the use of scientific advisory boards an invaluable tool in drug development dr robert miller chief...
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The Use of Scientific Advisory Boards
An Invaluable Tool in Drug Development
Dr Robert Miller
Chief Medical Officer
Fulcrum Pharma Developments
19th Annual EuroMeeting, Vienna 2007
Why
Appropriate advice is the key to successful drug development
Drug Development is:
> Expensive
> Time consuming
> Highly Competitive
> Resource Intensive
> Rewarding
> Dangerous
> Frustrating
Milestone Phase 0Entry into
ManPhase II
Phase III / Pre-
Submission
Lead Compound Selection
Developmental Pathway and Related Activities
Path Target pharmacology
In-vitro models
Drug substance manufacture and
formulation
Initial Target Profile
Output
Preliminary Product Development PlanManufacturing plans
Competitor analysisClinical Development Plan/Regulatory Strategy
Market reviewPeri-approval plan/Life
cycle management
Target Profile, Competitor analysis and Potential market
Corporate needs Budgeting and timelines
Toxicology
DMPK
Preclinical models for pharmacology
Potential therapeutic targets
Translational research and PD assays
Drug scale up
Regulatory considerations
Product appraisal
Target profile refinement
Therapeutic targets
Further preclinical studies
Biomarkers/PD assays
Factors for progress to Phase II
Target profile refinement
Initial therapeutic indications
Phase II Clinical trial design
Critical success factors for Phase III
Phase III strategy
Regulatory Strategy
Business strategy/licensing
Critical review of emerging data
Phase III trial designs
Regulatory issues for submission
Launch strategy
Peri-approval strategy
Publication planning
Where is the Expertise
>Pharmaceutical
Strategy
Trial Design
Logistics
Where is the Expertise
>Physician
Contemporary knowledge
Competitor studies
Clinical relevance
Why External Advice
> Contemporary “real life” information– By Indication– Medical Practise– Feasibility– Critical success factors
> Geographical perspective
> Competitor information
> Potential investigator
What External Advice
> One-on-one– “I know a few famous doctors – I’ll go and ask
them”– “I can take the advice I like best”– “Its cheaper”
> Advisory Boards– “Complicated”– “What if they don’t agree with my plan”– “Expensive”
“One-on-Ones”
What if they provide different advice?
Who do you believe?
Advisory Boardsaka. Focus Groups, Steering Groups
> Can provide all of the suggestions in one meeting
> Can “argue” against one point of view not universally held or not in the interests of the sponsor
> Speak through the moderator rather provide an oration
> Are with their peers so less likely to act as “prima donnas”
> Can provide a consensus if meeting properly handled
Consensus is Best
Advisory Boards
> The Make-up– Multinational and Multidisciplinary Composition– Current Clinical Practices– Trial design, relevant outcomes, target populations– Awareness of Competition / Treatment Trends
> The Conduct– Clear brief– Clear moderation and direction of the discussions– Consensus on path forward– Not a “rubber stamp”
> Potential for Advisers to be product champions
Path
Output
Corporate needs
Target pharmacology
In-vitro models
Drug substance manufacture and
formulation
Initial Target Profile
Toxicology
DMPK
Preclinical models for pharmacology
Potential therapeutic targets
Translational research and PD assays
Drug scale up
Regulatory considerations
Product appraisal
Target profile refinement
Therapeutic targets
Further preclinical studies
Biomarkers/PD assays
Factors for progress to Phase II
Target profile refinement
Initial therapeutic indications
Phase II Clinical trial design
Critical success factors for Phase III
Phase III strategy
Regulatory Strategy
Business strategy/licensing
Critical review of emerging data
Phase III trial designs
Regulatory issues for submission
Launch strategy
Peri-approval strategy
Publication planning
Preliminary Product Development PlanManufacturing plans
Competitor analysisClinical Development Plan/Regulatory Strategy
Market reviewPeri-approval plan/Life
cycle management
Target Profile, Competitor analysis and Potential market
Budgeting and timelines
Milestone Phase 0Entry into
ManPhase II
Phase III / Pre-
Submission
Lead Compound Selection
Scientific AB
Target pharmacology
In-vitro models
Toxicology
DMPK
Preclinical models for pharmacology
Potential therapeutic targets
Translational research and PD assays
Scientific/Clinical AB
Target profile refinement
Initial therapeutic indicationsTherapeutic targets
Further preclinical studies
Biomarkers/PD assays
Factors for progress to Phase II
Clinical AB
Phase II Clinical trial design
Critical success factors for Phase III
Phase III strategy
Regulatory Strategy
Business strategy/licensing
Critical review of emerging data
Phase III trial designs
Regulatory issues for submission
Peri-approval AB
Launch strategy
Peri-approval strategy
Publication planning
When and What Advisory Boards
Why Do Advisory Boards Work
> They provide: – a forum for the discussing development – reality check for plans and potential studies
> They can:– identify issues and provide solutions– validate the development programme – provide product championship
> They are critical because……
Planning is the Key
Not everyone has the same ideas!
Development Strategy Planning
Target Profile/MIRS
Ongoing clinical trial dataCurrent Information
Target Profile
> Label Claims:– Indications for use
– Dosage, route and regimen
– Efficacy and safety
– Product quality
– Risk:benefit
> Marketing Profile:– Features and benefits
– Comparison to alternative treatments
– Pharmacoeconomics
> Commercial Targets:– Time to market
– Licensing plans
– Financial
– COG
– NPV
– Sales
– Market share
– Licensing value etc
– Patent life
Tools - MIRS
Designs/data
Completed/ongoing studies
Publications
GuidelinesPrecedents
New Studies
Refutation
Scientific precedent
Re-analysis
Expert opinion
Issues
ChallengesRisks
Comparisons
ConflictingResults
Questions
Claims
Features and benefits
Advantages
Interpretations
Conclusions
SupportResponse/Rationale
IssuesMessages
Development Strategy Planning
Advisory Board(s)
Target Profile/MIRS
Ongoing clinical trial dataCurrent Information
Development Strategy Planning
Advisory Board(s)
Target Profile/MIRS
Ongoing clinical trial data
Gap Analysis
Current Information
Investigator input
Strategy Plan
Protocols
Life Cycle Planning
Advisory Board(s)
Target Profile/MIRS
Ongoing clinical trial data
Gap Analysis
Current Information
Investigator input
Life-cycle team review
Life Cycle Strategy Plan
Summary
> Advisory Boards are an excellent way of gauging opinion> They avoid the difficulties of one-to-one meetings where
individual experts' opinions differ> They allow a consensus approach to development> They can be tailored according to the stage of
development, the nature of the product and the breadth of discussions required
> Independent moderation of Boards is an excellent way of getting a unbiased advice
> The use of these boards should be factored into developments plans whether they be early or life-cycle
Robert M MillerFulcrum Pharma DevelopmentsHemel HempsteadUnited Kingdom
Tel: +44 870 710 4360+1 919 226 1440 ext 325
e-mail: [email protected]
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