BCG Overview Of Services And Value Propositions 2010
- 1. Overview of Services and Value Propositions 2010
- 2. Q: Besides Being Blockbuster Products, What Do These Have in Common? $4.4B* $1.5B* Drug-eluting coronary stent $3.8B* $3.6B* A: Before they could be sold, approval was required by the Food and Drug Administration or associated regulatory agency outside the U.S. * 2008 annual sales
- 3. Biologics Consulting Group: 17+ Years of Expertise in Helping Companies Move Technology into the Marketplace Facilitates and accelerates product development by: • Deep understanding of FDA regulations • Identification, design, and review of the key steps required for testing in man • Anticipation of land-mines in the road to commercialization • Practical advice and solutions tailored to a company’s product development plans Alexandria (Headquarters) Beijing Boston Raleigh San Francisco Tokyo
- 4. BCG Offers Comprehensive Regulatory Strategic Planning Industries Services Biologics Audits and Inspections Combination Products Clinical Development Devices Pharmacology/Toxicology Diagnostics Process validation Drugs Product Development Program Management Regulatory Support All Major Markets, incl: Training programs Autoimmunity/Transplantation U.S. Agent Cancer Cardiac disease Diabetes/Metabolic disease Infectious disease Inflammatory disease Neurological disease Orthopedics
- 5. Regulatory Affairs Impact Key Early Development Milestones Lead ID Proof-of-concept IND Basic Research Development Preclinical • Pre-IND support • Line development • Pre-clinical plan Pre-IND meeting with FDA • CMO selection • Clinical synopsis • Method development • CRO selection • Gap analysis • Development plan • Pre-IND Follow-up • Protocol Preparation • CMO/CRO Mgmt • Quality Systems Implementation • Method Qualification
- 6. Regulatory Affairs Impact Key Clinical Development Milestones 1st-in-human P1 P2 BLA/NDA Clinical Development FDA Filing, Preclinical Approval & Phase 1 Phase 2 Phase 3 Launch Initial IND End of Phase 2 Market Safety submission meeting Application update submission • IND preparation • Ongoing submission • Clinical site selection • IND support • Clinical trial support Pre-BLA or -NDA meeting
- 7. How big can we make our role? A Poor Regulatory Strategy Has a Significant, Negative Financial Impact CAUSE EFFECT ▼ Clinical Hold Private company: ▼ Refusal to file Bridge financing may be needed ▼ Complete Response Letter Down round may occur Flat round may occur ▼ Warning Letter IPO/M&A unlikely ▼ Bridging Studies ▼ New product Designation Public company: Decreased market cap ▼ Comparability Studies Secondary offering unlikely ▼ Clinical Supply Shortages Loss of confidence by ▼ Non-approval public markets
- 8. BCG Consultants Possess Extensive Regulatory Affairs Experience
- 9. BCG Consultants Represent Both FDA and Industry Experience
- 10. BCG: Continual Increase in Clients Served (2000-2008) Most projects come from: 439 • Word-of-mouth 397 • Referrals • Repeat business 312 265 Continual growth and increased 218 revenues for 16 straight years, 186 despite: 160 • No marketing department 115 • No sales department 77 • No business development department
- 11. BCG Has Extensive, Successful Submission Experience (2002-2007) To sell a drug , the FDA must approve a: • Biologics License Application (BLA), or • New Drug Application (NDA) Paper Submissions To sell a device, the FDA must approve a: INDs (non-CTD and CTD) 60 • 510k, or BLAs/NDAs/PMAs 1 • Pre-Marketing Approval (PMA) To test a product in humans, the FDA Electronic Submissions must allow an: eINDs and related Amendments (non-CTD) 9 • Investigational New Drug (IND) Application eINDs and related Amendments (CTD) 14 In 2009, FDA issued a guideline eBLAs/NDAs 4 (Guidance Document) for Common Technical Documents (CTDs) • CTDs are standards used by U.S., Japan, and Europe to expedite human studies FDA and other world bodies are rapidly moving towards electronic submissions – and away from paper
- 12. Commercial Sector: Project Examples Client Project Summary On-site biologics training regarding vaccine manufacturing Top-5 Public Pharmaceutical Firm (U.S.) technologies and regulatory compliance. Due diligence for strategic partnership. Evaluated raw material control, manufacturing, in-process control, specifications and Top-5 Public Pharmaceutical Firm (Japan) analytical methods, cell bank status, and development and commercialization strategy. Alliance executed. Top-5 Public Biotechnology Firm Review and gap analysis of commercial stability programs. Top-5 Public Biotechnology Firm Process Validation Master Plan for pandemic flu. Developed a strategy and plan for writing a BLA for an assay to Top-5 Drug Development Contract screen injectable drugs, biologics, medical devices, and raw Research Organization materials for the presence of bacterial endotoxin. Application received FDA approval. Assay currently marketed. Electronic publishing of their IVIG BLA in eCTD format for Large, Private Device Firm submission to CBER, FDA. Craft Product Development Plan (PDP), including: (1) scale-up Small, Private, Infectious Disease and validation, and (2) qualification/validation of the test Biotechnology Firm methods for product characterization, and potency evaluation.
- 13. Government and Public Sector: Project Examples Client Project Summary Audit blood product manufacturing facility. American Red Cross Assess current level of compliance with – and corrective actions regarding – applicable US regulatory requirements for licensure, Assess capabilities and needs for various clinical programs and Major, World-wide Infectious Disease manufacturing facilities related to producing treatments for key Foundation developing-world diseases. Regulatory support leading to the filing of an Investigation New State of Texas’ University Research Drug (IND) application with the FDA for a phase 1 clinical trial of System a virus-based treatment for Cystic Fibrosis. National Institutes of Health’s Grand Pre-clinical and clinical development support for medical Program in Global Health investigators, to get promising research to human studies. Determine appropriate levels of quality systems for various St. Jude Children’s Research Hospital tissue-based therapies, and whether they adhere to FDA mandates. Pan-Provincial Vaccine Product development support for initiatives involving several Enterprise/University of British Columbia potentially pandemic diseases.
- 14. BCG has Extensive, Comprehensive Consulting Expertise Consulting Areas: AUDITS AND INSPECTIONS PHARMACOLOGY/TOXICOLOGY • Inspection of manufacturing facilities • Program planning to support clinical • Audit of system/process validation studies product development • Evaluation of company Quality System • Writing of the pharm/tox section of INDs, BLAs, NDAs • Evaluation of company procedures • Review and interpretation of toxicology • Good Laboratory Practices (GLP) Audits study data with respect to potential risk • Quality Systems Audits • Participation in due diligence programs for perspective new products CLINICAL DEVELOPMENT • GLP audits • Assistance in responding to questions • Development of comparability protocol from FDA: • Writing of product label » Center for Biologics Evaluation and Research (CBER) » Center for Drug Evaluation and Research (CDER) • Recommendation/interface with toxicology » Center for Devices and Radiological Health (CDRH) Contract Research Organizations (CROs) • Biostatistics • Participation in FDA meetings • Case Report Form (CRF) design • Clinical Development Plan • Clinical Development Support • Investigator’s Brochure (IB) preparation • Medical writing, grant preparation • Phase I/II study design • Protocol design and preparation • Phase I study site selection
- 15. BCG has Extensive, Comprehensive Consulting Expertise PROCESS VALIDATION • Preclinical • Evaluate process validation status • Regulatory • Identifying critical process parameters and • Clinical Development designing appropriate validation studies • An integrated stand-alone document tying • Apply statistical methods to the evaluation all the four main areas of product of process validation data development with budgets, tasks, and timelines through Phase 1 or beyond • Assist in preparing process validation related submissions to CBER/CDER/CDRH Why Develop a Product Development Plan? • Validation Master Plan • Planning is crucial at every stage of • Facility Validation development, particularly at the outset o Equipment Validation • Provides a concise detailed analysis of o Utilities Validation your product and the roadmap to market o Cleaning Validation • Clearly states developmental objectives and crucial milestones PRODUCT DEVELOPMENT • Presents a single (or multiple, if desired) • A "roadmap" for your product's focused regulatory strategy for presenting development your product to the FDA • A concise, product-focused, strategic • Presents strategies for dealing with document laying out the path to market potential roadblocks and hurdles in the approval or licensure product development process • A detailed analysis of your product status • Lays out accurate and realistic budgets and and developmental requirements, including timelines through clinical development the four primary aspects of product development: • Manufacturing
- 16. BCG has Extensive, Comprehensive Consulting Expertise PROGRAM MANAGEMENT TRAINING PROGRAMS • Prepare Product Development Plan • Develop specific seminars and workshops • Prepare Project Timeline • BCG has offered courses such as: • Provide oversight and ongoing quality o "The Biologics Approval Process” review and adjustment of project o “Systems Approach to Process • Identify external contractors Validation” • Pre-clinical, Clinical, and CMC coordination o “Clinical Study Documents – • Central liaison with contractors and Regulatory Requirements and sponsor Review.” • Monitoring of project resources, tasks and timeline U.S. AGENT • Preparation and management of budget • U.S. Agent for filing INDs • Provide project updates/reports • U.S. Agent for Foreign Establishment Registration and Listing REGULATORY SUPPORT • Pre-IND and IND support • BLA/NDA support • Device support • Electronic submissions • Request for Orphan Drug designation • Preparation of Drug Master Files
- 17. Summary Highly experienced experts from bench to market/patient FDA/Industry experience provides best of both perspectives Expertise covering a wide range all aspects of numerous major markets, including: – Biologics – Pharmaceuticals – Medical devices – Combination products
- 18. BCG Contacts James G. Kenimer, PhD Ronald A. Marchesani CEO Head, West Coast Operations jkenimer@bcg-usa.com rmarchesani@bcg-usa.com Corporate Office West Coast Office 1317 King Street 1840 Gateway Drive, Alexandria, VA 22134 Suite 200 www.bcg-usa.com www.bcg-usa.com P: 800-485-0106 P: 650-378-1303 703-739-5695