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Washington Times - Responsible Medicine

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Washington Times - Responsible Medicine

By U.S. Senator Tom Coburn, M.D.
Washington Times

Nearly two-thirds of the drugs prescribed for children have not been studied and labeled for pediatric use. As a practicing physician, I know the importance of accurate clinical information about a drug's use in pediatric populations. The smaller body mass and higher metabolic rates of children mean they often respond differently to drug dosing than adults do. A drug that is safe and effective in adults may not always be safe for children. The question is not whether we should study the safety of drugs for children, but how we make that research happen.

In 1997, Congress considered this issue and created an incentives program for companies to study the use of their drugs in pediatric populations. The program offers additional six-month patent protection or "exclusivity" to drug manufacturers to help recoup the costs of investing in these critical pediatric studies. It's a win-win situation: Drug companies have an incentive to invest time and extra resources for a small share of the market, and, more importantly, children get the research they need.

That incentives program, today called the Best Pharmaceuticals for Children Act (BPCA), has generated more clinical information for the pediatric population than any other legislative or regulatory effort to date.

Over the last 10 years, the Food and Drug Administration (FDA) has sent more than 300 written requests to drug sponsors for more than 700 pediatric studies. The FDA has received more than 400 proposed pediatric-study requests and 144 completed studies have been submitted to the FDA. Twenty-five drugs now contain the right dosage information for children, 35 products have better safety data and the studies showed that 24 products should not be used by children for safety reasons. By contrast, in the six years before the program began only 11 drugs were studied in children. The success of BPCA has equipped doctors with accurate information about which drugs and which doses work for children.

Some may wonder if drug companies should be doing this type of research even without incentives. This policy is a good one, but the real question is how do we ensure this testing for children gets done? Instead of mandating studies and watching pharmaceutical companies find loopholes to protect their bottom line, the BCPA approach has proven that it does get these studies done.

Drug companies have not just used this incentive for their most lucrative drugs, but have used this tool to study drugs that treat rare disease conditions in children. In fact, out of the 200 best-selling drugs in the United States, only one out of every 10 pediatric exclusivity drugs were "blockbusters" in 2004.

Despite BPCA's success, there are efforts to diminish its ability to encourage new research. Legislation proposed by Sen. Christopher Dodd would create more government busywork in micromanaging drug companies — these efforts miss the point of BPCA. BPCA is not about profits for drug companies, it is about ensuring children get the same research that adults expect and demand. The current program — in place today — is working. I am concerned that well-intentioned but misguided efforts to change it will produce fewer results for children.

Witnesses before a recent hearing in the Senate Health, Education, Labor and Pensions Committee, on which I serve, stated that changing the structure of the incentives that are producing real results today for children is a "risky experiment." The problem is not excess profits, but ensuring all Americans have access to affordable drugs. I welcome that broader debate in the future, but I cannot support those who want to play the politics of drug pricing when it comes to making sure children get the critical safety information they deserve.

The Journal of the American Medical Association recently found that "The greatest return of the exclusivity program is the benefits derived in obtaining new information relevant and applicable toward the care of children, and this benefit should not be compromised."
As Congress considers the reauthorization of BPCA, my colleagues and I will also consider the reauthorization of the Pediatric Research Equity Act (PREA). The two programs have created an effective two-pronged approach: PREA provides the FDA with the rarely used authority to require pediatric studies in certain cases; and BPCA goes beyond PREA to encourage companies to ask: "Where are the unmet needs for children?" In order to maintain that results-based approach, the two programs must continue to work in tandem.

Some have proposed ending the successful BPCA incentive program in 2012, while permanently authorizing PREA's authority to mandate. I am concerned this approach will ultimately lessen the number of new pediatric studies and also restrict children's access to cutting-edge medical technology. In order to make sure children have the research and drugs they need to win difficult battles against cancer or diabetes, the two programs should both be re-examined in 2012 or both be made permanent. I hope my colleagues will remember Primum non nocere, "First, do no harm."

Sen. Tom Coburn, Oklahoma Republican, is a member of the Judiciary Committee, Homeland Security and Governmental Affairs Committee, the Indian Affairs Committee and the Committee on Health, Education, Labor and Pensions.


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