{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,22]],"date-time":"2026-01-22T17:52:04Z","timestamp":1769104324253,"version":"3.49.0"},"reference-count":50,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2020,7,9]],"date-time":"2020-07-09T00:00:00Z","timestamp":1594252800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100002429","name":"Amgen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002429","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008637","name":"Center for Strategic Scientific Initiatives, National Cancer Institute","doi-asserted-by":"publisher","award":["1\u2010R01\u2010CA157458\u201001A1"],"award-info":[{"award-number":["1\u2010R01\u2010CA157458\u201001A1"]}],"id":[{"id":"10.13039\/100008637","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmaceutical Statistics"],"published-print":{"date-parts":[[2020,11]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>In most drug development settings, the regulatory approval process is accompanied by extensive studies performed to understand the drug's pharmacokinetic (PK) and pharmacodynamic (PD) properties. In this article, we attempt to utilize the rich PK\/PD data to inform the borrowing of information from adults during pediatric drug development. In pediatric settings, it is especially crucial that we are parsimonious with the patients recruited for experimentation. We illustrate our approaches in the context of clinical trials of cinacalcet for treating secondary hyperparathyroidism in pediatric and adult patients with chronic kidney disease, where we model both parathyroid hormone (efficacy endpoint) and corrected calcium levels (safety endpoint). We use population PK\/PD modeling of the cinacalcet data to quantitatively assess the similarity between adults and children, and use this information in various hierarchical Bayesian adult borrowing rules whose statistical properties can then be evaluated. In particular, we simulate the bias and mean square error performance of our approaches in settings where borrowing is and is not warranted to inform guidelines for the future use of our methods.<\/jats:p>","DOI":"10.1002\/pst.2043","type":"journal-article","created":{"date-parts":[[2020,7,10]],"date-time":"2020-07-10T05:31:38Z","timestamp":1594359098000},"page":"882-896","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Pharmacokinetic\/pharmacodynamic data extrapolation models for improved pediatric efficacy and toxicity estimation, with application to secondary hyperparathyroidism"],"prefix":"10.1002","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5480-4731","authenticated-orcid":false,"given":"Cynthia","family":"Basu","sequence":"first","affiliation":[{"name":"Pfizer Inc. San Diego California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaoye","family":"Ma","sequence":"additional","affiliation":[{"name":"Genentech Inc. San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"May","family":"Mo","sequence":"additional","affiliation":[{"name":"Amgen Inc. Thousand Oaks California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hong Amy","family":"Xia","sequence":"additional","affiliation":[{"name":"Amgen Inc. Thousand Oaks California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Brundage","sequence":"additional","affiliation":[{"name":"Department of Experimental and Clinical Pharmacology College of Pharmacy, University of Minnesota Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mahmoud","family":"Al\u2010Kofahi","sequence":"additional","affiliation":[{"name":"Department of Experimental and Clinical Pharmacology College of Pharmacy, University of Minnesota Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bradley P.","family":"Carlin","sequence":"additional","affiliation":[{"name":"Counterpoint Statistical Consulting, LLC Edina Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,7,9]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.ki.5002287"},{"key":"e_1_2_11_3_1","unstructured":"Sensipar.http:\/\/www.sensipar.com\/about-sensipar\/how-sensipar-works.html. Accessed June 28 2017."},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1093\/ndt\/13.8.1923"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1124\/jpet.103.057273"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(04)16942-0"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-642-20195-0_11"},{"key":"e_1_2_11_8_1","first-page":"2121055685","volume-title":"Transforming Clinical Research in the United States. Challenges and Opportunities: Workshop Summary","author":"English R","year":"2010"},{"key":"e_1_2_11_9_1","volume-title":"Proceedings of the ASA Biopharmaceutical Conference","author":"Viele K","year":"2016"},{"key":"e_1_2_11_10_1","first-page":"107","article-title":"Best pharmaceuticals for children act","author":"Food and Drug Administration","year":"2002","journal-title":"Public Law"},{"key":"e_1_2_11_11_1","first-page":"97","article-title":"Orphan drug act","author":"Food and Drug Administration","year":"2013","journal-title":"Public Law"},{"key":"e_1_2_11_12_1","first-page":"108","article-title":"Pediatric research equity act","author":"Food and Drug Administration","year":"2003","journal-title":"Public Law"},{"key":"e_1_2_11_13_1","unstructured":"Food and Drug Administration.Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices: Draft Guidance for Industry and Food and Drug Administration Staff; 2015.www.fda.gov\/downloads\/MedicalDevices\/DeviceRegulationandGuidance\/GuidanceDocuments\/UCM444591.pdf. Accessed June 28 2017"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1201\/EBK1439825488"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1201\/b14884"},{"key":"e_1_2_11_16_1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/j.2517-6161.1972.tb00885.x","article-title":"Bayes' estimates for the linear model","volume":"34","author":"Lindley DV","year":"1972","journal-title":"J R Stat Soc Ser B"},{"key":"e_1_2_11_17_1","unstructured":"Food and Drug Administration.Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials.http:\/\/www.fda.gov\/medicaldevices\/deviceregulationandguidance\/guidancedocuments\/ucm071072.htm. Accessed June 28 2017."},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1214\/ss\/1009212673"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1541-0420.2011.01564.x"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1214\/12-BA722"},{"key":"e_1_2_11_21_1","unstructured":"Food and Drug Administration.Guidance for Industry: Population Pharmacokinetics; 1999.https:\/\/www.fda.gov\/downloads\/drugs\/guidances\/UCM072137.pdf. Accessed June 28 2017."},{"key":"e_1_2_11_22_1","doi-asserted-by":"publisher","DOI":"10.2307\/2532547"},{"key":"e_1_2_11_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jspi.2007.06.017"},{"key":"e_1_2_11_24_1","doi-asserted-by":"publisher","DOI":"10.1080\/10543400701514080"},{"key":"e_1_2_11_25_1","doi-asserted-by":"publisher","DOI":"10.1201\/b15054"},{"key":"e_1_2_11_26_1","doi-asserted-by":"publisher","DOI":"10.1093\/biomet\/84.2.429"},{"key":"e_1_2_11_27_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.12352"},{"key":"e_1_2_11_28_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.3588"},{"key":"e_1_2_11_29_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.13900"},{"key":"e_1_2_11_30_1","doi-asserted-by":"publisher","DOI":"10.1002\/jcph.460"},{"key":"e_1_2_11_31_1","doi-asserted-by":"publisher","DOI":"10.1002\/jcph.314"},{"key":"e_1_2_11_32_1","doi-asserted-by":"publisher","DOI":"10.1201\/b13613"},{"key":"e_1_2_11_33_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10928-012-9240-x"},{"key":"e_1_2_11_34_1","doi-asserted-by":"publisher","DOI":"10.1002\/bdd.575"},{"key":"e_1_2_11_35_1","doi-asserted-by":"publisher","DOI":"10.1023\/B:JOPA.0000012998.04442.1f"},{"key":"e_1_2_11_36_1","doi-asserted-by":"publisher","DOI":"10.1023\/B:JOPA.0000012999.36063.4e"},{"key":"e_1_2_11_37_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10928-005-0065-8"},{"key":"e_1_2_11_38_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40262-017-0550-4"},{"key":"e_1_2_11_39_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12106"},{"key":"e_1_2_11_40_1","doi-asserted-by":"publisher","DOI":"10.1080\/10543400701668274"},{"key":"e_1_2_11_41_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2010-3487"},{"key":"e_1_2_11_42_1","unstructured":"Food and Drug Administration.Physiologically Based Pharmacokinetic Analyses \u2013 Format and Content: Guidance for Industry; 2018.http:\/\/www.fda.gov\/media\/101469\/download. Accessed March 16 2020."},{"key":"e_1_2_11_43_1","doi-asserted-by":"publisher","DOI":"10.1289\/ehp.00108s3451"},{"key":"e_1_2_11_44_1","unstructured":"Amgen Inc.Sensipar: Highlights of Prescribing Information;2004.https:\/\/www.pi.amgen.com\/~\/media\/amgen\/repositorysites\/pi-amgen-com\/sensipar\/sensipar_pi_hcp_english.ashx. Accessed February 25 2020."},{"key":"e_1_2_11_45_1","doi-asserted-by":"publisher","DOI":"10.1177\/1740774513483934"},{"key":"e_1_2_11_46_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1541-0420.2007.00888.x"},{"key":"e_1_2_11_47_1","doi-asserted-by":"publisher","DOI":"10.1214\/12-BA720"},{"key":"e_1_2_11_48_1","volume-title":"Proceedings of the ASA Biopharmaceutical Section FDA\u2010Industry Statistics Workshop","author":"Pennello G","year":"2016"},{"key":"e_1_2_11_49_1","unstructured":"European Medicines Agency.Reflection Paper on the Use of Extrapolation in the Development of Medicines for Paediatrics; 2018.https:\/\/www.ema.europa.eu\/documents\/scientific-guideline\/adopted-reflection-paper-use-extrapolation-development-medicines-paediatrics-revision-1_en.pdf. Accessed March 16 2020."},{"key":"e_1_2_11_50_1","unstructured":"International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use.E11(R1): Clinical Investigation of Medicinal Products in the Pediatric Population; 2014.https:\/\/database.ich.org\/sites\/default\/files\/E11_R1_Concept_Paper_0.pdf. Accessed March 16 2020."},{"key":"e_1_2_11_51_1","unstructured":"Food and Drug Administration. E11(R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population: Guidance for Industry; 2018.https:\/\/www.fda.gov\/media\/101398\/download. Accessed March 16 2020."}],"container-title":["Pharmaceutical Statistics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpst.2043","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pst.2043","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pst.2043","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pst.2043","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,8,9]],"date-time":"2024-08-09T20:13:09Z","timestamp":1723234389000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pst.2043"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,7,9]]},"references-count":50,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2020,11]]}},"alternative-id":["10.1002\/pst.2043"],"URL":"https:\/\/doi.org\/10.1002\/pst.2043","archive":["Portico"],"relation":{},"ISSN":["1539-1604","1539-1612"],"issn-type":[{"value":"1539-1604","type":"print"},{"value":"1539-1612","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,7,9]]},"assertion":[{"value":"2019-07-01","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-05-23","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-07-09","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}