TY - JOUR
T1 - Orphan drug development
T2 - the increasing role of clinical pharmacology
AU - Ahmed, Mariam A.
AU - Okour, Malek
AU - Brundage, Richard
AU - Kartha, Reena V.
N1 - Publisher Copyright:
© 2019, Springer Science+Business Media, LLC, part of Springer Nature.
PY - 2019/10/1
Y1 - 2019/10/1
N2 - 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.
AB - 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.
KW - Model informed drug discovery and development
KW - Modeling and simulations
KW - Orphan drugs
KW - Rare diseases
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U2 - 10.1007/s10928-019-09646-3
DO - 10.1007/s10928-019-09646-3
M3 - Review article
C2 - 31338634
AN - SCOPUS:85069529331
SN - 1567-567X
VL - 46
SP - 395
EP - 409
JO - Journal of Pharmacokinetics and Pharmacodynamics
JF - Journal of Pharmacokinetics and Pharmacodynamics
IS - 5
ER -