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

Jane P.F. Bai, Jeffrey S. Barrett, Gibert J. Burckart, Bernd Meibohm, Hari Cheryl Sachs, Lynne Yao

Research output: Contribution to journalReview article

18 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)447-454
Number of pages8
JournalAAPS Journal
Volume15
Issue number2
DOIs
StatePublished - Apr 1 2013

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Rare Diseases
Biomarkers
Newborn Infant
Pharmaceutical Preparations
Consumer Product Safety
Orphan Drug Production
Critical Illness
Pediatrics
Drug Therapy
Therapeutics

All Science Journal Classification (ASJC) codes

  • Pharmaceutical Science

Cite this

Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants. / Bai, Jane P.F.; Barrett, Jeffrey S.; Burckart, Gibert J.; Meibohm, Bernd; Sachs, Hari Cheryl; Yao, Lynne.

In: AAPS Journal, Vol. 15, No. 2, 01.04.2013, p. 447-454.

Research output: Contribution to journalReview article

Bai, Jane P.F. ; Barrett, Jeffrey S. ; Burckart, Gibert J. ; Meibohm, Bernd ; Sachs, Hari Cheryl ; Yao, Lynne. / Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants. In: AAPS Journal. 2013 ; Vol. 15, No. 2. pp. 447-454.
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