Essentials for rapid and cost‐effective product developmentDr. Ronald van der GeestUMCN, June 2012
The Long Road to a New    Medicine...                                                            NDA/MMA                  ...
Creating added value 3 Dimensions: time, cost & risk
How to get there?      The essentials:            IPCapital           Expertise
Capital• Sufficient funding  – Simple rule of thumb: 2‐3M projects get into     trouble, 6‐8M projects don’t• Based on a t...
Expertise       development/business plan• Sufficient detail for all aspects tox, CMC,   clinical, regulatory, etc.• P&R… ...
Expertisedevelopment/business plan
Development plans compared…Program I (1996)        Program II (2002)       Program III (2008)4 phase I trials        6 pha...
Organisation                            CEO                Development management boardProtocol review committee          ...
Organisation II                                             Consultant                       ConsultantVery  limitedin‐hou...
Organisation III                       BOD/Investors                                       Legal counselSMO   CRO         ...
The project team...       Who need to be in?          Management/Planning            Clinical operations          Non-clin...
The project team...• Core team staffing as early as possible   ‐ completeness  ‐ availability  ‐ continuity within the tea...
The project team…• High level of ownership• Fast and efficient• Focus on high‐risk development• Biggest risks:  ‐ running ...
Getting started• Sorting out the regulatory process• Scientific advice  – “We are going to have a chat with FDA”  – “Rubbi...
The regulatory process                                  NCE no/yes                    BE/hybrid                           ...
The regulatory processScientific advice – 2 example questions:“It is the sponsor’s position that the completed nonclinical...
PKPD modeling implementation  4-way cross-over, double blind, placebo controlled                        2.5 mg IV         ...
PKPD modeling implementation
Clinical Utility Index Balancing Efficacy  and Safety in Drug Development Decision Making
Contact detailsRonald van der Geest, PhDPartner & General Manager+31 6 523 901 30Ronald@3d‐pxc.comMaidstone 48A, 5026 SK T...
The Long Road to a New Medicine...        Questions?
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3D Farm Exchange

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

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3D Farm Exchange

  1. 1. Essentials for rapid and cost‐effective product developmentDr. Ronald van der GeestUMCN, June 2012
  2. 2. The Long Road to a New  Medicine... NDA/MMA Clinical Data  AnalysisFull 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. 3. Creating added value 3 Dimensions: time, cost & risk
  4. 4. How to get there? The essentials: IPCapital Expertise
  5. 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. 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. 7. Expertisedevelopment/business plan
  8. 8. Development plans compared…Program I (1996) Program II (2002) Program III (2008)4 phase I trials 6 phase I trials 2 phase I trials1 phase II trial 0 phase II trials 1 phase II trial2 phase III trials 2 phase III trials2 open label trialsdevelopment duration: development duration: development duration:Est. 5‐6 yrs Est. 4 yrs Est. 2 yrsEst. budget: Est. budget: Budget:105 M 60 M 15 M
  9. 9. Organisation CEO Development management boardProtocol review committee Core development team Sub teams Collaborative teams ….
  10. 10. Organisation II Consultant ConsultantVery  limitedin‐house staff CRO CRO CRO CRO CRO Consultant Consultant
  11. 11. Organisation III BOD/Investors Legal counselSMO CRO MT SAB administration PROJECT TEAM
  12. 12. The project team... Who need to be in? Management/Planning Clinical operations Non-clinical/Toxicology CMC Regulatory affairs
  13. 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. 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. 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. 16. The regulatory process NCE no/yes BE/hybrid  Full package application BE study  Hybrid study  development  development  implications &   implications &  design design
  17. 17. The regulatory processScientific 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. 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. 19. PKPD modeling implementation
  20. 20. Clinical Utility Index Balancing Efficacy  and Safety in Drug Development Decision Making
  21. 21. Contact detailsRonald van der Geest, PhDPartner & General Manager+31 6 523 901 30Ronald@3d‐pxc.comMaidstone 48A, 5026 SK Tilburg  Molenweg 50, 5349 AC OssTel: +31 (0)13 534 8272 Tel: +31 (0)6 55 71 86 66Website:www.3d‐pxc.com Website:www.3d‐pxc.com
  22. 22. The Long Road to a New Medicine... Questions?

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