A Panel Discussion
21st Annual ACPU Meeting
NIH Clinical Center
April 26, 2012
1
Format of Session
 Introduction & Background
 Panelists comments and generation of topics to
discuss
 What 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 ACPU
 Drug Development Landscape
 Phase I Market
 Market Trends
 Opportunities
3
Ask the Experts
 Broad perspective of expertise
 Academia
Rob Califf, Carl Peck, [Stephen Spielberg]
 CROs
 Oren Cohen, Royce Morrison
 Pharma, big & small
 Matt Troyer (John Wagner), Diane Jorkasky
 Regulatory
 Stephen Spielberg, [Carl Peck]

4
Drug Development Landscape
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
Getz, ACPU Oct 2010
 Regulatory conservatism
 Public discontent
“Patent Cliff”
6
Downsizing of Pharma
Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
7
Declining New Drugs per R&D $
Kaitin, CP&T, 2010; 87(3):356-361
8
R&D Cost Drivers
 Chronic 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
Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
10
Global Phase I Facilities
Getz, Overview of the Phase I Market, Oct 2010 ACPU Meeting
11
Phase I CPUs & Number of Beds
Getz, Overview of the Phase I Market, Oct 2010, ACPU Annual Meeting
12
Trends in the Phase I Market
 Heightened safety concerns
 Emphasis on hospital and in-patient settings
 Shift to US and Canada for time and cost
advantages post EU-Directive
 Increasing proportion of patients vs. NHV
 Modified and combination protocol designs
 Transfer operating risk to CROs
 Growing 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…”]
Greenberg, DIA Global Forum, Feb 2012
14
Pressures in Clinical Research
 Off shoring clinical trials
 Transfer operating risk to CROs & partnering with
AMCs
 Personalized 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 Pharma
15
Commercialization Conditions
 Economic crunch on Pharma productivity
 Consolidation of Pharma companies
 Restrictive price controls
 Healthcare reform uncertainty and its adverse
impact
 Depressed traditional markets & growing
emerging markets
 Patent cliff and revenue at risk
16
R&D Operating Conditions
 Lower success rates
 Declining levels of innovation
 Increase in biologics and biosimilars
 Rapidly rising R&D costs, and outsourcing
demand
 Regulatory conservatism
17
Trends in the Phase I Market
 CPUs from Pharma owned and operated to CROs
 Heightened safety concerns
 Emphasis on hospital and in-patient settings
 Shift to US and Canada for time and cost advantages
post EU Directive
 Increasing proportion of patients vs. NHV
 Combination protocol designs, incl SAD/MAD/POC
& adaptive designs
 Growing role of community-based investigative sites
18
Why these Panelists?
 Rob Califf: clinical research in AMC
 Oren Cohen: heads phase I in leading global CRO
 Diane Jorkasky: duel CPU operations and pharma
 Royce Morrison: clinician, CPU PI, CRO CMO
 Carl Peck: FDA CDER, drug development ‘guru’
 Stephen Spielberg: academic, pharma, now FDA
 John Wagner (Matt Troyer): current sponsor view
19
Why these Panelists?
Among the 7 panelists:
 Over 180 years experience in clinical
research in over 1000 clinical trials
 Co-authored over 2000 Publications
 Given over 3000 Presentations
 Participated in hundreds of INDs and NDAs
in various capacities
20
Let’s get going ….
21