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Human clinical data is the critical proof for new medical treatments. The current pharma, venture capital and NIH funding environments make it more important than ever for capital efficient clinical trial design and funding. Here are some best practices in mapping new treatments to clinical applications to accelerate clinical trial data.
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Driving AcceleratedClinical Data
CIMIT Innovation Grand RoundsMass General Hospital
May 4, 2010
ZEN CHUWyss Institute
Harvard Medical SchoolOffice of Technology Development
Best Practices for Med TechAccelerated De-Risking
• Challenges Today
• Innovation Landscape & Risks
• Teams & Conflict of Interest
• Clinical Innovation Rules & Tradeoffs
• Panel Discussion
Good Business IS Good Medicine
Challenges In Driving Clinical Data
• Clinical effectiveness is not enough• Pharma & venture funding tight• Publications & research polluted with noise• High sensitivity to Conflict of Interest• FDA reviews more conservative, higher hurdles• Payers & Clinicians demand Evidence-Based Data• Health reform & Comparative Effectiveness impact today
More important than ever to matchFocused solution to clinical champions
Clinical Innovation LandscapeMatch Resources to Opportunity Size
Market Size of Opportunity
Low
C
apita
l Req
uire
d H
igh
Big OpportunitiesComplex Solutions,
Fragmented Services
Philanthropy/NIHBasic Research GrantsUnknown Mechanisms
$100MM $1B
Large EstablishedCompanies
Integrate Whole Solutions
Venture-BackedCompanies
Focused Teams to Prioritize & Execute
Discrete Product
Product-Line ExtensionsLicense or Product Acq
Royalties
Humanitarian Exemption,
Venture Philantropy
Stem Cells
Philanthropy/NIHBasic Research GrantsUnknown Mechanisms
Gates Foundation
Off-Label Uses
Ortho LumbarDisc
Market Size of Opportunity
Low
C
apita
l Req
uire
d H
igh
Big OpportunitiesComplex Solutions,
Fragmented Services
$100MM $500MM
RNAi for AMD
Cystic Fibrosis
IschemicStroke
Gastric Bypass
Unfocused & Requires
Segmentation
AorticAbdominalAneurysm
Segment To FocusPatients, Disease State, Providers
Disease SegmentationMap Unmet Clinical Needs To Development Path
• Disease Natural History & Diagnostics• Comorbidities & Adjacencies• Physician Referral Patterns• Untreatable Patient Subsegments• Humanitarian Exemption
Focus Segments onHigh Impact & High Value
Market Analysis CriticalEnables Path To Clinical Efficacy
Proven Clinical Adoption Drivers1) Patient pain & outcomes
2) Therapeutic procedure time
3) System costs over entire disease cycle
Comparative Effectiveness
Will Impact Prioritization
Return On Investment Drivers
• Choose Clinical Endpoints with
Fastest & Highest ROI– Hospital stay– Re-admission & revision– Complications & errors– Recurrence– Early detection (prevention coming?)
• Key Players– Patients, Payors, Physicians, Hospitals
Analysis Is Paramount
Efficacy is NecessaryBut Insufficient For
Translating to Widespread Practice
Value Inflection Points
Time to Market & FDA Approval
but also
Time to First Human Data&
Time to Clinical Adoption
Val
ue
TimeCritical Milestones
Acquisition/IPO Exit Reimbursement Published clinical
results Market Introduction
Salesforce hired Europe & Japan distribution
FDA Approval
Manufacturing ramp Human pivotal study Corporate partnership First human data First Hiccup & Danger of Momentum Loss
CEO hired Pilot manufacturing Patent grant First FDA meeting Regulatory opinionsMoney raised & team hired
Animal testing Clinician feedback Patent disclosure Prototype/Reduction to Practice Market AnalysisIdea
Accelerate Clinical ImpactTime is Life
COMPRESS TIME TO MARKETFASTER CURES & ROI
Which Risks to Prioritize?V
alue
Time
Market RiskReimbursement
Physician & Patient AdoptionPackaged Solutions
Regulatory RiskSafety & Efficacy
Management Risk
Technology IP Risk
Investor Risk PrioritiesV
alue
Time
Market RiskReimbursement
Physician & Patient AdoptionPackaged Solutions
Regulatory RiskSafety & Efficacy
Management Risk
Technology IP Risk
Largest Risks Must Be Addressed & De-Risked Up Front Before Funding
Investors & Companies Willing to Take These Risks
Build Team To ExecuteV
alue
Time
Market RiskReimbursement
Physician & Patient AdoptionPackaged Solutions
Regulatory RiskSafety & Efficacy
Management Risk
Technology Risk2) Te
am E
xecu
tion
1) P
lan in
Rev
erse
Teams Drive Data
• Clinical & Business teams
• Focus & segment the opportunities
• Build teams to match opportunity type
• Complement inventors with team
BUILD TEAMS THAT UNDERSTAND
THE RISK / EXECUTION TRADEOFFS
Building Clinical Team WhileNavigating Conflict of Interests
• Transparency in relationships & publication• Equity holders cannot drive clinical data• Avoid equity & minimize cash consulting• Find & Engage Clinical Champions• Match patients to risk/benefit• Negotiate IP rights up front
CAPITAL EFFICIENT & CREDIBLE DATA
7 Rules of Innovation
• Minimize Time To Demonstration• Build Smallest Possible Team• Develop Only Bare Minimum• Iterate Frequently Before Mfg Controls• Never Develop What Can Be Bought• Insist on Highest Quality Workmanship• Prove One Idea/Variable At A Time
EMPHASIS ON SPEED & PROOF
Clinical Data TradeoffsImpact & Speed versus Cost
CLINICAL EFFICACY
• Acceptable endpoints
• Risk / Benefit analysis
• Random / Blinded / Controls
• Statistical power / Sample size
SPEED
• Clinical data timeline
• Fundable interim endpoints
• Trial complexity & IRBs
• Approval, Adoption, Reimburse
COST EFFECTIVENESS
• Controls & Competitors
• Cost per Patient
• Procedure time savings
• Total Episode cost & ROI
ADDRESSABLE PATIENT POPUL.
• Segment Inclusion/Exclusion
• Labeling Restrictions
• Comorbidities, non-responders
• New delivery channels
BALANCE COSTS & RISKS
Time is LifeACCELERANT DECCELERANT
REGULATORY Device 510(k)Euro CE Literature ClaimsPredictive animal models
New materials & claimsUnclear endpoints
CLINICAL Existing human dataReproducible published studiesAcute episodic symptoms
Non-superiorityEntrenched behaviorChronic disease endpoints
REIMBURSEMENT Existing Codes DRGsExisting insurer coverage policiesPrivate-pay, consumer
Cost-Benefit StudiesNew codes required
MARKET Reduce skills neededPhysician buying powerUnambiguous diagnostic
Capital equipment purchase No physician benefit
SALES Focused physician popul.Existing distribution
Learning curvesEntrenched behavior
Incremental vs BreakthroughPlenty of Incremental Value, Different Strategy
METRIC INCREMENTAL BREAKTHROUGHFunding $$ $$$$$$
Market Size Varies depending on funding needed
>$100MMNo current therapies
Time NeededAdoption & Exit
ShorterMatch opportunity to strategy
LongerRegulatory path dependent
Value @ Exit Less More
Major Risks Window of opportunityExecution quality & critical pathCompetition
Technical/Clinical unknownsMarket adoption hurdlesReimbursement
Success Factors Focused executionManagement experienceManufacturing economics
Patents, Franchise ValueMarket adoption, Std of CareInvestor expectations
Patent Protection Less More
Competition More Less
Clinical InnovationCritical Success Factors
• Rigorous Analysis• Focused Plan To Early Proof• Credible & Creative Team • Adapt With New Data• Evidence-Based Business• Desire to help patients
Innovation Grand Rounds Panel Discussion:
Driving Clinical ProofZEN CHU
Wyss InstituteHarvard Medical School
Office of Technology Development
HELEN COLQUHOUN, MDFounder & CEO, Pleiad Devices
DAVID REED, MD, MBA Serial Medical Entrepreneur
AARON SANDOSKIManaging Director, Norwich Ventures
