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

  • Review Article
  • Theme: Challenges and Opportunities in Pediatric Drug Development
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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.

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Author information

Authors and Affiliations

  1. Office of Clinical Pharmacology, Center for Drug Evaluation and Research, FDA, Silver Spring, Maryland, USA

    Jane P. F. Bai & Gibert J. Burckart

  2. Pediatric Pharmacology Research Unit, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA

    Jeffrey S. Barrett

  3. Department of Pharmaceutical Sciences, College of Pharmacy, University of Tennessee Health Science Center, Memphis, Tennessee, USA

    Bernd Meibohm

  4. Pediatric and Maternal Health Staff, Office of New Drugs, Center for Drug Evaluation and Research, FDA, Silver Spring, Maryland, USA

    Hari Cheryl Sachs & Lynne Yao

Authors
  1. Jane P. F. Bai
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  2. Jeffrey S. Barrett
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  3. Gibert J. Burckart
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  4. Bernd Meibohm
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  5. Hari Cheryl Sachs
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  6. Lynne Yao
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Corresponding authors

Correspondence to Jane P. F. Bai or Lynne Yao.

Additional information

Guest Editors: Bernd Meibohm, Jeffrey S. Barrett, and Gregory Knipp

Disclaimer

The views expressed in this article are not intended to represent the views of the US Food and Drug Administration.

This manuscript is partially based on the AAPS symposium at the 2009 AAPS annual meeting in Los Angeles, California, USA.

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Bai, J.P.F., Barrett, J.S., Burckart, G.J. et al. Strategic Biomarkers for Drug Development in Treating Rare Diseases and Diseases in Neonates and Infants. AAPS J 15, 447–454 (2013). https://doi.org/10.1208/s12248-013-9452-z

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  • DOI: https://doi.org/10.1208/s12248-013-9452-z

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