21st century cpu – the future of clinical pharmacology units and early phase clinical research
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21st Century CPU – The Future of Clinical Pharmacology Units and Early Phase Clinical Research. A Panel Discussion 21 st Annual ACPU Meeting NIH Clinical Center April 26, 2012. Format of Session. Introduction & Background Panelists comments and generation of topics to discuss - PowerPoint PPT PresentationTRANSCRIPT
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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
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“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
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“Patent Cliff”
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Downsizing of Pharma
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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
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Declining New Drugs per R&D $
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Kaitin, CP&T, 2010; 87(3):356-361
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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
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Growing Protocol Complexity
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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
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Global Phase I Facilities
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Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
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Phase I CPUs & Number of Beds
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Getz, Overview of the Phase I Market, Oct 2010, ACPU Annual Meeting
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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
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The Times They Are a-changin’ [“you better start swimming or you’ll sink like a stone…”]
14Greenberg, DIA Global Forum, Feb 2012
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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
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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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