Descripton of current basic needs and challenges of precision medicine with some concepts. Special foccus on pricing & reimbursement.

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Precision medicine
From population-based to precision-based approach
• Definition
• Need for individual therapies
• Current Challenges
• Holistic concept
• Clinical pathway
• Economic impact
Kurt R. Mueller, PhD MSc BBA
PM-concepts-TYE-es

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Definition of precision medicine
“Precision Medicine (PM) is an emerging approach for
disease treatment and prevention that takes into account
individual variability in genes, environment, and lifestyle for
each person” (Genetics Home Reference)

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Health
Care
Genetic- and
Life Style
Conditions Risk
Assessment
Prevention
Monitoring
Disease or
Accident
Diagnosis
Primary
Care
Medical
Specialist
Hospital
Rehabilitation
Home Care
Nursing
Home
Palliative
Care
.
.
.
.
.
Genetics start before any health care interventions
Genetic disposition – risk- assessment - self responsibility
• Health Care – Goals
o From neonatology until end of life
o Reduce morbidity & mortality
o Prolong disability free life expectancy
o Reduce burden of disease
o Maintain or increase survival
• Health Care – Challenges
o Growth of health care costs for the society
and individuals
o Define value & reward for innovations
o Equal and fast access to new and innovative
health care interventions
o Fragmentations and variability of health
care services
o Disinvestment of low value care and
reduction of inefficiencies
Intervention & care Genetics & preventions

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Treatment should get specific to individuals
37.6%
22.1%
19.1%
11.4%
9.8%
Lifestyle Genetics
Socio-economics Health Care
Einvironment
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
Responder Non-Responder
Factors impacting health Percentage of Non-Responders
Key drivers for health are lifestyle and
genetics and not health care provision.
Source: Marty F and Salvi M. Wirtschaft bringt Gesundheit,
economiesuisse, Dossier Politik 08/2018, 2018
We have to move from a population- to
a precision- based approach
Source: Sandra L. Kweder, MD, Deputy Director,
Europe Office US-FDA
https://www.ema.europa.eu/en/documents/presentation/presentation-
us-food-drug-administration-fda-precision-medicine-initiative-patients-
consumers_en.pdf

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Current drug treatment challenges
1. One size fits all has hardly be an effective strategy in different industries *
2. Difficulties to separate responder from non-responder
3. Many diseases remain unclear: «idiopathic & essential»
4. Hard to figure out which patient will suffer from ADR
5. According to the WHO around 20-40% health care resources are wasted
to inefficiencies: treating non-responder is one of the elements
6. The value of prognostic factors from subgroup analysis is limited to
predict treatment response
7. Prognostic factors identified by subgroup analysis are insufficient to
predict response
Source: * https://www.statnews.com/2018/05/23/personalized-medicine-future-cost-value/

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Reimbursement is the key challenge
We should manage precision medicine as a holistic concept (next
slide) where savings are coming mainly from prevention in order to
finance targeted therapies (pharmaLevers).
“With the rising importance and budget impact of precision
medicine, the current pricing and coverage approach will
need to be redefined” (1)
“Stakeholders indicate the paramount challenge to Precision
Medicine is reimbursement, not scientific and technical
hurdles” (2)
(1) Degtiar I. A review of international coverage and pricing strategies for personalized medicine and orphan
drugs. Health Policy (New York) [Internet]. 2017;121(12):1240–8. Available from:
https://doi.org/10.1016/j.healthpol.2017.09.005
(2) Pothier KC. Personalizing Precision Medicine: A Global Voyage from Vision to Reality. 1st Edition.
Hoboken, NJ 07030, USA: John Wiley & Sons, Inc; 2017. 261 p

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The holistic concept of precision medicine

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Clinical pathway – process
Ideally a patient will monitor or track his health and disease status
over time with Health Care Apps and adjust his activities and
behaviors as agreed with his physician who continuously stores such
data in the EHR.
At a certain point patient's clinical and lab data will be compared with
health, disease and multi-omics data from biobanks.
However, this is seldom a one-time assessment as clinical and
laboratory patterns frequently change over time.
Altogether, AI will continuously calculate and define the most
appropriate clinical pathway for this individual patient.
The success of treatment will be monitored by RWE which will
determine the level of dynamic P&R.
The process is illustrated in the following slide

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Genomics & AI will define patients clinical pathway
Fore more information:
https://www.linkedin.com/pulse/precision-medicine-key-challenges-kurt-r-m%C3%BCller/

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Today, we simple do not know the economic
consequences of precision medicine
• Higher costs
o More patients will be diagnosed
o More patients get costly
PM-therapies which will be often combined
• Lower costs
o More untreated patients to avoid
unnecessary treatment
o Avoiding unsuccessful treatment
o Less ADR and hospitalization due to
pharmacogenetics therapies
• Fragmented care
o Genomics based treatment algorithm will
lead to many different clinical pathways
with different benefits and costs
o Too small population to compare the
economic consequences of different clinical
pathways
or
?
?

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Newsletter – Precision Medicine
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