Originally written by CDER and CBER officials and industry experts, the newly-updated Biologics Development: A Regulatory Overview offers an extensive examination of the FDA’s regulations and guidelines for biologic products, from preclinical testing to post-marketing regulatory requirements, and from user fees to electronic submissions. The book also provides a detailed look inside the approval pathway for today’s biological products along with an analysis of each stage of the biological product development process, including:
CDER organization and processes for regulating and reviewing therapeutic biological products;
CDER’s processes for regulating and reviewing cellular/tissue-based products and gene therapies, vaccines, and blood products.;
How CDER and CBER have evolved their procedures and requirements to address new challenges including risk management priorities and internal agency initiatives.;
Applications of updated ICH GCP E6 and E8 guidelines to biological products development.;
Emerging standards for the clinical and nonclinical testing of biological products, including combination products and biosimilars.
- Chapter 1
- What is a Biologic?
- A Brief History of Biologics Regulation in the United States
- Recent Changes in the Government’s Approach to Biologics Regulation
- Regulatory Definition of a Biological Product
- General Comparison of Drug and Biologic Characteristics
- Product Classification and the FDA’s Intercenter Agreements
- The Importance of Product Classification: Impact on Development Strategy
- The Specter of “Follow-On” Biologics
- References
- Chapter 2
- Preclinical Safety Assessment of Therapeutic Proteins and Monoclonal Antibodies
- Preclinical Development of Biologics
- Biologics-Specific Concepts
- Preclinical Studies to Support the Safety of Biologics
- The Timing of Preclinical Studies Relative to Clinical Trials
- Summary
- References
- Chapter 3
- The Biological IND
- FDA Organization and IND Submissions
- Types of IND Submissions
- Before Submitting an IND
- Example Meeting Request Sheet
- The Original IND Submission
- Contents of the IND
- INDs for Gene Therapies
- The Submission and Review of the IND
- Amendments to the IND
- Electronic INDs
- Summary
- Acknowledgments
- References
- Chapter 4
- The Biological IND Review Process
- The FDA and Regulating Biological Products: Recent History and Agency Structure
- FDA Announces CDER/CBER Consolidation
- A Look at the Structure of CBER and CDER for IND Reviews
- CBER Structure
- The Office of Tissues and Advanced Therapies (formerly known as Office of Cellular, Tissue and Gene Therapies).82
- CDER’s Structure for Biological IND Reviews
- The Review Process for INDs
- CBER and the IND Review Process
- CDER and the IND Review Process for Biological Therapeutic Products
- The IND and the 30-Day IND Review Clock
- The Clinical Hold
- IND Status
- Biologics Development: A Regulatory Overview
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- Chapter 5
- Clinical Testing of Biologically Derived Therapeutics
- Phases of Clinical Drug Development
- References
- Chapter 6
- The Clinical Evaluation of Preventive Vaccines
- Federal Regulations Pertaining to Vaccines
- U.S. FDA Regulations Relevant to the Manufacture,
- Product Quality and Clinical Testing of Vaccines*
- Overview of Clinical Evaluation
- Phases of Clinical Development
- Serious Adverse Events (SAEs) During Clinical Development
- Additional Considerations for Safety Data
- FDA Advisory Committee Deliberations
- Postlicensure Studies
- Postlicensure Surveillance
- Regulatory Initiatives for Accelerated Vaccine Development During COVID-19 Pandemic
- Acknowledgments
- References
- Chapter 7
- Good Clinical Practices (GCP)
- Information Sheets: Guidance for Institutional Review Boards and Clinical Investigators
- Responsibilities of the Sponsor
- Responsibilities of Investigators
- The Institutional Review Board (IRB)
- Informed Consent
- References
- Chapter 8
- The Biological License Application (BLA)
- A Short History of the FDA’s Licensing Process for Biologics
- An Introduction to the BLA: Content and Format Requirements and Form 356h
- The Content of the BLA
- CBER and the eBLA Program
- Amending the License Application
- References
- Chapter 9
- Manufacturing Arrangements For Biological Products
- Biologics Establishment Registration
- Human Cells, Tissues and Cellular and Tissue-Based Products (HCT/Ps) Establishments
- The Biotech Explosion, Industry Consolidation, and Biological Manufacturing
- The FDA Redefines “Manufacturer”
- Sole Manufacturing
- Current Cooperative Manufacturing Arrangements for Licensed Biologics
- Facility Inspections
- References
- Table of Contents
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- Chapter 10
- The Biological License Application (BLA) Review Process
- The BLA Review Process
- The BLA Managed Review Process Within CBER
- Sponsor Rights during the License Application Review Process
- References
- Chapter 11
- Post-licensure Requirements
- Adverse Experience-Related Regulatory Definitions
- Changes in AE Reporting Requirements
- Expedited Reporting of Additional Safety Information
- Lot Release
- Current Good Manufacturing Practice (cGMP) Standards for Biological Products
- Written Procedures for the investigation should include the following:
- Written records of the investigation should include:
- General Reporting Requirements
- Phase 4 Study Commitments
- References
- Chapter 12
- Bioresearch Monitoring Program for Biologics
- A Brief History of the Bioresearch Monitoring Program
- Recent Issues for the FDA’s BIMO Program
- An Overview of CBER’s Bioresearch Monitoring Program
- The Clinical Investigator Compliance Program
- The Clinical Sponsor/Monitor Compliance Program
- The Institutional Review Board (IRB) Compliance Program
- The Nonclinical Laboratory Compliance Program
- CDER’s Bioresearch Monitoring Program
- References
- Chapter 13
- Biologics and the Regulation of Combination Products
- Defining Biologics, Drugs, and Devices
- CBER Action Plans
- Combination Products and Gaining CBER Approval
- Safe Medical Devices Act (SMDA) of 1990
- Table. RFD Determinations by OCP and Workload Trends: FY 2013 to FY 2018
- Table. Workload at OCP by Combination Product Category Number in FY 2018. Combination
- PDUFA and Various Classes of CBER-Regulated Products
- Risk–Based Quality and Safety Management throughout the Lifecycle of Combination Products
- References
- Chapter 14
- Special Topics in the Development of Biologically Derived Therapeutics
- References
- Biologics Development: A Regulatory Overview
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- Chapter 15
- Biosimilar Products
- Biosimilar Development, Review, and Approval Processes
- Interchangeable Products: Definition, Regulatory Review and Approval Process
- Post-marketing or Post-authorization Surveillance Requirements
- Nomenclature
- FDA-Approved Biosimilar Products
- The Biologics Price Competition and Innovation Act
- An Abbreviated Approval Pathway for Biological Products
- References
- About Barnett International
- About Cambridge Healthtech Institute
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