To mark the 10-year anniversary of the law that encourages drugmakers to study their meds in children, the Tufts Center for the Study of Drug Development did a little research. This is of interest because the Best Pharmaceuticals for Children Act, which grants an extra six months of patent exclusivity in exchange for pediatric studies, is up for renewal in October.

Supporters contend the law yields important dosing and safety information for a population that would, otherwise, not have access to certain medicines. Critics say the law is a boondoggle that allows drugmakers, at relatively low cost, to ring the register a while longer and chalk up big profits, at least on best-selling drugs. As October looms, the jockeying has begun.

Here’s what the think tank, which receives significant industry support, found:

- The average cost to complete pediatric research on already marketed prescription drugs rose nearly eight times between 2000 and 2006, and the increase, in nominal dollars, went from $3.93 million in 2000 to $30.82 million in 2006;

- Studies were undertaken on more than 100 diseases and conditions, and have led to new labeling for 120 new or already approved drugs for use in children;

- Efficacy and safety studies now account for 40 percent of all pediatric studies conducted, up from 25 percent in 2000;

- Time required to complete a study and submit a final report nearly doubled since 2000;

- The mean number of patients required for studies rose 178 percent between 2000 and 2006, and the mean number of studies per FDA request rose 60 percent

“While the cost to complete pediatric studies has soared, drug companies are not letting that get in their way,” says Tufts CSDD’s Christopher Milne (no Pooh jokes, please). “The Best Pharmaceuticals for Children Act seems to be doing its job, which is to generate more pediatric studies. The bottom line is that better prescribing information and more formulations are being developed for children.”

A recent JAMA study (subscription required) advocated a similar conclusion after conducting an economic analysis of 9 drugs used to treat depression, bacterial infections, ADHD, acid reflux, diabetes, high blood pressure, fragile bones, tumors and asthma. The authors, whose work was supported by NIH grants, maintained the law provides a useful public health benefit. “This benefit should not be compromised,” they wrote, by reducing patent exclusivity to three months.

However, the researchers also found the return on investment associated with pediatric studies was all over the map, ranging from a $9 million loss to a $507 million gain and, not surprisingly, they acknowledged those six extra months of patent protection may provide a windfall for blockbuster drugs. By the way, five of the 10 researchers disclosed subsantial ties to every big drugmaker, collectively, including research grants, stock holdings and consulting income. So, theoretically, they stand to gain if incentives are maintained, rather than reduced or eliminated.

The Tufts study marks the second time in recent weeks that researchers with industry ties have advocated for maintaining the status quo. Certainly, the argument for public health benefits is reasonable and well-founded. And more information is always better. The act does serve an important purpose.

However, there are issues to probe: the wisdom of testing certain meds in children; the timely disclosure of all pertinent clinical-trial data associated with these studies, and the extent to which blockbusters may be favored for study purposes. Before the act is renewed, these items should be examined closely. After all, this is supposed to be about putting kids first.

[tags]FDA, Pediatric Studies, Tufts[/tags]