CMC Workshop: From Drug Development to Global Supply to Patients

Day 1 Monday, April 15, 2013

• 8:30AM - 8:45AM

  Welcome and Introduction

  
  Session Chair(s):

  • Yasmin de Faria Krim, PharmD,MSc
    Manager, Global CMC Regulatory Affairs
    Janssen Pharmaceutical Companies of Johnson & Johnson, Belgium

  Welcome and Introduction
• 8:45AM - 10:15AM

  Session 1: Globalization in the CMC Area - Collaboration Between Regulatory Agencies

  
  Session Chair(s):

  • Jean-Louis Robert, PhD
    Head, Medicines Control Laboratory
    National Health Laboratory, Luxembourg

  From the hurdles of getting global regulatory approvals through updates on the EMA/FDA parallel assessment pilot program for QbD submissions or global activities for GMP inspection, this session will focus on globalization of activities in the area of both CMC assessment and GMP inspections. Current and future challenges as well as opportunities to facilitate global approvals of drugs will be addressed through feedback from representatives from both Industry and Agencies. A Question/Answer session will provide attendees with the opportunity to comment and share their thoughts on current initiatives.

  Speaker(s):

  • Challenges in Getting Global Regulatory Approvals
    Ganapathy Mohan, PhD
    Executive Director Global CMC, Pharmaceutical and Devices
    Merck, Sharp and Dohme, Corp., US, United States
  • Lessons Learned Developing a Global QbD Pilot for Biologics
    Lynne Krummen
    Vice President, Technical Regulatory, Biologics
    Genentech, A Member of the Roche Group, United States
  • FDA Update on Inspection Globalization Activities
    Diana Amador Toro, PhD
    Director, New Jersey District, Office of Regulatory Affairs
    FDA, United States
  • Q&A Panel (including above speakers)
    Elaine Morefield, PhD
    Deputy Office Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States
• 10:45AM - 12:15PM

  Session 2A: Challenges and Opportunities for the API Supply Chain Following ICH Q11

  
  Session Chair(s):

  • Timothy J.N. Watson, PhD
    Research Fellow, GCMC Advisory Office
    Pfizer, Inc., United States

  This session will feature speakers willing to expand upon some of the more challenging issues for the API supply chain as they relate to ICH Q11 (Development and Manufacture of Drug Substances). ICH Q11 provides opportunities for improving regulatory global harmonization expectations for dug substance development and manufacture. However, implementation of ICH Q11 may not necessarily align with region to region interpretations and/or current regulations. For example, the interpretation and assessment of criticality of quality attributes and process parameters, the justification of a design space based on small-scale models, the development of a holistic control strategy, and the role of post-approval process verification have varied interpretations form both industry and regulatory authorities.

  Speaker(s):

  • Impact of Control Strategy on API - the ICH Q11 View
    Betsy P. Fritschel, MS
    Director, Regulatory Intelligence, Quality & Compliance Worldwide
    Johnson & Johnson, United States
  • Control Strategy: Developing, Interpreting, and Sharing the Data Rich Content of an Enhanced Submission
    Kevin Seibert, PhD,MS
    Senior Research Advisor, Chemical Product R&D
    Eli Lilly and Co., US, United States
  • Design Space - The Challenges of Balancing Knowledge, Risk and Uncertainty
    John Lepore, PhD
    Senior Director, Chemical Process Development and Commercialization
    Merck & Co. Inc., United States
• 10:45AM - 12:15PM

  Session 2B: Pharmacopoeias: Collaboration Across Borders

  
  Session Chair(s):

  • Erin Wang, MS,RAC
    Associate Consultant – Compendial Affairs, Global Quality Laboratory
    Eli Lilly and Company, US, United States

  Pharmacopeias are recognized sources of public standards for pharmaceutical products, ingredients, components, and agreed common practices. Compendial standards are typically comprised of test procedures and associated acceptance criteria. These standards are enforced by regulatory agencies, and are quality standards to be met by all manufacturers/applicants for certain territories. Companies that wish to market products globally must have a robust strategy for meeting compendial requirements for each market. Collaboration between regulators, industry, and pharmacopeias is crucial to ensure that these standards serve to benefit public health, both regionally and internationally. This session will provide an overview of the United States and European compendial science activities. The industry presentation will share experiences in managing public standard implementation globally. Panel discussion will allow participants to have deeper interactions with the speakers and the fellow participants.

  Speaker(s):

  • USP Compendial Activites
    Shawn Dressman, PhD
    Vice President, Chemical Medicines, USP-NF
    United States Pharmacopeia, United States
  • European Pharmacopoeia Overview (presentation will be delivered via webinar)
    Michael Wierer, PhD
    Deputy Head, European Pharmacopoeia Department
    European Directorate for the Quality of Medicines and HealthCare, France
  • Compendial Affairs
    Craig Hamilton, PhD
    Director - Operational Excellence, Global Quality Laboratory
    Eli Lilly and Company, United States
  • Compendial Affairs
    Erin Wang, MS,RAC
    Associate Consultant – Compendial Affairs, Global Quality Laboratory
    Eli Lilly and Company, US, United States
• 10:45AM - 12:15PM

  Session 2C: Clinical Trials and Marketing Applications in Latin America CMC Considerations

  
  Session Chair(s):

  • Rebecca E. Komas, MS
    Director, CMC Advocacy
    GlaxoSmithKline, United States

  ANVISA regulators will discuss the CMC and Clinical Trials requirements in the Latin America region. Industry speakers will share opportunities, challenges and recent experience with clinical trial and marketing applications. This will include experience with recent guidance such as the API Stability Guidance Resolution 45 issued August 2012. This will be followed by a panel discussion including additional industry experts. An opportunity for the audience to master the intricacies of CMC aspects of clinical trial and marketing applications in the Latin America market with special focus on Brazil.

  Speaker(s):

  • CMC Requirements in Brazil- Regulatory Aspects
    Ana Carolina Araujo, PharmD
    Sanitary Surveillance and Regulation Specialist - Pharmacist, COPRE, GTFAR
    Brazilian Health Surveillance Agency-Anvisa, Brazil
  • Clinical Trial Requirements in Brazil - CMC Considerations
    Fanny N. Viana, PharmD
    Sanitary Surveillance and Regulation Specialist - Pharmacist, COPEM, GESEF
    Brazilian Health Surveillance Agency-Anvisa, Brazil
  • Industry Experience with Clinical Trial and Marketing Applications in Latin America
    Rebecca E. Komas, MS
    Director, CMC Advocacy
    GlaxoSmithKline, United States
  • Panel Industry Representative
    Ganapathy Mohan, PhD
    Executive Director Global CMC, Pharmaceutical and Devices
    Merck, Sharp and Dohme, Corp., US, United States
  • Panel Industry Representative
    Marcio Andre Silva
    Regulatory Manager for GSK Brazil
    GlaxoSmithKline Brazil Ltda, Brazil, Brazil
• 1:30PM - 3:00PM

  Session 3A: Global Supply Chain: Counterfeits

  
  Session Chair(s):

  • Ganapathy Mohan, PhD
    Executive Director Global CMC, Pharmaceutical and Devices
    Merck, Sharp and Dohme, Corp., US, United States

  In this session, the speakers will focus on counterfeits in the pharmaceutical global supply chain, their impact and initiatives to counter this continuously growing problem. Strategies to stay ahead of counterfeits are definitively a challenge. Initiatives taken in the area of packaging to prevent counterfeiting through innovative techniques have shown to be promising however more and more fighting counterfeits in the supply chain will rely on advanced technologies

  Speaker(s):

  • Assurance of Quality in the Global Supply Chain
    Jennifer Finnegan McCafferty, PhD
    Vice President, External Quality, Global Manufacturing & Supply
    GlaxoSmithKline, US, United States
  • Global Supply Chain Security with Focus on EU Falsified Medicine Directive
    Michael Rose, MS
    Vice President, Supply Chain Visibility
    Johnson and Johnson Health Care Systems, Inc., United States
• 1:30PM - 3:00PM

  Session 3B: Impurities - Genotoxic Impurities

  
  Session Chair(s):

  • Stephen Miller, PhD
    CMC Lead, Office of New Drug Quality Assessment, CDER
    FDA, United States

  Genotoxic compounds may be used as synthetic intermediates, reagents, etc, or may be formed as by-products during the manufacture of pharmaceuticals. Maintaining any genotoxic impurities at or below levels that are acceptable presents challenges to industry and to regulators due to the need for highly sensitive analytical methods, and the diversity of structures and reactivity. This session will present current approaches under consideration for an ICH guideline, and discuss experience from both the industry and regulatory perspectives on the control of genotoxic impurities in pharmaceuticals.

  Speaker(s):

  • Genotoxic Impurities: EU Experience and Comparison with the Current Discussion at ICH
    Jean-Louis Robert, PhD
    Head, Medicines Control Laboratory
    National Health Laboratory, Luxembourg
  • Genotoxic Impurities - An Industry Perspective
    Kate Arnot, MSc
    CMC Director, Global Regulatory Affairs
    AstraZeneca, United Kingdom, United Kingdom
  • Genotoxic Impurities as Critical Quality Attributes
    Stephen Miller, PhD
    CMC Lead, Office of New Drug Quality Assessment, CDER
    FDA, United States
• 1:30PM - 3:00PM

  Session 3C: Clinical Trials in the European Union (Biologicals)

  
  Session Chair(s):

  • Jilla Boulas, MS
    Director
    Voisin Consulting, Inc., United States

  This session will focus on the EU guideline for CMC requirements for clinical trials for Biologicals, released in May 2012. Experiences and perspectives will be shared on the key elements when compiling and maintaining CMC documentation for investigational medicinal products. An update on the proposed EU Clinical Trials Regulation and its opportunities for the future and Biologicals in particular will also be discussed: simplified submission, authorization procedures, etc.

  Speaker(s):

  • An Industry View Point On The CMC Requirements For Clinical Trials In The EU (Biotherapeutics)
    Stefanie B. Pluschkell, PhD
    Senior Director, Global Biotherapeutics, Policy and New Product Development
    Pfizer Inc, United States
  • CMC Aspects Of Clinical Trial Applications: What The Regulators Ask - An Industry Perspective
    Khandan Baradaran, PhD
    Associate Director Regulatory Affairs
    Biogen Idec, United States
• 3:30PM - 5:30PM

  Session 4A: Drug Shortages

  
  Session Chair(s):

  • Norman R. Schmuff, PhD
    Associate Director for Product Quality
    FDA, United States

  Drug and other medical product shortages have the potential to adversely affect patient care by delaying treatment or forcing the use of second-choice products. Some recent shortages have involved drugs for life-threatening conditions and, in some cases, the product in shortage has been the only product for the patient’s condition. This is a significant public health problem, one that deserves the concerted attention of government and industry. In this session causes, consequences, and FDA action to address and prevent drug shortages will be discussed.

  Speaker(s):

  • Current Trends in Drug Shortages -- A University of Utah Drug Information Perspective
    Erin Fox, PharmD
    Director, Drug Information Service
    University of Utah Hospitals and Clinics, United States
  • FDA's Role in Mitigating Drug Shortages
    Valerie Jensen, RPh
    Associate Director, CDER Drug Shortage Staff
    FDA, United States
  • Economic and Technological Drivers of Generic Sterile Injectable Drug Shortages
    Marta E. Wosinska, PhD
    Director for Economics Staff, Office of Planning and Analysis, CDER
    FDA, United States
• 3:30PM - 5:30PM

  Session 4B: Impurities - Metal Impurities

  
  Session Chair(s):

  • John F. Kauffman, PhD,MBA
    Research Chemist, Office of Pharmaceutical Science, CDER
    FDA, United States

  This session will provide a status update on the ICH Q3D guideline on metal impurities in pharmaceuticals and a description of the IPEC Q3D Information Exchange Request documents. In addition, examples of risk-based approaches to controls on metal impurities in pharmaceuticals will be described. At the conclusion of the presentations, the panel of speakers will be available to answer questions from the audience.

  Speaker(s):

  • Approaches To Assess And Control Metal Impurities In Drug Products
    Mark G. Schweitzer, PhD
    Director, NCE Analytical R&D LC
    AbbVie, Inc., United States
  • ICH Q3D Metal Impurities – Excipient Realities and Challenges
    Katherine L. Ulman
    Associate Scientist & Global Regulatory Compliance Manager
    Dow Corning, United States
  • Metal Analysis in Pharmaceuticals: Impending Changes and Potential Strategies to Address Them
    Nancy Lewen
    Principal Scientist
    Bristol-Myers Squibb, United States
• 3:30PM - 5:30PM

  Session 4C: Clinical Trials Submissions in Asia Pacific Region

  
  Session Chair(s):

  • Lin-Jau (Christine) Wu Anderson, MS,RAC
    Senior Research Scientist, Global Regulatory Affairs CMC
    Eli Lilly and Company, United States

  There are many new regulatory developments in the Asia Pacific region. In order to have a successful clinical trial application in this region, the sponsors need to stay on top of the changes and new requirements. This session will cover the most recent hot topics in China, South Korea, India, Taiwan, Hong Kong, Malaysia, etc., regarding the clinical trial submission.

  Speaker(s):

  • Clinical Trial Application in Asia Pacific Region other than China
    Xiling Song, MS
    Regulatory Product Manager, Pharma Technical Regulatory
    Genentech, Inc., United States
  • Clinical Trial Application in China
    Chi-Wan Chen, PhD
    Executive Director, Global CMC
    Pfizer Inc, United States
  • Key Regulatory Development in China
    Min Gui, PhD
    Director, CMC Asia Pacific, China CMC, Global Regulatory Sciences
    Bristol-Myers Squibb, China

Day 2 Tuesday, April 16, 2013

• 8:00AM - 9:30AM

  Session 5: Clinically Relevant Specifications

  
  Session Chair(s):

  • Nagesh Bandi, PhD
    Senior Manager, Global CMC
    Pfizer Pharmaceuticals, Inc., United States

  Due to the critical role that dissolution plays in the bioavailability of the drug, in vitro dissolution can serve as a relevant predictor of the in vivo performance of the drug product. This plenary session will discuss relevant factors that should be considered in developing a clinically relevant dissolution method and specifications. The role of dissolution in the development of the appropriate design space will also be illustrated. Establishing the appropriate dissolution specifications will assure that the manufacture of the dosage form is consistent and successful throughout the product’s life cycle and that each dosage unit within a batch will have the same pharmaceutical qualities that correspond to those shown to have an adequate safety and efficacy profile.

  Speaker(s):

  • Speaker
    Richard T. Lostritto, PhD,MS
    Associate Director, Office of New Drug Quality Assessment, DPAMS, CDER
    FDA, United States
  • How Do We Achieve Clinical Relevancy And Is This A Regulatory Requirement?
    Ganapathy Mohan, PhD
    Executive Director Global CMC, Pharmaceutical and Devices
    Merck, Sharp and Dohme, Corp., US, United States
  • Challenges for Developing Dissolution Methods with Clinically Relevant Specifications
    Roy De Maesschalck, PhD
    Associate Director, Dissolution testing
    Janssen Pharmaceutical Companies - Johnson&Johnson, Belgium
• 10:00AM - 12:00PM

  Session 6A: Regional Approaches to Bioequivalence

  
  Session Chair(s):

  • Anther C. Keung, PhD
    Director, Clinical Pharmacology Leader
    Janssen Pharmaceutical Companies of Johnson and Johnson, United States

  Bioequivalence (BE) studies are typically performed following CMC changes either as bridge studies between clinical trial and to-be-marketed formulations or during lifecycle management (post-approval changes). In addition, BE studies are also performed to support Biowaiver strategy for combination products. The design, performance, and evaluation of bioequivalence studies have received major attention lately. This session proposes to focus on the difference in bioequivalence studies requirements across regions/countries based on the more general and global requirements.

  Speaker(s):

  • The WHO Approach to Bioequivalence: The Global Perspective
    Jan Welink
    Senior Pharmacokinetic Assessor
    Medicines Evaluation Board, Netherlands
  • Brazilian (Anvisa) Requirements for Bioequivalence Studies
    Marcia Sayuri Takamatsu Freitas
    Partner - Owner
    BE Consulting, Brazil, United States
  • Japan (PMDA) Requirements for Bioequivalence Studies
    Vijay Tammara, PhD
    Vice President, Regulatory Affairs
    Nuron Biotech Inc., United States
• 10:00AM - 12:00PM

  Session 6B: Control Strategy - Lessons Learned

  
  Session Chair(s):

  • Elaine Morefield, PhD
    Deputy Office Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States

  This session will cover industry and regulator experiences with implementing integrated control strategies for small molecule and biologic products. Control strategy discussion may include process controls, in process tests, design space and real time release testing. Drug substance and drug product considerations will be covered. Parenteral and solid oral dosage form examples will be presented.

  Speaker(s):

  • Regulatory Considerations for Control Strategy
    Elaine Morefield, PhD
    Deputy Office Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States
  • Experiences in Establishing a Commercial Control Strategy for a Small Molecule Product
    John Groskoph, MBA
    Senior Director, Global CMC
    Pfizer Inc, United States
  • Considerations for Establishing a Control Strategy for a Biologic Product Throughout Development
    Allison Wolf, MS
    Research Scientist, Global Regulatory Affairs CM&C
    Eli Lilly & Company, United States
• 10:00AM - 12:00PM

  Session 6C: Antibody-Drug Conjugates (ADC)

  
  Session Chair(s):

  • Thirunellai G Venkateshwaran, PhD
    Director Pharma Technical Regulatory
    Genentech, A Member of the Roche Group, United States
  • Sarah C. Pope Miksinski, PhD
    Acting Director, Div.1, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States

  The goal of this session is to discuss the current status of the various control systems for Antibody-drug conjugates (ADCs) and some of the typical challenges that are encountered during their development and commercialization. ADCs, monoclonal antibodies (MAbs) coupled to cytotoxic agents, utilize the specificity of a monoclonal antibody to deliver a cytotoxic drug to tumor cells. Since an ADC product is a mixture of conjugated species, appropriate assays are key to characterize and release ADC products as well as identifying potential critical quality attributes (CQAs) for the conjugates, including those for the antibody, the cytotoxic agent, and the linker.

  Speaker(s):

  • Antibody Drug Conjugates – Current Status of Regulations
    Debasis Ghosh
    Office of New Drug Quality Assessment, CDER
    FDA, United States
  • Antibody Drug Conjugates – Current Status of Regulations
    Marjorie A. Shapiro, PhD
    Chief, Laboratory of Molecular and Developmental Immunology, OBP, CDER
    FDA, United States
  • Analytical Characterization and Control Strategies for Antibody Drug Conjugates
    Heyi Li, PhD
    Senior Principal Scientist
    Analytical R&D, BioTx PS; Pfizer, Inc., United States
• 1:00PM - 3:00PM

  Session 7A: Pediatrics: Challenges and Specific Requirements

  
  Session Chair(s):

  • Jenny Walsh, PhD
    Director
    Jenny Walsh Consulting Ltd., United Kingdom

  The development of paediatric medicines can be particularly challenging, since it is necessary to consider the diversity of this patient population in terms of physiological and biological maturation, compliance challenges such as acceptable palatability and potential safety concerns associated with the required excipients. This session will discuss some of these challenges in more detail and provide an overview of the European perspective including Paediatric Investigation Plans (PIPs). An update on the joint AAPS/Pediatric Task Force Points to Consider Document will also be provided.

  Speaker(s):

  • Challenges Associated with Developing Medicines for Children - An Industry Perspective
    Gossett Campbell, PhD
    Scientific Investigator, Formulation and Process Analysis
    GlaxoSmithKline, United States
  • EU Paediatric Investigation Plans
    Mike H Saleh, MS
    Director, Global Chemistry, Manufacturing and Controls
    Pfizer, Inc., United States
  • AAPS/Pediatric Task Force Points to Consider Paper on Pediatric Formulations Development
    Georgia Charkoftaki, PhD
    Research Associate Laboratory of Biopharmaceutics and Pharmacokinetics
    National and Kapodistrian University of Athens, Greece
• 1:00PM - 3:00PM

  Session 7B: Control Strategy in Continuous Manufacturing

  
  Session Chair(s):

  • Thirunellai G Venkateshwaran, PhD
    Director Pharma Technical Regulatory
    Genentech, A Member of the Roche Group, United States

  Continuous manufacturing is the ability to process materials to a defined end state without any process interruptions. Continuous processes while prevalent in industries such as petrochemical manufacturing are a fairly innovative concept in the Pharmaceutical Industry. It is often seen as the next step in the cGMP’s for the 21st century initiative and is currently being explored by a number of companies. The advantages of continuous processing while many are also accompanied by some uncertainties. The purpose of this session is to focus on continuous manufacturing process and control strategies for continuous processing. The challenges as well as opportunities of continuous manufacturing will be discussed as part of the session.

  Speaker(s):

  • Continuous Processing – A Regulatory Perspective
    Sharmista Chatterjee, PhD
    Chemist, Office of New Drug Quality Assurance, CDER
    FDA, United States
  • Implementation of Continuous Manufacturing in the Pharmaceutical Industry: Challenges and Opportunities
    Nirdosh K. Jagota, PhD
    Vice President and Global Head - Small Molecules
    Genentech, A Member of the Roche Group, United States
  • Continuous Manufacturing: Is it Feasible or Not?
    James Evans, PhD
    Associate Director, Novartis-MIT Center for Continuous Manufacturing
    Massachusetts Institute of Technology, United States
• 1:00PM - 3:00PM

  Session 7C: Biosimilars

  
  Session Chair(s):

  • Gregory C. Davis, PhD
    Consultant
    Davis Consulting Services, United States

  This session will examine the EU, Canada, and US regulatory pathways for the registration of biosimilars and discuss lessons learned from the implementation and application of these pathways to product reviews. The speakers will also update any anticipated changes to the pathways or to existing guidance.

  Speaker(s):

  • EU Regulatory Update On Guidance And Experience With Biosimilars (Presentation will be given remotely via webinar)
    Peter J Richardson, PhD
    Head of Biologics, Quality of Medicines Sector
    European Medicines Agency, European Union, United Kingdom
  • Canadian Approach to the Regulation of Subsequent Entry Biologics (Biosimilars)
    Anthony Ridgway, PhD
    Senior Regulatory Scientist, Office of the Director
    Health Canada, Canada
  • FDA Update on the Implementation of the Biosimilar Approval Pathway
    Steven Kozlowski, MD
    Director, Office of Biotechnology Products, CDER
    FDA, United States
• 3:30PM - 5:00PM

  Session 8: New Technologies

  
  Session Chair(s):

  • Fernando Muzzio, PhD
    Director, NSF ERC on Structured Organic Particulate Systems; Professor II
    Rutgers University, United States

  New technologies are rapidly gaining momentum in pharmaceutical manufacturing including new manufacturing and mathematical modeling approaches. Continuous manufacturing methods have attracted enormous interest in recent years. In this and in other areas, a growing toolbox of particle engineering methodologies are enabling both more effective manufacturing (by allowing the user to design particles with tailored properties) and more accurate drug release profiles (by allowing tighter control of particle size, particle-substrate interactions, etc). To help support these and other technologies, sophisticated modeling approaches have evolved. In this session, we will review recent advances from both a technological and regulatory perspective.

  Speaker(s):

  • New Technologies For Continuous Manufacturing
    Fernando Muzzio, PhD
    Director, NSF ERC on Structured Organic Particulate Systems; Professor II
    Rutgers University, United States
  • Mathematical Models for Enhanced Control of Pharmaceutical Manufacturing
    Christine M. V. Moore, PhD
    Acting Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States
  • New Particle Engineering Technologies For Modified Release Applications
    Raj Dave, PhD
    Distinguished Professor of Chemical, Biological and Pharmaceutical Engineering
    New Jersey Institute of Technology, United States

Day 3 Wednesday, April 17, 2013

• 8:00AM - 9:30AM

  Session 9A: Innovative Approaches for Stability Programs

  
  Session Chair(s):

  • Kenneth Waterman, PhD
    President
    Freethink Technologies, Inc., United States

  Stability methodologies for drug substances and drug products continue to evolve both from a scientific and regulatory perspective. This session will explore advances in scientific understanding of stability using the Accelerated Stability Assessment Program (ASAP) and how it impacts decisions in drug development. The session will also explore the changing ways that pharmaceutical companies look to meet their regulatory and ethical commitments while reducing low-value and often time-consuming testing.

  Speaker(s):

  • Global Acceptance Of Lean Stability Strategies: Still Just A Pipe-Dream Or Are We Making Real Progress?
    Stephen T Colgan, PhD
    Senior Director
    Pfizer Inc, United States
  • Using Scientific Methods To Model Shelf-Life For Solid Drug Substances And Drug Products
    Kenneth Waterman, PhD
    President
    Freethink Technologies, Inc., United States
  • Using Predictive Stability To Enhance Product Knowledge
    Zhixin Jessica Tan, PhD
    Principal Scientist
    Amgen Inc., United States
• 8:00AM - 9:30AM

  Session 9B: Post-Approval Change Management Protocols

  
  Session Chair(s):

  • Moheb M. Nasr, PhD,MS
    Vice President, Regulatory CMC Strategy
    GlaxoSmithKline, United States

  A post-approval change management protocol describes changes that a company would like to implement during the lifecycle of the product and how these would be prepared and verified. The use of post-approval protocols can greatly facilitate life-cycle management of CMC. However, the utilization of post-approval management protocols has not been widely embraced by industry. Additional experience and regulatory clarity could speed up implementation. Leading regulators, from Europe and United States, and industry speakers will share their perspectives on the utilization of post-approval management protocols, outline implementation challenges and provide suggestions to facilitate preparation and approval. Presentations will be followed by panel discussions

  Speaker(s):

  • Regulatory Consideration For The Development And Filing Of Post-Approval Management Protocols – EU Perspective
    Jean-Louis Robert, PhD
    Head, Medicines Control Laboratory
    National Health Laboratory, Luxembourg
  • Regulatory Consideration For The Development And Filing Of Post-Approval Management Protocols – FDA Perspective
    Christine M. V. Moore, PhD
    Acting Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States
  • Utilization Of Post-Approval Management Protocols In The Implementation Of Quality By Design (Qbd) For Biological Drug Products
    Lynne Krummen
    Vice President, Technical Regulatory, Biologics
    Genentech, A Member of the Roche Group, United States
• 8:00AM - 9:30AM

  Session 9C: Advanced-Therapy Medicinal Products (ATMP)

  
  Session Chair(s):

  • Deborah Hursh, PhD
    Senior Investigator, CBER
    FDA, United States

  Interest in ATMP’s continues to grow, with the recent authorization of Glybera in the EU and there are a substantial number of products under development. The Committee for Advanced Therapies at the EMA has received a large number of scientific advice requests and FDA has seen increasing numbers of Investigational New Drug Applications and more advanced Phase trials. This experience shows that common issues arise for CMC related to characterization, consistency and comparability, particularly for potency assays. The current regulatory climate will be explored and examples discussed to highlight these aspects.

  Speaker(s):

  • CMC Regulatory Considerations for Advanced Therapies
    Deborah Hursh, PhD
    Senior Investigator, CBER
    FDA, United States
  • Development Experience Gained From The EU Authorization Glybera
    Harald Petry, PhD
    VP, Director Research and Development
    uniQure B.V, Netherlands, Netherlands
  • Speaker
    Robert A Preti, PhD
    President & CSO
    PTC Cell Therapy Services, United States
• 9:45AM - 11:15AM

  Session 10A: Shipments Monitoring and Temperature Control

  
  Session Chair(s):

  • Kenneth Waterman, PhD
    President
    Freethink Technologies, Inc., United States

  While storage and shipping conditions are qualified based on stability studies, invariably in an increasingly dynamic world market, materials will experience excursions. The responsible company needs to justify the acceptability (or make a decision to discard) of any such drug products. This session will discuss the scientific and regulatory basis for determining the appropriate course of action.

  Speaker(s):

  • Storage Temperature × Humidity Design Space And Logistics Excursions
    William R. Porter, PhD
    Principal Scientist
    Peak Process Performance Partners, United States
  • Dealing With Temperature Excursions During Shipment And Storage
    Manuel Zahn, DrSc,PhD
    Managing Director
    3R Pharma Consulting GmbH, Germany
  • Use Of Stability Modeling As Part Of Qbd To Determine The Impact Of Excursions On Drug Substances And Drug Products
    Kenneth Waterman, PhD
    President
    Freethink Technologies, Inc., United States
• 9:45AM - 11:15AM

  Session 10B: Process Validation: Maintaining the State of Validation in the Continuous Improvement Paradigm

  
  Session Chair(s):

  • Prabu Nambiar, PhD,MBA,RAC
    Principal, Syner-G Pharma
    CMC Consulting, LLC, United States

  Continuous improvement (CI) is one of the key benefits of drug development under the QbD based drug development paradigm. The traditional process validation requirements might be interpreted to be limiting in terms of the industry’s ability to realize the full benefits of the CI process. In this regard, FDA and EMA have issued a draft process validation guidance document that provide for the possibility of continuous process verification (CPV) to cover an alternative approach to process validation based on a continuous monitoring of manufacturing performance. The FDA draft guidance has focused on process validation as a three stage process – Process Design, Process Qualification and Continuous Process Verification. The objective of this session is to explore how companies can use the CPV approach to maintain the state of validation and be in full compliance with regulatory requirements.

  Speaker(s):

  • Continuous Improvement and Continuous Process Verification: FDA Perspectives
    Kelli F. Dobilas
    Supervisory Investigator, NWJ-DO, ORA
    FDA, United States
  • Maintaining the State of Validation in an Outsourced Manufacturing Paradigm
    Eda Ross Montgomery, PhD
    Senior Director
    Shire Pharmaceuticals, United States
  • Innovative Approaches To Process Validation
    Thirunellai G Venkateshwaran, PhD
    Director Pharma Technical Regulatory
    Genentech, A Member of the Roche Group, United States
• 9:45AM - 11:15AM

  Session 10C: Drug/Device Combination Products

  
  Session Chair(s):

  • Douglas Mead
    Director, CMC Global Regulatory Affairs
    Janssen Pharmaceutical Companies of Johnson&Johnson, United States

  Evolving regulations and requirements related to drug/device combination products have increased the complexity of their development, including questions of their use in clinical trials, design validation, regulatory submission content, and post market changes. This session will focus on the latest changes and trends in regulatory requirements that are impacting the introduction of new drug delivery technologies. In addition to increasingly specific technical requirements, health authorities are also starting to expect that to-be-marketed presentations be used in clinical trials and that the usability of these devices be assessed in formal human factors studies. Session speakers will address the current regulatory landscape for these requirements and strategies that may successfully meet them.

  Speaker(s):

  • FDA Updates to Combination Product Regulations
    Patricia Y. Love, MD,MBA
    Deputy Director, Office of Combination Products, OC
    FDA, United States
  • Human Factors Study Requirements For Drug/Device Combination Products
    Mark Marley
    Principal Research Scientist - Regulatory-Devices
    Eli Lilly and Company, United States
  • Clinical Trial Challenges Related To Drug/Device Combination Products
    Donna French, PhD
    Senior Director
    Genentech, a Member of the Roche Group, United States
  • Clinical Trial Challenges Related To Drug/Device Combination Products
    Le Dao
    Principle Research Associate
    Genentech, a Member of the Roche Group, United States
• 11:30AM - 12:00PM

  Session 11: Panel Discussion

  
  Session Chair(s):

  • Elaine Morefield, PhD
    Deputy Office Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States

  This session will provide participants with a last opportunity for questions as a wrap-up of the 2.5 day workshop.

  Speaker(s):

  • Panel Speakers
    Jean-Louis Robert, PhD
    Head, Medicines Control Laboratory
    National Health Laboratory, Luxembourg
  • Panel Speakers
    Diana Amador Toro, PhD
    Director, New Jersey District, Office of Regulatory Affairs
    FDA, United States
  • Panel Speakers
    Ana Carolina Araujo, PharmD
    Sanitary Surveillance and Regulation Specialist - Pharmacist, COPRE, GTFAR
    Brazilian Health Surveillance Agency-Anvisa, Brazil
  • Panel Speakers
    Christine M. V. Moore, PhD
    Acting Director, Office of New Drug Quality Assessment, OPS, CDER
    FDA, United States