General Updates | The challenges with pediatric trials in oncology
In this video, Gwen Nichols, MD, The Leukemia & Lymphoma Society, Rye Brook, NY, discusses the challenges with pediatric trials in oncology, highlighting the difficulties in combining data sets from different regions due to varying standards of care and discrepancies in clinical endpoints. Dr Nichols emphasizes that these challenges result in underpowered studies, which hinder the approval of novel therapies for pediatric patients. This interview took place virtually.
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Transcript
Well, there are many, many excellent pediatric consortia that do clinical trials for new therapies and for how to utilize available therapies. They, unfortunately, while they’re consortia, they’re still too small to meet the kind of data requirements for regulatory approval, and also, we realized, when I brought together a group of pediatric leukemia experts from Europe, the US, Australia, and Canada, we realized that there are different standards of care in terms of what therapies are used because different drugs are approved in different regions...
Well, there are many, many excellent pediatric consortia that do clinical trials for new therapies and for how to utilize available therapies. They, unfortunately, while they’re consortia, they’re still too small to meet the kind of data requirements for regulatory approval, and also, we realized, when I brought together a group of pediatric leukemia experts from Europe, the US, Australia, and Canada, we realized that there are different standards of care in terms of what therapies are used because different drugs are approved in different regions. And there is no one way that relapse disease is treated, nor is there even an agreement on endpoints. So, the endpoint – one great example is what is complete remission in Germany is a completely different platelet count than what is complete remission in Philadelphia. So, how do you combine data sets together when it’s different drugs, different endpoints? You can’t. And so, everything in pediatrics has been underpowered. And unfortunately, that means that new therapies aren’t getting approved. Lots of studies are done, but either they end early or they aren’t of sufficient size to change what the standard of care is. And that is only going to get worse as we get better at subsetting our cancers with our precision medicine and our ability now to really understand the molecular biology of some of these tumors.
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