TDM

Children suffering with pediatric type 2 diabetes need more treatment options that have been tailored specifically for them. There is currently only one oral medication on the market, metformin, that has been studied in children formally. Other treatments are either not prescribed to children or are given to them based solely on studies in adults, leaving doctors to make their best guess at an appropriate dose. 

TDMWe need to do better. Pediatric type 2 diabetes is a growing healthcare issue and we need to involve more children in type 2 diabetes clinical research. 

Admittedly, such research can be complicated. It is difficult to recruit children for almost any type of clinical study, and pediatric type 2 diabetes is particularly challenging. While this disease is a growing problem, the overall number of pediatric patients is still relatively small, there are often socio-economic barriers to enrolling these patients, and researchers are competing for the few patients available. 

There are also the concerns about involving children as trial subjects, particularly if the research involves discomfort, pain or fear, with little benefit to the subject. Pharmacokinetic (PK) studies can be particularly unappealing because they involve needle sticks, with no promise of therapeutic benefit to patients because only one or a few doses of the drug are given. That’s a tough sell for parents deciding whether to burden their child and their family with participation in a clinical trial. 

Despite these barriers, such research is essential. As Margaret Hamburg, commissioner of the Food and Drug Administration (FDA) noted in a recent post on the FDA’s blog, “Without the information gained from testing children in clinical trials, health care professionals and parents alike (can) only guess when trying to gauge the correct dosages for children.” 

That uncertainty is unacceptable to patients, their families and the regulators. Hamburg and FDA scientists are actively focused on ensuring that pediatric studies are designed and conducted in accord with current scientific understanding of the characteristics that make children unique. And they are looking for treatment options for children. 

To accomplish this goal, we need to be more innovative in the way we design these trials, and in the way we attract the patients – and their families – to these trials.

One approach is to minimize the number of patients enrolled in PK trials, by using physiologically based pharmacokinetic (PBPK) modeling to zero in on the correct dose. This dose is then tested in the trial and if confirmed to be correct, the need to test other dose levels is eliminated, along with the need to recruit additional patients, every one of whom is precious in this difficult-to-recruit patient population. 

Another strategy that can address the recruiting challenges researchers face is a rolling study design. In this scenario, patients enrolled into the PK study, would continue treatment into the longer term study to test the efficacy of the drug (an interim dose adjustment may be necessary after the PK results are evaluated). This approach is more attractive to patients and their families because it offers the child hope of therapeutic benefits. At the same time, researchers benefit from a single recruiting process for both trials, which means they don’t lose those valuable recruits. 

These types of innovative approaches accommodate the needs of patients, families and researchers, while increasing the promise of a more productive trial experience.

Topics in this blog post: Clinical Trials, Diabetes, Pediatric Type 2 Diabetes