StATS: Therapeutic orphans (November 3, 2006).

As an introduction to a talk I am giving about research concerns involving children, I have to start off by pointing out that children are "therapeutic orphans." Historically, they have been, to a large extent, left behind by the medical research community, forcing pediatricians to make difficult choices for the care of their patients. As a simple example of this problem, a group of researchers in Australia examined the product information (PI) for 1,497 prescription medications and found that most of the PIs were unhelpful for children.

The proportions, for each age group, of PIs with inadequate paediatric dosing information were: < 1 month (80.5%), 1–3 months (79.1%), 3 months–2 years (77.5%), 2–6 years (73.2%), and 6–12 years (71.6%). The proportions, for each age group, of PIs that gave specific paediatric dosing information but did not provide a paediatric dosage form were: < 1 month (26.5%), 1–3 months (25.1%), 3 months–2 years (23.3%), 2–6 years (21.9%), and 6–12 years (24.0%). (Source: Dosing information for paediatric patients: are they really “therapeutic orphans”? Tan E, Cranswick NE, Rayner CR, Chapman CB. MJA 2003; 179 (4): 195-198. [Full text] [PDF]).

The reasons for this are complex and no one party is entirely to blame (Editorial Comment: Therapeutic Orphans. Shirkey H. Pediatrics 104(3): 583-584. [Full text] [PDF]). There are some proposed solutions, which I support, but these solutions are not without controversy. But one thing I want to dispel is the notion that research in children is impossible or even difficult.

Research in children is not impossible. You need to respect the differences. You can't blindly force-fit an adult research model onto children. But I want to encourage everyone to pay close attention to the pediatric field and leave my talk thinking "Yes, I understand the special issues associated with research in children and I'm ready to get to work."

Creative Commons License This work is licensed under a Creative Commons Attribution 3.0 United States License. It was written by Steve Simon on 2006-11-03, edited by Steve Simon, and was last modified on 2010-04-01. Send feedback to ssimon at cmh dot edu or click on the email link at the top of the page. Category: Children in research

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