CAN WE TRANSFORM HEALTHCARE IN THE PUBLIC INTEREST NOW?
ScienceDriven January, 2012
AGENDA: USE WHAT WE ALREADY HAVE FOR PROSPECTIVE HEALTH NOW
DEFINE Model and Assessments based on Knowledge
Exchange between Patient/Family & Multidisciplinary Care Team
DESIGN Framework for New Service Development for Personalized Healthcare
Delivery
DELIVER Higher value, more efficient KNOWLEDGE EXCHANGE
On Target: Save Lives and Let the “Rising Star” Clinicians Thrive
On Time: Get Research Breakthroughs to Patients Sooner
On Budget: Avoid and Defer Costs
DEFINE A MODEL FOR NEW SERVICE DEVELOPMENT IN A FOR-MISSION ENTERPRISE
Excellent
Science
Biologically Inspired
Translation
Personalized Health Care
HOW THIS PROCESS MAPS TO MISSIONS
* “Clinical” and “laboratory” refer to the locations where research takes place
ACADEMICHEALTHCARE
PROCESS
Patient care
x
Research x x xLearning / Mentoring
x x x
Emerging
*
Practitioneridentifies unmet need
SIMILARITY TO NEW PRODUCT DEVELOPMENT IN A FOR-PROFIT ENTERPRISE
In an enterprise, need a balance of people. ALL are needed.
*See Steven Wheelwright and Kim Clark “Revolutionizing Product Development” (1992)
Each innovates differently
Inventor Adapterdiscovers re-purposes
ACADEMIC HEALTHCARE
PROCESS
Research
Development
Production
NEW PRODUCTDEVELOPMENT*
Excellent Science
Biologically Inspired
Translation
Personalized Healthca
re
Excellent
Science
Biologically Inspired
Translation
Personalized Health Care
Clinical Innovatio
n
DISTINCTION OF NEW SERVICE DEVELOPMENTIN A FOR-MISSION ENTERPRISE
CYCLE DRIVES HEALTHCARE TRANSFORMATION
To renew cycle, need:
1. ResourcesNote: Cycle does not violate
laws of thermodynamics.
2. Flexible, appropriate performance assessments
3. Renewable support for clinician-investigators
To allocate limited life-saving resources that transform healthcare in the public interestDEFINE FLEXIBLE, APPROPRIATEPERFORMANCE ASSESSMENTS
RIGHTRESOUR
CESTO THE
RIGHT PEOPLE
AT THE
RIGHTTIME
Multiple Stakeholders: Challenge for Implementing Prospective Health in the Clinic
Patient/ Family
Multi. CareTeam
CMSInsurance
PharmaBiotech
Devices
Diagnostics
EmployersHospitals
Physicians
Patients
NIH
Senate & House
FDA
1. Each stakeholder has its own, appropriate interests and priorities
2. Stakeholders may contribute to prospective health, but will not have clinical adoption as their primary goal.
HOW WILL THIS BE SOLVED?
New clinical services must put PATIENT/FAMILY & MULTI. CARE TEAM back at the center of healthcare.
Historically, trade sustains self-renewing societies
Trade involves more than goods and commerce Translation of language Acceptance of culture Exchange of knowledge
3000 BC 2000 BC 1000 BC 1000 AD 2000 AD0
Old Kingdom New Middle Kingdom
EGYPT Kingdom Republic Empire ROME
1st 2nd BRITAIN
Knowledge Exchange: Unit Operation of Real-world Healthcare
Knowledge
EXCHANGE
Patient /Family
Multidisciplinary Care
Team
GOAL:Sustainable, high-performance KNOWLEDGE EXCHANGE
URGENT NEED:Renew and refocus healthcare on
The Generality of Knowledge Exchange
As a unit operation, KNOWLEDGE EXCHANGE BETWEEN SERVICE TEAMS AND SERVICE USER is of broad generality for development of mission-driven services
A. For HEALTH, renew and refocus on knowledge exchange between multi teams of health coaches/mentors/advocates/advisors and healthy people/their families.
B. For EDUCATION, renew & refocus on knowledge exchange between teachers and students/families.
C. For LEARNING, renew & refocus on knowledge exchange among learners/do-ers.
D. For FAMILIES, renew and refocus on knowledge exchange between parents and daughters/sons.
For product manufacturing, measure 3 dimensions “On target, on time, on budget” Faster,
better, cheaper
For services with knowledge exchange as unit operation: Performance = f (Value, Efficiency) +
Observation Value = Quality Efficiency = Unit Operation =
Knowledge Exchange Cost Unit Time Time
For dynamic R&D, “on target” is captured in Observation
We become what we measure
Numbers Don’t Tell the Whole Story
Observation = Summary of unmet need,
lesson learned, next experiment, or suggested improvement
Like a comment in chart or lab notebook Knowledge waiting to be exchanged Most important measure for R&D dashboard
Novel Measures Reported by
assess relative change in
Value = Quality Efficiency = Knowledge Exchange Cost Time provide Observations for ongoing evaluation
Simple Assessments for Experiments in New Service Development
Patient/Family
Multi. CareTeam
AGENDA: USE WHAT WE ALREADY HAVE FOR PROSPECTIVE HEALTH NOW
DEFINE Model and Assessments based on Knowledge Exchange
between Patient/Family & Multidisciplinary Care Team
DESIGN Framework for New Service Development to
Deliver Personalized Healthcare DELIVER
Higher value, more efficient KNOWLEDGE EXCHANGE On Target: Save Lives and Let the “Rising Star” Clinicians
Thrive On Time: Get Research Breakthroughs to Patients Sooner
On Budget: Avoid and Defer Costs
* From bipartisan “Genomics & Personalized Medicine Act of 2007” co-sponsored by Burr (R-NC) and Obama (D-IL)
per·son·al·ized med·i·cine *
The application of genomic and molecular data to:
- better target the delivery of health care - facilitate the discovery and clinical testing
of new products - help determine a person’s predisposition
to a particular disease or condition
Both Retrospective AND Prospective Clinical Innovation Is Needed
Retrospective ProspectiveCohort size Large SmallResearch method
Deductive Inductive
Level of analysis
Population Patient
Hypothesis Hypothesis-testing Hypothesis-generatingTypical analysis
Association of SNPs with traits
Co-analysis of clinical & patient-reported measures
Sample areas
Cardiovascular, psychiatry
“Traitors & invaders” *
“Right drug for right patient at the right time”
“How many CRs does it take to be statistically significant?”
* Pathology of cancer cells (traitors) and infectious diseases (invaders), as well as immune and metabolic responses to both, are sufficiently understood for meaningful interpretation of prospective studies.
Personalized Medicine System applied to Patients diagnosed with Cancer
Personalized Medicine System
Patient Needs
1. Engaging the patient
Should I be checked?
2. Systems diagnosis I am not my tumor
3. Therapy decision support
What is best for me?
4. Outcomes analysis Is it best for others like me?
5. Molecular actuarial* Transparent, up-front price
based on scientific evidence
* Note: Outside healthcare, risks to life and health are empirically estimated (see links in http://law.vanderbilt.edu/faculty/faculty-personal-sites/w-kip-viscusi/biography/index.aspx )
B. Actionable
Pathology Report
2. Systems Diagnosis
C. Learning Health
Data System
D. Estimated Value
of Risk to Life/Health
Connecting the Dots for a Prototype ofPersonalized Healthcare Delivery
E. Allocation of Life-saving Resources
5. MolecularActuarial
A. Local
Referral
1. Engaging the Patient
Personalized Medicine
Analysis & Interpretation
• Prospective• Multiplexed• Patient-
level
3. Therapy Decision
Support
4. Benefit-Cost
Analysis
= Human Experience = Informatics Infrastructure
AGENDA: USE WHAT WE ALREADY HAVE TO DELIVER PERSONALIZED HEALTHCARE NOW
DEFINE Model and Assessments based on Knowledge Exchange
between Patient/Family & Multidisciplinary Care Team
DESIGN Framework for New Service Development to Deliver Personalized
Healthcare
DELIVER Higher value, more efficient KNOWLEDGE
EXCHANGE On Target: Save Lives and Let the “Rising Star” Clinicians
Thrive On Time: Get Research Breakthroughs to
Patients Sooner On Budget: Avoid and Defer Costs
One Starting Point: Systems Diagnosis Informs Personalized Therapy Decision Support
Clinical Pathway for Systems Diagnosis Use companion biomarkers as prototypes
Working Prototype of Therapy Decision Support at the Point of Care Disseminate best practice of knowledge
exchange between patient/family and multidisciplinary care team
B. Actionable
Pathology Report
2. Systems Diagnosis
C. Learning
Health Data System
D. Estimated Value
of Risk toLife/Health
E. Allocation of Life-saving Resources
5. MolecularActuarial
A. Local
Referral
1. Engaging the Patient
3. Therapy Decision
Support
4. Benefit-Cost
Analysis
21
Real-World Unwarranted Variation (
ScienceDriven
Exam
ple
of
Cu
rren
t C
are
1. Surgery (1st hospital) to remove GI tumor and colon2. Chronic meds for digestive symptoms3. Patient seeks opinions for months4. Surgery (2nd hospital): Liver too damaged for aggressive resection5. Meds to manage infection6-9. Radiology (3rd hospital): Chemo delivered to liver; 4 invasive treatments
Cost to date: $600,000
Patient JM (reported in WSJ, May 2010) with abdominal pain; scan reveals gastrointestinal tumor;
liver metastases
Pers
on
alize
d
Med
icin
e
1. Community oncology team & patient/family exchange knowledge.
2. Tumor biopsy sent out for Systems Diagnosis. 3. Interpretive report yields personalized diagnosis4. Multidisciplinary therapy decision support yields personalized therapy plan5. Short-course radiation treatment sensitizes tumor to pathway inhibitor6. Surgery to remove tumor remaining after treatment
Cost to date: $100,000
Personalized
Medicine
Example of Current
Care
22
Potential Economic Value: Personalized Dx & Personalized Tx
Rough Extrapolation of Case Study1. Cost Reduction from Personalizing Treatment
of One Patient Diagnosed with GI Cancer$500,000
2. Annual New Cases of GI Cancer in US 270,000
$500,000 270,000 = $135 billion annually
Why Now?
“. . . an innovation that reduced overall cancer death rates by only 1% would be worth almost $500 billion or about 6% of GDP. Reducing age-specific death rates from a single category of cancer such as breast or digestive cancer by 10% would have a similar value.”
-- Kevin M. Murphy & Robert Topel (1999) “The Economic Value of Medical Research”
23
Cu
rre
nt
Care
Pers
on
ali
zed
M
ed
icin
e
Female Asian Never-Smokers
clinical diagnosis of non-small cell lung
cancer
1. Treatment with chemotherapy.
2. Overall 5 yr survival rate <10%.1. Molecular tests of tumor biopsy reveal ALK mutation in
personalized diagnosis. 2. As part of personalized therapy plan, team & patient/family share decision to enroll in clinical trial of ALK pathway inhibitor3. 65% of patients enrolled in trial respond to ALK pathway inhibitor.
Value of lives saved estimated at >$100B per year
Personalized Therapy Decision Support at the Point-of-Care
SHORT-TERM
EFFECTS
LONG-TERM
EFFECTS
COST TO
MEMBER
Standard of Care $10,000
Clinical TrialsPathway
Inhibitor 1Antibody ACombination X
$0
$0
$0
Palliative Care at Home
$ 5,000
Proposed Interactive Screen(used by Patient/Family and Multidisciplinary Team)
Business Interest
Benefit
Quality Reduce unwarranted variation in care
Market Share
Speed discoveries to patients most likely to respond (e.g., Tiacci et al NEJM: hairy cell leukemia)
Profitability
See J&J’s credo for a proven model in addition to profit maximization
Risk Prospective outcomes analysis for molecular actuarial
Patient/family and multidisciplinary team share decision-making and accountability
Time Up-front, transparent pricing (with real-time claims adjudication)
Personalized Therapy Decision Support at the Point-of-Service: Benefits
What it Will Take to Finish Prototype(or Who Will Pay to Transform Healthcare in the Public Interest?)
ScienceDriven
1. AGILE “SWAT” TEAMS collaborate to connect existing solutions
2. DE-IDENTIFIED STRATEGIC PHILANTHROPY is most likely funding mechanism.
3. OBJECTIVE: WORKING PROTOTYPE 1 yr after funding received
4. Prototype provides real-world basis for business plan: 21st century FFRDC
Role* No. of FTEs
Programmers (existing start-up)
18
Cross-Functional Program Managers
2
Execution Team ScienceDriven, Medical, Technical
3
Biomedical Informatics Advisors Systems Pathology Expert-moderated Collaboratories Research Processes
4
Financial Planning & Analysis 2
Legal / Business Portal 3
TOTAL (@ $300K per fully loaded FTE)
$9.9M
* All FTEs are real people, not position descriptions
27
ScienceDriven
“A well run system with average practitionersdelivers better healthcare than
a poorly run system with outstanding practitioners.”
--Institute of Medicine Member & HHMI Investigator
28
Unwarranted Variation
Rates of common surgical procedures among Medicare patients for 306 referral regions
Mulley A G BMJ 2009;339:bmj.b4073
29
Regional Informatics
Imagine a rural farmer who visits a community oncologist. . .
MidSouth eHealth Alliance Primary care physicians for 900,000 Memphis
residents have an e-Health record system that lets them see what Vanderbilt physicians see, at lower cost
Turnkey, portable infrastructure for cost-effective health information exchange
Established by Vanderbilt Center for Better Health in 2004
http://www.markfrisse.com/presentations/2008-11-10-amia.pdf
Navigating the Interface of Clinical Research and Routine Patient Care
CLINICAL RESEARCH
ROUTINE PATIENT CARE
Health IT Patient consents to include research results in eMR
Shift to mass-customized/personalized car care informed by knowledge exchange between patient/family and multi team
Payers Industry pays routine care, drug or device. Philanthropy funds some rising stars and innovation.Which federal agency funds clinical investigation to address unmet medical need?
Reimbursement determined by CMS and payers . Currently, RVU-based. Shift to performance-based healthcare and risk evaluation (using measures consistent with rest of federal gov’t)
Regulatory
Protection of human subjects determined by patient/family and practice team.CAP, CLIA for research tests
FDA approves efficacy. Shift to product safety testing by UL. Shift to accelerated clinical guidelines developed by trial PIs.
Some Guiding Principles
1. Do no harm.2. There are no villains.3. Play to strengths.4. Learn by doing.5. Use what we’ve got.6. Better together.7. Hope over fear.