F By Dr. Jeffrey Kahn
ederal policy for biomedical research on children has come to a crossroads because of a convergence of circumstances, and the next step is vitally important. For starters, the U.S. Senate is about to consider a bill that would require all drugs sold and marketed in the United States to be tested on children as well as adults.
It might seem surprising that this isn’t already the case, but most drugs prescribed for children have been tested in controlled settings on adults only. Doctors then order lower doses for children, but it’s an inexact science because children are not just small versions of grown-ups.
So a policy that requires testing explicitly for safety and efficacy in children is long overdue, and this bill – stemming from a recent rule by the Federal Drug Administration – would make it happen.
This policy decision comes at a critical time in biomedical research.
Gene therapy research has been put on hold after what appeared to be success stories – in three children with a genetic type of immune deficiency – suffered a setback when one of the children developed leukemia from the treatment. And the family of a deceased artificial heart recipient is suing. They say the patient didn’t understand all that was involved in the research project.
As these cases should teach us, research we hope will improve drugs for future children requires risks to those involved in the research today. Should children be exposed to these risks of research so that other children can benefit?
As a society, we want the benefits of biomedical research, and we want those benefits to be shared as widely as possible – especially with the most vulnerable. But benefits come with costs, one of which might be injury to those very people we hope to help.
Our research policies have focused on protecting children as much as possible from risks. Unfortunately, that didn’t mean children weren’t harmed. It means the harm occurred when drugs developed for adults were used in children through trial and error and without the benefit of a controlled scientific setting.
We now have an opportunity to change that and test drugs for children just like we test drugs for adults – on the populations that will eventually use them and in controlled settings.
But that doesn’t mean we should do it in just the same ways as we do in adults. Since children can’t decide for themselves whether to participate in drug trials, we should make sure parents or guardians understand all the risks and benefits. For example, parents shouldn’t receive benefits, such as payments, in return for their children participating. Also, the risks should be markedly less than those we tolerate in adult drug trials. This might mean testing drugs on children later in the testing process, after early-stage trials on adults have proven safety. That way we can protect children in research while reaping the benefits of pediatric research.
The reality is that if we want a future that includes effective drugs for our children, it’s time to move past pint-sized drug research policy.
