• Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Scientific Publications by FDA Staff

  • Print
  • Share
  • E-mail

Search Publications



Starting Date

Ending Date

Order by

Entry Details

AAPS J 2013 Apr;15(2):447-54

Strategic Biomarkers for Drug Development in Treating Rare Diseases and Diseases in Neonates and Infants.

Bai JP, Barrett JS, Burckart GJ, Meibohm B, Sachs HC, Yao L


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.

Category: Journal Article, Review
PubMed ID: #23334978 DOI: 10.1208/s12248-013-9452-z
Includes FDA Authors from Scientific Area(s): Drugs
Entry Created: 2013-01-22 Entry Last Modified: 2013-05-29