{ "id": "RL33986", "type": "CRS Report", "typeId": "REPORTS", "number": "RL33986", "active": false, "source": "EveryCRSReport.com, University of North Texas Libraries Government Documents Department", "versions": [ { "source": "EveryCRSReport.com", "id": 409704, "date": "2012-06-25", "retrieved": "2016-04-07T00:02:49.955603", "title": "FDA\u2019s Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective", "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\u2019s processes for approval and regulation of drugs: \nCRS Report R41983, How FDA Approves Drugs and Regulates Their Safety and Effectiveness, by Susan Thaul.\nCRS Report RL33986, FDA\u2019s Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective, by Susan Thaul.\nCRS Report R42130, FDA Regulation of Medical Devices, by Judith A. Johnson.\nCRS Report R42508, The FDA Medical Device User Fee Program, by Judith A. Johnson.\n(Note: The rest of this report has not been updated since November 10, 2011.)\nWith 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\u2019 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\u2019s impact. The report also discusses pediatric drug issues that remain of concern to some in Congress.\nMost 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\u2014that is, how much gets into a patient\u2019s system and is available for use\u2014varies in children for reasons that include a child\u2019s 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.\nDrug 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\u2019s market exclusivity for that product for six months, not approving the sale of another manufacturer\u2019s product during that period. In 2002, BPCA (P.L. 107-109) reauthorized this program for five years.\nIn 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.\nIn 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\u2014including the higher prices paid by government\u2014compare 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?", "type": "CRS Report", "typeId": "REPORTS", "active": false, "formats": [ { "format": "HTML", "encoding": "utf-8", "url": "http://www.crs.gov/Reports/RL33986", "sha1": "22dfce0e68fad6fc2e5cee70cc7116b7865a8196", "filename": "files/20120625_RL33986_22dfce0e68fad6fc2e5cee70cc7116b7865a8196.html", "images": null }, { "format": "PDF", "encoding": null, "url": "http://www.crs.gov/Reports/pdf/RL33986", "sha1": "4337985db463f1080ac90763acf78b1efb3bc094", "filename": "files/20120625_RL33986_4337985db463f1080ac90763acf78b1efb3bc094.pdf", "images": null } ], "topics": [] }, { "source": "University of North Texas Libraries Government Documents Department", "sourceLink": "https://digital.library.unt.edu/ark:/67531/metadc822699/", "id": "RL33986_2008Dec02", "date": "2008-12-02", "retrieved": "2016-03-19T13:57:26", "title": "FDA\u2019s Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective", "summary": "The Food and Drug Administration (FDA) has approved for adult use many drugs never tested in\r\nchildren. Yet clinicians often prescribe them for children believing that the safety and effectiveness demonstrated with adults probably reasonably transfers to younger patients. The data show that this is not always true. To encourage industry to develop drugs and medical devices for pediatric use, Congress has established three programs. The Food and Drug Administration Amendments Act of 2007 (FDAAA, P.L. 110-85) reauthorized and strengthened two laws addressing drugs\u2014the Best Pharmaceuticals for Children Act (BPCA) of 2002 and the Pediatric Research Equity Act (PREA) of 2003\u2014and enacted a new law addressing devices\u2014the Pediatric Medical Device Safety and Improvement Act (PMDSIA) of 2007. The historical approach of this report allows an understanding of how and why Congress took these steps.", "type": "CRS Report", "typeId": "REPORT", "active": false, "formats": [ { "format": "PDF", "filename": "files/20081202_RL33986_76419e532fa147d8d678c48533f74d4dbd32bfec.pdf" }, { "format": "HTML", "filename": "files/20081202_RL33986_76419e532fa147d8d678c48533f74d4dbd32bfec.html" } ], "topics": [ { "source": "LIV", "id": "Drug industry", "name": "Drug industry" }, { "source": "LIV", "id": "Medicine", "name": "Medicine" }, { "source": "LIV", "id": "Drug law and legislation", "name": "Drug law and legislation" }, { "source": "LIV", "id": "Consumer protection", "name": "Consumer protection" } ] }, { "source": "University of North Texas Libraries Government Documents Department", "sourceLink": "https://digital.library.unt.edu/ark:/67531/metadc820482/", "id": "RL33986_2007May01", "date": "2007-05-01", "retrieved": "2016-03-19T13:57:26", "title": "FDA\u2019s Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective", "summary": null, "type": "CRS Report", "typeId": "REPORT", "active": false, "formats": [ { "format": "PDF", "filename": "files/20070501_RL33986_4f3f05e84bb7442ebeefe53c1118eb26727abafc.pdf" }, { "format": "HTML", "filename": "files/20070501_RL33986_4f3f05e84bb7442ebeefe53c1118eb26727abafc.html" } ], "topics": [] } ], "topics": [ "Economic Policy", "Health Policy" ] }