FDA's Regulation of Regenerative Medicine including Stem Cell Treatments, Tissue Engineering and Gene Therapies

Registration Process: 8:30 AM – 9:00 AM

Session Start Time: 9:00 AM

Introduction Human Regenerative Medicine: Science and the Law

• FDA’s approval process for regenerative medicine products
• FDA’s Regulation of Human Cells, Tissues or Cellular or Tissue-based Products (“HCT/P”)
• The application of the FFDC Act and the PHS Act
• The criteria for determining how a product will be regulated
• The “Minimal Manipulation” Standard
• Approval of Human Regenerative Treatments
• FDA’s regulatory approval pathway for HCT/Ps
• The new drug and biologic approval pathways
• The various existing regulatory drug approval pathways (the “full” NDA v. the 505(b)(2) application) and the biologic approval pathways (BLA v. biosimilars) will be discussed.
• The NDA and BLA review and approval processes including a discussion of available options, application components, relevant meetings, timing, costs and approval requirements.
• Obtaining designation and approval as an Orphan Drug Product
• Survey of the types of HCT/Ps and those indications receiving designation and approval as Orphan Drugs
• The process for obtaining orphan drug designation and approval
• The incentives accompanying orphan drug designation and approval
• Available Marketing Exclusivity
• Regulating Some Regenerative Treatments as Medical Devices
• Products and accessories being regulated as medical devices
• Identifying such products for regulatory purposes
• Overview of FDA’s medical device clearance and approval pathways
• Strategies for marketing and selling such products
• The cGMPs and cGLPs Applied to Regenerative Treatments and Therapies
• How to implement appropriate cGMPs and cGLPs applicable to the manufacturing, production, control, packaging and distribution of HCT/Ps
• Some of the topics to be covered: material sourcing, characterization, manufacturing, sterility and quality controls
• Standard Operating Procedures or SOPs

Bringing Regenerative Medicine Products to Market: Four Key Components

• Conducting Clinical Trials to Support Product Approvals
• Factors to consider when designing study protocols, engaging Clinical Research Organizations and investigators, and conducting clinical studies.
• Labeling, Marketing and Advertising
• Definitions and Regulation
• Applicable Statutory and Regulatory Requirements
• Federal Agency Shared Jurisdiction
• Examples of Non-Compliance Labeling and Marketing
• FDA Enforcement Action and Effective Strategies for Mitigating Such Risk
• Types of enforcement action
• A survey of recent FDA enforcement activities
• Recommendations for mitigating the risk of FDA and FTC enforcement action
• Strategies for preventing products from being deemed “adulterated” or “misbranded”
• Intellectual Property Considerations
• U.S. patent rules applicable to stem cells and other regenerative products
• Relevant Patent Laws and Cases
• A survey of past important legal cases A
• Applicability of patent protection to drug marketing and commercialization, and the concept of Patent Restoration.

Introduction Veterinary Regenerative Medicine

• The Center for Veterinary Medicine’s (“CVM”)
• Regulatory Jurisdiction: FDA v. USDA
• CVM’s Organization
• USDA’s Organization
• Statutory and Regulatory Overview
• The Federal, Food, Drug and Cosmetic Act
• The Virus, Serum and Toxin Act of 1913
• Relevant Regulations
• Guidance Documents and Policy Statements
• The Regulation of Veterinary Cellular Products and Therapies
• Regulatory Status
• Definitions: Drug v. Biologic
• The Veterinary Drug Approval Process
• Approval Process
• NADA Approval Process
• User Fees and Waiver Request
• Sections of an NADA
• Product Characterization, CMC, Safety, Efficacy, etc
• Managing Veterinary Clinical Trials
• 7 Major Phases:
• Planning
• Study Initiation
• In-life Activities
• Site close-out
• Data management
• Biostatistical analysis
• Report writing
• Labeling, Marketing and Advertising
• Definitions and Examples
• Labeling
• Immediate Container, Outer Packaging, Package Insert, Client Information Sheet, Shipping Label
• Marketing Materials
• Brochures, Leaflets, Press Releases, Flyers, Audio and Visual Pieces
• Advertising
• Written Media, Broadcast Media, Internet Media, Social Media
• Standards to Avoid Misbranding
• Truthful, Substantiated, and Not Misleading
• Effective Communication, Risk Communications, Risks and Benefits
• Timing of Review: NADA/ANADA Filing, Initial Publication/Broadcast, Disseminations Following Changes, Drug Experience Reports
• Post Approval Surveillance: Traditional Media Surveillance, Internet and Social Media Surveillance, Industry and Professional Events, Internal Company Materials
• Extra-Label Drug Use: Animal Medicinal Drug Use Clarification Act of 1994
• Veterinary Drug User Fees and Fee Reductions and Waivers
• Animal Drug User Fee Act (ADUFA) – Applies to Innovators Only
• Animal Generic Drug User Fee Act (ADGUF) – Applies to Generic Manufacturers
• Types of User Fees
• Types of Fee Waivers and Reductions
• Procedures, Timing and FDA Evaluation of Waivers or Reductions

Karl M. Nobert is a food and drug regulatory attorney with the Nobert Group LLC. Mr. Nobert provides regulatory advice and counseling to both large and small domestic and international pharmaceutical companies on regulatory issues related to the U.S. Food and Drug Administration’s review and approval of new drug products. This includes counseling clients on FDA’s regulation of prescription branded and generic drugs, and over-the-counter drug products. He also has considerable experience related to the regulation of biologics including cellular and genetic therapies, and veterinary drug products. He frequently presents and has published on such topics.