Testing Drugs for US Children in Poor Nations Raises Ethical Questions

A law designed to accelerate the development of new drugs for children in the US is financing trials in developing countries, where medicine might not become available.

According to a study published in Pediatrics, Sara K. Pasquali, MD, of Duke University Medical Center, and colleagues found that the majority of published trials conducted under 1997 legislation called the Pediatric Exclusivity Provision were carried out at least partly in developing or transitioning nations, such as Uganda and India.

“The trend that we describe brings up some scientific and ethical problems,” said Pasquali in an online report. “Oftentimes, access to a study may be the only access to medical care a family has,” she said of trial participants in poor countries. However, once testing is complete, it is unknown whether effective drugs will be marketed in the country in question, and whether they will be affordable.

In their evaluation of the Pediatric Exclusivity Provision—which provides economic incentives to pharmaceutical companies to conduct drug studies with children—the researchers extracted data from published studies containing the results of trials conducted in 1998—2007, including therapeutic area of drug studied, number of patients enrolled, number of sites, and location where the study was conducted, if reported. Of the 174 trials that were included, 65% were conducted outside the US, and 11% did not include any sites in the United States. Of the 54 countries represented, 38% of trials enrolled patients in a developing nation, including more than one-third of infectious disease, cardiovascular, and allergy/immunology trials.

The purpose of the Pediatric Exclusivity Provision is to provide “economic incentives to pharmaceutical companies to conduct drug studies with children,” said the authors.

Because many diseases are rare in childhood, clinical trials tend to target adults only, meaning that results doesn’t automatically translate to pediatric populations. The pediatric provision grants companies an extra 6 months of patent life if they test their drugs in kids as well. Thus far, the patent extensions have netted drugmakers an estimated $14 billion dollars, according to the FDA, while more than 150 drugs have been approved for children since 1997.

For more:

• Read the original study, “Globalization of Pediatric Research: Analysis of Clinical Trials Completed for Pediatric Exclusivity,” in Pediatrics
• View a related study, “Pediatric Exclusivity Provision of 1997: Statistical Testing for its efficacy using the Probit Model”
• Learn about the “Effect of the Pediatric Exclusivity Provision on children's access to medicines”
• Read about “Drug Research and Children”