Biopharmaceutical Manufacturing, Volume 1

Name: Biopharmaceutical Manufacturing, Volume 1 | Regulatory processes
Brand: Institute of Physics Publishing
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Regulatory processes

Sarfaraz K. Niazi Sunitha Lokesh(Autor*in)

Institute of Physics Publishing

Erschienen am 31. Dezember 2021

350 Seiten

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978-0-7503-3175-3 (ISBN)

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Inhalt

Intro
Preface
Outline placeholder
Overview
Definitions
The impact of biopharmaceuticals
Biopharmaceutical development
Global markets
About this book
Acknowledgments
Author biographies
Sarfaraz K Niazi
Sunitha Lokesh
Chapter 1 Introduction to biopharmaceuticals
1.1 Overview
1.1.1 Biosimilars
1.1.2 Intellectual property
1.1.3 Regulations
1.2 Biopharmaceutical molecules
1.2.1 Overview
1.2.2 Molecular mechanism
1.2.3 Therapeutic classification
1.2.4 Hormone peptide drugs
1.2.5 Human hematopoietic factor
1.2.6 Human cytokines
1.2.7 Human plasma protein factor
1.2.8 Human bone formation protein
1.2.9 Recombinant enzymes
1.2.10 Antibodies
1.3 Protein structure and properties
1.3.1 Primary structure
1.3.2 Secondary structure
1.3.3 Tertiary structure
1.3.4 Quaternary structure
1.3.5 Post-translational modification (PTM)
1.3.6 Association and aggregation
1.4 Pharmacokinetic manipulations
1.4.1 Protein modification to increase the duration of action
1.5 Immunogenicity
1.5.1 The immune system
1.5.2 Antibodies
1.5.3 Antigens
1.5.4 Biopharmaceutical immunogenicity
1.5.5 Immunogenicity testing
1.6 Recombinant expression
1.6.1 Understanding DNA and RNA
1.7 Gene and cell therapy
1.7.1 Gene editing
1.8 Conclusion
Chapter 2 Antibody biopharmaceuticals
2.1 Overview
2.1.1 Naming
2.1.2 Commercial antibodies
2.2 The immune system
2.2.1 Innate immune system
2.2.2 Adaptive immune system
2.3 Monoclonal antibodies
2.3.1 Mode of action
2.4 Types of antibodies
2.4.1 Bispecific (BsAbs)
2.4.2 Fab fragments and single-chain antibodies
2.4.3 Humanized and chimeric mAbs
2.4.4 Affinity maturation
2.4.5 Antigenized antibodies
2.4.6 IgG1 fusion proteins
2.4.7 Drug or toxin conjugation
2.5 Development of antibodies
2.5.1 Endogenous method: single B-cell
2.5.2 Exogenous methods
2.5.3 Mouse hybridoma
2.5.4 Transgenic mice
2.5.5 Surface display libraries
2.5.6 Recombinant expression
2.6 Conclusion
2.7 Databases relevant to antibodies
Chapter 3 Gene and cell therapy
3.1 Overview
3.2 Gene therapy
3.2.1 Methodologies
3.2.2 Risks of gene therapy
3.2.3 Gene-editing technologies
3.2.4 Vector manufacturing
3.2.5 DNA vaccines
3.2.6 mRNA vaccines
3.3 Cell therapy
3.3.1 Types of cell therapies
3.3.2 CAR-T treatment (cellular immunotherapy)
3.3.3 Allogenic cell therapy
3.4 Regulatory considerations
3.4.1 Development and characterization of cell populations for administration
3.4.2 Characterization and release testing of cellular gene therapy products
3.4.3 Additional possible applications: radioisotopes or toxins added to cell preparations
3.4.4 Vector production, characterization, and release testing for gene therapy
3.4.5 Construction and characterization of vectors
3.4.6 Vector manufacturing system
3.4.7 Viral master banks
3.4.8 Lot-to-lot distribution vector testing and specifications
3.4.9 Issues in gene therapy related to specific vector classes
3.5 Conclusion
Chapter 4 Formulation of biopharmaceuticals
4.1 Overview
4.2 Structural instability
4.2.1 Basis
4.2.2 Physical degradation
4.2.3 Chemical degradation
4.3 Formulation composition
4.3.1 Route of administration
4.3.2 Excipients and properties
4.3.3 Liquid formulations
4.3.4 Lyophilized formulations
4.4 Delivery routes
4.4.1 Intravenous
4.4.2 Subcutaneous
4.4.3 Oral
4.4.4 Nasal/pulmonary
4.4.5 Transdermal
4.4.6 Ocular
4.4.7 Rectal
4.5 Formulation technologies
4.5.1 Hydrogels and in situ forming gels
4.5.2 Nanoparticles
4.5.3 Liposome
4.5.4 Higher concentration formulations
4.6 Examples of formulation
4.7 Conclusion
Appendix A: Physicochemical properties of proteins and peptides approved by FDA
Chapter 5 Biopharmaceutical development cycle
5.1 Overview
5.2 The US drug development process
5.2.1 Early drug discovery
5.2.2 Pharmacopoeia
5.2.3 Preclinical research
5.2.4 Investigational new drug application
5.2.5 Clinical research
5.2.6 Regulatory review, approval, and post-marketing safety surveillance
5.3 Drug development in Europe
5.3.1 Research initiation
5.3.2 Testing new medicines
5.3.3 EMA influence in development
5.3.4 Scientific advice
5.3.5 Scientific advice for studies
5.3.6 EMA providing other advice
5.3.7 How is scientific advice paid for?
5.3.8 What happens during scientific advice?
5.3.9 Who's involved in scientific advice?
5.3.10 Is there any involvement of patients in scientific advice?
5.3.11 Is it true that providing scientific advice has an impact on the EMA's assessment of the medicine?
5.3.12 What does the EMA say about the results of scientific advice?
5.3.13 What safeguards are in place to protect experts' independence during scientific advice?
5.3.14 Details of the procedure based on scientific advice
5.3.15 What happens before a medicine evaluation begins?
5.3.16 In a marketing authorisation application, what information must be provided?
5.3.17 What sources do you have for medical data?
5.3.18 What is the fundamental premise that underpins a medicine's evaluation?
5.3.19 Who is responsible for evaluating marketing authorisation applications?
5.3.20 How does the CHMP work?
5.3.21 Is it possible for the CHMP to ask for extra information during the evaluation?
5.3.22 What other resources does the CHMP have?
5.3.23 What role do patients and healthcare providers play?
5.3.24 What safeguards are in place to protect the independence of experts?
5.3.25 How did the CHMP arrive at its final conclusion?
5.3.26 What information is made public throughout the examination of new medicines and after a decision is reached?
5.3.27 A medicine's evaluation calendar
5.3.28 Further assessment
5.3.29 Discussion and comment at the end
5.3.30 Re-examination
5.3.31 Who grants EU-wide marketing authorization?
5.3.32 Who makes decisions on patient access to medicines?
5.3.33 How is the safety of a medicine ensured once it has been put on the market?
5.4 Japanese drug approval process
5.4.1 Overview
5.4.2 New drug approval (NDA) application
5.5 Conclusion
Chapter 6 Biosimilar biopharmaceuticals
6.1 Overview
6.2 Biosimilarity
6.3 Regulatory guidance
6.3.1 Background
6.3.2 Latin America
6.3.3 Canada
6.3.4 The World Health Organization (WHO)
6.3.5 India
6.3.6 Australia
6.3.7 Japan
6.3.8 Korea
6.3.9 Iran
6.3.10 EU
6.3.11 United States
6.4 Development master plan
6.4.1 Choice of product
6.4.2 Qualification of product
6.4.3 Public domain knowledge
6.4.4 Manufacturing plan and facility
6.4.5 Analytical testing
6.4.6 Nonclinical pharmacology
6.4.7 Clinical pharmacology
6.4.8 Clinical immunogenicity
6.4.9 Residual uncertainty
6.4.10 Clinical safety and efficacy
6.4.11 Post-market surveillance
6.5 Interchangeability and substitution
6.6 Analytical assessment
6.6.1 Introduction
6.6.2 Sources of variation
6.6.3 Product- or process-related substances
6.6.4 Method sensitivity
6.6.5 Comparative testing
6.6.6 Side-by-side testing
6.6.7 Heterogeneity
6.6.8 Structure confirmation
6.6.9 Acceptance criteria
6.6.10 Orthogonal testing
6.6.11 Accountability of lots
6.6.12 Critical quality attributes
6.6.13 Reference standard
6.6.14 The finished drug product
6.6.15 Excipients
6.6.16 Stability
6.6.17 ICH
6.6.18 Examples of test methods
6.6.19 Risk assessment
6.6.20 Statistical considerations
6.7 Functional assessment
6.7.1 Orthogonal studies
6.7.2 Lots tested
6.8 In vivo assessment
6.8.1 Determination of the needs for in vivo studies
6.8.2 In vivo animal studies
6.9 Clinical pharmacology assessment
6.9.1 Introduction
6.9.2 Scope of studies
6.9.3 Study plan
6.9.4 Dose selection
6.9.5 Route of administration
6.9.6 Pharmacokinetic parameters
6.9.7 New approach to PK analysis
6.9.8 PK/PD waivers
6.9.9 Pharmacodynamic parameters
6.9.10 Statistical treatment of PK and PD results
6.9.11 Assay considerations
6.9.12 Reserve samples
6.9.13 Examples of pharmacokinetic studies
6.10 Clinical immunogenicity assessment
6.10.1 Introduction
6.10.2 Immunogenicity investigation
6.10.3 Safety and efficacy correlation
6.10.4 Management of immunogenicity testing
6.10.5 Examples of studies
6.11 Clinical efficacy assessment
6.11.1 Introduction
6.11.2 Residual uncertainty
6.11.3 Waivers
6.11.4 Study types
6.11.5 Selection of study protocols
6.12 Extrapolation of clinical data across indications
6.12.1 Extrapolation across indications
6.12.2 Additional conditions of use
6.13 Conclusion
Chapter 7 Intellectual property issues for scientists
7.1 Overview
7.1.1 Patent landscape
7.2 Patent law basics
7.2.1 Pharmaceutical patenting practices
7.3 US patent elements
7.3.1 Title of invention
7.3.2 Cross-reference to related applications
7.3.3 Statement regarding federally sponsored research or development
7.3.4 Background of the invention
7.3.5 Brief summary of the invention
7.3.6 Background of the invention
7.3.7 Brief description of the drawing
7.3.8 Detailed description of the invention
7.3.9 Claim or claims
7.3.10 Abstract of the disclosure
7.3.11 Drawings
7.3.12 Oath or declaration
7.3.13 Sequence listing (when necessary)
7.4 Types of patents
7.4.1 Unpatentable inventions
7.4.2 Software patents
7.4.3 Medical method patents
7.4.4 Business method patents
7.4.5 Utility model in the EU
7.4.6 Provisional application
7.5 Comparison of patent laws
7.5.1 Jurisdiction
7.5.2 The Patent Cooperative Treaty
7.5.3 First to invent rule
7.5.4 First to file rule
7.5.5 Best mode requirement
7.5.6 Patent publication
7.5.7 Rights conferred
7.5.8 Opposition after grant
7.5.9 Inventive step
7.5.10 Two-part claim
7.5.11 Patent assignment
7.5.12 Patent infringement
7.6 Biological patents
7.7 Monoclonal antibody technology
7.8 Antisense technology
7.9 Transgenic plants
7.10 Exclusivities for biological products
7.11 Broad coverage
7.12 The Purple Book
7.13 Patent term extension
7.14 Patent term adjustment
7.14.1 Factors affecting a patent term
7.14.2 Pharmaceutical patent variations
7.14.3 Annuity fees and term computation
7.14.4 Information on aspects of the patent term
7.14.5 USPTO web sources
7.14.6 Other web registers
7.14.7 Commercial online files
7.15 Freedom-to-operate (FTO) opinions
7.15.1 Submarine patents
7.15.2 System expression patents
7.15.3 Process patents of originator
7.15.4 Third-party process patents
7.15.5 Formulation composition
7.15.6 Lifecycle formulation projections
7.15.7 Alternative offering
7.15.8 Indications and dosage
7.15.9 Delivery devices
7.15.10 Developing freedom-to-operate
7.16 Conclusion
Outline placeholder
Preface
Chapter 1
Chapter 2
Chapter 3
Chapter 4
Chapter 5
Chapter 6
Chapter 7

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