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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 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]