21st Century CPU

1
21st Century CPU The Future of Clinical
Pharmacology Units and Early Phase Clinical
Research

• A Panel Discussion
• 21st Annual ACPU Meeting
• NIH Clinical Center
• April 26, 2012

2
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

3
Overview of the Phase I Market

• Ken Getz, Tufts CSDD Oct 2010 ACPU
• Drug Development Landscape
• Phase I Market
• Market Trends
• Opportunities

4
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

5
Drug Development Landscape

• Commercialization Conditions
• Restrictive price controls
• Healthcare reform uncertainty and adverse impact
• Depressed global markets
• High-level of revenue at risk
• RD Operating Conditions
• Low success rates
• Declining levels of innovation
• Rapidly rising RD costs
• Regulatory conservatism
• Public discontent

Getz, ACPU Oct 2010
6
Patent Cliff
7
Downsizing of Pharma
Getz, Overview of the Phase I Market, Oct 2010
ACPU Meeting
8
Declining New Drugs per RD
Kaitin, CPT, 2010 87(3)356-361
9
RD 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
10
Growing Protocol Complexity
Getz, Overview of the Phase I Market, Oct 2010
ACPU Meeting
11
Global Phase I Facilities
Getz, Overview of the Phase I Market, Oct 2010
ACPU Meeting
12
Phase I CPUs Number of Beds
Getz, Overview of the Phase I Market, Oct 2010,
ACPU Annual Meeting
13
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
14
The Times They Are a-changin you better start
swimming or youll sink like a stone
Greenberg, DIA Global Forum, Feb 2012
15
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

16
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

17
RD Operating Conditions

• Lower success rates
• Declining levels of innovation
• Increase in biologics and biosimilars
• Rapidly rising RD costs, and outsourcing demand
• Regulatory conservatism

18
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

19
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

20
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

21
Thank you

• Lets get going .