2. The Long Road to a New
Medicine...
NDA/MMA
Clinical Data
Analysis
Full Development Registration
Studies in 100‐300
Candidate Medicine Tested in
Patients (Phase II)
3‐10,000 Patients (Phase III)
Large Amounts of
Candidate Medicine Extensive
Studies in Healthy Synthesized Safety
Volunteers (Phase I) Studies
Formulations
Exploratory Development Developed Candidate
Early Safety
Studies
Project Team Synthesis
Screening
and Plans of Compounds
Discovery
5. 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
6. Expertise
development/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
8. Development plans compared…
Program I (1996) Program II (2002) Program III (2008)
4 phase I trials 6 phase I trials 2 phase I trials
1 phase II trial 0 phase II trials 1 phase II trial
2 phase III trials 2 phase III trials
2 open label trials
development duration: development duration: development duration:
Est. 5‐6 yrs Est. 4 yrs Est. 2 yrs
Est. budget: Est. budget: Budget:
105 M 60 M 15 M
9. Organisation
CEO
Development management board
Protocol review committee
Core development
team
Sub teams
Collaborative teams
….
10. Organisation II
Consultant
Consultant
Very limited
in‐house staff
CRO CRO CRO CRO CRO
Consultant
Consultant
11. Organisation III
BOD/Investors
Legal counsel
SMO CRO MT SAB
administration
PROJECT TEAM
12. The project team...
Who need to be in?
Management/Planning
Clinical operations
Non-clinical/Toxicology
CMC
Regulatory affairs
15. 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
16. The regulatory process
NCE no/yes
BE/hybrid
Full package
application
BE study Hybrid study
development development
implications & implications &
design design