A Panel Discussion

21st Annual ACPU MeetingNIH Clinical Center

April 26, 2012

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Format of SessionIntroduction & BackgroundPanelists comments and generation of topics

to discussWhat has changed, is changing, will

change?

Panel Discussion on leading topics Identify attributes of ‘ideal’ CPU

What would you build with a blank check?

Audience questions 2

“Overview of the Phase I Market”Ken Getz, Tufts CSDD; Oct 2010 ACPUDrug Development LandscapePhase I MarketMarket TrendsOpportunities

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Ask the ExpertsBroad perspective of expertise

AcademiaRob Califf, Carl Peck, [Stephen Spielberg]

CROsOren Cohen, Royce Morrison

Pharma, big & smallMatt Troyer (John Wagner), Diane Jorkasky

RegulatoryStephen Spielberg, [Carl Peck]

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Commercialization Conditions Restrictive price controls Healthcare reform

uncertainty and adverse impact

Depressed global markets High-level of revenue at risk

R&D Operating Conditions Low success rates Declining levels of

innovation Rapidly rising R&D costs Regulatory conservatism Public discontent

Drug Development Landscape

Getz, ACPU Oct 2010

“Patent Cliff”

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Downsizing of Pharma

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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting

Declining New Drugs per R&D $

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Kaitin, CP&T, 2010; 87(3):356-361

R&D Cost DriversR&D Cost DriversChronic and complex indications

Clinical trial size

Protocol design complexity

Patient recruitment/retention

High cost discovery/research tools

Regulatory demands

Market oriented studies

Late-stage attrition

Getz, ACPU Oct 2010

Growing Protocol Complexity

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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting

Global Phase I Facilities

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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting

Phase I CPUs & Number of Beds

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Getz, Overview of the Phase I Market, Oct 2010, ACPU Annual Meeting

Trends in the Phase I MarketHeightened safety concernsEmphasis on hospital and in-patient settingsShift to US and Canada for time and cost

advantages post EU-DirectiveIncreasing proportion of patients vs. NHVModified and combination protocol designsTransfer operating risk to CROsGrowing entry by for-profit, community-

based investigative sites

Source: Tufts CSDD interviews

The Times They Are a-changin’ [“you better start swimming or you’ll sink like a stone…”]

14Greenberg, DIA Global Forum, Feb 2012

Pressures in Clinical ResearchOff shoring clinical trialsTransfer operating risk to CROs & partnering

with AMCsPersonalized medicine – fad or future?Advances in genomics, proteomics,

biomarkers, imaging, diseases models, bioinformatics, etc.

Regulatory and social pressures on safety for longer term studies

Public discontent with Pharma15

Commercialization ConditionsEconomic crunch on Pharma productivityConsolidation of Pharma companiesRestrictive price controlsHealthcare reform uncertainty and its

adverse impactDepressed traditional markets & growing

emerging marketsPatent cliff and revenue at risk

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R&D Operating ConditionsLower success ratesDeclining levels of innovationIncrease in biologics and biosimilarsRapidly rising R&D costs, and outsourcing

demandRegulatory conservatism

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Trends in the Phase I MarketCPUs from Pharma owned and operated to CROsHeightened safety concernsEmphasis on hospital and in-patient settingsShift to US and Canada for time and cost

advantages post EU DirectiveIncreasing proportion of patients vs. NHVCombination protocol designs, incl

SAD/MAD/POC & adaptive designsGrowing role of community-based investigative

sites

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Why these Panelists?Rob Califf: clinical research in AMCOren Cohen: heads phase I in leading global CRODiane Jorkasky: duel CPU operations and pharmaRoyce Morrison: clinician, CPU PI, CRO CMOCarl Peck: FDA CDER, drug development ‘guru’Stephen Spielberg: academic, pharma, now FDAJohn Wagner (Matt Troyer): current sponsor view

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Why these Panelists?Among the 7 panelists:

Over 180 years experience in clinical research in over 1000 clinical trials

Co-authored over 2000 PublicationsGiven over 3000 PresentationsParticipated in hundreds of INDs and NDAs

in various capacities

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Let’s get going ….

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