Orphan drug development: the increasing role of clinical pharmacology

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

Research output: Contribution to journalReview article

Abstract

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
Volume46
Issue number5
DOIs
StatePublished - Oct 1 2019

Fingerprint

Orphan Drug Production
Clinical Pharmacology
Rare Diseases
Drug Approval
Economics
Clinical Trials
Technology
Growth
Research
Pharmaceutical Preparations
Population

Keywords

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

Cite this

Orphan drug development : the increasing role of clinical pharmacology. / Ahmed, Mariam A.; Okour, Malek; Brundage, Richard; Kartha, Reena V.

In: Journal of Pharmacokinetics and Pharmacodynamics, Vol. 46, No. 5, 01.10.2019, p. 395-409.

Research output: Contribution to journalReview article

@article{ce165840951c4886a29c6e795f911e30,
title = "Orphan drug development: the increasing role of clinical pharmacology",
abstract = "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.",
keywords = "Model informed drug discovery and development, Modeling and simulations, Orphan drugs, Rare diseases",
author = "Ahmed, {Mariam A.} and Malek Okour and Richard Brundage and Kartha, {Reena V.}",
year = "2019",
month = "10",
day = "1",
doi = "10.1007/s10928-019-09646-3",
language = "English (US)",
volume = "46",
pages = "395--409",
journal = "Journal of Pharmacokinetics and Pharmacodynamics",
issn = "1567-567X",
publisher = "Springer New York",
number = "5",

}

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.

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

UR - http://www.scopus.com/inward/record.url?scp=85069529331&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85069529331&partnerID=8YFLogxK

U2 - 10.1007/s10928-019-09646-3

DO - 10.1007/s10928-019-09646-3

M3 - Review article

C2 - 31338634

AN - SCOPUS:85069529331

VL - 46

SP - 395

EP - 409

JO - Journal of Pharmacokinetics and Pharmacodynamics

JF - Journal of Pharmacokinetics and Pharmacodynamics

SN - 1567-567X

IS - 5

ER -