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Prepared by Blandine Bourgoin, Innovation Analyst
For PRIME Paris Region International Mission Enterprise
June 2015
Personalized Medicine in the U.S.
A. Why Personalize Medicine ?
B. What is it ?
C. The Ecosystem
D. From an Economic Perspective
E. Challenges and Future Opportunities
SUMMARY
Imprecision Medicine
The right time
Why Personalize Medicine ?
Today’s Imprecision Medicine
Approved drugs in the US
Adverse effect No adverse effect
Hospitalized
Patients
7%
Troubles of adverse drugs reaction:
http://www.medscape.com/viewarticle/834273#vp_2
16%
Cost per Raw Megabase of DNA Sequence
Personalized Medicine - Example in Cancer
Benefits of Personalized Drugs in some Cancers
Munoz, J. & Kurzrock, R. (2012) Targeted therapy in rare cancers—adopting the orphans Nat. Rev. Clin. Oncol. doi:10.1038/nrclinonc.2012.16
What is Personalized Medicine ?
• “Tailoring of medical treatment to the
individual characteristics of each
patient”
President's Council of US Advisors
• “Choosing treatments that work well
with a person's genomic profile or with
certain characteristics in the person's
blood proteins or cell surface proteins“
• FDA
• “Healthcare that is informed by each
person’s unique clinical, genetic, and
environmental information”
• American Medical Association
Many Definitions
• “Providing the right treatment to the
right patient at the right dose at the
right time”
European Union
• “A medical model that proposes the
customization of healthcare - with
medical decisions, practices, and/or
products being tailored to the
individual patient”
Wikipedia
• “A form of medicine that uses
information about a person’s genes,
proteins, and environment to prevent,
diagnose, and treat disease.” NIH
Many Areas for a Huge Subject
Personalized
Medical Devices
Oncological
Vaccination
Gene TherapyTissue EngineeringRisk Profiling
Diagnostic
Data Management
Digital Health
Detect
early
Diagnose
accurately
Select
treatment
Monitor /
adjust
treatment
Prevent
A New Approach in Modern Medicine
Integrating Multiple Data
Genetic make-up
Key biomarkers
Treatment history
Behavioural preferencesEnvironmental factors
Multiple Stakeholders
Policy
makers
Patients Biotechs
Pharma
Cies
Providers Payers
Physicians Patients
From patients
To patients
ITCs
The Ecosystem
DIAGNOSTIC
3 TYPES OF STARTUPS
THERAPEUTIC DATA
President Obama’s Precision Medicine Initiative
• Voluntary
National
Research
Cohort
• 1 million
volunteers
Creation
COHORT
• With NCI
• Building a
Cancer
Knowledge
Network
Focus
CANCER
• Input from
Experts
• Patients
groups,
bioethicists,
advocates…
PRIVACY
Modernization
• FDA thinks
of new
evaluation
of NGS
REGULATORY
Public /
Private
partnerships
PARTNERSHIP
State of the Union Address, 20 January 2015, USA — Budget : 215 M$
https://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative
130 M$70 M$ 10 M$5 M$
Apple
Researchkit Open Source Project:
– Creation of Apps for medical
research
– Easy sharing
DELL
N°1 IT Service Provider in Healthcare:
– Data storage
– Predictive analytics solution
– Health technology academy
IT Companies – Big Players
Google Could Have the Tools to Integrate this Data
From an Economic Perspective
Funding
• Big Fund Raising:
• IPO :
EXAMPLES
56 M$
50 M$ 44 M$
52M$
IPO 2005
19.55M$
IPO 2015
10M$
Roche 2014
150M$
IPO 2015
• Diagnostic products and services:
Oncology alone : All:
> By 2018
• Key drivers of growth:
– Payors mandating Dx to ensure proper use of
therapeutics
– Improvements in the underlying technology
and quality of Dx testing
– Physicians’ need for the information
generated by Dx to provide optimal
treatment
– Increasingly informed and active patients
– Advances in digital and info techno
Market Size
$6
Drugs in pharma pipeline
With companion diagnostic
Without
Roche pipeline
$3
Challenges and Future Opportunities
• Scientific:
– New targets, new companion diagnostics
– Data management (standardization, access…)
• Political:
– When ?
– How, what means ?
• Economical :
– Expenses, investment, public, private…
• Social :
– Ethical considerations
– Privacy but patient tracking, access to actionable knowledge
– Changes in attitude
Challenges
Potential Development in Oncology
Future Areas of Personalized Medicine
Immunotherapy for cancer • Raised 51$M in their Series D round
A New Thing that Could Surpass PM
Sources
 http://www.personalizedmedicinecoalition.org/Userfiles/PMC-
Corporate/file/pmc_case_for_personalized_medicine.pdf
 http://www.paristechreview.com/2011/11/08/revolution-personalized-medicine/
 http://en.wikipedia.org/wiki/Personalized_medicine
 http://2015sv.pmwcintl.com/program19115.pdf
 Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product
Development.http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421
.pdf
 Personalized medicine: Time for one-person trials, Nicholas J. Schork, 29 April 2015
About PRIME & French Tech Hub
PRIME is a transatlantic innovation platform
promoting the Paris region as a center of
innovation and engaging in activities to
encourage and incentivize American companies
and organizations to invest in the Paris region.
PRIME is a subsidiary of the Paris Region
Economic Development Agency.
French Tech Hub serves as the U.S. Hub for
entrepreneurial and innovative French
companies. We provide custom-tailored services
to help our clients strategize, launch, expand, and
achieve their business potential.
For more info,
contact Marie-Perrine Durot
marie-perrine.durot@parisregionentreprises.org
For more info,
contact Marie Buhot-Launay
marie.buhotlaunay@frenchtechhub.com

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Personalized medicine in the US

  • 1. Prepared by Blandine Bourgoin, Innovation Analyst For PRIME Paris Region International Mission Enterprise June 2015 Personalized Medicine in the U.S.
  • 2. A. Why Personalize Medicine ? B. What is it ? C. The Ecosystem D. From an Economic Perspective E. Challenges and Future Opportunities SUMMARY
  • 3. Imprecision Medicine The right time Why Personalize Medicine ?
  • 4. Today’s Imprecision Medicine Approved drugs in the US Adverse effect No adverse effect Hospitalized Patients 7% Troubles of adverse drugs reaction: http://www.medscape.com/viewarticle/834273#vp_2 16%
  • 5. Cost per Raw Megabase of DNA Sequence
  • 6. Personalized Medicine - Example in Cancer
  • 7. Benefits of Personalized Drugs in some Cancers Munoz, J. & Kurzrock, R. (2012) Targeted therapy in rare cancers—adopting the orphans Nat. Rev. Clin. Oncol. doi:10.1038/nrclinonc.2012.16
  • 8. What is Personalized Medicine ?
  • 9. • “Tailoring of medical treatment to the individual characteristics of each patient” President's Council of US Advisors • “Choosing treatments that work well with a person's genomic profile or with certain characteristics in the person's blood proteins or cell surface proteins“ • FDA • “Healthcare that is informed by each person’s unique clinical, genetic, and environmental information” • American Medical Association Many Definitions • “Providing the right treatment to the right patient at the right dose at the right time” European Union • “A medical model that proposes the customization of healthcare - with medical decisions, practices, and/or products being tailored to the individual patient” Wikipedia • “A form of medicine that uses information about a person’s genes, proteins, and environment to prevent, diagnose, and treat disease.” NIH
  • 10. Many Areas for a Huge Subject Personalized Medical Devices Oncological Vaccination Gene TherapyTissue EngineeringRisk Profiling Diagnostic Data Management Digital Health
  • 12. Integrating Multiple Data Genetic make-up Key biomarkers Treatment history Behavioural preferencesEnvironmental factors
  • 13. Multiple Stakeholders Policy makers Patients Biotechs Pharma Cies Providers Payers Physicians Patients From patients To patients ITCs
  • 15. DIAGNOSTIC 3 TYPES OF STARTUPS THERAPEUTIC DATA
  • 16. President Obama’s Precision Medicine Initiative • Voluntary National Research Cohort • 1 million volunteers Creation COHORT • With NCI • Building a Cancer Knowledge Network Focus CANCER • Input from Experts • Patients groups, bioethicists, advocates… PRIVACY Modernization • FDA thinks of new evaluation of NGS REGULATORY Public / Private partnerships PARTNERSHIP State of the Union Address, 20 January 2015, USA — Budget : 215 M$ https://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative 130 M$70 M$ 10 M$5 M$
  • 17. Apple Researchkit Open Source Project: – Creation of Apps for medical research – Easy sharing DELL N°1 IT Service Provider in Healthcare: – Data storage – Predictive analytics solution – Health technology academy IT Companies – Big Players
  • 18. Google Could Have the Tools to Integrate this Data
  • 19. From an Economic Perspective
  • 21. • Big Fund Raising: • IPO : EXAMPLES 56 M$ 50 M$ 44 M$ 52M$ IPO 2005 19.55M$ IPO 2015 10M$ Roche 2014 150M$ IPO 2015
  • 22. • Diagnostic products and services: Oncology alone : All: > By 2018 • Key drivers of growth: – Payors mandating Dx to ensure proper use of therapeutics – Improvements in the underlying technology and quality of Dx testing – Physicians’ need for the information generated by Dx to provide optimal treatment – Increasingly informed and active patients – Advances in digital and info techno Market Size $6 Drugs in pharma pipeline With companion diagnostic Without Roche pipeline $3
  • 23. Challenges and Future Opportunities
  • 24. • Scientific: – New targets, new companion diagnostics – Data management (standardization, access…) • Political: – When ? – How, what means ? • Economical : – Expenses, investment, public, private… • Social : – Ethical considerations – Privacy but patient tracking, access to actionable knowledge – Changes in attitude Challenges
  • 26. Future Areas of Personalized Medicine
  • 27.
  • 28. Immunotherapy for cancer • Raised 51$M in their Series D round A New Thing that Could Surpass PM
  • 29. Sources  http://www.personalizedmedicinecoalition.org/Userfiles/PMC- Corporate/file/pmc_case_for_personalized_medicine.pdf  http://www.paristechreview.com/2011/11/08/revolution-personalized-medicine/  http://en.wikipedia.org/wiki/Personalized_medicine  http://2015sv.pmwcintl.com/program19115.pdf  Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development.http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421 .pdf  Personalized medicine: Time for one-person trials, Nicholas J. Schork, 29 April 2015
  • 30. About PRIME & French Tech Hub PRIME is a transatlantic innovation platform promoting the Paris region as a center of innovation and engaging in activities to encourage and incentivize American companies and organizations to invest in the Paris region. PRIME is a subsidiary of the Paris Region Economic Development Agency. French Tech Hub serves as the U.S. Hub for entrepreneurial and innovative French companies. We provide custom-tailored services to help our clients strategize, launch, expand, and achieve their business potential. For more info, contact Marie-Perrine Durot marie-perrine.durot@parisregionentreprises.org For more info, contact Marie Buhot-Launay marie.buhotlaunay@frenchtechhub.com

Hinweis der Redaktion

  1. 16% among approved drugs in the US Almost 7% of hospitalized patients associated with adverse drug reactions More than 100 000 deaths 4 to 7 billions $ / year in 2014 EX: ABILIFY, 7,54billions$ 8,8 millions of prescriptions
  2. Propeller health > ASTHMA tracker
  3. http://www.ddw-online.com/personalised-medicine/p92845-overview-of-companion-diagnostics-in-the-pharmaceutical-industry.spring-10.html
  4. understanding variants, treating genomic and molecular data http://www.syapse.com http://oneome.com/
  5. Create apps for medical research and easy sharing. Real time dynamic tasks, surveys… Mobile healthcare market expected to be around 9 billions$ DELL GARTNER conseil dans les domaines technos
  6. Same idea of superposition of layers But GIS organized by geographical position And IC overlays in complex ways Google Ventures 36% Health TRANSITION : AMAZON COULD BE NEXT MET FDA leadership
  7. Genomic Health: 52 Million of VC finding – IPO in 2005 raising more than 60 million Precision for Medicine was the only early-stage company to make the cut (the other nine were in expansion or later stage).  Besides Atlanta-based AirWatch, it was the only non-California-, non-New York City-based company. The Maryland-based Precision for Medicine was also the only biotech to make the ranks. J.H. Whitney – as well as the VC100 chart-topping Oak Investment Partners – invested $150 million in first-round funding. Precision for Medicine was founded in 2012, shortly after co-founders Mark Clein and Ethan Leder sold United BioSource Corp (UBC), a drug research and development company, to Medco for $730 million. The co-founders of UBC (which has since become an Express Scripts company) remained in management to ease the transition. It was then they were swept by Medco’s attention to personalized medicine, or precision medicine: the idea of starting with incremental information about an individual patient to determine best treatments, as opposed to handing them prescription drugs that are given broadly across populations. “The more we looked at this concept the more we were convinced that this was the most important trend affecting medicine in the next 10 to 20 years,” says Clein. "We identified this large trend that’s starting in the research world, and spreading to the common practice of medicine."