Expediting Drugs and Biologics Development

Published Sep 01, 2021

Length 630 Pages

SKU # BNT17523519

Description

From the initial planning to the NDA/BLA review process, Expediting Drug and Biologics Development shows you how to use reverse-engineering techniques to drive and improve each aspect of a drug and biologic product development program’s design and implementation. Written by dozens of leading experts, this book is a real-world “doer’s” guide. It provides templates, forms, and tools to assist those “in the trenches” of new drug and biologic development today. With this book, you will learn how to:

Make planning the central part of all aspects of drug and biologics development.

Establish a Target Product Profile (TPP) to critically evaluate the needs of the evolving package insert and eventual marketing application before getting deeply into clinical trials.

Understand that the clinical development program dictates much of the nonclinical development program, and that both dictate the chemistry, manufacturing and controls development program.

Involve thoughtful ethics in the planning and execution of clinical research.

Leverage standardization to drive and expedite the entire development process, from the development of clinical trial protocols to the development of clinical data presentations.

Critically assess the needs of a final study report before developing the clinical protocol.

Chapter 1: Beginning at the Destination: A Corporate Culture: Begin at the Destination; View from the Corner Office; Target Product Profile; Regulatory Strategy; Product Development Plan; The Big Picture; Too Big to Fail; Planning, Planning, and More Planning; Planning Versus Execution; About the Author
Chapter 2: The Target Product Profile: Need for Planning; Need for Communication; What the TPP Is; What the TPP Is Not; TPP in the Press and Regulatory Agency Guidance; Implementation, and the Pharmaceutical Company Perspective; What Can a TPP Look Like?; Wrap-Up; About the Authors
Chapter 3: The Common Technical Document: What Is a CTD/eCTD?; Key Terms at a Glance; Organization; Reference Tools; Roadmaps for the NDA/BLA in CTD Format – Module 1; Begin with the End in Mind; Marketing Application Form – Form FDA 356h; Product Description; Established and Proprietary Names; Dosage Form and Route of Administration; Indication; viii Published by:; New Drug Development: A Regulatory Overview; Marketing Application Types; 505(b)(1) NDA; 505(b)(2) NDA; ANDA; 351(a) BLA; 351(k) BLA; Combination Products; Cross References; Rx, OTC; Regulatory Information; Target Product Profile — Annotated Labeling; Comprehensive Table of Contents; Format and Content of the CTD – Modules 2 to 5; Chemistry (Quality); Modules 2 and 3; Tips for Efficiency; CTD Table of Contents (TOC); Labeling; Regulatory Interactions and Guidelines; Novel Excipients; Abbreviations; Hypertext Linking Consistency; Tabular Summaries; Nonclinical (Safety); Modules 2 and 4; Tips for Efficiency; CTD Table of Contents (TOC); Labeling; Regulatory Interactions and Guidelines; Abbreviations; Hypertext Linking Consistency; Tabular Summaries; Report Summaries/Synopses; Literature Reports; Clinical (Efficacy); Modules 2 and 5; Tips for Efficiency; CTD Table of Contents (TOC); Table of Contents; Published by: ix; Labeling; Regulatory Interactions and Guidelines; Abbreviations; Hypertext Linking Consistency; Tabular Summaries; Report Summaries/Synopses; Literature Reports; FDA Review; Summary; References; About the Authors; Acknowledgments
Chapter 4: Expedited Development of New Drugs and Biologics: Introduction; Tracing the Roots of the FDA; The Origin of Expedited Programs; Concepts for Expedited Programs; Serious Condition; Available Therapy; Unmet Medical Need; The Specifics of the Expedited Programs; Fast Track Designation; Breakthrough Therapy Designation; Accelerated Approval Pathway; Priority Review Designation; Summary and Conclusions; About the Author
Chapter 5: Clinical Trials Enrolling Children: Rationale for Pediatric Studies; Protecting Children Participating in Clinical Research; Definitions; IRB Approval Process for Pediatric Research; Evaluation of Efficacy in Pediatric Research; Extrapolation of Adult Efficacy Data to the Pediatric Population; Evaluation of Safety in Pediatric Research; Considerations of Trial Design; Timing of Initiation of Pediatric Studies in Product Development; x Published by:; New Drug Development: A Regulatory Overview; Development of Pediatric Formulations; Federal Initiatives to Promote Pediatric Development; Best Pharmaceuticals for Children Act; The Pediatric Research Equity Act; Summary and Conclusions; About the Author; Acknowledgments
Chapter 6: Phase 1 Clinical Studies: Phase 1 Studies; Phase 1 Sites; Phase 1 Staff; Site Training and Experience; Safety in Phase 1 Studies; Sponsor Opportunities; Protocol Execution: A Day in the Life; Lessons Learned; About the Authors; Acknowledgments
Chapter 7: Nonclinical Development: Introduction; Nonclinical Testing: Introduction and Brief History; Nonclinical Development: A Multidisciplinary Integration; Formulating a Development Plan; Guiding Principles for Nonclinical Planning; Drugs: Nonclinical Safety Testing; General Nonclinical Testing Requirements; Studies Expected Prior to Phase 1 Testing; Pharmacology Studies; Safety Pharmacology Studies; Pharmacokinetics and ADME; Genetic Toxicity; General Toxicity Studies; Studies Expected During Clinical Development; Pharmacology Studies; Safety Pharmacology Studies; Pharmacokinetics and ADME; Genetic Toxicity; Table of Contents; Published by: xi; General Toxicity Studies; Developmental and Reproductive Toxicity; Special Toxicity; Additional Studies Expected to Support a Marketing Application; General Toxicity Studies; Carcinogenicity; Developmental and Reproductive Toxicity; Juvenile Toxicity; Combination Toxicology Studies; Biologics: Nonclinical Safety Testing; Overview of Nonclinical Testing for Biologic Products; Key Principles for the Nonclinical Development of Biologics; Relevant Animal Model; Immunogenicity; Dose Selection and Safety Margin Calculation; Product Characterization; Product-Specific Nonclinical Issues for Biologics; Monoclonal Antibody (mAb) Products; Recombinant Proteins and Peptides; Somatic Cell Therapy; Gene Therapy; Preventive and Therapeutic Vaccines; Oligonucleotides; Nonclinical Safety Testing: General Technical and Scientific; Considerations; General Study Design Concepts; Species Selection; Number of Animals; Use of Recovery Groups; Dose Selection; Route of Administration; Dose Volumes and Good Practices; Dosing Regimen; Extrapolating Doses Between Species; Establishing Timelines; Typical Cost Estimates; Outsourcing Safety Studies; Summary; xii Published by:; New Drug Development: A Regulatory Overview; References; About the Authors; Acknowledgments
Chapter 8: Integration of CMC into Drug Development:: Strategies for Success and Tactics for Avoiding Common Pitfalls; Introduction; The CMC Development Plan; Never Allow API or CTM to Be Rate Limiting; (or Always Make More); Learn Where the Minefields Are; Know Your Compound; Know Your Dosage Form; Learn to Efficiently Manage Risk for CMC Activities; Biologics; Small Molecules; Phase-Appropriate Emphasis (Too Little or Too Much?); The IND CMC Section Is Just the Tip of the Iceberg; Plan for Success; Adopting Smart Development Practices; Conclusion; About the Author; Acknowledgments
Chapter 9: The Sponsor/Contract Research Organization Partnership: Introduction; How to Pick a CRO; The Proposal Process; Option A; Pros; Cons; Option B; How to Pick a CRO Under Option B; The Bid Defense; Formal Transfers of Obligations; Project Management; The Sponsor Advocate; Sponsor/CRO Meetings; Clarity of Invoices; Table of Contents; Published by: xiii; Closing Remarks; About the Authors
Chapter 10: eCTD Submissions to the FDA: Background; Terminology; Tools; Planning; Team Members; Planning Process; Planning Content; Authoring; Compilation and Publishing; Archiving; Conclusion; About the Author
Chapter 11: Meeting with the FDA: Why Meet and How to: Communicate; Introduction; FDA Guidance; Types of Meetings; Type A Meetings; Type B Meetings; Type C Meetings; Meeting Request; Meeting Package; Industry and Regulator Observations on Meetings; Industry Perspective; Pre-Meeting Planning; During the Meeting; Regulator Perspective; Closing Thoughts; About the Authors
Chapter 12: Investigational New Drug Application: IND Content, Format, and Timeline; IND Content; IND Format; IND Review Clock; xiv Published by:; New Drug Development: A Regulatory Overview; Clinical Hold; ClinicalTrials.gov: Registration, Updates, and Informed Consent; Requirements; IND Amendments; Protocol Amendments; Information Amendments; IND Safety Reporting; Annual Reports; Administrative Considerations; Resources; About the Author
Chapter 13: Title 21 CFR Part 11: Introduction; Background and History; Applicability; Part 11 Requirements; Requirements for Electronic Records; Validation; Authorized Access; Audit Trails; Sequence Checks; Device Checks; Record Copies; Record Protection and Retention; Training; Controls Over System Documentation; Open Systems; Requirements for Electronic Signatures; Identity Verification; Signature Execution; Signatures on the Record; Digital Signatures; Additional Considerations; Application Inventory; System Description; User Training; Risk Determination; Programming Tools; Table of Contents; Published by: xv; Scanned Records; EDC Systems; Other Record Systems; Email; Global Records and Regulations; Other US Regulations; Regulatory Guidances; Company Size; Outsourcing and Third Parties; The Future; About the Author; Acknowledgments
Chapter 14: The Investigator’s Brochure: Introduction; The Initial IB and the IND; Updating the IB; Safety Updates; Timing of Regular Updates; Evolution of the IB During Development; Administrative Issues; Summary; About the Author
Chapter 15: Clinical Study Reports: Introduction; Establishing the Format and Content; ICH E3: Structure and Content of Clinical Study Reports; Overall Development of a CSR; Using a CSR Template; Statistical Analysis Plan; Format and Content of a CSR; Outline; CSR Shell; Results; Summary Source Tables and Serious Adverse Event Narratives; Source Documents; Synopsis; Clinical Study Report Writing; xvi Published by:; New Drug Development: A Regulatory Overview; Overall CSR Development Timelines; Project Planning; Establishing Roles and Responsibilities; Developing Timelines; Clinical Study Report Development Process; Prior to Study Completion; At Study Completion; Database Lock to Final Analyses; Technical Issues Related to the Content of a CSR; Summary; About the Author
Chapter 16: Analysis Plans: Analysis Plans Defined; Standalone SAP; Statistical Guidelines; SAP Elements That Demand Special Attention; Definition of the Research Question and Estimands; An Example of an Estimand; The Process for Defining the Research Question and Estimand; Sensitivity and Supportive Analyses; Sample Size Calculations, Power, and Type I and Type II Errors; Clinical Outcome Assessments; Conclusion; About the Author
Chapter 17: An Integrated Approach to Data Management: Background and Industry Landscape; The New World of Technology and Its Impact; Putting the Data Puzzle Back Together; The New Data Tracking and Reporting Tools; Defining an Integrated Data Management Solution; Initial Protocol Strategy Meeting; Data Collection Requirements; Types of Data Collection Systems and Processes; eCRF Data; eCOA Data; Wearable Device Data; Data Tracking Requirements; Table of Contents; Published by: xvii; Data Cleaning Requirements; Data Analysis/Reporting Requirements; Implementation and Validation; The Role of Data Management in the Clinical Trials Process; Collaboration Is the New Integration; Data Stewardship; Summary & Conclusion; About the Authors
Chapter 18: The Protocol: A Clinical/Statistical Collaboration: The Protocol Strategy Meeting; The Participants; The Agenda; Objectives; Endpoints/Variables; Design; Entry Criteria; Schedule of Assessments; Data Presentations; End Products; Standard Protocol Content; Minimum Criteria; Adequate and Well Controlled; Protocol Content Considerations; Standard Protocol Format; Title Page; Investigator Protocol Signature Page; Table of Contents; Body of the Text; Protocol Synopsis (Summary); Standard Protocol Template; Drafting an Individual Study Protocol; Conclusion; About the Authors
Chapter 19: Data Content and Design: Introduction; What Data Should Be Captured?; Data Design; xviii Published by:; New Drug Development: A Regulatory Overview; Tools of the Trade; Standard Formatting; Help the User Avoid Discrepancies; Adverse Event Capture; Date and Time; Templates; Standard Formatting for CRFs; Consider the Source; Implement the Standards; Basic CRF Design Principles; Standard Case Report Forms; Demographic Data; Medical History; Inclusion/Exclusion; Medication Log; Clinical Laboratory Data; Adverse Event CRF; Termination CRF; Comment Fields; Study-Specific Case Report Forms; Implementation; Data Team; Conclusion; About the Author
Chapter 20: Informed Consent, IRB Review, and Ethics: History and Regulatory Background; Preparing the Informed Consent Draft; Authors of the Informed Consent Document; Informed Consent Document Process; Enhancing Comprehension of the Informed Consent Form: Decreasing the; Reading Level; Elements of Informed Consent; Basic Elements of Informed Consent; Certificates of Confidentiality; Additional Elements of Consent; Documentation of Consent; Terminology; Process After Drafting the Informed Consent Form; Table of Contents; Published by: xix; Study Site Investigator’s Review of the Draft Informed Consent Document; and Preparation for IRB Approval; Review of the Research Project by the IRB; Criteria for IRB Approval; IRB Approval Letter; Inclusion of Vulnerable Populations; Pediatric Studies; Women of Child-Bearing Potential and Minorities; Other Considerations; IRB Review of Multi-Site Studies; Sponsor’s File Copy; Direct Communication Between Sponsors and IRBs; Sponsors’ Access to IRB Written Procedures, Minutes, and Membership; Rosters; The International Council for Harmonisation; Conclusions; About the Author; Acknowledgments
Chapter 21: Clinical Trial Operations & Project Management: Introduction; Protocol Development; Study Planning; End-to-End Timeline; Feasibility; Enrollment Forecasting; Service Provider Selection; Study Kick-Off and Team Engagement; Study Execution: Start-Up Phase; Study Plan Development & Team Training; Investigator Site Evaluation/Selection, Initiation, and Activation; Investigator Meeting; Study Execution: Maintenance Phase; Site & Subject Tracking: Utilizing Metrics; Investigational Product Supply Management; Data Monitoring; Executing Clinical Database Lock; Study Reporting & Close-Out; Results Reporting; xx Published by:; New Drug Development: A Regulatory Overview; Clinical Study Report Development; Investigator Site Close-Out; Lessons Learned; Conclusion; About the Authors; Acknowledgments
Chapter 22: Clinical Site Budgets and Contracts: Introduction; Clinical Trial Agreements; Clinical Trial Agreement Template; Clinical Trial Agreement Considerations; Third Party Agreements; Governing Laws; Indemnification; Clinical Trial Participant Rights; Clinical Trial Agreement Negotiations; Clinical Site Budgets; Clinical Site Budget Template; Per Patient Cost Benchmarking; Indirect Costs & Fees; Investigator Payment Administration; Clinical Site Budget Negotiations; Conclusion; About the Author; Acknowledgments
Chapter 23: Clinical Trial Monitoring: The Clinical Research Associate (CRA); Background and Terminology; Roles and Responsibilities of the CRA; Site Selection Visits; Background Information for Site Selection Visits; Site Selection Visit: Preparation; Site Selection Visit: Conduct; Site Selection Visit: Follow-Up; Site Initiation Visit; Background Information for Site Initiation Visits; Site Initiation Visit: Preparation; Table of Contents; Published by: xxi; Site Initiation Visit: Conduct; Site Initiation Visit: Follow-Up; Periodic Site Visits; Background Information for Periodic Site Visits; Source Data Review and Source Data Verification; Risk-Based Monitoring; Periodic Site Visit: Preparation; Periodic Site Visit: Conduct; Periodic Site Visit: Follow-Up; Termination Site Visits; Background Information for Termination Site Visits (TSV); Termination Site Visit: Preparation; Termination Site Visit: Conduct; Termination Site Visit: Follow-Up; Decentralizing Clinical Trials; Career Opportunities as a CRA; The Site’s Perspective on the Role of a CRA; About the Author
Chapter 24: Evaluation of Safety During Pharmaceutical Product: Development and Lifecycle; Introduction; Background; Strategy for the Evaluation of Clinical Safety; Compound Source/Structure; Formulation; Late Discoveries in Chemistry, Manufacturing, and Controls for the; Formulation; Route of Administration; Duration of Administration; Pharmacology; Late Discoveries from Pharmacology; Toxicology; Late Discoveries from Toxicology; Regulatory Expectations/Requirements/Standards; Alert Reporting; Serious; Unexpected; Associated; xxii Published by:; New Drug Development: A Regulatory Overview; Reporting Requirements; Breaking Blinds; Clinical Effects; Phase 1; Starting Dose in Phase 1; Multiple Doses in Phase 1; Use of Phase 1 Data; Phase 2; Phase 2a; Use of Phase 2a Data; Phase 2b; Use of Phase 2b Data; Phase 3; Phase 3a; Use of Phase 3a Data; Phase 3b; Use of Phase 3b Data; Phase 4; Postmarket (Phase 4) Studies Required upon Product Approval; Postmarket (Phase 4) Studies Required after Product Approval; Use of Phase 4 Data; Risk Evaluation and Mitigation Strategy; Role of the Medical Monitor; Summary; References; About the Author; Acknowledgments
Chapter 25: The FDA Marketing Application Review Process: Legislation, Policies and Procedures; Organization of the Review Process; Application Receipt and Processing; Filing Determination and Review Planning; The Administrative Filing Review of the Application; Internal Review Schedule Planning; Review; FDA Application Review Philosophy; 120-Day Safety Update; Table of Contents; Published by: xxiii; Mid-Cycle Meeting; Inter-Review Occurrences and Communications with the Company; 546; Obtaining Advice/Input for the Review Division; Labeling Meeting, Negotiations, and Philosophy; Late-Cycle Meeting; End of Review Activities; Wrap-Up Meeting; Final Action; Possible FDA Actions on a Pending Marketing Application; Working Towards a Good Review Cycle; About the Authors
Chapter 26: Preparing for FDA Inspection: Introduction; Prepare Early and Often; Internal Audit; External “Third-Party” Audit; The FDA Inspector’s Frame of Reference; The FDA Inspector’s Frame of Mind; Mulder’s Law; Genesis Factor; Stephens’ Legacy; Hoover Hypothesis; Murphy’s Law Correlate; Preparing the Clinical Study Site; Preparing the Sponsor or CRO; The FDA Inspection; Long Before the Visit; Procedure; Personnel; Inspection Coordinator; Responsible Parties; Reception; Study Staff or Project Team; Facilities; Just Before the Visit; During the Visit; After the Visit; xxiv Published by:; New Drug Development: A Regulatory Overview; Conclusion; About the Author; Acknowledgments
Chapter 27: Preparing for an FDA Advisory Committee Meeting: Overview; About FDA Advisory Committees; Who Is on an Advisory Committee?; Subject Matter Experts; Industry Representative; The Chair; Committee Members; Designated Federal Officer; A “Typical” Advisory Committee Meeting; Meeting Space; Meeting Agenda; Materials and Timing Leading Up to an AC Meeting; Preparing for This High-Profile, High Stakes Event; Timeline; Define Strategy and Argument; Develop the Materials; Delivery; Mock Panels; Presenters; Q&A Moderator; Other Team Members; Communicating Effectively with the Committee; Know Thy Audience; Know Thy Message; Questions & Answers; Open Public Hearing; Best Practices for Advisory Committee Success; About the Author; Acknowledgments; Index; About Barnett International; About Cambridge Healthtech Institute

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