A hierarchical Bayesian approach for combining pharmacokinetic/pharmacodynamic modeling and Phase IIa trial design in orphan drugs: Treating adrenoleukodystrophy with Lorenzo’s oil

Journal of Biopharmaceutical Statistics

Volumn 26, Issue 6, 2016, Pages 1025-1039

  Basu, Cynthia ^a   Ahmed, Mariam A ^b,c   Kartha, Reena V ^b   Brundage, Richard C ^b   Raymond, Gerald V ^d   Cloyd, James C ^b   Carlin, Bradley P ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

^c HELWAN UNIVERSITY   (Egypt)

^d UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

Adaptive clinical trial design; Bayesian hierarchical modeling; Lorenzo s oil; orphan disease; pediatric drug development; PK PD modeling; X ALD

Indexed keywords

ERUCIC ACID; LORENZO OIL; VERY LONG CHAIN FATTY ACID; DRUG COMBINATION; TRIOLEIN;

ADRENOLEUKODYSTROPHY; ARTICLE; BAYES THEOREM; CONCENTRATION AT STEADY-STATE; CONCEPTUAL FRAMEWORK; DRUG EFFICACY; HUMAN; MALE; PHARMACODYNAMICS; PHARMACOKINETICS; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; SIMULATION; BLOOD; DOSE RESPONSE; DRUG COMBINATION; DRUG MANUFACTURE; METHODOLOGY;

ADRENOLEUKODYSTROPHY; BAYES THEOREM; CLINICAL TRIALS, PHASE II AS TOPIC; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG COMBINATIONS; ERUCIC ACIDS; HUMANS; MALE; ORPHAN DRUG PRODUCTION; RESEARCH DESIGN; TRIOLEIN;

EID: 84989809572     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2016.1226326     Document Type: Article

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