Children and Clinical Studies: For parents and caregivers



pediatric clinical trials are important for enabling a better future for our children it is with clinical research and clinical trials that we make significant advances in medicine the changes that I've seen in the time that I've been in medicine have been absolutely mind-boggling children who when I started in Pediatrics no one would ever have expected them to survive are routinely surviving and heading off to kindergarten and graduating from high school we've made enormous progress improving the outcomes or preterm infants and four-term infants with a variety of problems we have lots of discoveries that have led to basically the eradication of many infectious diseases tetanus pertussis measles mumps rubella one area that we made really huge strides in is the use of inhaled nitric oxide to relax the blood vessels to the lungs when that first came out it was really I remember using it was a miracle the average life expectancy for someone with cystic fibrosis was 7 years of age these days the average life expectancy is 37 that's a really dramatic change and it represents a whole variety of studies the only way that those studies could have been done those trials was excellent science and parents who were willing to say you know who would listen and say okay I want to give my baby a chance all too often we hear that children are small adults and we know that's not true some diseases occur in children that don't occur in adults at all some diseases occur both in children and adults for instance asthma but actually behave somewhat differently in children than in adults and finally children often metabolize drugs quite differently from adults and so we really need to know something very specific about how a given therapy or a given set of therapies is handled by a child compared to an adult the medications that we use in children are medications that have been approved by the FDA for use in humans but most of them have not been tested in children because historically that either wasn't required or it wasn't feasible and so we borrow those medications and we borrow the literature and the information that help us to choose a medication for adults and we just do our best the only way to examine the scientific value of a new therapy is by a pediatric clinical trial with a parent when you go to them with a clinical research study you're getting them at a very vulnerable time you never expect that you're gonna have a sick baby all of these situations are very stressful and I think in terms of being successful in getting the parents and the families to participate in research it takes a lot of one-on-one communication one of the commonest concerns that parents have is when they hear the word clinical trials that is equated with experimentation it evokes a sense of fear and that's natural no parent really wants their child to be experimented on the parent wants to be sure that they're doing the right thing for their child and that they're not putting their child in any danger putting their child in any increased risk sometimes parents might think that the investigator is more worried about their study than their child they want to know okay so what's the benefit is the benefit going to be immediate to them or is the benefit in general going to be applied to a group that they're part of what happens sometimes is patients are reluctant to participate because they think the standard of care is perfectly fine and then you have to explain to them that for us the standard of care hasn't been tested that well we need to understand whether there are more effective therapies and they may benefit them and there are overall health it's a very hard decision for parents to make to enroll a child that small and that vulnerable in a clinical trial but we can't improve their therapies unless we actually study the therapies that we're using for them so we see it as a particularly strong responsibility to fully engaged the families in these conversations so that they understand what we're doing we meet with them to go over the whole idea of the trial the goals the outcomes the processes in a non-binding mechanism they're given information in English and Spanish or other languages if need be for the parent who is uncertain we don't want to pressure them in any way we understand I mean this is their baby and we want them to be very thoughtful about the process if they don't want to do it it's not going to change our care for that baby at all people who go into clinical research in Pediatrics or a very special breed of people they have a very very powerful commitment to the children who are in their studies and with whom they're dealing I think most paediatric investigators really consider the children and their parents and families to be Co investigators in the studies most of us are parents and so we asked ourselves would I enroll my child into this particular study and if I couldn't say yes as a parent I probably wouldn't be doing that study we're doing as much as we can to safeguard that children that are involved and reward the trust and loyalty of our parents there are very rigorous set of federal regulations that guide enrollment recruitment and conduct of pediatric research our first responsibility is to the safety of the child and that safety over always anything that might be required in the protocol all of these review processes are done to make sure that for every participant the potential benefit far exceeds and potential risk parents and the children actually are very interested to be part of these studies they look up and say I have a health problem if that study might help me that would be wonderful but there's an amazing sense of altruism so they look behind them and say what I'd really like to do though is to solve this problem or make the life just a little bit easier for children and families who are coming behind me they are really very interested in furthering medical knowledge to help other families coming after them when you help children who are sick and often have otherwise fatal diseases and you can cure their disease they live wonderful full healthy lives a dad said to me dr. Wagner you have to save my baby and you know in those moments at two o'clock in the morning that's when someone reaches out to you and you think outside of the box so to me that's my passion and I think it's a privilege and an honor to not only take care of babies but to in doing research and conducting research you can affect thousands of children if you design a study that's insightful and it has a huge impact parents are really the essential piece to this whole puzzle without them and children we wouldn't be able to answer a lot of the questions that we need to answer they really are heroes when you stop and think about it to offer up their children their families their time to research and to help not only perhaps themselves and their children but to help children that they've never met before there is hope that there's a better day out there and the way that we're going to get there is through this carefully designed trial we'll know an answer and that will allow us to go to the next step so it's building a series of foundations that will take us to a better tomorrow if you're a parent of a sick child that's what you hope for is a better tomorrow you

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