Pediatric Testing Requirement Would Be Wrong Medicine

Published January 1, 2003

If you’ve filled a prescription recently, you may have had a nasty surprise. Antibiotics or a tube of steroid cream used to cost a few bucks, but now the prices are soaring. A few examples of the cost of a week’s supply of drugs: the antibiotic Biaxin, $63; Celebrex for arthritis, $85; and Procrit, for anemia, $218.

Why the high price tags? In large part, it’s your tax dollars at work. Government regulation imposes enormous “taxes” on drug development, and those taxes are ultimately passed along to consumers as higher prices. They also decrease innovation and the number of drugs that are developed, putting us all at risk.

Pediatric Testing

A prime example is the Food and Drug Administration’s 1997 announcement that drug companies would be required to test in children the medicines they sell for adults. The good news is that a federal court nullified the policy. “The pediatric rule exceeds the Food and Drug Administration’s statutory authority and is therefore invalid,” said Judge Henry H. Kennedy Jr.

The bad news its that Sen. Hillary Clinton (D-New York), who championed the policy during the Clinton years, is now proposing to make the testing requirements law. She still doesn’t get it.

The one-size-fits-all requirement for pediatric trials ignores the realities of drug testing. It may actually be detrimental to kids and delay the availability of new drugs, because FDA can withhold approval for adult uses while the required data from pediatric studies are being collected.

Moreover, the regulation is a rigid, centralized government solution to a non-problem, according to many pediatricians. Even the FDA concedes physicians commonly and safely prescribe pain relievers, asthma drugs, antihistamines, antibiotics, and other therapeutics millions of times annually for children, despite the clinical trials of those products having been performed only in adults.

Even if additional testing of drugs in children were needed, there are more imaginative and effective ways to accomplish it. Federal law already offers financial incentives, in the form of extended market exclusivity, to companies that voluntarily perform pediatric testing.

In addition, the FDA could simply require a prominent label or logo on drug preparations whose safety and efficacy have not yet been determined in children, or the agency could regularly publish a list of such drugs. This would make parents and physicians aware that data from child testing are not available; they, in turn, could exert moral and economic pressure on drug companies to obtain it.

Clinton seems to be unaware of the nuances of drug development. Creating a dosage form appropriate for children is often especially challenging, for a number of reasons. Can the active ingredient be incorporated in a chewable or syrup form? Will it have special storage requirements and adequate shelf-life? Does it taste good enough so kids will actually take it?

A pediatric form of Glaxo Wellcome’s antibiotic, Ceftin, required more than double the cost and man-hours to develop than did its adult formulation. The same company also experienced serious problems in finding effective preservatives for its pediatric syrup form of Epivir, an AIDS drug, even after the adult formulation had been fully developed.

Clinical trials are especially difficult to perform with children. For ethical reasons, testing is done in patients, not in healthy volunteers. Study participants may be scarce because a disease is rare in children and patients are few and far between, or because parents are reluctant to enroll their sick children in an “experiment.”

Finally, for the purposes of drug testing, “children” is by no means a homogenous category. It encompasses several groups that are physiologically and metabolically distinct–newborns, infants, preschoolers, primary-schoolers, and teens. Moreover, children may pass through two or more age groups during the course of a multi-year clinical study, confounding data analysis.

High Price to Pay

As regulatory requirements are ratcheted ever upward, drugs are moving more sluggishly through the clinical testing pipeline. The total time required for drug development, from synthesis of the molecule to the patient’s bedside, has almost doubled during the past three decades, from 8.1 to 15.2 years. And the United States’s $800 million tab for each drug approved is by far the most expensive in the world.

When regulation is excessive, everyone is a loser–except government regulators themselves. Money spent by the government, industry, and consumers on FDA regulation is money not spent on something else, including the ability to protect public health in other ways.

The FDA’s pediatric testing requirement increases costs to consumers, makes guinea pigs of our children, and puts everyone at risk as the introduction of new drugs is delayed. It is a disturbing example of Big Brother deciding where private sector R&D resources are best spent. Instead of becoming law, it should be allowed to expire quietly.

Dr. Henry I. Miller, a physician, is a fellow at the Hoover Institution. He was an FDA official from 1979-1994. He can be reached by email at [email protected].

For more information …

Dr. Henry I. Miller’s book, To America’s Health: A Proposal to Reform the FDA, is available in paperback for $10.47 from Point your Web browser to