Precision medicine 2014

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

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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


other

omics

Precision

Medicine

Other

Big-

DATA

जेम्स, कृपया फिर से ईधन देना आपके रक्त में शकक रा कम है

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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.


Create Application

POC for longitudinal, intra-individual multi-omics profiling’s value proposition Exponential value growth by the generation of content from data

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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


A paradigm shift in R&D: Intraindividual multi-omics analysis replaces population based medicine

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Intra-individual, long-term Big-DATA analytics generates

new insight in human biology and pathologies


Multi-omics and Big Data analysis: Biological dynamics and complexities require new data architectures and analytics

10

Data analytics challenges:

• up to 100 terabyte per patient measurement

(i.e. 50 samples over time)

• nonlinear dynamics


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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


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


The route to precision medicine What the diagnostics companies need:

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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



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



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


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

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Launch of a Transformative Health Care

Initiative: The National Biomarker

Development Alliance (NBDA) 1/2014


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


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


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


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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’


Alan Curtis Kay

Health & Medicine

Precision medicine 2014