No child's play: Testing prescription drugs in children is unethical, critics say.
AP Photo/Oscar Sosa
Nearly 40 years ago, a medical practitioner named Harry Shirkey coined the term 'therapeutic orphans' to describe how children are overlooked in medical research. Shirkey's observation has stood the test of time: according to estimates by the US Food and Drug Administration (FDA), nearly 80% of all drugs prescribed for children have never been tested in children, and are used 'off-label' for unapproved treatments.
But that could all change soon. If a new bill approved by the US Senate in March becomes law, the FDA can demand that companies test certain drugs—both new and already marketed—in children. A second initiative, announced in January by the US Department of Health and Human Services (HHS), calls for tests of 12 drugs commonly prescribed for children. Government-funded tests of those drugs are set to begin later this year.
The HHS list includes the widely used antibiotic azithromycin, the anticoagulant heparin, and lithium, a drug commonly prescribed for bipolar disorder. Although none of those drugs are new to the market, the tests will provide crucial knowledge of how the drugs affect children, HHS officials say. But many of these drugs have been used in children for years, which begs the questions—why these drugs, and why now?
"In a way we're doing drug development backwards for kids, taking drugs to market and making sure they're safe later," says Don Mattison, senior scientific advisor to the US National Institute of Child Health and Development. But even if they are untimely, the tests are necessary, Mattison says. "It is an attempt to redress essentially a subsequent gap in pediatric knowledge and available formulations."
The list reflects off-patent drugs with the highest testing priority in children, Mattison notes. "These are not necessarily unsafe drugs," he adds. "There might just be a lot of interest in their use."
Many medical professionals support testing drugs for children, in children, to avert deadly medical mistakes. But regardless of any benefits, pediatric tests are unethical, argues Jane Orient, director of the Association of American Physicians and Surgeons. "Children are real people too, not guinea pigs or mice," Orient says. Instead, she says, the best option is for pediatricians to monitor and follow the long-term effects of drugs.
The HHS tests are called for by the Best Pharmaceuticals For Children Act (BPCA), signed into law over a year ago. BPCA authorizes private contractors, working under HHS supervision, to evaluate drugs that have not been tested in children. Because patents for the listed drugs have expired, however, pharmaceutical companies have no incentive to conduct the tests, leaving the government to foot the bill. To encourage participation from the pharmaceutical sector, BPCA allows the government to offer a 6-month patent extension to companies who opt to conduct their own clinical trials.
Companies may also have no choice but to comply if the proposed Pediatric Research Equity Act becomes law. The act originated in 1998 as the Pediatric Rule but in 2002, the Association of American Physicians and Surgeons, the Competitive Enterprise Institute and Consumer Alert had the rule overturned on the grounds that the FDA lacked the statutory authority to require pediatric tests. Taking the matter to Congress, rather than to the courts, HHS pushed for the new legislation to clearly establish the FDA's authority.
In a statement, the bill's sponsor, Republican Senator Mike DeWine wrote, "We are introducing this bill so that our children are no longer a therapeutic afterthought. Children are not small adults. They metabolize drugs very differently and require different doses than adults."
Tests supervised by the HHS will begin this year, with a budget of $25 million in 2003 and a potential budget of $50 million in 2004. Another $18 million is to be provided for review by the FDA. Each drug will undergo about two years of testing, followed by FDA evaluation; the list of drugs will be updated every year.
