Susan Thaul
Specialist in Drug Safety and Effectiveness
Summary Update: On June 20, 2012, the House of Representatives passed, by voice vote and under suspension of the rules, S. 3187 (EAH), the Food and Drug Administration Safety and Innovation Act, as amended. This bill would reauthorize the FDA prescription drug and medical device user fee programs (which would otherwise expire on September 30, 2012), create new user fee programs for generic and biosimilar drug approvals, and make other revisions to other FDA drug and device approval processes. It reflects bicameral compromise on earlier versions of the bill (S. 3187 [ES], which passed the Senate on May 24, 2012, and H.R. 5651 [EH], which passed the House on May 30, 2012). The following CRS reports provide overview information on FDA’s processes for approval and regulation of drugs:
- CRS Report R41983, How FDA Approves Drugs and Regulates Their Safety and Effectiveness, by Susan Thaul.
- CRS Report RL33986, FDA’s Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective, by Susan Thaul.
- CRS Report R42130, FDA Regulation of Medical Devices, by Judith A. Johnson.
- CRS Report R42508, The FDA Medical Device User Fee Program, by Judith A. Johnson.
(Note: The rest of this report has not been updated since November 10, 2011.)
With the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), Congress authorized the Food and Drug Administration (FDA) to offer drug manufacturers financial and regulatory incentives to test their products for use in children. Congress extended both programs with the FDA Amendments of 2007 (FDAAA) and, because of the programs’ sunset date, must act before October 1, 2012, to continue them. This report presents the historical development of BPCA and PREA, their rationale and effect, and FDAAA’s impact. The report also discusses pediatric drug issues that remain of concern to some in Congress.
Most prescription drugs have never been the subject of studies specifically designed to test their effects on children. In these circumstances, clinicians, therefore, may prescribe drugs for children that FDA has approved only for adult use; this practice is known as off-label prescribing. Although some clinicians may believe that the safety and effectiveness demonstrated with adults would hold for younger patients, studies show that the bioavailability of drugs—that is, how much gets into a patient’s system and is available for use—varies in children for reasons that include a child’s maturation and organ development and other factors. The result of such off-label prescribing may be that some children receive ineffective drugs or too much or too little of potentially useful drugs; or that there may be side effects unique to children, including effects on growth and development.
Drug manufacturers are reluctant to test drugs in children because of economic, ethical, legal, and other obstacles. Market forces alone have not provided manufacturers with sufficient incentives to overcome these obstacles. BPCA and PREA represent attempts by Congress to address the need for pediatric testing. FDA had tried unsuccessfully to spur pediatric drug research through administrative action before 1997. With the FDA Modernization Act of 1997 (FDAMA, P.L. 105- 115), Congress provided an incentive: if a manufacturer completed pediatric studies that FDA requested, the agency would extend the company’s market exclusivity for that product for six
months, not approving the sale of another manufacturer’s product during that period. In 2002, BPCA (P.L. 107-109) reauthorized this program for five years.
In 1998, to obtain pediatric use information on the drugs that manufacturers were not studying, FDA published the Pediatric Rule, which required manufacturers to submit pediatric testing data at the time of all new drug applications. In 2002, a federal court declared the rule invalid, holding that FDA lacked the statutory authority to promulgate it. Congress gave FDA that authority with PREA (P.L. 108-155). PREA covers drugs and biological products and includes provisions for deferrals, waivers, and the required pediatric assessment of an approved marketed product.
In extending BPCA and PREA in 2007, Congress considered several issues: Why offer a financial incentive to encourage pediatric studies when FDA has the authority to require them? How does the cost of marketing exclusivity—including the higher prices paid by government—compare with the cost of the needed research? What percentage of labeling includes pediatric information because of BPCA and PREA? Do existing laws provide FDA with sufficient authority to encourage pediatric studies and labeling? Is FDA doing enough with its current authority? The 112th Congress will likely consider those questions as well as others: What information do clinicians and consumers need and how could industry and government develop and disseminate it? How can Congress balance positive and negative incentives to manufacturers for developing pediatric information to use in labeling? How could Congress consider cost and benefit when it deals with reauthorizing legislation in 2012?
Date of Report: June 25, 2012
Number of Pages: 28
Order Number: RL33986
Price: $29.95
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