3D Farm Exchange Essentials for rapid and cost‐effective product development Dr. Ronald van der Geest

1. Essentials for rapid and cost‐effective
product development
Dr. Ronald van der Geest
UMCN, June 2012

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

3. Creating added value
3 Dimensions: time, cost & risk

4. How to get there?
The essentials:
IP
Capital Expertise

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

7. Expertise
development/business plan

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

13. 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

14. 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

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

17. 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?”

18. PKPD modeling implementation
4-way cross-over, double blind, placebo controlled
2.5 mg IV
+ placebo
2.5 mg + 16 5.0 mg +
placebo placebo
IV subjects IV
Placebo +
placebo
IV

19. PKPD modeling implementation

20. Clinical Utility Index
Balancing Efficacy and Safety in Drug Development
Decision Making

21. Contact details
Ronald van der Geest, PhD
Partner & General Manager
+31 6 523 901 30
Ronald@3d‐pxc.com
Maidstone 48A, 5026 SK Tilburg Molenweg 50, 5349 AC Oss
Tel: +31 (0)13 534 8272 Tel: +31 (0)6 55 71 86 66
Website:www.3d‐pxc.com Website:www.3d‐pxc.com

22. The Long Road to a New
Medicine...
Questions?