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Biopharmaceutical Innovation:
The Complexity of The R&D Process
WIPO IGC 21
Geneva, 17 April 2012
Markus Boehringer, Roche
Many fascinating projects –
Pegasys
Xeloda
medicalinnovation
idea
improved healthcarebetter quality of life
many fascinating projects –
Pegasys
Xeloda
medicalinnovation
idea
improved healthcarebetter quality of life
some bioactive compound
some testing formulation
a layman’s perspective
many fascinating projects –
Pegasys
Xeloda
medicalinnovation
idea
the reality is different
highly complex multidisciplinary R&D
frontier science & high-technology
many fascinating projects –
Pegasys
Xeloda
medicalinnovation
idea
highly complex multidisciplinary R&D
frontier science & high-technology
high cost
long-term
high risk
the reality is different
STOP
STOP
STOP
STOP
STOP
many fascinating projects –
Pegasys
Xeloda
medicalinnovation
idea
but only a few are successful
STOP
STOP
STOP
STOP
STOP
project
lead molecule
drug candidate
prep for
clin tests
clin development:
Phase I Phase II Phase III
R D
medicalinnovation
idea
concept
project
lead molecule
drug candidate
prep for
clin tests
R D
medicalinnovation
idea
US$ 1‘300‘000‘000
cumulated costs / successfully launched drug
sustainability of R&D
Phase I Phase II Phase III
clin development:
concept
Pharma R&D metrics of success
TargetAssess
LeadIdent
LeadOpt
EIH Enable
PhaseI Reg
PhaseIII
PhaseII to Market
New MedProposal
Research clin. development
60
40
25
1510
4.5 2 1.5 1
ca 100-300 people
needed in research
to produce
1 clin.cand. / year
10 ideas
1 project
0.071
safety efficacy
>2 4 1 7time years
>10 years of patent life expired
� A highly networked multi-disciplinary environment!
Drug Discovery Todaygenetics
modeling
pharmaco-genomicsmol.design
toxicogenomics
behaviorpharmacy
biotechnology
chem.inform.
genomics
proteomics
biostructure
measuring tech.
physics
chemistry
pharma-cologybiology
biophysics
phys.chem.
biochemistry
analytics
informatics
comput.chem.
bioinformaticstoxicology
pk/pd
metabolism
� A multitude of cost intensive technologies!
Drug Discovery Today
in vivo
cell biology
histology
mol.bio
proteinbiochem
µ-spotting
454
genomics
proteomics
biotechnologyprotein expression
proteinX-ray
uHTS
geneexpression
profiles
imagingMRI & fMRI
imagingCLSM
bio-analyticsMS
virtuallibraries
sRCD
CAMM
chem WS
SPR AUC
protein-ligand
Additional Complexity: Multidimensional OptimizationTarget
Assessment
Lead
Identification
Lead
OptimizationPhase 0 Phase I
Database
Synthesis
Assay
SAR
DesignAssay
SAR
Assay
SAR
Assay
SAR
Assay
SAR
Assay
SAR
Why so complex?Why these high costs?
• “Low hanging fruits are gone” – technologies, translational approaches to overcome
• Increasing demands:
e.g. T2D: need to demonstrate not just glucose lowering, but benefit on mortality and morbidity. � longer studies, more patients, higher costs
• Natural products:
– Scientifically of value– Complex optimization required!!
(oral bioavailability, PK, selectivity, …)– Complex synthesis!!– Reliability of supply
N
OOH
OH
NH2OH
OH
S
O
O
ONH
O
NH
NH
O
COOHcyclothialidine
Pharmaceutical R&D: Summary
• Less than 1 out of 60 research projects started will deliver a drug to the market.
• Less than 1 out of 10 molecules starting in clinical trials makes it to the market.
• More than 10 years from project start to entering the market.
• Period of patent protection for a drug entering
the market: less than 10 years.
• The costs for bringing 1 new drug to the market: USD 1’300’000’000.
<1/60
<1/10
>10 yrs
<10 yrs
1.3 bil USD
Still many unmet medical needs - complex –
frontier science – high-tech – expensive –
high-risk