Study examines movement of pediatric drug testing outside the U.S.

"A law intended to speed up development of new drugs for U.S. kids has ended up financing clinical trials in poor countries, where the medicines might never become available," suggest the authors of a study published online Monday in the journal Pediatrics, Reuters reports (Joelving, 8/23).

The U.S. statute - called the Pediatric Exclusivity Provision - "provides six months of patent exclusivity to pharmaceutical companies to conduct safety and efficacy studies of drugs in children," according to a Duke Medicine press release. This study marks the "first [attempt] to examine pediatric research moving overseas, which is a trend that has been previously reported in adult clinical research," the release states (8/23).

As described in the journal, the authors analyzed 174 published studies "containing the main results of trials conducted in 1998-2007 under the Pediatric Exclusivity Provision." Of the studies that reported the site or sites where trials took place, "65% were conducted in 1 country outside the United States, and 11% did not include any sites in the United States. Fifty-four countries were represented, and 38% of trials enrolled patients in 1 site located in a developing/transition country, including more than one-third of infectious disease, cardiovascular, and allergy/immunology trials," the authors report (Pasquali et al., 8/23).

"[D]rugs against infectious diseases were most likely to be tested in the developing world, closely followed by heart, allergy and arthritis medications," Reuters continues (8/23). The authors of the study caution their findings may underestimate the globalization of pediatric clinical trials, because roughly half of the trials carried out under the program remain unpublished, according to the Duke release.

"The trend that we describe in [the article] brings up some scientific and ethical problems," said Sara Pasquali, Duke University Medical Center pediatrician and lead author of the study. She attributes the number of trials held outside the U.S. to the lower costs of conducting research abroad and fewer regulatory requirements. In the release, Pasquali also notes, "Whether it's valid to extrapolate the results from trials conducted in other countries is not known. The efficacy of a medication may depend on genetic background and access to health care resources, among other factors, which may differ across countries" (8/23).

In developing countries, "[o]ftentimes, access to a study may be the only access to medical care a family has," Pasquali said, according to Reuters. "Once the testing is done, however, it's unclear if effective drugs will be marketed in the country in question, and whether they will be affordable," the news service writes.

"We are now using vulnerable people in vulnerable countries as drug laboratories," Harvard Medical School's Marcia Angell, who was not involved in the new research, said, according to Reuters. The article includes comments by a spokesman from the Pharmaceutical Research and Manufacturers of America, who states that researchers follow principles, similar to those used in the U.S., when conducting clinical trials overseas. The piece also includes comments by a scientist involved in drug development, who describes some of the cultural differences between participants in clinical trials in developing countries compared to those in the U.S. (8/23).

The authors of the study offer several recommendations for conducting pediatric clinical trials outside the U.S., including: "Requiring pharmaceutical companies to describe how the proposed study population matches the intended market for the drug being tested," and "[i]mproving mechanisms of global regulatory oversight with input from all stakeholders," according to the press release (8/23).