{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T12:15:40Z","timestamp":1791375340548,"version":"4.3.1"},"reference-count":15,"publisher":"American Academy of Pediatrics (AAP)","issue":"5","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2011,11,1]]},"abstract":"<jats:sec>\n                    <jats:title>OBJECTIVES:<\/jats:title>\n                    <jats:p>In 1994, the US Food and Drug Administration (FDA) proposed an approach, based on extrapolation of efficacy findings from adults to the pediatric population, to maximize the use of adult data and other data when designing pediatric drug-development programs. We examined the experience of the FDA in using extrapolation to evaluate how and when it was used and any changes in scientific assumptions over time.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>METHODS:<\/jats:title>\n                    <jats:p>We reviewed 370 pediatric studies submitted to the FDA between 1998 and 2008 in response to 159 written requests (166 products) issued under the Pediatric Exclusivity Provision. We identified cases in which efficacy was extrapolated from adult data or other data, we categorized the type of pediatric data required to support extrapolation, and we determined whether the data resulted in new pediatric labeling.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>RESULTS:<\/jats:title>\n                    <jats:p>Extrapolation of efficacy from adult data occurred for 82.5% of the drug products (137 of 166). Extrapolation was defined as complete for 14.5% of the products (24 of 166) and partial for 68% of them (113 of 166). Approaches to extrapolation changed over time for 19% of the therapeutic indications studied (13 of 67). When extrapolation was used, 61% of the drug products (84 of 137) obtained a new pediatric indication or extension into a new age group; this number decreased to 34% (10 of 29) when there was no extrapolation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>CONCLUSIONS:<\/jats:title>\n                    <jats:p>Extrapolating efficacy from adult data or other data to the pediatric population can streamline pediatric drug development and help to increase the number of approvals for pediatric use.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1542\/peds.2010-3487","type":"journal-article","created":{"date-parts":[[2011,10,24]],"date-time":"2011-10-24T20:59:48Z","timestamp":1319489988000},"page":"e1242-e1249","source":"Crossref","is-referenced-by-count":283,"title":["Extrapolation of Adult Data and Other Data in Pediatric Drug-Development Programs"],"prefix":"10.1542","volume":"128","author":[{"given":"Julia","family":"Dunne","sequence":"first","affiliation":[{"name":"Office of Pediatric Therapeutics, Office of the Commissioner,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William J.","family":"Rodriguez","sequence":"additional","affiliation":[{"name":"Office of Pediatric Therapeutics, Office of the Commissioner,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Dianne","family":"Murphy","sequence":"additional","affiliation":[{"name":"Office of Pediatric Therapeutics, Office of the Commissioner,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B. Nhi","family":"Beasley","sequence":"additional","affiliation":[{"name":"Divisions of Cardiorenal Products,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gilbert J.","family":"Burckart","sequence":"additional","affiliation":[{"name":"Office of Clinical Pharmacology, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane D.","family":"Filie","sequence":"additional","affiliation":[{"name":"Anesthesia, Analgesia, and Rheumatology Products,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda L.","family":"Lewis","sequence":"additional","affiliation":[{"name":"Antiviral Products,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hari C.","family":"Sachs","sequence":"additional","affiliation":[{"name":"Pediatric and Maternal Health Staff,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philip H.","family":"Sheridan","sequence":"additional","affiliation":[{"name":"Neurology Products,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Starke","sequence":"additional","affiliation":[{"name":"Pulmonary and Allergy Products, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lynne P.","family":"Yao","sequence":"additional","affiliation":[{"name":"Gastroenterology Products, and"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2011,11,1]]},"reference":[{"issue":"1","key":"2021120900345027600_B1","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1016\/S0022-3476(68)80414-7","article-title":"Therapeutic orphans","volume":"72","author":"Shirkey","year":"1968","journal-title":"J Pediatr"},{"issue":"3","key":"2021120900345027600_B2","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1542\/peds.104.S3.585","article-title":"An update on the therapeutic orphan","volume":"104","author":"Wilson","year":"1999","journal-title":"Pediatrics"},{"key":"2021120900345027600_B3","article-title":"Specific requirements on content and format of labeling for human prescription drugs: revision of \u201cpediatric use\u201d subsection in the labeling: final rule","volume":"59","author":"Food and Drug Administration","year":"1994","journal-title":"Fed Regist"},{"key":"2021120900345027600_B4","unstructured":"Food and Drug Administration. Food and Drug Administration Modernization Act of 1997. Available at: www.fda.gov\/RegulatoryInformation\/Legislation\/FederalFoodDrugandCosmeticActFDCAct\/SignificantAmendmentstotheFDCAct\/FDAMA\/FullTextofFDAMAlaw\/default.htm. Accessed March 7, 2011"},{"key":"2021120900345027600_B5","unstructured":"Food and Drug Administration. Best Pharmaceuticals for Children Act. Available at: www.fda.gov\/RegulatoryInformation\/Legislation\/FederalFoodDrugandCosmeticActFDCAct\/SignificantAmendmentstotheFDCAct\/ucm148011.htm. Accessed March 7, 2011"},{"key":"2021120900345027600_B6","unstructured":"Food and Drug Administration. Food and Drug Administration Amendments Act of 2007. Available at: www.fda.gov\/downloads\/Drugs\/DevelopmentApprovalProcess\/DevelopmentResources\/UCM049870.pdf. Accessed March 7, 2011"},{"key":"2021120900345027600_B7","unstructured":"Food and Drug Administration. Breakdown of requested studies report. Available at: www.fda.gov\/Drugs\/DevelopmentApprovalProcess\/DevelopmentResources\/ucm050001.htm. Accessed March 7, 2011"},{"key":"2021120900345027600_B8","unstructured":"Food and Drug Administration. Guidance for Industry: Exposure-Response Relationships: Study Design, Data Analysis, and Regulatory Applications. Washington, DC: Food and Drug Administration; 2003. Available at: www.fda.gov\/downloads\/Drugs\/GuidanceComplianceRegulatoryInformation\/Guidances\/UCM072109.pdf. Accessed March 7, 2011"},{"issue":"3","key":"2021120900345027600_B9","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1067\/mhj.2002.124402","article-title":"The Pediatric Randomized Carvedilol Trial in Children With Heart Failure: rationale and design","volume":"144","author":"Shaddy","year":"2002","journal-title":"Am Heart J"},{"issue":"10","key":"2021120900345027600_B10","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1001\/jama.298.10.1171","article-title":"Carvedilol for children and adolescents with heart failure: a randomized controlled trial","volume":"298","author":"Shaddy","year":"2007","journal-title":"JAMA"},{"issue":"4","key":"2021120900345027600_B11","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1111\/j.1365-2125.2007.03046.x","article-title":"Population pharmacokinetics and dose simulation of carvedilol in paediatric patients with congestive heart failure","volume":"65","author":"Albers","year":"2008","journal-title":"Br J Clin Pharmacol"},{"issue":"3","key":"2021120900345027600_B12","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1007\/s00380-008-1102-5","article-title":"Efficacy and safety of carvedilol for heart failure in children and patients with congenital heart disease","volume":"24","author":"Nishiyama","year":"2009","journal-title":"Heart Vessels"},{"issue":"4","key":"2021120900345027600_B13","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1161\/HYPERTENSIONAHA.107.108886","article-title":"Pediatric antihypertensive trial failures: analysis of endpoints and dose range","volume":"51","author":"Benjamin","year":"2008","journal-title":"Hypertension"},{"key":"2021120900345027600_B14","doi-asserted-by":"crossref","first-page":"555","DOI":"10.1542\/peds.114.S2.555","article-title":"The fourth report on the diagnosis, evaluation, and treatment of high blood pressure in children and adolescents","volume":"114","author":"National High Blood Pressure Education Program Working Group on High Blood Pressure in Children and Adolescents","year":"2004","journal-title":"Pediatrics"},{"issue":"3","key":"2021120900345027600_B15","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1016\/j.rdc.2007.07.006","article-title":"Juvenile idiopathic arthritis","volume":"33","author":"Weiss","year":"2007","journal-title":"Rheum Dis Clin North Am"}],"container-title":["Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/128\/5\/e1242\/845018\/zpe011110e1242.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/128\/5\/e1242\/845018\/zpe011110e1242.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,9]],"date-time":"2021-12-09T01:38:02Z","timestamp":1639013882000},"score":1,"resource":{"primary":{"URL":"https:\/\/publications.aap.org\/pediatrics\/article\/128\/5\/e1242\/30987\/Extrapolation-of-Adult-Data-and-Other-Data-in"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,11,1]]},"references-count":15,"journal-issue":{"issue":"5","published-online":{"date-parts":[[2011,11,1]]},"published-print":{"date-parts":[[2011,11,1]]}},"URL":"https:\/\/doi.org\/10.1542\/peds.2010-3487","relation":{},"ISSN":["0031-4005","1098-4275"],"issn-type":[{"value":"0031-4005","type":"print"},{"value":"1098-4275","type":"electronic"}],"subject":[],"published":{"date-parts":[[2011,11,1]]}}}