Dr. Bertil Lindmark discusses personalized medicine from an industry perspective. He argues that while personalized medicine aims to provide the right prevention and treatment for each patient, applying it to all situations may lead to overly expensive therapies for small patient groups and neglect of broader patient needs. For pharmaceutical companies, the "sweet spot" of personalized medicine is small, requiring a balance between development costs, therapeutic effects, and market size. Effective collaboration is needed between industry and academia to focus efforts on both specific disease areas with high costs and predictability, as well as broader indications where incremental innovation can still be profitable.

1. 1 1
Dr Bertil E. Lindmark, MD, PhD
Global Head of R&D, CSO
Almirall
Personalized medicine:
an industry view
13th June 2014

2. 2
The Philosphy – the concept
The right prevention and
treatment
for the right patient at the right
time
EFPIA: Strategic Research
Agenda for
Innovative Medicines Initiative 2,
2014
The simple version:
The right therapy
at the right dose and for the right
patient
The right prevention and treatment
at the right dose and with the right
assessment of treatment effect and side
effects at the right cost for the right patient
with the right adherence support with the right
level of outcomes
etcetera

3. 3
The concept
Health care resource
Most optimal health
outcome:
• Society
• Individual

4. 4
The conundrum
Care
Medical therapy Complex system of intervention
delivery, patient adaptation, and
support systems and adherence
structures and complex outcomes:
FEV1
Health
Happiness
Revolving HCU
Life expectancy
Self management
Employment
Family stability
Child rearing
Car driving
Accidents
Personal economy

5. 5
Personalization
Phenotypic definition Omics definition
• High degree of prediction of indivuals of meaningfully “like” cohort
• High degree of prediction of likelihood of high value outcomes

6. 6
“Likeness”
Meaningful
likeness of
pathobiology
Meaningful
likeness of
disease
manifestations
and risk
Meaningful
likeness of
disease
drivers
Meaningful
likeness of
therapeutic
response
Meaningful
likeness of
disease
markers
Meaningful
likeness of
markers of
theraputic effect
Meaningful
probability of
designing and
developing a
meaningful
therapy

7. 7
Industry parameters
• Overall cost, time, risk, differentiation, exclusivity
• Derisking possibilities: does phase 1 derisk phase 2, and does phase 2 derisk phase 3?
• Cost and risk including market assess studies (MAPR)
• Competitors (all sorts – medical and non-medical intervention, procedures)
• Mechanistic markers, POC markers and how validated are they vs phase 3 primary
endpoints
• How well is population defined, - is there a simple practical low cost unequivocal test?
• Who will be the customer and the user– patient? Hospital? Specialist Physician? or
GP?
Insurer? Government? Local care system? Who do I sell to? Who decides?
From where can I get the value?
• Business case: capability, marketing capacity, overall investment, population size,
price, MAPR risk, NPV, payback time, IRR, do we have better areas to invest in?
Sweet spot needs careful definition – where all these factors intersect
This is important signal for academic reserach: focus in areas where
Business makes sense! Or not?

8. 8
Optimisation for the few and
disoptimization for the many?
Mass of COPD patients
Price
Size of population
Transformational
highly predictive,
high cost therapy
Mass therapy, low
cost, less precision,
no or maybe
incremental
innovation

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Some conclusions:
• Personalized medicine is fine as a concept
• If the concept is applied for all and everything we may
• Get too expensive therapy for small segments of patients but neglect of the
need of the masses
• Expectation of transformational therapy leading to cynicism and discarding
of incremental innovation
• Industry can support some certain parts of the concept
but the sweet spot is quite small
• Right relationship between development cost, therapeutic effect and market
size
• Effective collaboration between industry and academia need
focussed efforts in COPD both in:
• Partialities of COPD with high risk and high cost and high degree of
treatment predicatibility- concept of Personalized Likeness
• Broader indications where definitions need to be broad enough and
definition of ambition in terms of treatment efficacy well defined, so that
incremental innovation can be achieved and be profitable
• Read outs that permit early derisking are key (fail cheaply)

10. 10
size does not fit all

11. Thank
You!

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