UC CAI
University of California Center
for Accelerated Innovation
Face-To-Face Meeting
October 14, 2013
Michael Palazzolo
Local and National Announcements
CTSA Central
NBC Channel 4 News
• Posted by UCLA on
Sept 30, 2013 (day
before the government
shutdown)
• Aired October 1, 2013
Notice of Award
Notice of Award: Key Points
•
•
•
•
•
•
Year 1 Budget Period 9/26/13-7/31/14
Key Personnel
100-day Implementation plan due 10/25/13
Interim Progress Report in Year 1 due 3/1/14
Cost-share report due with FFR
Program Steering Committee Meeting
10/29-10/30/13
Introductions
Contents
•
•
•
•
•
•
Introduction to leadership
Goals
Governance
Technology Solicitation & Selection
Technology Development
Skills Development
Leadership
UC CAI
Michael Palazzolo, MD, PhD
Center Director, UC CAI
Professor, UCLA
Tomas Ganz, MD, PhD
Center Associate Director
Professor, UCLA
Leadership
Executive Committee
Gary S. Firestein, MD
Lars Berglund, MD, PhD
Dan Cooper, MD
Executive Committee Chair
Director, UCSD Clinical and
Translational Research Institute
Director, UCLA Clinical
and Translational Science Center
Director, UCLA Institute for Clinical
and Translational Science
Steven Dubinett, MD
Clay Johnston, MD, PhD
Director, UCLA Clinical
and Translational Science Institute
Director, UCSF Clinical and
Translational Science Institute
Site Leaders
Campuses
Laura Marcu, PhD
UC Davis
Steven George, MD, PhD Tomas Ganz, MD, PhD
UC Irvine
UCLA
Sotirios Tsimikas, MD Joseph Witztum, MD
UCSD
UCSD
June Lee, MD
UCSF
Shaun Coughlin, MD, PhD
UCSF
Domain Leaders
Diseases
Tomas Ganz, MD, PhD
Shaun Coughlin, MD, PhD
Blood Disorders
and Resources
June Lee, MD
Cardiovascular Disease
Lung and Sleep Diseases
Sotirios Tsimikas,MD
Cardiovascular Disease
Domain Leaders
Platforms
Laura Marcu, PhD
Sotirios Tsimikas,MD
Shaun Coughlin, MD, PhD
Co-Leader,
Devices and Tools
Co-Leader,
Diagnostics
Co-Leader,
Therapeutics
StevenGeorge,MD,PhD
Joseph Witztum, MD
June Lee, MD
Co-Leader,
Devices and Tools
Co-Leader,
Diagnostics
Co-Leader,
Therapeutics
Domain/Site Leaders
• At least one site leader for each campus
• At least one domain leader for each
platform (drugs, devices, diagnostics) and
disease (heart, lung and sleep, blood)
• Ensure the pool of candidate technologies
is large
• Oversee proposal solicitation process
• Recruit study sections
• Recruit and oversee project development
teams on respective campuses
Skills Development Program
• Catalogs entrepreneurial course
offerings on all five campuses
• Matches innovators to mentors
• Conducts webinars, symposia
Vish Krishnan
Leader, Skills Development Program
UCSD
Goals
Goal 1
• Engage University of California heart
lung and blood disease innovators
through a comprehensive education,
training and mentorship program.
Goals
Goal 2
• Solicit and select technologies with high
commercial potential that align with
NHLBI’s mission and address unmet
medical needs or significant scientific
opportunity.
Goals
Goal 3
• Incubate our most promising
technologies in accordance with
industry requirements to facilitate their
translation to commercial products that
improve patient care and enhance
health.
Goals
Goal 4
• Create a high-performing, sustainable
infrastructure that will serve as a model
to academic research centers.
Governance
External
Selection
Committee
Executive Committee
External Advisory
Board
Business Review
Panel
Center Director
Skills Development
Program
Associate Director
Domain Areas
Therapeutics
Diagnostics
Devices
Domain/
Site Leaders
Projects
Cardiovascular
Lung & Sleep
Disorders
Blood
Diseases
Program Resources
Administrative
& Budgetary
Support
Website & Data
Management
Project
Management
Industry
Relations & IP
CTSA
Infrastructure
Evaluation
& Tracking
External Selection Committee
• No fewer than 5 members
• Size and composition depends on RFAs
under review
• Appointed by Executive Committee
• May be present and former faculty
and/or industry leaders
• Must be external to institution(s)
External Advisory Board
•
•
•
•
Consists of no fewer than 5 members
Experienced business leaders
Includes NHLBI Program Officer
Advice about operations, project
development
Catherine Mackey, PhD
Former Senior VP, Pfizer
Founder, MindPiece Partners
Francis Duhay, MD
VP Medical Affairs and CMD,
Edwards Lifesciences
Lawrence Souza, PhD
Former Senior VP, Amgen
Founder, Coastview Capital,
Business Review Panel
• Five members
• VCR on each campus appoints one
member
• Evaluate Center’s progress toward
sustainability
Bill Ouchi, PhD
UCLA Initial Chair
Anderson School
CAI Administration
• Administration is based at UCLA
– Anne Skinner is Administrative Director
• UCLA CTSI maintains CAI website
– CAI information, RFP, project-tracking
– Doug Bell, MD, PhD leads
• UCLA CTSI conducts evaluation with
UC BRAID
– Pamela Davidson, PhD, MSHS leads
Governance Tasks for First 100 Days
• Executive Committee names at least 5
members to the External Selection
Committee
• VCRs at UCD, UCSD, UCI, UCSF each
name 1 faculty to Business Review
Panel
• Name at least 2 members to External
Advisory Board.
Governance Tasks for First 100 Days
• 1 meeting: Executive Committee,
Center Director, Associate Director &
External Advisory Board
• 3 meetings (1 per month): Center
Director, Associate Director & Executive
Committee
• 3 meetings (1 per month): Center
Director, Domain and Skills
Development Leaders
Administrative Tasks for First 100 Days
•
•
•
•
Initiate intercampus sub-awards
Identify campus administrative leads
Build website
Begin coordination/communication
process
Discussion
• Process for naming External Advisory
Board members
• Do we want more than 5 EAB
members?
• Deadlines for naming members to EAB,
Business Advisory Panel, and External
Selection Committee
Technology Selection Overview
5-Step Process
• Solicit 2-page pre-applications
• 1st Review: Review Panels assembled by
Domain Leaders review pre-applications and
invite full applications
• 2nd Review (Leadership Review): Executive
Committee, Center Director, Assoc. Director,
Domain Leaders and ad hoc reviewers select
full proposals
• 3rd Review (External Review): External Selection
Committee scores proposals and sends to
NHLBI
• 4th Review: NHLBI makes final selection
Technology Selection Overview
RFP
Pre-application
Pre-application
Review
Full
Application
Leadership
Review
External Selection
Committee Review
NHLBI Review
Technologies
Selected for
Entrance to Center
Technology Selection Timeline
• Solicitations occur three times a year, one
for each platform (therapeutics, devices,
diagnostics)
• RFPs for the platforms run concurrently
• Time from solicitation to prioritization by
External Selection Committee takes 7 mos.
• Up to 3 technologies enter Center in yr.-1
• Awards of up to $200K
Solicit
PreApplication
Review
PreApplication
Develop and
Submit Full
Application
First Review
of
Full
Application
ESC Review
of
Application
1 month
1 month
3 months
1 month
1 month
Pre-application and Review
RFP
Pre-application
Pre-application
Review
Full
Application
Leadership
Review
External Selection
Committee Review
NHLBI Review
Technologies
Selected for
Entrance to Center
Eligibility
• Faculty in all series and ranks at UC Davis,
UC Irvine, UCLA, UC San Diego, and UC San
Francisco
• Postdoctoral scholars are eligible to submit
applications as Co-PI with a faculty PI
• Projects with existing or imminent target
validation and a clear clinical indication
• Patents or patent applications are filed or
potential for obtaining defensible intellectual
property is strong
Solicitation Process
• Broad solicitation
• Added focus on cardiology, pulmonary,
hematology, cardiothoracic surgery, etc.
• Centralized RFP
• Webinar on submission process
• Campuses to provide potential applicants with
referrals for help with business plans, grant
writing, IP, licensing, team building
• Innovators may apply for Catalyst Grants from
their campus CTSA to support team building
2-page Pre-application
• Centralized online submission
• The two-page pre-application contains:
–
–
–
–
description of the invention
its potential market and impact
description of competitive landscape
whether pre-clinical or early clinical proof of
concept is achieved or imminent
– what is needed to make the invention licensable
– proposed budget
• Review Panels (one for each platform
technology) review pre-applications for
scientific merit and commercial potential
Review Panels
• Selected by Domain Experts
• Includes external experts from industry
and internal or external academic
experts
• Domain Experts do not participate in
pre-application review
• At least some Review Panel members
should be expert in platform under
review
Technology Solicitation Tasks
for First 100 Days
• Develop RFP for 2-page
pre-application
• Create Webinar about submission
process
• Develop online submission “package”
• Each campus site develop plan for
advertising RFP
• Recruit Review Panel members
Discussion
• Review criteria for pre-application
• Scoring for pre-application
• Local or central pre-application review?
• Size of review panel(s)?
• Begin with one platform or solicit all
three at once?
• Are campuses prepared to provide
guidance with IP, licensing, etc.?
• Date for RFP release
Technology Selection: Full Application
RFP
Pre-application
Pre-application
Review
Full
Application
Leadership
Review
External Selection
Committee Review
NHLBI Review
Technologies
Selected for
Entrance to Center
Full Application
• By invitation
• Central RFP submission
• Full-proposal format:
– Summary, including objectives
– Background, including research strategy
– Product development & commercialization
– Strategic partnerships
– Budget
• 3 months to submit
Full Application
• Review Criteria
– Unmet medical need
– Development feasibility
– Commercial attractiveness
– Intellectual property status
– Relevance to NHLBI mission
– Metrics for success
o Evidence of target validation (therapeutic)
o Time and cost of prototyping (device)
o Combination of the above (diagnostic)
Full Application: Leadership Review
• Reviewers
– Executive Committee
– Center Director and Assoc. Director
– Domain Leaders for disease and platforms
under review
– As needed: Ad hoc reviewers from
academia, industry, venture capital with
specific expertise in the diseases and
platforms under review.
• 1 month to conduct Leadership Review
Full Application:
External Selection Committee
• Prioritize applications received from
Leadership Review
• Same review criteria as Leadership Review
• 1 month to review
• Submit recommendations to NHLBI
• Reviewers
– No fewer than 5 members; present and former
faculty and/or industry leaders
– Must be external to institution(s)
Full Application: Tasks for First 100 Days
• Develop RFP for full applications
• Develop online submission
“package” for full application
• Begin to identify ad hoc reviewers
for Leadership Review
Discussion
• Limit the number of applicants who
are invited to submit full proposals?
• How many pages for full proposal?
• Include NIH review criteria?
• Scoring system?
Technology Selection: Second Chances
RFP
Pre-application
Pre-application
Review
Full
Application
Referral
Leadership
Review
Referral
External Selection
Committee Review
Consultation
Award
NHLBI Review
Technologies
Selected for
Entrance to Center
Consultation Awards
• Eligibility
– Proposal not selected for Center
– Leadership Review or External Review
recommends Consultation Award
consideration
• Funded by campus CTSAs
• Amount and duration of awards vary
– Most awards for 3-6 months
• Recipients must agree to resubmit and
target a specific RFP for resubmission
Consultation Awards
• Consultation Awards will address gaps
in following areas:
– In vivo proof of principle
– Hypothesis testing
– IP assessment
– Target product profile discussion
– Regulatory assessment
– Further development planning
• Skills Development Program will match
awardees with mentors and expertise
Consultation Awards:
Tasks for First 100 Days
• Each CTSA determines feasibility of offering
Consultation Awards
• Skills Development Program begins to identify
expertise in the following:
– In vivo proof of principle
– Hypothesis testing
– IP assessment
– Target product profile discussion
– Regulatory assessment
– Further development planning
Discussion
• Mechanics of Consultation Award
– Application process or automatic review based
on referral?
– Standard or local criteria?
– Timeframe for review?
– Who conducts review?
• Size of Consultation Award
– Set an upper limit or local decision?
• Resubmission Process
– Pre-application or directly to full review?
Technology Development Process
Technology
Enters
Center
Project
Design
Project
Design
Team
Project Plan
Product
Development
Licensing
Project Management Team
Exit
Center
Technology Development
• Two phases
– Project design
– Project management
• Collaboration with campus IP office
during entire development process is
critical
• Simultaneous skills development
– We develop the innovation and the
innovator
Project Design
• Center Director names Project Design
Team
– Platform expert(s)
– Disease expert(s)
– Campus Technology Transfer Officer
– Project manager
– Innovator
• Two months to develop project plan
• Must be approved by Center Director
Elements of Project Plan
• Product profile and exit strategy
• Task definition
• Resource identification
• Identification of critical path and milestones
• Gantt charts for
–
–
–
–
–
task responsibility and ordering
time to task completion
activity-based budgets
process for tracking project against milestones
process for periodic review
Product Profile Example
Device Development
Profile
Unmet
Clinical
Needs
Testing /
Validation
IP/FDA
Technology /
Components
Hardware /
Software
Validation
Intellectual
Property
Cost Breakout
Animal Model
FDA
Device Class
Approval Path
Good
Laboratory
Practices
Pilot Human
Study
Commercial
Partner /
FDA
Gantt Chart
Q1
Procedure
Quality
Control/Records
Components/Assembly
Testing/Validation
Animal model
Evaluation-decision
Pilot study-human trial
Commercial partner
FDA
Q2
Q3
Q4
Jan Feb Mar Apr May Jun July Aug Sept Oct Nov Dec
Year 2
Project Management
• Site leader assembles team to manage product
development
• Team includes at a minimum:
–
–
–
–
–
Site leader
Project manager
Innovator
Campus Technology Transfer Officer
Mentors assigned to innovator by Skills Development
Program
•
Team may also include ad hoc disease, platform
or business experts assigned by Site leader
•
Project managers report to Site leaders
Monitoring Progress
• Continually assess progress against
milestones
• Terminate projects that do not make
adequate progress
• Use risk-mitigation strategies to permit
multiple paths to success
• Center Director is final authority for go/
no-go decisions
Go/No-Go Decisions
Potent,
Selective
Compounds
Test PK
Properties
All
Compounds
Sufficient t ½
and oral
bioavailability
Intraperitoneal
In Vivo
Assays
Oral In Vivo
Assays
Sufficient
Therapeutic
Index
Insufficient
Therapeutic
Index
Sufficient
Therapeutic
Index
Insufficient
Therapeutic
Index
Go
No
Go
No
Exit Strategies
Development at the
Center
Continued
evaluation by
Center leadership
Exit Processes
Licensing
Project aborted by PI
Further management
by Technology
Transfer Office
Licensing not
achieved
Development
Outside the Center
Review by
Technology
Transfer
Office
Low future
interest
Return IP
High future interest
Further incubation or
marketing
Technology Development:
Tasks for First100 Days
• UCLA and UCSF identify possible project
managers
• Each campus begins to identify internal and
external disease, platform and business experts
for project design and project management
teams
• Site leaders liaise with campus Technology
Transfer Office
• Establish committee of Technology Transfer
Officers from each campus to advise the Center.
• Campuses identify cores that will be available to
CAI innovators
Technology Development Discussion
• How do we make unique cores on each of
our campuses visible to innovators at all
campuses?
– Can CTSA Translational Technologies and
Resources Programs coordinate this effort?
• What must be done to make cores
available to all innovators with minimal red
tape?
• Process for organizing committee of
Technology Transfer Officers from each
campus
Skills Development