Investigator Initiated-Sponsored Research (IISR)

State of the Industry and the Need for Global Standards and Metrics

Alexander Kostek and DeeAnn Tinjum

Disclaimer

The views and opinions expressed by the presenter and set forth in the following PowerPoint slides are those of the individual presenters and should not be attributed to Pfizer Inc., or any of their respective directors, officers, employees, or any organization with which the presenter is affiliated.

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Objectives Understand the current state of IISR programs globally

and the need for minimum standards and comprehensive programs

Describe the current regulatory and legal environment for IISR programs and understand current industry led initiatives for IISR standards

Explain the mission and vision for IISRA

What is IISR? Investigator Initiated Research/Studies/Trials

Investigator Sponsored Research/Studies/Trials

Physician Initiated Research/Studies/Trials

Physician Sponsored Research/Studies/Trials

Investigator Initiated Sponsored Research (IISR)

Current Global Environment Lack of clear, specific Regulatory Guidelines for IISR Varying global definitions of sponsorship Unfamiliarity with research by Investigators Inconsistencies in Industry policies Inconsistencies in Industry resources & support

Regulatory & Legal Considerations Food, Drug and Cosmetic Act International Conference Harmonization (ICH)/Good

Clinical Practice (GCP) Guidelines US Department of Health & Human Services Federal Code of Regulations (CFR Section 21) OIG: Compliance Program Guidance Sunshine Act

Federal Anti- Kickback Statute

Regulatory & Legal Considerations, cont. False Claims Act (FCA) Foreign Corrupt Practices Act (FCPA) European Unions Clinical Trial Directive European Medical Research Council (EMRC) of the

European Science Foundation PhRMA and AdvaMed Codes

Proposal Submission Identify submission method Ensure transparency & availability to the public

Provide templates and clear directions Document receipt Ensure completeness of proposal

Proposal Review Committee reviews using well defined criteria:

Company Strategy Scientific Merit Patient Safety Fair Market Value (FMV) Expertise of the Investigator Scientific, Legal and Regulatory considerations

Committee decision is documented Decision communicated to the submitter

Example of Review Process Research interest received

Medical Affairs does initial review,

application packet sent if warranted

Submitter completes standard IISR

application form Team Review

Decision communicated to submitter

Denial

Submitter updates proposal

Conditionally Approved

Submitter provided IISR Start Up

Packet

Finalize Contract / Legal Review

Approved (and communicated to submitter)

IISR authorized and research

initiated

Deferred (IISR to be re-evaluated by team)

Proposal Review, cont. Provide a IISR packet Company specific

Review Protocol Prior to IRB/EC submission

Record comments Communicate to submitter

IISR Agreement Develop an IISR agreement template including: ◦ Compliance with laws and regulatory requirements ◦ Support provided by company & how it is provided ◦ IND or IDE submission, if applicable ◦ Registration of study ◦ Safety reporting ◦ Study progress reports from sponsor ◦ Publication expectations ◦ Intellectual Property ◦ Company rights for review prior to submissions ◦ Indemnification ◦ Term and termination

Overall Program Management Communicate with the sponsor-investigator Collect study progress reports Distribute funding and/or product Maintain study documents Ensure Safety Reporting Develop Metrics Obtain deliverables for internal review Perform close out

IISRA - Mission Statement

IISRA is an independent association of professionals dedicated to promoting excellence in investigator initiated-sponsored research (IISR) programs. Our commitment is to support members through the development and implementation of educational activities, professional knowledge standards, and ethical practices.

Membership

Approximately 300 professionals Pharma, biotech, device, academia, CROs, technology vendors,

consultants Growing European Interest

Benefits: Communication forum for consensus building Networking with global IISR professionals Tools and information Access to IISRA Position Statements and Guidelines Stay abreast of new trends/benchmarking Free to join: www.iisra.org.

IISRA Activities Standards and Best Practices Guidance Documents Position Statements

Implementation of IISRA By-Laws Pulse on Compliance/Ethics Issues Surveys and Benchmarking Leadership Presence at National Organizations/Conf. Networking Job Postings (Future)

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2011/2012 Strategic Goals

Broaden Communication Among Members Advance the Value of the IISRA Org Sustain IISRA’s Growth

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Goal: Broaden Communications

Increase IISR presence and community dialogue through on-line media Accomplishments IISRA group established on LinkedIn (Jan 2011) Updated www.iisra.org website (Feb 2011)

Focus IISR presence at industry conferences in the USA Accomplishments (Chair/Speaker/Moderator) Center for Business Intelligence (CBI)

Standards/Best Practice Webinar_Sponsor is SteepRock (Jan 2011) Collaborative Group Initiative (Mar 2011, Oct 2011) CBI IIS Conference (June 2011)

ExL Pharma IIT Conf in USA (Apr 2011, Sept 2011, Apr 2012) Q1 Productions_Medical Device in USA (Sept 2011) DIA - IIS Trial Master File (TMF) Content Model (Jan 2012) ACRP – Abstract and Oral Presentation (April 2012)

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Broaden Communications, Con’t

Support IISRA values Accomplishments Conduct IISRA annual elections (2011; 2012)

IISRA By-Laws (July 2011)

Increase exposure to non-industry partners (government, academia) Future Regulatory links and training slides for Investigators Greater interface and input with FDA

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Goal: Advance Value of IISRA Drive the creation / completion of new position statements and guidelines

Accomplishments Metrics Guidance Document (Mar 2011)

IISRA surveys - future guidance topics (Apr and June 2011)

Ongoing committees:

Industry collaboration with Cooperative Groups

Polaris initiates FMV (interviews in May 2011; draft Jan 2012)

Initiating four new documents

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Advance Value of IISRA (Con’t) Expand the usages / offerings through IISRA.org Future Job postings Networking

Create additional value for the biopharm and device industries, and also for government / academia Future 2012 member survey Links to portals for industry grant applications

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Goal: Sustain IISRA Growth Achieve a target membership of 500 registrations globally

Accomplishment: Membership doubled from approx 180 to 300 members

Increase presence in Europe, Asia/Pacific & emerging markets

Accomplishments: DIA_Europe: Intro of IISRA to EU community (Mar 2011) Q1 Productions (Frankfurt, Germany) – Chair/Speaker IISRA Board increased from one to four EU members

Plan and execute at least two (2) IISRA general sessions 2012 annual meeting via conference attendance and webinar

Build strategic partnerships (FDA, Medical Affairs)

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Initiatives With Other Organizations

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National Organizations

Inter- National Conferences

Industry Leaders Technology Vendors

National Cancer Institute (NCI)

Industry_Posting of Grant Applic. Links

Polaris

Clinical Trials Network (CTN)

Committees for Position/Guidance Docs

SteepRock

DIA (USA) DIA (Europe) MedNet Solutions

ACRP

ExL Pharma IIT ExL Pharma IIT (Europe)

CBI ISS CBI Coop Groups

Q1 Prod (USA) Q1 Prod

IISRA Board of Directors (2012-2013) Chairperson: Karen Bartels (AstraZeneca) Officers: President: Anton Ehrhardt (Sanofi) Vice President: Ran Frenkel (Clinipace_Europe) Treasurer: Nader Molavi Secretary: Patricia Westergren (MedNet) Executive Committee: Executive Director: Alex Kostek (Pfizer) Members: Dan Bromberg (Polaris) Jocelyn Ulrich (HGSI) Ornah Levine-Dolberg (Lundbeck) Jonathan Chambers (Pierre-Fabre) Ouadah Hadjebi (Editor, IST Journal)

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IISRA

Guidance Documents / Position Statements

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Guidance Documents and Position Statements

Completed Documents - Currently Available on the IISRA Website (www.iisra.org) IISRA Mission Statement Standards and Best Practices Metrics Guidelines Setting Up an IISR Program Statement on Fair Market Value Future Documents - IISRA conducted a survey at the ExL Pharma and CBI Conferences in 2011 to determine topics of interest

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Survey Results: Desired Topics New Committee Initiatives: Fair Market Value (FMV) - Initiated Collaborative Initiative with NCI - Initiated Contract Bottlenecks Publications CTMS System for IISR Programs Sunshine Act

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A thank you for their contributions! Cynthia Barbitsch Karen Bartels Shiferaw Kibriye Michael Montgomery Wendy Moore Matthew Silverman DeeAnn Tinjum Mary Voehl Pat Westergren