Michael Palazzolo

UC CAI

Face-To-Face MeetingOctober 14, 2013

University of California Center for Accelerated Innovation

Local and National Announcements

• Posted by UCLA on Sept 30, 2013 (day before the government shutdown)

• Aired October 1, 2013

CTSA Central NBC Channel 4 News

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

Michael Palazzolo, MD, PhDCenter Director, UC CAI

Professor, UCLA

Leadership

UC CAI

Tomas Ganz, MD, PhDCenter Associate Director

Professor, UCLA

Leadership

Executive Committee

Lars Berglund, MD, PhDDirector, UCLA Clinical

and Translational Science Center

Steven Dubinett, MDDirector, UCLA Clinical

and Translational Science Institute

Gary S. Firestein, MDExecutive Committee ChairDirector, UCSD Clinical and

Translational Research Institute

Clay Johnston, MD, PhDDirector, UCSF Clinical and

Translational Science Institute

Dan Cooper, MDDirector, UCLA Institute for

Clinical and Translational Science

Site Leaders

Campuses

June Lee, MDUCSF

Shaun Coughlin, MD,

PhDUCSF

Sotirios Tsimikas, MDUCSD

Joseph Witztum,

MDUCSD

Laura Marcu, PhDUC Davis

Steven George, MD,

PhDUC Irvine

Tomas Ganz, MD, PhDUCLA

Domain Leaders

Diseases

June Lee, MDLung and Sleep

Diseases

Shaun Coughlin, MD,

PhDCardiovascular

Disease

Sotirios Tsimikas, MDCardiovascular

Disease

Tomas Ganz, MD, PhDBlood Disorders and Resources

Domain Leaders

Platforms

June Lee, MDCo-Leader,

Therapeutics

Shaun Coughlin, MD,

PhDCo-Leader,

Therapeutics

Sotirios Tsimikas, MDCo-Leader, Diagnostics

Joseph Witztum, MDCo-Leader, Diagnostics

Laura Marcu, PhDCo-Leader,

Devices and Tools

Steven George, MD,

PhDCo-Leader,

Devices and Tools

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 KrishnanLeader, 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

Center Director

Associate Director

Domain/Site Leaders

Cardiovascular

Therapeutics Devices

Lung & Sleep Disorders

BloodDiseases

Diagnostics

Domain Areas

Projects

Program Resources

Administrative& Budgetary

Support

Website & Data Management

Industry Relations & IP

CTSA Infrastructure

Evaluation & Tracking

Project Management

External Selection

Committee

Business Review Panel

Skills Development Program

External Advisory Board

Executive Committee

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, PhDFormer Senior VP, Pfizer

Founder, MindPiece Partners

Francis Duhay, MDVP Medical Affairs and CMD,

Edwards Lifesciences

Lawrence Souza, PhDFormer 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, PhDUCLA Initial ChairAnderson 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

Pre-applicationReview

RFP Pre-application

FullApplication

External Selection Committee Review

NHLBI Review

Leadership 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 $200KSolicit Pre-

Application

Review Pre-

Application

1 month

Develop and Submit Full Application

First Review of

Full Application

ESC Review of

Application

1 month 1 month 1 month3 months

Pre-application and Review

Pre-applicationReview

RFP Pre-application

FullApplication

External Selection Committee Review

NHLBI Review

Leadership 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

Pre-applicationReview

RFP Pre-application

FullApplication

External Selection Committee Review

NHLBI Review

Leadership 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 successoEvidence of target validation (therapeutic)

oTime and cost of prototyping (device)

oCombination 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

Pre-applicationReview

RFP Pre-application

FullApplication

External Selection Committee Review

NHLBI Review

Leadership Review

Technologies Selected for

Entrance to Center

Referral

Referral

Consultation Award

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 EntersCenter

Project Design Project Plan LicensingProduct

DevelopmentExit

Center

Project Management TeamProject Design Team

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 ExampleDevice Development

Profile

Unmet Clinical Needs

Technology / Components

Cost Breakout

Good Laboratory Practices

Testing / Validation

Hardware / Software Validation

Animal Model

Pilot Human Study

IP/FDA

Intellectual Property

FDA Device Class

Approval Path

Commercial Partner /

FDA

Gantt Chart

 Q1 Q2 Q3 Q4 Year 2

Procedure Jan Feb Mar Apr May Jun July Aug Sept Oct Nov Dec     

Quality Control/Records                              

Components/Assembly                 

Testing/Validation                 

Animal model               

Evaluation-decision           

Pilot study-human trial               

Commercial partner             

FDA             

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 DecisionsPotent,

Selective Compounds

Test PK Properties

All Compounds

Intraperitoneal

In Vivo Assays

Sufficient Therapeutic

Index

Go

Insufficient Therapeutic

Index

No

Sufficient t ½ and oral

bioavailability

Oral In Vivo Assays

Sufficient Therapeutic

Index

Go

Insufficient Therapeutic

Index

No

Exit Strategies

Continued evaluation by

Center leadershipLicensing

Review by Technology

Transfer Office

High future interest

Further incubation or marketing

Development at the Center Exit Processes Development

Outside the Center

Project aborted by PI

Further management by Technology Transfer Office

Licensing not achieved

Low future interest

Return IP

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