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Overview of Servicesand Value Propositions
2010
Q: Besides Being Blockbuster Products,What Do These Have in Common?
$4.4B*
$3.8B*$3.6B*
Drug-elutingcoronary stent
$1.5B*
A: Before they could be sold, approval was required by the Food and Drug Administration orassociated regulatory agency outside the U.S.
* 2008 annual sales
Alexandria (Headquarters) Beijing Boston
Raleigh San Francisco Tokyo
• 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
Biologics Consulting Group:17+ Years of Expertise in Helping Companies
Move Technology into the Marketplace
Facilitates and accelerates product development by:
BCG Offers ComprehensiveRegulatory Strategic Planning
Audits and Inspections Clinical Development Pharmacology/Toxicology Process validation Product Development Program Management Regulatory Support Training programs U.S. Agent
Biologics Combination Products Devices Diagnostics Drugs
Industries Services
All Major Markets, incl: Autoimmunity/Transplantation Cancer Cardiac disease Diabetes/Metabolic disease Infectious disease Inflammatory disease Neurological disease Orthopedics
Regulatory Affairs ImpactKey Early Development Milestones
BasicResearch Development Preclinical
• Pre-IND Follow-up• Protocol Preparation• CMO/CRO Mgmt• Quality Systems Implementation• Method Qualification
Pre-IND meeting with FDA• Line development• CMO selection• Method development
• Gap analysis• Development plan
• Pre-IND support• Pre-clinical plan• Clinical synopsis• CRO selection
Lead ID Proof-of-concept IND
Preclinical
Regulatory Affairs ImpactKey Clinical Development Milestones
Clinical Development
Phase 1 Phase 2 Phase 3
FDA Filing,Approval &Launch
1st-in-human P1 P2 BLA/NDA
• IND preparation
• Clinical site selection
Initial IND submission
• Ongoing submission• IND support• Clinical trial support
End of Phase 2 meeting
Pre-BLA or -NDA meeting
Market Application submission
Safety update
A Poor Regulatory Strategy Has a Significant, Negative Financial Impact
▼ Clinical Hold
▼ Refusal to file
▼ Complete Response Letter
▼ Warning Letter
▼ Bridging Studies
▼ New product Designation
▼ Comparability Studies
▼ Clinical Supply Shortages
▼ Non-approval
Private company: Bridge financing may be needed Down round may occur Flat round may occur IPO/M&A unlikely
Public company: Decreased market cap Secondary offering unlikely Loss of confidence by
public markets
How big can we make our role?
CAUSE EFFECT
BCG Consultants PossessExtensive Regulatory Affairs Experience
BCG Consultants RepresentBoth FDA and Industry Experience
BCG: Continual Increase in Clients Served(2000-2008)
115
77
160186
218
265
312
397
439Most projects come from:• Word-of-mouth• Referrals• Repeat business
Continual growth and increased revenues for 16 straight years, despite:
• No marketing department• No sales department• No business development
department
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)
To sell a device, the FDA must approve a:• 510k, or • Pre-Marketing Approval (PMA)
To test a product in humans, the FDAmust allow an:
• Investigational New Drug (IND) Application
In 2009, FDA issued a guideline (Guidance Document) for Common Technical Documents (CTDs)
• CTDs are standards used by U.S., Japan,and Europe to expedite human studies
Paper SubmissionsINDs (non-CTD and CTD) 60
BLAs/NDAs/PMAs 1
Electronic SubmissionseINDs and related Amendments (non-CTD) 9
eINDs and related Amendments (CTD) 14
eBLAs/NDAs 4
FDA and other world bodies are rapidly moving towards electronic submissions –and away from paper
Commercial Sector:Project Examples
Client Project Summary
Top-5 Public Pharmaceutical Firm (U.S.) On-site biologics training regarding vaccine manufacturing technologies and regulatory compliance.
Top-5 Public Pharmaceutical Firm (Japan)
Due diligence for strategic partnership. Evaluated raw material control, manufacturing, in-process control, specifications and 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.
Top-5 Drug Development Contract Research Organization
Developed a strategy and plan for writing a BLA for an assay to screen injectable drugs, biologics, medical devices, and raw materials for the presence of bacterial endotoxin. Applicationreceived FDA approval. Assay currently marketed.
Large, Private Device Firm Electronic publishing of their IVIG BLA in eCTD format for submission to CBER, FDA.
Small, Private, Infectious Disease Biotechnology Firm
Craft Product Development Plan (PDP), including: (1) scale-up and validation, and (2) qualification/validation of the test methods for product characterization, and potency evaluation.
Government and Public Sector:Project Examples
Client Project Summary
American Red CrossAudit blood product manufacturing facility. Assess current level of compliance with – and corrective actions regarding – applicable US regulatory requirements for licensure,
Major, World-wide Infectious Disease Foundation
Assess capabilities and needs for various clinical programs and manufacturing facilities related to producing treatments for key developing-world diseases.
State of Texas’ University ResearchSystem
Regulatory support leading to the filing of an Investigation New Drug (IND) application with the FDA for a phase 1 clinical trial of a virus-based treatment for Cystic Fibrosis.
National Institutes of Health’s Grand Program in Global Health
Pre-clinical and clinical development support for medical investigators, to get promising research to human studies.
St. Jude Children’s Research HospitalDetermine appropriate levels of quality systems for various tissue-based therapies, and whether they adhere to FDA mandates.
Pan-Provincial Vaccine Enterprise/University of British Columbia
Product development support for initiatives involving several potentially pandemic diseases.
Consulting Areas: AUDITS AND INSPECTIONS
• Inspection of manufacturing facilities • Audit of system/process validation studies • Evaluation of company Quality System • Evaluation of company procedures • Good Laboratory Practices (GLP) Audits • Quality Systems Audits
CLINICAL DEVELOPMENT• Assistance in responding to questions
from FDA:» Center for Biologics Evaluation and Research (CBER)» Center for Drug Evaluation and Research (CDER)» Center for Devices and Radiological Health (CDRH)
• Biostatistics • 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
PHARMACOLOGY/TOXICOLOGY• Program planning to support clinical
product development• Writing of the pharm/tox section of INDs,
BLAs, NDAs • Review and interpretation of toxicology
study data with respect to potential risk• Participation in due diligence programs for
perspective new products• GLP audits• Development of comparability protocol• Writing of product label• Recommendation/interface with toxicology
Contract Research Organizations (CROs)• Participation in FDA meetings
BCG has Extensive, Comprehensive Consulting Expertise
BCG has Extensive, Comprehensive Consulting Expertise
PROCESS VALIDATION• Evaluate process validation status• Identifying critical process parameters and
designing appropriate validation studies • Apply statistical methods to the evaluation
of process validation data • Assist in preparing process validation
related submissions to CBER/CDER/CDRH • Validation Master Plan• Facility Validation
o Equipment Validationo Utilities Validationo Cleaning Validation
PRODUCT DEVELOPMENT• A "roadmap" for your product's
development• A concise, product-focused, strategic
document laying out the path to market approval or licensure
• A detailed analysis of your product status and developmental requirements, including the four primary aspects of product development:
• Manufacturing
• Preclinical• Regulatory• Clinical Development• An integrated stand-alone document tying
all the four main areas of product development with budgets, tasks, and timelines through Phase 1 or beyond
Why Develop a Product Development Plan?• Planning is crucial at every stage of
development, particularly at the outset• Provides a concise detailed analysis of
your product and the roadmap to market• Clearly states developmental objectives
and crucial milestones• Presents a single (or multiple, if desired)
focused regulatory strategy for presenting your product to the FDA
• Presents strategies for dealing with potential roadblocks and hurdles in the product development process
• Lays out accurate and realistic budgets and timelines through clinical development
BCG has Extensive, Comprehensive Consulting Expertise
PROGRAM MANAGEMENT• Prepare Product Development Plan• Prepare Project Timeline• Provide oversight and ongoing quality
review and adjustment of project• Identify external contractors• Pre-clinical, Clinical, and CMC coordination• Central liaison with contractors and
sponsor• Monitoring of project resources, tasks
and timeline• Preparation and management of budget• Provide project updates/reports
REGULATORY SUPPORT• Pre-IND and IND support• BLA/NDA support• Device support• Electronic submissions• Request for Orphan Drug designation• Preparation of Drug Master Files
TRAINING PROGRAMS• Develop specific seminars and workshops• BCG has offered courses such as:
o "The Biologics Approval Process”o “Systems Approach to Process
Validation”o “Clinical Study Documents –
Regulatory Requirements and Review.”
U.S. AGENT• U.S. Agent for filing INDs• U.S. Agent for Foreign Establishment
Registration and Listing
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
Summary
BCG Contacts
James G. Kenimer, [email protected]
Ronald A. MarchesaniHead, West Coast [email protected]
Corporate Office1317 King StreetAlexandria, VA 22134www.bcg-usa.comP: 800-485-0106
703-739-5695
West Coast Office1840 Gateway Drive, Suite 200www.bcg-usa.comP: 650-378-1303