What External Advice - InternetVIZ newsletter

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Transcript What External Advice - InternetVIZ newsletter

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
Appropriate advice is the key to successful
drug development
Why
Drug Development is:
> Time consuming
> Highly Competitive
> Resource Intensive
> Dangerous
> Rewarding
> Frustrating
> Expensive
Developmental Pathway and Related Activities
Milestone
Path
Lead
Compound
Selection
Entry into
Man
Phase 0
Toxicology
Product appraisal
Target
pharmacology
DMPK
In-vitro models
Preclinical models for
pharmacology
Target profile
refinement
Therapeutic targets
Drug substance
manufacture and
formulation
Potential therapeutic
targets
Further preclinical
studies
Initial Target Profile
Translational research
and PD assays
Biomarkers/PD
assays
Drug scale up
Regulatory
considerations
Preliminary Product Development Plan
Manufacturing plans
Factors for
progress to Phase II
Phase II
Target profile
refinement
Critical review of
emerging data
Initial therapeutic
indications
Phase III trial
designs
Phase II Clinical trial
design
Regulatory issues
for submission
Critical success
factors for Phase III
Launch strategy
Phase III strategy
Peri-approval
strategy
Regulatory Strategy
Publication planning
Business
strategy/licensing
Competitor analysis
Clinical Development Plan/Regulatory Strategy
Output
Target Profile, Competitor analysis and Potential market
Corporate needs
Phase III /
PreSubmission
Budgeting and timelines
Market review
Peri-approval plan/Life
cycle management
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 Boards
aka. 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
When and What Advisory Boards
Milestone
Path
Lead
Compound
Selection
Entry into
Man
Phase 0
Phase II
Phase III /
PreSubmission
Target profile
refinement
Critical review of
emerging data
Therapeutic
Therapeutic targets
targets
Initial therapeutic
indications
Phase III trial
designs
Potential therapeutic
targets
Further
Further preclinical
preclinical
studies
studies
Phase II Clinical trial
design
Regulatory issues
for submission
Translational research
and PD assays
Biomarkers/PD
Biomarkers/PD
assays
assays
Critical success
factors for Phase III
Launch strategy
Factors
Factors for
for
progress
progress to
to Phase
Phase IIII
Phase III strategy
Peri-approval
strategy
Regulatory Strategy
Publication planning
Toxicology
Product appraisal
Target
pharmacology
DMPK
In-vitro models
Preclinical models for
pharmacology
Target profile
refinement
Drug substance
manufacture and
formulation
Initial Target Profile
Drug scale up
Regulatory
considerations
Preliminary Product Development Plan
Manufacturing plans
Business
strategy/licensing
Competitor analysis
Clinical Development Plan/Regulatory Strategy
Market review
Peri-approval plan/Life
cycle management
Output
Target Profile, Competitor analysis and Potential market
Corporate needs
Budgeting and timelines
Scientific AB
Scientific/Clinical AB
Clinical AB
Peri-approval AB
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
Current Information
Ongoing clinical trial data
Target Profile/MIRS
Target Profile
>
>
Label Claims:
–
–
–
–
–
Indications for use
Dosage, route and regimen
Efficacy and safety
Product quality
Risk:benefit
Commercial Targets:
–
–
–
–
>
Time to market
Licensing plans
Financial
– COG
– NPV
– Sales
– Market share
– Licensing value etc
Patent life
Marketing Profile:
–
–
–
Features and benefits
Comparison to alternative treatments
Pharmacoeconomics
Tools - MIRS
Messages
Issues
Response/
Rationale
Support
Claims
Issues
New Studies
Designs/data
Features and
benefits
Challenges
Risks
Refutation
Completed/
ongoing
studies
Advantages
Comparisons
Scientific
precedent
Publications
Interpretations
Conflicting
Results
Conclusions
Questions
Re-analysis
Expert
opinion
Guidelines
Precedents
Development Strategy Planning
Current Information
Ongoing clinical trial data
Target Profile/MIRS
Advisory
Board(s)
Development Strategy Planning
Current Information
Ongoing clinical trial data
Target Profile/MIRS
Gap Analysis
Investigator
input
Strategy Plan
Protocols
Advisory
Board(s)
Life Cycle Planning
Current Information
Ongoing clinical trial data
Target Profile/MIRS
Gap Analysis
Life Cycle Strategy Plan
Life-cycle team review
Investigator
input
Advisory
Board(s)
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
Contact
Robert M Miller
Fulcrum Pharma Developments
Hemel Hempstead
United Kingdom
Tel:
+44 870 710 4360
+1 919 226 1440 ext 325
e-mail: [email protected]