, right?
0:31:00
Dr. Cynthia Gerhardt: Yeah. That I think is often challenging, because some of the kinds of treatments or devices that we may be testing are maybe a little bit more complicated. And even the best of us have a hard time when we go to the doctor and hear about all of this medical information.
So as part of the process of having our studies reviewed through the Institutional Review Board, they make sure that the materials that we provide families to explain the study and the consent forms are at a readable level. So that it's very easy in plain language for them to understand. And we train our teams to be able to walk families through and answer questions when we're going through that consenting process.
So they, as I mentioned before, fully understand what the participation means to them and then what the risks and benefits are. And for research with kids, we're really clear that benefits really need to outweigh the risks of their participation. And so, it's not common that we would have a significant, severe, high-risk kind of study happening.
0:32:11
Most of the types of risks that we may go through might be boredom from filling out questionnaires, or it might be kind of minor symptoms or reaction to side effects from a medication. But we definitely need to kind of go through all of that so families are fully informed and they can make that decision of their own free will.
Dr. Mike Patrick: One thing we've not talked about yet, is a blinded study versus not blinded. But then, as parents get enrolled and you're moving forward, there may be some instances when you aren't really sure if your child's getting the new treatment or not, right, Myeshia? How do you explain that?
Myeshia Harmon: Yes, I mean, there are times where you can be blinded where the pharmacy or the parents may not know or there's just one party that's blinded. And then there's times where you could be double blinded in a study where no one knows, whether that is the study team and the pharmacy, or the study team and the parents and families. We may not know what medication is being given, so that way at the end of the study, kind of compare those two cohorts or arms and see what the outcome is.
0:33:17
So there is several times where you can be on that type of study. And usually what happens is that you're randomized to one of those arms, which means it's chance. Most times it goes into a computer system where it's putting in the information and the computer spits out what arm, based on criteria or different things, what arm that patient should go on.
So it is not the study team picking. We're literally putting your information into some sort of computerized system and you are picked at random. That's literally what it means to be on a randomized trial. You may go either way, depending on how the computer spits that out.
Dr. Mike Patrick: And once you are on an arm, you're stuck on that arm, right? There's no switching from one arm to another. If you find out like, "Oh, my child got treatment A, but I really wanted them to get treatment B." You can't move, right?
0:34:13
Myeshia Harmon: In most instances, you can't move arms. But I will highlight that everyone has the option to withdraw a study if they are randomized. If it is a situation where they know the treatment that they're randomized to and it is not what they wanted, they are allowed to withdraw. We don't encourage that because we want the data to be balanced.
But any patient which, we want to highlight that anyone who is involved in clinical research has the option to withdraw at any time and should have an informed conversation with their medical providers about next steps. What does it mean if they withdraw from the study? What are the other options? They should be discussing that. But yes, they do have the option that if they're not happy with what they were randomized to, they can withdraw.
Dr. Mike Patrick: And the important thing here really, is those open channels of communication and really explaining to folks right from the beginning everything about this trial, what to expect, but then continuing to have those conversations when questions come up.
0:35:14
Also, during the trial and at the conclusion of the trial, do families just have to wait for a study to be published to find out the results? Or do you share the data that you're collecting during the trial or at the conclusion of the trial? Or does it really just depend on the study in question?
Myeshia Harmon: Well, the federal regulations require that, in our informed consent that we make sure that any significant new findings we will share with the family. That can be in a variety of mechanisms. We can send a patient family letter. There may be an amendment to the trial because there was a new finding where we might need to change the original protocol.
Informed consent doesn't just happen at the beginning. It is an ongoing process. Your treating physician, your principal investigator, your study team should have ongoing conversations with you about the nature of the trial, what's happening, if there's a new risk, if there is a reason to close the study down early. All of that type of information should be communicated to our patients and families to let them know what's going on with the trial.
0:36:21
We may provide a telephone call. Some of our consortiums may send a letter or a note, letting folks know what's going on with the study. But your physician and your study team should be updating you during the progression of the trial.
And then at the end, the final results should be made available to everyone via lay summary. It may be published in a medical journal. It may be presented at a conference. A sponsor, if the medication has done well and they're going to go after a new drug approval, they may put out a press release.
So there's all sorts of vehicles and mechanisms for patients and families to find out what's going on. Pretty much most of those trials and those publications are written in a way that you cannot identify yourself as a participant in the trial.
0:37:11
For smaller trials, folks may be able to say. Sometimes trial participants, they make friends with other families if they're all going to the same treating institution for a trial. But, no layperson or the public should not be able to identify our clinical research participants.
Dr. Mike Patrick: That's a really important point. We think about HIPAA and privacy as it relates to medical care, but that also relates to medical research too, right? Yeah, so really very important.
So, we share information with families in one way or another. And then the other place where it's going to be important to share results coming out of a trial is going to be with the community and with the medical community, but maybe even with the community at large.
So Cynthia, how do we go about sharing the results of research with the medical community and the general public?
0:38:06
Dr. Cynthia Gerhardt: Well, I think that sometimes can be a challenge too, as much as we try and get the word out. Marketing and communications have been wonderful at Nationwide Children's and sharing and disseminating some of our findings from our research.
But, the investigators are often networking and sharing this information at national and international scientific conferences where they're presenting their work and talking about that, publishing in journals that other scientists and clinicians and providers are reading. And there is a push now to really get our science into the communities faster, so that we're really translating the work that we're doing in the lab to the community and improving care more quickly.
And so, there's more funding for what we call implementation trials, which are kind of real-world trials, where we're not just doing our science in a lab, in the hospital, or at a particular institution, but we're actually working with providers, in the community, to teach them how to deliver this care or to offer these procedures or devices to kids and families directly.
0:39:18
And I think that's a really important change in the way we do our science to help expedite that process, to ensure that families in the communities that we care for are having meaningful changes and improvements in their health because of our discoveries.
Dr. Mike Patrick: Yeah, absolutely. Traditionally, the main way that this information was disseminated was through peer reviewed journal articles. And of course that still happens and it's still a very important part of the advancement of medicine.
For one, it's peer reviewed. So that not only now do you have your institutional review board looking at things, but now after the fact, you also have folks from other institutions also looking through and making sure that they agree with the science and that it was done in a sound fashion.
0:40:07
And then, also anything that's in peer review journals can be easily looked up on down the road. There's a historical data that basically gets published so that you're able then to look things up and find it. So all important.
On the other hand, when you look at the number of physicians, nurses, other researchers who use digital media and social media, having it in the journal is important. But maybe it gets out there sooner and in a more personal fashion through things like this, like a podcast.
And so that's really one way in which the dissemination of information has changed quite a bit over the last ten to 20 years.
Dr. Cynthia Gerhardt: That's absolutely true. I think sometimes word spreads a little bit faster when you go viral…
[Laughter]
Dr. Cynthia Gerhardt: In social media. And you do, it is an easier way. It's how so many people communicate with each other now, and it's a wonderful way to share.
Dr. Mike Patrick: Have any of your PI's done TikTok videos yet?
0:41:07
[Laughter]
Dr. Cynthia Gerhardt: We do have some folks that do that. And I will say a lot of our investigators are also called to participate in national or international working groups that are creating guidelines for care based on what the findings are.
So those types and advocating for policy changes both at local and state government levels, but also nationally. And I think that really makes a big difference in improving public health and the health of our kids and families.
Dr. Mike Patrick: Yeah, that really is so important at the professional level, because a lot of times, the individual physicians who may be in private practice or working in a subspecialty clinic that's very busy, one of the primary ways that they get their information is from their professional society's journal of choice.
So, for the American Academy of Pediatrics, that would be Pediatrics is the name of the journal. And oftentimes, there are clinical practice guidelines and clinical pathways that get published in that journal. And that often is the result of committees looking at the results that have been published on particular things to come to some consensus.
0:42:15
And that's really then how research is translated into practice through these clinical practice guidelines put out by professional organizations. And as you said, that takes doctors and researchers serving on those committees to synthesize all that information into a recommendation.
And that's another really important piece of all of this. And I think for the public in general, there's been some skepticism I feel like, especially during the pandemic, because recommendations changed and then some folks would say, "Well, you didn't know what you were talking about." And, "Why should we trust you now with this new recommendation?"
But when you really consider all of the thought and work from the very first idea of a study and then designing it, getting it through review boards, getting the data or getting it funded, getting the data, getting it published disseminated and then having enough people to agree with what you've done to make it into a practice guideline, that's a whole huge series that I feel if you're going to trust something that should be trusted. Wouldn't you agree?
0:43:22
Dr. Cynthia Gerhardt: Absolutely. It is a very long process that's vetted many times along the way. And we're always learning. So there are changes we really take to heart and information we get from children and families to continuously improve the care that we're providing.
And we do what we can to provide the best care at any moment in time, but then we want to do better. We always want to do better. And we always want to help ensure that we're providing the best care possible, so that we get the best outcomes.
Dr. Mike Patrick: Yeah, absolutely. And sometimes, if recommendations need to change, they need to change. The first recommendation was based on the best information we had at that time. And the next recommendation that may be very counter to the first one is based on newer, better data. And there may be a third one out there that we don't know about, but we just try to get better with each step along the way.
0:44:19
And I think that's what happened with the pandemic too, with do you wear a mask? Do you not wear masks? How long do you wear masks? Just that as we learn things, we implemented them. And sure, that's going to change as we learn more things, but that's the nature of science.
Dr. Cynthia Gerhardt: That's absolutely true. And I think what's really great about clinical research and our clinical science is that families and kids get to participate in it. And I've met so many families that just really appreciate the opportunity to be able to pay it forward. They know that their child's getting the care that they're getting now because so many other families before them participated in a clinical trial or participated in a research study that made a difference.
And so, they know that their participation will help the next family or the next child in that position. And, I really am proud of the fact that we do that and that we offer that opportunity to our families at Nationwide Children's.
0:45:13
Dr. Mike Patrick: Yeah, that is so important. Speaking of clinical trials at Nationwide Children's Hospital, I'm going to put a link in the show notes to clinical research at Nationwide Children's Hospital. And you'll be really surprised at just how much research is happening here.
Basically, once you get to that page, there's a link to each of our subspecialties, everything from adolescent medicine to emergency medicine, certainly hematology, oncology, neonatology, orthopedic surgery. Just the list goes on and on and on. And I would encourage folks to head over to pediacast.org.
This is Episode 538, and you'll find a link to the Clinical Research page here at our hospital. But Myeshia, what are some of the studies that are ongoing that folks may be interested in learning more about by visiting that page?
0:46:06
Myeshia Harmon: Absolutely, as you alluded to, we have a plethora of clinical research that's happening at Nationwide Children's Hospital. For example, Dr. Karen McCoy is looking at a phase three study that's evaluating the safety and tolerability of combination therapy for adolescents in cystic fibrosis.
She has been here for quite some time, and she's looking to see what's the benefit in evaluating these subjects and looking at the clinical data and presenting the study to obtain the pharmacokinetic data. Pharmacokinetics is really looking at how a medication may work in a child's body. And when we're doing that, we're taking samples or doing certain types of research procedures to see how that medication is working for our patients.
Over in our behavioral health sector, they are looking at the effectiveness of adding a novel digital storybook intervention platform to look at therapy to improve speech for our kiddos that are suffering with hearing loss. So they're using a story platform to try to improve outcomes for those children.
0:47:14
Our Gene Therapy team has a very vast portfolio. They're looking at gene therapy for patients with muscular dystrophy. We have long-term follow-up studies for kiddos that are also suffering from muscular dystrophy. We're doing natural history studies.
Our IGM sector, is right now involved in an NCH-led collaboration for molecular characterization initiative. That is looking to improve diagnosis for childhood cancer. Our IGM labs and that team, they are responsible for that initiative for all of the hospitals that are participants in our Children's Oncology Group. And folks can learn more about Children's Oncology Group, online. We're all participating in that consortium.
0:48:06
We're also having studies that are in our HemOnc Department, we have Dr. Setty, who's looking at how to have immunotherapy in combination with drugs for patients with relapsed or refractory, pediatric bone, and soft tissue sarcomas. So that's in our cancer sector.
Dr. Ranalli is looking at universal donor in NK cell infusions in combinations with chemotherapy for people with relapsed neuroblastoma.
We have gene therapy for sickle cell now. Dr. Rangarajan is looking at that. Sickle cell patients don't have a lot of therapy options. So right here at Nationwide Children's Hospital, we are looking at new and novel therapeutics to help that very wide and very large patient population.
We also have various studies that Clinical Research Services is overseeing throughout the hospital. Again, it's our centralized Clinical Research Group that is there to help you from the time of study concept till it's time to enroll patients and do that data collection and data analysis.
0:49:07
So, like you said, there's a lot of information at that website where folks can see the type of research that we're doing here. And if they have any questions, feel free to reach out, and we'll make sure that we get them to the right study team and the right contact to be able to have more knowledge about that research.
Dr. Mike Patrick: Yeah, and again, I'll put a link to the Clinical Research page at Nationwide Children's Hospital in the show notes over at pediacast.org, so folks can find that very easily.
Of course, advancing pediatric medicine is reason enough to get involved in a clinical trial. Is there any other compensation that goes along with it? Can folks get like an Amazon gift card or something? Or is there something else that you get other than just the knowledge that you've contributed to the future?
Dr. Cynthia Gerhardt: Yes, most of our studies do have some reimbursement for participants in terms of their time and the energy that they spend with us to participate.
0:50:08
And some of that is free medical care in the context of a clinical trial. But also, there may be a small toy or gift for the child who participates to recognize their contribution, as well as a monetary gift for families.
Dr. Mike Patrick: Yeah, absolutely. Once again, we'll have links for you over at the show notes, not only for the Clinical Research page where you can find a trial, perhaps, that you would like to be a part of, but we also have some other great links for you as well. We'll have one, the ResearchMatch that we had talked about earlier. Also, the Pediatric Trials Network.
There's also some great information from healthychildren.org from the American Academy of Pediatrics called "Should My Child Join a Clinical Trial?" "Doing the Work to Improve Diversity in Pediatric Clinical Trials" is also published by the AAP, and evidence-based guidelines for pediatric clinical trials from the NIH.
0:51:03
And so if you are a researcher or pediatrician, you've thought about maybe embarking on a clinical trial and adding your voice to the research community, there's some great information there. And we'll put all of those links in the show notes.
So Dr. Cynthia Gerhardt, a chief clinical research officer for Nationwide Children's Hospital, and Myeshia Harmon, Director of Clinical Research Operations for Hematology, Oncology and Bone Marrow Transplant here at Nationwide Children's, thank you both so much for stopping by today.
Dr. Cynthia Gerhardt: Thanks so much, Dr. Mike. We appreciate it.
Myeshia Harmon: Thank you so much for having us. Thank you.
[Music]
0:52:05
Dr. Mike Patrick: We are back with just enough time to say thanks, once again, to all of you for taking time out of your day and making PediaCast a part of it. Really do appreciate that.
Also, thank you to our guests this week, Dr. Cynthia Gerhardt, Chief Clinical Research Officer, and Myeshia Harmon, Director of Clinical Research Operations for Hematology, Oncology and Bone Marrow Transplant, both of them from Nationwide Children's Hospital.
Don't forget, you can find us wherever podcasts are found. We're in the Apple and Google podcast apps, iHeartRadio, Spotify, SoundCloud, Amazon Music and most other podcast apps for iOS and Android.
Our landing site is pediacast.org. You'll find our entire archive of past programs there, along with show notes, our Terms of Use Agreement, and our handy contact page if you would like to suggest a future topic for the show.
Reviews are helpful wherever you get your podcasts. We always appreciate when you share your thoughts about the show.
And we love connecting with you on social media. You'll find us on Facebook, Twitter, LinkedIn and Instagram. Simply search for PediaCast.
0:53:03
Also, don't forget about our sibling podcast, PediaCast CME. That stands for Continuing Medical Education. It is similar to this program. We do turn the science up a couple of notches and offer free continuing medical education credit for those who listen.
And that includes physicians of course, but also nurse practitioners, physician assistants, nurses, pharmacists, psychologists, social workers, and dentists. And since Nationwide Children's is jointly accredited by all of these professional organizations, it's likely we offer the exact credits you need to fulfill your state's continuing medical education requirements. Of course, you want to be sure the content of the episode matches your scope of practice.
Shows and details are available at the landing site for that program, pediacastcme.org. You can also listen wherever podcasts are found, simply search for PediaCast CME. And did I mention? It's free and it is Category 1 Credit.
All right, thanks again for stopping by. And until next time, this is Dr. Mike saying stay safe, stay healthy, and stay involved with your kids. So long everyone.
0:54:02
[Music]
Announcer 2: This program is a production of Nationwide Children's. Thanks for listening. We'll see you next time on PediaCast.
