3d farm exchange
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UMCN, June 2012
Essentials for rapid and cost‐effective product developmentDr. Ronald van der Geest
The Long Road to a New Medicine...
Registration
Discovery
NDA/MMA
Full Development
Exploratory Development
Project Team and Plans
Synthesis of Compounds Screening
Early Safety Studies
Formulations Developed
Studies in Healthy Volunteers (Phase I)
Studies in 100‐300 Patients (Phase II)
Candidate Medicine Tested in 3‐10,000 Patients (Phase III)
Clinical Data Analysis
Large Amounts of Candidate Medicine
SynthesizedExtensive Safety Studies
Candidate
Creating added value
3 Dimensions: time, cost & risk
How to get there?
The essentials:
IP
ExpertiseCapital
Capital
• Sufficient funding– Simple rule of thumb: 2‐3M projects get into trouble, 6‐8M projects don’t
• Based on a thought‐trough development plan– Include scenario’s
– Plan for the best
– Ask funding for the worst
– It’s about managing expectations of shareholders
Expertisedevelopment/business plan
• Sufficient detail for all aspects tox, CMC, clinical, regulatory, etc.
• P&R… as much as possible
• CRO‐shopping: – We have seen anything in the range of 70k to 1.5M
Expertisedevelopment/business plan
Development plans compared…
Program I (1996) Program II (2002) Program III (2008)
4 phase I trials1 phase II trial2 phase III trials
2 open label trials
6 phase I trials0 phase II trials2 phase III trials
2 phase I trials1 phase II trial
development duration:
Est. 5‐6 yrs
development duration:
Est. 4 yrs
development duration:
Est. 2 yrs
Est. budget:
105 M
Est. budget:
60 M
Budget:
15 M
Organisation
CEO
Development management board
Core developmentteam
Sub teams
Collaborative teams
….
Protocol review committee
Organisation II
Very limitedin‐house staff
CRO CROCROCRO CRO
Consultant
Consultant
Consultant
Consultant
Organisation III
BOD/Investors
MT SAB
Legal counsel
administration
CROSMO
PROJECT TEAM
The project team...
Who need to be in?
Management/Planning
Clinical operations
Non-clinical/Toxicology
CMC
Regulatory affairs
The project team...
• Core team staffing as early as possible
‐ completeness
‐ availability
‐ continuity within the team
‐ full alignment with the company goals
‐ allow flexibility within the team
The project team…
• High level of ownership
• Fast and efficient
• Focus on high‐risk development
• Biggest risks:‐ running out of money‐ lack skills / over‐confident‐ too limited number of projects / risk of failure
Getting started
• Sorting out the regulatory process
• Scientific advice– “We are going to have a chat with FDA”
– “Rubbish‐in – rubbish‐out” principle
– Based on a solid plan
– Role play
– Negotiate
– Regulators are not the experts
The regulatory process
NCE no/yes
BE/hybrid application
BE study development implications &
design
Hybrid study development implications &
design
Full package
The regulatory process
Scientific advice – 2 example questions:
“It is the sponsor’s position that the completed nonclinical safety studies are adequate to support a MAA in the proposed indication. Does the Agency concur with the sponsor’s position?”
“Does the Agency agree that the proposed program of clinical pharmacology studies is adequate and is sufficient to support a MAA in the targeted indication?”
PKPD modeling implementation
16 subjects
2.5 mg IV + placebo
5.0 mg + placebo
IV
Placebo + placebo
IV
2.5 mg + placebo
IV
4-way cross-over, double blind, placebo controlled
PKPD modeling implementation
Clinical Utility Index Balancing Efficacy and Safety in Drug Development Decision Making
Maidstone 48A, 5026 SK Tilburg Tel: +31 (0)13 534 8272Website:www.3d‐pxc.com
Contact details
Ronald van der Geest, PhDPartner & General Manager+31 6 523 901 30Ronald@3d‐pxc.com
Molenweg 50, 5349 AC OssTel: +31 (0)6 55 71 86 66Website:www.3d‐pxc.com
The Long Road to a New Medicine...
Questions?
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