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Precision Medicine Foundations for a new ecosystem to deliver future health care services
Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease Committee on a
Framework for Development a New Taxonomy of Disease; National Research Councile
http://www.ucsf.edu/sites/default/files/documents/new-taxonomy.pdf
Preparing for Precision Medicine World Economic Forum November 2012
http://www3.weforum.org/docs/WEF_GAC_PreparingPrecisionMedicine_2012.pdf
Precision Medicine © Dr. Thomas Wilckens, InnVentis
Precision Medicine: an attempt of a definition
Precision Medicine is an approach:
to discover and develop medicines, vaccines or routes of intervention
(behavior, nutrition, etc.) that enable disease prevention and deliver
superior therapeutic outcomes for patients, by integrating “Big DATA”,
clinical, molecular (multi-omics including epigenetics), environmental and
behavioral information to understand the biological basis of disease.
This effort leads to better selection of disease targets and identification of
patient populations that demonstrate improved clinical outcomes to novel
preventive and therapeutic approaches.
In order to achieve this goal novel data architectures/analytics and
mobile ICT technologies are instrumental to enable implementation
and delivery of Precision Medicine 24/7 anywhere anytime worldwide
2 Precision Medicine © Dr. Thomas Wilckens
3
The changing landscape of R&D and beyond: Big-Data analytics will disrupt current R&D processes requiring new paradigms and data architectures
Erich Schadt; The changing privacy landscape in the era of big data
Mol Syst Biol. 2012; 8: 612.
The route to precision medicine (adopted from Clayton Christensen):
mobile information,-communication technologies (mICT), new mobile diagnostics (mDx) & Big Data
as game changers of existing products/services and business models
Symptom
-based
(Trial &
Error)
Algorithm
-based Pattern-
based
Intuition
Medicine Precision
Medicine
Evidence-
based
Medicine
Yesterday & emerging markets Today
Tomorrow
With the advent of mobile ICT enabled solutions, emerging markets will
leapfrog western world today‘s medicine and create new business models
Available
data sets
Action
*omics mobile ICT
Big-data
5
Integrated health care 24/7 anywhere: A vision
Successful commercial strategies require the creation of a new ecosystem to deliver future services
Precision Medicine © Dr. Thomas Wilckens
Key elements:
• Fast real-time access to databases
• Algorithms
• Reliable and validated reference databases on customers
requirements & health status
• Interface to enable new „sticky services“ (third parties)
Convergence of diagnostics with Big-Data and algorithms:
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Genome
25.000 genes
Proteome
1 million
Molecular
imaging
Metabolome
> 6000 SM
Molecular
fingerprint
Multi-omics individual molecular fingerprints Algorithms &
intelligent, real time
assistance
Precision Medicine © Dr. Thomas Wilckens
other
omics
Precision
Medicine
Other
Big-
DATA
जेम्स, कृपया फिर से ईधन देना आपके रक्त में शकक रा कम है
60
mobile ICT enables Precision Medicine 24/7 anywhere: Application of rules, algorithms and reference databases : diabetes care as a role model
Rules &
Algorithms
Algorithms & Avatar MDs: Advise insulin quantity and nutrition from historic patient data, current blood metabolic status, physical activity etc.
Precision Medicine © Dr. Thomas Wilckens
Create Application
POC for longitudinal, intra-individual multi-omics profiling’s value proposition Exponential value growth by the generation of content from data
8
The biggest value growth in health care resides in the generation of proprietary “biomarkers”,
actionable information and a knowledge base for algorithm-based Precision Medicine
Personal omics profile (iPOP), an analysis that combines
genomic, proteomic, metabolomic, and autoantibody profiles
from a single individual can be used to interpret healthy and
diseased states by connecting genomic information with
additional dynamic omics activity.
Cell 148 , 1293–1307, March 16, 2012
Precision Medicine © Dr. Thomas Wilckens
A paradigm shift in R&D: Intraindividual multi-omics analysis replaces population based medicine
9
Intra-individual, long-term Big-DATA analytics generates
new insight in human biology and pathologies
Precision Medicine © Dr. Thomas Wilckens
Multi-omics and Big Data analysis: Biological dynamics and complexities require new data architectures and analytics
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Data analytics challenges:
• up to 100 terabyte per patient measurement
(i.e. 50 samples over time)
• nonlinear dynamics
Precision Medicine © Dr. Thomas Wilckens
11
Real-time analytics requires new data architecture and analytics capacities Complex biology follows chaos rather linear mathematics
ICT Challenges:
• in-memory reference databases
• Safe, secure encrypted data
transmission, storage & back-ups
• Patient lifetime data storage
• New algorithms including
quality control
• …
adopted from Mias and Snyder,
Clinical Pharmacology & Therapeutics 2013, 93/1: 29-32
Precision Medicine © Dr. Thomas Wilckens
Dada Mining, Expert System, Therapy, Context
matching
Data management, Personal health records
Monitoring, Analytics, distributed, secure data
storage and „HealthSecurity“
Mobile und portable vital data measurement,
Preprocessig and control
CASE-specific: User interface, Data representation, Alarm,
Parameters
Data transport, near field communication, mobil and fix
Non invarsive Sensoric
Lab on a chip Technologies
Pilo
t im
ple
men
tati
on
in s
elec
ted
tar
get
mar
ktes
The integrated diagnostics/monitoring technology & value map Structure, competences, plattforms, up-scaling & value added services in a lean pilot project
Ho
rizo
nta
l cro
ss s
ectio
na
l fu
nctio
ns
User- and administration interface
Value added services (VAS) and
APPs , Algorithm-based Medicine
Central platform functionalities with general application
features; i.e.:
• Secure data collection, storage, managment and
extraction/analysis
• Monitoring and alarm
• Personalised health records
• Expertsystems for datamining, epidemiologic
analysis, therapeutic decisions etc.
• Datatransmission via operator
• Care management
Target case specific diagnostics/sensor and actuator
at the frontend
• Single point measurement
• Repeated measurement (chronic monitoring)
• Continuous measurement and monitoring Frontend tech
(non) invasive
sensors
Still missing: IT-platforms, validated front end data, reference databases,
algorithms and business models Precision Medicine © Dr. Thomas Wilckens
The route to precision medicine What pharma and nutraceuticals need:
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Biomarkers for patient stratification
To increase efficacy and/or safety
To increase success rate in drug/product development
To increase clinical utility / value of marketed drugs i.e. anti-TNF
To rescue failed drugs
Integrated services for fast decision making
Biomarkers for monitoring of clinical efficacy
Novel therapeutic targets/concepts
IT solution to enable Big-Data management & Precision Medicine
*omics insight; what do the data mean; i.e. actionable information
Reliable and valid data to create new products and services
Precision Medicine © Dr. Thomas Wilckens
The route to precision medicine What the diagnostics companies need:
14
Access to novel, emerging markets
Concepts to secure established markets
Concepts to create value from Big-Data
IT-solutions to enable integrated diagnostics & disease management
Novel concepts on value-added services alongside current business
Reliable and valid data to create new products and services
Precision Medicine © Dr. Thomas Wilckens
The route to precision medicine What the health care industries needs:
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Products and services to limit health care expenditures
Product and services enabling disease prevention
Products and services to compare clinical efficacy of competing concepts
IT-solutions to enable large scale Big data analysis (including costs)
Intelligent diagnostics support systems (data flodding reduction)
Novel concepts on value-added services alongside current business
Reliable and valid data to create new products and services
Precision Medicine © Dr. Thomas Wilckens
New data and standardized processes (SOP) are essential for success: Establishing data bases and algorithms for Precision Medicine requires highest validity and reliability
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Nature 03 April 2013
If a job is worth doing, it is worth
doing twice
Researchers and funding agencies
need to put a premium on ensuring
that results are reproducible
Precision Medicine © Dr. Thomas Wilckens
A lack of
standardized procedures,
well documented
patient collectives
and
reproducible results
is the
key problem in biomarker
and drug discovery
Standards and SOPs on the horizon to secure value today? The case for vertically integrated pilot projects
17
Launch of a Transformative Health Care
Initiative: The National Biomarker
Development Alliance (NBDA) 1/2014
Precision Medicine © Dr. Thomas Wilckens
A sea of standards for omics data: sink or swim? JD Tenenbaum et. al. Am Med Inform Assoc, 09/2013
Problems with scientific research
How science goes wrong
Scientific research has changed the
world. Now it needs to change itself
10/2013
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Implementing Symbiotic Innovation to ensures scientific leadership
Project specific management teams, will orchestrate the virtual R&D process securing quality control
CROs to become part of a value chain and the ROI by participating in the project solving a task
Participation anticipates acceptance of a master-contract a priori, which eliminates endless negotiations
Successful
PoC
Biomarkers
IT Plattform for
Precision medicine
A project’s specific tasks are orchestrated science and technology leaders
Big-data, IT-platform, pattern-recognition, algorithms
Clinical Chemistry
Antibody Analytics
Metabolomics
Genomics
Sequencing
Proteomics
Precision Medicine © Dr. Thomas Wilckens
Other omics
Science Google
Ontologies
Logistics/QM/SOPs
Inflammation
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Clinical
data
Ontologies
Literature
Proteomics
Clinical
chemistry Antibody
analysis
Arthritis
Genomics Metabolomics
Biomarkers
Proprietary reference database
Services
Big-DATA
TRAPS Anti-TNF
1. Patient selection • Disease duration
• Treatment regimen
• Drug
• …
2. Sample • Collection & logistics
• Processing
• Storage
3. Data • Format
• …
4. Data intergration • Validation
• ….
5. Analytics & Algorithms
Implementation in a vertically integrated, scalable pilot project: R&D approaches lack of standardized, integrated SOPs and a coherent analytics architecture
Precision Medicine © Dr. Thomas Wilckens
Other
*omics
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Stratification
Monitoring
Database
Biomarkers
Compagnien Dx
Drugs/Nutraceuticals
Services
InnVentis’ products enable new product/services development Exponential value growth by serving growth poised industries needs
Precision Medicine © Dr. Thomas Wilckens
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InnVentis reduces risks, costs, time and increases the chance for success Successful commercial strategies require sustainable proactive industry engagement in innovating health care
Charles Darwin
P. Talaga in Drug Discovery Today 9/2009, Share or die!:
The best way to predict the future is to invent it
‘It is not the strongest of the species that survives,
nor the most intelligent,
but the one most responsive to change’
Precision Medicine © Dr. Thomas Wilckens
Alan Curtis Kay