Document Detail

Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants.
MedLine Citation:
PMID:  23334978     Owner:  NLM     Status:  MEDLINE    
There are similar challenges in developing a product designed to treat patients with a rare disease and drugs to treat critically ill neonates and infants. Part of the challenge in developing such products as well as identifying the optimal dosing regimen for the treatment of young children arises from the complex interrelationship between developmental changes and changes in biomarkers responsive to drug therapy. These difficulties are further compounded by our lack of understanding of the key physiological factors that cause the differences in clinical responses between adults and neonates and infants. Regulatory efforts have succeeded in overcoming these challenges in many areas of pediatric and orphan drug development. Strategic applications of biomarkers and surrogate endpoints for the development and approval of a product used to treat an orphan disease will be highlighted with examples of approved products. Continued efforts are still needed to fill in our knowledge gap and to strategically link biomarkers and surrogate endpoints to clinical responses for rare diseases and diseases affecting neonates and infants.
Jane P F Bai; Jeffrey S Barrett; Gibert J Burckart; Bernd Meibohm; Hari Cheryl Sachs; Lynne Yao
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Publication Detail:
Type:  Journal Article; Review     Date:  2013-01-19
Journal Detail:
Title:  The AAPS journal     Volume:  15     ISSN:  1550-7416     ISO Abbreviation:  AAPS J     Publication Date:  2013 Apr 
Date Detail:
Created Date:  2013-04-05     Completed Date:  2013-10-22     Revised Date:  2014-01-23    
Medline Journal Info:
Nlm Unique ID:  101223209     Medline TA:  AAPS J     Country:  United States    
Other Details:
Languages:  eng     Pagination:  447-54     Citation Subset:  IM    
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MeSH Terms
Age Factors
Biological Markers / analysis*
Critical Illness
Drug Approval
Drug Discovery* / legislation & jurisprudence
Drug-Related Side Effects and Adverse Reactions
Endpoint Determination
Infant, Newborn
Infant, Newborn, Diseases / diagnosis,  drug therapy*,  metabolism*
Orphan Drug Production* / legislation & jurisprudence
Patient Safety
Predictive Value of Tests
Rare Diseases / diagnosis,  drug therapy*,  metabolism*
Risk Factors
Severity of Illness Index
Treatment Outcome
United States
United States Food and Drug Administration
Reg. No./Substance:
0/Biological Markers

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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