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.