Here's the lead from a story that ran earlier this week in the Philadelphia Inquirer on a new study showing that nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA:
"Most children treated at major pediatric hospitals are given medicines not approved by the Food and Drug Administration for use in patients so young. The study, in today's Archives of Pediatrics & Adolescent Medicine, found that the sickest children and those undergoing surgery were most likely to get a so-called off-label drug. But altogether, nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA."
Why is this so? Well it's because of the big, bad, evil pharmaceutical industry don't ya know:
"The risk and benefits of many drugs in children are poorly studied, often because the drugmakers had little financial incentive to do so."
Well, not precisely. It's not the financial incentives so much as the difficulty relative to the return. I know, that sounds like verbal gymnastics. Try this -- the real issue is that FDA permitted pediatric clinical trial design makes it difficult if not impossible to precisely determine which medicines in what dosages work in specific circumstances in specific pediatric patient populations. In other words, if personalized medicine in adults is difficult, in children it is nearly impossible.
Yet another important reason for the FDA to embrace adaptive clinical trial design and to become a research hub for 21st century clinical trial design. And that, of course, is part of what makes the Critical Path program so, well, critical to the future of America's health.
Here's a link to the Inquirer article:
http://www.philly.com/mld/inquirer/16841956.htm
According to Samir S. Shah, the study's lead author and infectious-disease doctor at Children's Hospital of Philadelphia, "We don't know whether in the absence of off-label use there would have been fewer deaths or more deaths. I suspect that in many instances the drugs were beneficial and in a smaller number of cases the drugs were harmful."
That's good news. What would be even better news would be getting the right drug in the right dose to the right child at the right time based on sound science and under the FDA's imprimatur.
Tempus fugit.
"Most children treated at major pediatric hospitals are given medicines not approved by the Food and Drug Administration for use in patients so young. The study, in today's Archives of Pediatrics & Adolescent Medicine, found that the sickest children and those undergoing surgery were most likely to get a so-called off-label drug. But altogether, nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA."
Why is this so? Well it's because of the big, bad, evil pharmaceutical industry don't ya know:
"The risk and benefits of many drugs in children are poorly studied, often because the drugmakers had little financial incentive to do so."
Well, not precisely. It's not the financial incentives so much as the difficulty relative to the return. I know, that sounds like verbal gymnastics. Try this -- the real issue is that FDA permitted pediatric clinical trial design makes it difficult if not impossible to precisely determine which medicines in what dosages work in specific circumstances in specific pediatric patient populations. In other words, if personalized medicine in adults is difficult, in children it is nearly impossible.
Yet another important reason for the FDA to embrace adaptive clinical trial design and to become a research hub for 21st century clinical trial design. And that, of course, is part of what makes the Critical Path program so, well, critical to the future of America's health.
Here's a link to the Inquirer article:
http://www.philly.com/mld/inquirer/16841956.htm
According to Samir S. Shah, the study's lead author and infectious-disease doctor at Children's Hospital of Philadelphia, "We don't know whether in the absence of off-label use there would have been fewer deaths or more deaths. I suspect that in many instances the drugs were beneficial and in a smaller number of cases the drugs were harmful."
That's good news. What would be even better news would be getting the right drug in the right dose to the right child at the right time based on sound science and under the FDA's imprimatur.
Tempus fugit.
