Pediatric Versus Adult Clinical Trials--Benchmarking the Costs and Complexity

Do clinical trials involving children cost more than those involving adults? Are they more complex? And what are the trends—have pediatric trials become more expensive and complicated than adult ones? Until now, sponsors haven’t been able to answer these questions. In fact, very little has been known about the differences in cost and complexity between pediatric and adult clinical trials. To shed some light on these questions, we recently conducted an extensive analysis of adult versus pediatric trials using Medidata’s PICAS® database, a rich source of data used to help budget clinical studies. Specifically, the analysis focused on differences in study design, protocol complexity and costs per patient (CPP). The results were intriguing.

The PICAS database contains more than 27,000 clinical study protocols and nearly a quarter of a million negotiated investor grants from over 80 countries. Drawing on this wealth of data, our analysis showed that the CPP of pediatric versus adult clinical trials is surprisingly similar. However, adult clinical trials appear to be getting more complex (involving a higher number of procedures). Our analysis also showed that adult trials actually have a lower cost per procedure than pediatric trials.

We came to these conclusions using a dataset consisting of Phase II and III studies. We further segmented the dataset into two timeframes, 2006-09 and 2010-13, to be able to spot any trends in the cost and complexity of adult versus pediatric clinical trials. Pediatric trials were defined as those with a patient population under 18 years of age.

Using the PICAS database, we were able to define and measure the costs and complexity of clinical trials in a variety of ways, which helped create a clearer picture of trends in pediatric versus adult studies. In our analysis, we measured:

• Protocol complexity – a measure of the effort required by the site staff to implement the clinical procedures included in the study protocol. Also known as site work effort (SWE).

• Unique & total clinical procedures – the number of clinical procedures performed on each patient.

• CPP – total cost of one patient at one site.  This cost may include the total costs for clinical procedures, other direct costs, IRB fees, overhead and central lab costs.

• Cost per procedure – a relative cost for each clinical procedure performed on each patient. This is calculated by dividing clinical procedure total per patient cost by the total number of clinical procedures performed on one patient.
  

Our analysis showed that across all therapeutic areas, the median pediatric CPP was 6% less than the adult CPP in the 2006-09 dataset. This gap increased only slightly to 9% in the 2010-13 dataset. Both pediatric and adult CPPs were higher in 2010-13 compared with 2006-09, with pediatric CPP increasing 10.8% and adult CPP rising 13.8%.

By contrast, the difference in SWE between pediatric and adult trials was much greater. Between 2006-09 and 2010-13, the complexity of adult clinical trials increased 8 percent, while the complexity of pediatric clinical trials fell 3%. In other words, adult clinical trials were 40% more complex than pediatric trials in the 2010-13 dataset.

We analyzed this trend further by looking at the number of unique and total procedures performed on each patient. We found the same trend as with the SWE data. Between the 2006-09 and 2010-13 datasets, the number of unique and total procedures in adult clinical trials rose 10 and 15%, respectively. On the other hand, in pediatric trials, the number of unique procedures remained relatively unchanged, while the number of total procedures rose only 3%. By 2010-2013, the median number of unique and total procedures in adult trials was 36% and 58% higher, respectively, than in pediatric trials.

However, it was when we examined clinical trial costs in the context of trial complexity that interesting insights emerged. By taking  clinical procedure total per patient cost and dividing it by the total number of procedures, we were able to obtain a relative cost for each clinical procedure being performed on a patient throughout the study (i.e. the cost per procedure). For the 2006-09 timeframe, the pediatric cost per procedure was actually 7% higher than for that of adult clinical trials and 50% higher for the 2010-13 timeframe.

This apparent disparity between the cost per patient and complexity in pediatric trials leads us to believe that other factors are behind the higher cost per procedure metrics when compared to adult trials.

While these cost and complexity metrics provide a good start to understanding the differences between adult and pediatric trials, further analysis will be needed to uncover other key trial and operational metrics. And these new metrics could help further improve how we design and conduct both pediatric and adult clinical trials in the future.

Rafael Campo ([email protected]) of Medidata