BioPharm
I N T E R N AT I O N A L
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T h e S ci e n ce & B usi n e ss o f B i o p h a r m a ce u t i c a ls
2 BioPharm International eBook March 2021 www.biopharminternational.com
Cover: wladimir1804 - Stock.adobe.com
QUALITY
Can FDA Reinvent On-Site Inspections?
Agnes Shanley 4
Growing Pipeline of Antisense
Oligonucleotides Prompts Guidance
Feliza Mirasol 6
Gene Therapies Target
Neurodegenerative Diseases
Feliza Mirasol 12
OPERATIONS
A Real-World Approach to
Evaluating Cleanroom Garments
Anne Marie Dixon-Heathman 14
QUALITY COLLABORATION
Special Section:
It Takes a Bio/Pharma Village
The editors of Biopharm International 22
Overcoming Challenges to Biopharmaceutical
Development and Manufacture with Science
and Risk-Based Strategies
Saroj Ramdas, Renata Varga, Martin Gastens, Twinkle R. Christian,
Bharat Jagannathan, Barbara Rellahan, and Jillian Brady 23
Enabling the Virtual Human
Through Physiologically-based
Pharmacokinetic Modeling
Heidi J. Einolf, Stephen D. Hall, Tracy Williams, Aarti Patel,
Christopher Gibson, Nancy G.B. Agrawal, and Jens Sydor 27
Extrapolating Data from Adult
Clinical Trials to Advance Pediatric
Drug Development
Sebastian Haertter, S.Y. Amy Cheung, Angela James, Ashley Strougo,
Christina Bucci-Rechtweg, Jing Liu, Meina Tao Tang, Paulien Ravenstijn,
Raafat Bishai, Satyendra Suryawanshi, and Solange Corriol-Rohou
31
Collaborative Efforts Address
Key Data Integrity Challenges
Ganapathy Mohan, Christopher Turner, Dennis O'Connor, Lisa Fink,
Thomas Purdue, Jeffrey Beebie, Kerri Robles, and Karen Waetje
36
RESOURCES
FDA 2021 Guidance Agenda Sees Focus on Generics 39
Resources, Guidelines, and Guidance Documents 42
Regulatory and Standard Setting Organizations 44
Ad Index 48
Regulatory Sourcebook
March 2021
Contents
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MJH LIFE SCIENCES
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EDITORIAL
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