personalized medicine bb june 2015
TRANSCRIPT
Prepared by Blandine Bourgoin, Innovation AnalystFor PRIME Paris Region International Mission Enterprise
June 2015
Personalized Medicine in the U.S.
A. Why Personalize Medicine ?B. What is it ?C. The EcosystemD. From an Economic PerspectiveE. 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.2012.160
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 preferences
Environmental 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 diagnosticWithout
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
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