Orphan drug development: the increasing role of clinical pharmacology

Mariam A. Ahmed, Malek Okour, Richard Brundage, Reena V. Kartha

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


Over the last few decades there has been a paradigm shift in orphan drug research and development. The development of the regulatory framework, establishment of rare disease global networks that support drug developments, and advances in technology, has resulted in tremendous growth in orphan drug development. Nevertheless, several challenges during orphan drug development such as economic constraints; insufficient clinical information; fewer patients and thus inadequate power; etc. still exist. While the standard regulatory requirements for drug approval stays the same, applications of scientific judgment and regulatory flexibility is significantly important to help meeting some of the immense unmet medical need in rare diseases. Clinical pharmacology presents a vital role in accelerating orphan drug development and overcoming some of these challenges. This review highlights the critical contributions of clinical pharmacology in orphan drug development; for example, dose finding, optimizing clinical trial design, indication expansion, and population extrapolation. Examples of such applications are reviewed in this article.

Original languageEnglish (US)
Pages (from-to)395-409
Number of pages15
JournalJournal of Pharmacokinetics and Pharmacodynamics
Issue number5
StatePublished - Oct 1 2019


  • Model informed drug discovery and development
  • Modeling and simulations
  • Orphan drugs
  • Rare diseases

PubMed: MeSH publication types

  • Journal Article
  • Review

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