Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants

AAPS Journal

Volumn 15, Issue 2, 2013, Pages 447-454

  Bai, Jane P F ^a   Barrett, Jeffrey S ^b   Burckart, Gibert J ^a   Meibohm, Bernd ^c   Sachs, Hari Cheryl ^d   Yao, Lynne ^a,d  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^d CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

biomarkers; infants; neonates; rare diseases

Indexed keywords

AGALSIDASE BETA; ALGLUCERASE; ARGATROBAN; BIOLOGICAL MARKER; CHOLESTEROL; CYCLOSPORIN; CYTOCHROME P450 2C19; CYTOCHROME P450 2E1; DRUG METABOLIZING ENZYME; ESOMEPRAZOLE; GENE ACTIVATED HUMAN GLUCOCEREBROSIDASE; GLOBOTRIAOSYLCERAMIDE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDE; HEMOGLOBIN; IMIGLUCERASE; IMIQUIMOD; LARONIDASE; LEVO IDURONIDASE; MIGLUSTAT; MILRINONE; MULTIDRUG RESISTANCE PROTEIN; ORPHAN DRUG; SOTALOL; TALIGLUCERASE ALFA; UNCLASSIFIED DRUG; VELAGLUCERASE;

AGE DISTRIBUTION; ANTIARRHYTHMIC ACTIVITY; BODY SIZE; CHOLESTEROL BLOOD LEVEL; CLINICAL PRACTICE; CLINICAL RESEARCH; CONDYLOMA ACUMINATUM; CONTINUOUS INFUSION; CRITICAL ILLNESS; DISEASE COURSE; DRUG APPROVAL; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISPOSITION; DRUG DISTRIBUTION; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG INDICATION; DRUG LABELING; DRUG PENETRATION; DRUG SAFETY; ENZYME REPLACEMENT; EVIDENCE BASED MEDICINE; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GASTROESOPHAGEAL REFLUX; GAUCHER DISEASE; GLOMERULUS FILTRATION RATE; GOVERNMENT REGULATION; HEMOGLOBIN BLOOD LEVEL; HUMAN; HURLER SYNDROME; HYPERCHOLESTEROLEMIA; HYPOPLASTIC LEFT HEART SYNDROME; INBORN ERROR OF METABOLISM; KIDNEY CLEARANCE; MOLLUSCUM CONTAGIOSUM; ONTOGENY; PATIENT CARE; PHARMACOGENETICS; PRESCRIPTION; RARE DISEASE; REVIEW; SURVIVAL;

AGE FACTORS; ANIMALS; BIOLOGICAL MARKERS; CRITICAL ILLNESS; DRUG APPROVAL; DRUG DISCOVERY; DRUG THERAPY; ENDPOINT DETERMINATION; HUMANS; INFANT; INFANT, NEWBORN; INFANT, NEWBORN, DISEASES; ORPHAN DRUG PRODUCTION; PATIENT SAFETY; PREDICTIVE VALUE OF TESTS; RARE DISEASES; RISK FACTORS; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84877066175     PISSN: 15507416     EISSN: None     Source Type: Journal    
DOI: 10.1208/s12248-013-9452-z     Document Type: Review

References (47)

1. EMA/794083/2009. Report on the survey of all paediatric uses of medicinal products in Europe. London, UK: Europena Mediciens Agency; 2010.
2. Sachs AN, Avant D, Lee CS, Rodriguez W, Murphy MD. Pediatric information in drug product labeling. JAMA. 2012;307(18):1914-5. Comparative study.
3. FDASIA (the Food and Drug Administration Safety and Innovation Act) http://insidefdagov:9003/ORA/ucm316580htm. Accessed Oct 2012.
4. Orphan Drug Act, Pub. L. 97-414. 96 Stat. 2049. (1984 as amended).
5. Cote TR, Xu K, Pariser AR. Accelerating orphan drug development. Nat Rev Drug Discov. 2010;9(12):901-2. Comment.
6. FDA U. Code of Federal Regulations Title 2: Adequate and well-controlled studies Pt. 314.126. US Government Printing Office, Washington, DC. 2009.
7. Biomarkers Definitions Working Group. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther. 2001;69(3):89-95. Review.
8. Najafian B, Svarstad E, Bostad L, Gubler MC, Tondel C, Whitley C, et al. Progressive podocyte injury and globotriaosylceramide (GL-3) accumulation in young patients with Fabry disease. Kidney Int. 2011;79(6):663-70. Multicenter study.
9. Cox TM. Biomarkers in lysosomal storage diseases. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry disease: perspectives from 5 years of FOS. Oxford: Oxford PharmaGenesis; 2006.
10. Guidance for industry. Qualification process for drug development tools. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/UCM230597.pdf. 2010. Accessed Jan 2013.
11. Dunne J, Rodriguez WJ, Murphy MD, Beasley BN, Burckart GJ, Filie JD, et al. Extrapolation of adult data and other data in pediatric drug-development programs. Pediatrics. 2011;128(5):e1242-9. Comparative study.
12. Register F. 21 CFR Parts 201, 312, 314 and 601 (DOCID: Fr02de98-24). http://wwwfdagov/ohrms/dockets/98fr/120298ctxt. 1998 December 2;63(231): 66631-72.
13. Blake MJ, Castro L, Leeder JS, Kearns GL. Ontogeny of drug metabolizing enzymes in the neonate. Semin Fetal Neonatal Med. 2005;10(2):123-38.
14. Kearns GL, Abdel-Rahman SM, Alander SW, Blowey DL, Leeder JS, Kauffman RE. Developmental pharmacology-drug disposition, action, and therapy in infants and children. N Engl J Med. 2003;349(12):1157-67.
15. Rhodin MM, Anderson BJ, Peters AM, Coulthard MG, Wilkins B, Cole M, et al. Human renal function maturation: A quantitative description using weight and postmenstrual age. Pediatr Nephrol. 2009;24(1):67-76.
16. de Wildt SN, Kearns GL, Leeder JS, van den Anker JN. Cytochrome P450 3A: Ontogeny and drug disposition. Clin Pharmacokinet. 1999;37(6):485-505.
17. Stevens JC, Hines RN, Gu C, Koukouritaki SB, Manro JR, Tandler PJ, et al. Developmental expression of the major human hepatic CYP3A enzymes. J Pharmacol Exp Ther. 2003;307(2):573-82.
18. Koukouritaki SB, Manro JR, Marsh SA, Stevens JC, Rettie AE, McCarver DG, et al. Developmental expression of human hepatic CYP2C9 and CYP2C19. J Pharmacol Exp Ther. 2004;308(3):965-74.
19. Hines RN. Developmental and tissue-specific expression of human flavin-containing monooxygenases 1 and 3. Expert Opin Drug Metab Toxicol. 2006;2(1):41-9.
20. de Wildt SN, Kearns GL, Leeder JS, van den Anker JN. Glucuronidation in humans. Pharmacogenetic and developmental aspects. Clin Pharmacokinet. 1999;36(6):439-52.
21. Strassburg CP, Strassburg A, Kneip S, Barut A, Tukey RH, Rodeck B, et al. Developmental aspects of human hepatic drug glucuronidation in young children and adults. Gut. 2002;50(2):259-65. Research support, non-U.S. Gov't.
22. Alcorn J, McNamara PJ. Ontogeny of hepatic and renal systemic clearance pathways in infants: part II. Clin Pharmacokinet. 2002;41(13):1077-94.
23. Alcorn J, McNamara PJ. Ontogeny of hepatic and renal systemic clearance pathways in infants: part I. Clin Pharmacokinet. 2002;41(12):959-98.
24. Leeder JS, Kearns GL, Spielberg SP, van den Anker J. Understanding the relative roles of pharmacogenetics and ontogeny in pediatric drug development and regulatory science. J Clin Pharmacol. 2010;50(12):1377-87. Research support, N.I.H., extramural.
25. Becker ML, Leeder JS. Identifying genomic and developmental causes of adverse drug reactions in children. Pharmacogenomics. 2010;11(11):1591-602. Research support, N.I.H., extramural research support, non-U.S. gov't review.
26. Tang L, Rines R, Schuetz E, Meibohm B. Age-associated expression of P-glycoprotein and MRP2 in human pediatric liver. Clin Pharmacol Ther. 2007;81:S101.
27. Nong A, McCarver DG, Hines RN, Krishnan K. Modeling interchild differences in pharmacokinetics on the basis of subject-specific data on physiology and hepatic CYP2E1 levels: A case study with toluene. Toxicol Appl Pharmacol. 2006;214(1):78-87.
28. Edginton AN, Schmitt W, Voith B, Willmann S. A mechanistic approach for the scaling of clearance in children. Clin Pharmacokinet. 2006;45(7):683-704.
29. Marshall JD, Kearns GL. Developmental pharmacodynamics of cyclosporine. Clin Pharmacol Ther. 1999;66(1):66-75.
30. Laer S, Elshoff JP, Meibohm B, Weil J, Mir TS, Zhang W, et al. Development of a safe and effective pediatric dosing regimen for sotalol based on population pharmacokinetics and pharmacodynamics in children with supraventricular tachycardia. J Am Coll Cardiol. 2005;46(7):1322-30.
31. Laer S, Barrett JS, Meibohm B. The in silico child: Using simulation to guide pediatric drug development and manage pediatric pharmacotherapy. J Clin Pharmacol. 2009;49(8):889-904.
32. Meibohm B, Laer S, Panetta JC, Barrett JS. Population pharmacokinetic studies in pediatrics: Issues in design and analysis. AAPS J. 2005;7(2):E475-87.
33. Bonate PL. The effect of collinearity on parameter estimates in nonlinear mixed effect models. Pharm Res. 1999;16(5):709-17.
34. Anderson BJ, Holford NH. Mechanistic basis of using body size and maturation to predict clearance in humans. Drug Metab Pharmacokinet. 2009;24(1):25-36.
35. Anderson BJ, McKee AD, Holford NH. Size, myths and the clinical pharmacokinetics of analgesia in paediatric patients. Clin Pharmacokinet. 1997;33(5):313-27.
36. Albers S, Meibohm B, Mir TS, Laer S. Population pharmacokinetics and dose simulation of carvedilol in paediatric patients with congestive heart failure. Br J Clin Pharmacol. 2008;65(4):511-22.
37. Barrett J. Modeling and simulation in pediatric research and development. In: Kimko H, Peck C, editors. Clinical trial simulations: Applications & trends. New York: Springer Science + Business Media; 2010.
38. Cohen-Wolkowiez M, Ouellet D, Smith PB, James LP, Ross A, Sullivan JE, et al. Population pharmacokinetics of metronidazole evaluated using scavenged samples from preterm infants. Antimicrob Agents Chemother. 2012;56(4):1828-37. Multicenter Study Research Support, N.I.H., Extramural.
39. Ginsberg G, Hattis D, Miller R, Sonawane B. Pediatric pharmacokinetic data: Implications for environmental risk assessment for children. Pediatrics. 2004;113(4 Suppl):973-83. Research support, non-U.S. gov't research support, U.S. gov't, non-P.H.S.
40. Yang F, Tong X, McCarver DG, Hines RN, Beard DA. Population-based analysis of methadone distribution and metabolism using an age-dependent physiologically based pharmacokinetic model. J Pharmacokinet Pharmacodyn. 2006;33(4):485-518. Research support, N.I.H., extramural.
41. Leong R, Vieira ML, Zhao P, Mulugeta Y, Lee CS, Huang SM, et al. Regulatory experience with physiologically based pharmacokinetic modeling for pediatric drug trials. Clin Pharmacol Ther. 2012;91(5):926-31.
42. Chen IL, Gao WY, Johnson AP, Niak A, Troiani J, Korvick J, et al. Proton pump inhibitor use in infants: FDA reviewer experience. J Pediatr Gastroenterol Nutr. 2012;54(1):8-14.
43. Zuppa AF, Nicolson SC, Adamson PC, Wernovsky G, Mondick JT, Burnham N, et al. Population pharmacokinetics of milrinone in neonates with hypoplastic left heart syndrome undergoing stage I reconstruction. Anesth Analg. 2006;102(4):1062-9. comparative study randomized controlled trial research support, N.I.H., extramural research support, non-U.S. gov't.
44. Paradisis M, Jiang X, McLachlan AJ, Evans N, Kluckow M, Osborn D. Population pharmacokinetics and dosing regimen design of milrinone in preterm infants. Arch Dis Child Fetal Neonatal Ed. 2007;92(3):F204-9. multicenter study research support, non-U.S. gov't.
45. Argatroban. http://www.accessdata.fda.gov/drugsatfda-docs/abel/2008/ 020883s014lbl.pdf. Drugs@FDA. Accessed 2012.
46. Davis JM, Connor EM, Wood AJ. The need for rigorous evidence on medication use in preterm infants: Is it time for a neonatal rule? JAMA. 2012;308(14):1435-6. research support, N.I.H., extramural.
47. Drugs@FDA. http://wwwaccessdatafdagov/scripts/cder/drugsatfda/indexcfm. Accessed Jan 2012.