title: Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants.
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.
authors: Office of Clinical Pharmacology, Center for Drug Evaluation and Research, FDA, Silver Spring, MD, USA. jane.bai@fda.hhs.gov
authors: Jane P F
authors: JP
authors: Bai
authors: Jeffrey S
authors: JS
authors: Barrett
authors: Gibert J
authors: GJ
authors: Burckart
authors: Bernd
authors: B
authors: Meibohm
authors: Hari Cheryl
authors: HC
authors: Sachs
authors: Lynne
authors: L
authors: Yao
mesh_terms: D000367:Age Factors; D000818:Animals; D015415:Biomarkers / Q000032:analysis*; D016638:Critical Illness; D017277:Drug Approval; D055808:Drug Discovery / Q000331:legislation & jurisprudence; D064420:Drug-Related Side Effects and Adverse Reactions; D023381:Endpoint Determination; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007232:Infant, Newborn, Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism*; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence; D061214:Patient Safety; D011237:Predictive Value of Tests; D035583:Rare Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism*; D012307:Risk Factors; D012720:Severity of Illness Index; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015415:Biomarkers