Volume 50, Issue 6

Search all Journal of Pharmacokinetics and Pharmacodynamics articles

December 2023

Special Issue: Model-Informed Drug Development in Rare Diseases

Issue Editor:

Rajesh Krishna

8 articles in this issue

Special issue: Model-informed drug development in rare diseases: connecting the dots in an information rich ecosystem
Rajesh Krishna
Editorial 27 April 2023 Pages: 425 - 427
Part of 1 collection:

Model-informed drug development in rare diseases
Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases
Mariam A. Ahmed
Janelle Burnham
Bilal AbuAsal
Review Paper 04 May 2023 Pages: 429 - 444
Part of 1 collection:

Model-informed drug development in rare diseases
Challenges, approaches and enablers: effectively triangulating towards dose selection in pediatric rare diseases
Chandrasekar Durairaj
Indranil Bhattacharya
Review Paper 09 June 2023 Pages: 445 - 459
Part of 1 collection:

Model-informed drug development in rare diseases
Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
Gilles Tiraboschi
David Marchionni
Fabrice Hurbin
Original Paper Open access 03 August 2023 Pages: 461 - 474
Part of 1 collection:

Model-informed drug development in rare diseases
Rare oncology therapeutics: review of clinical pharmacology package of drug approvals (2019–2023) by US FDA, best practices and recommendations
Amitava Mitra
Jong Bong Lee
Rajesh Krishna
Review Paper 04 November 2023 Pages: 475 - 493
Part of 1 collection:

Model-informed drug development in rare diseases
Empirical bayes approach for dynamic bayesian borrowing for clinical trials in rare diseases
Bernard Sebastien
Original Paper 06 May 2023 Pages: 495 - 499
Part of 1 collection:

Model-informed drug development in rare diseases
External control arms for rare diseases: building a body of supporting evidence
Artak Khachatryan
Stephanie H Read
Terri Madison
Review Paper Open access 24 April 2023 Pages: 501 - 506
Part of 1 collection:

Model-informed drug development in rare diseases
The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)
Jeffrey S. Barrett
Alexandre Betourne
Will Roddy
Review Paper Open access 02 May 2023 Pages: 507 - 519
Part of 1 collection:

Model-informed drug development in rare diseases

Volume 50, Issue 6

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