Five multivariate Duchenne muscular dystrophy progression models bridging six-minute walk distance and MRI relaxometry of leg muscles
Authors (first, second and last of 10)
Collection
Model-informed drug development in rare diseases
This collection on rare diseases has been assembled to provide a snapshot on the power and utility of systems thinking in rare diseases. Contributing authors have presented thought leadership on various topics such as Bayesian information borrowing, external controls, methods of data pooling across a variety of similar mechanisms, but also the choice of endpoints and clinical trial considerations that leverage natural history of diseases. They collectively present a snapshot on what is possible in rare disease development.
Editors
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Rajesh Krishna, PhD, FAAPS
Rajesh Krishna, PhD, FAAPS, is Senior Director, clinical pharmacology and lead of the integrated practice area on rare diseases at Certara Strategic Consulting. With over 25 years of combined pharmaceutical industry and consulting experience, he has contributed to over 40 INDs; over 200 Phase 1/1b studies; and to the worldwide registration of >15 NDAs/BLAs. As a prolific scientist, Raj has published 95 peer-reviewed publications, is a frequently invited speaker and educator, and has edited or co-edited 4 books on new drug development.
Articles (10 in this collection)
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Docetaxel, cyclophosphamide, and epirubicin: application of PBPK modeling to gain new insights for drug-drug interactions
Authors (first, second and last of 6)
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Rare oncology therapeutics: review of clinical pharmacology package of drug approvals (2019–2023) by US FDA, best practices and recommendations
Authors (first, second and last of 5)
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Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
Authors (first, second and last of 10)
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Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases
Authors (first, second and last of 4)
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The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)
Authors (first, second and last of 8)
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