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Agenda: Workshop on Venture Philanthropy Strategies Used by Patient Organizations to Support Translational Research

Meeting Goals

  • Convene a group of key stakeholders and experts representing a variety of patient organizations involved in venture philanthropy supported translational research to identify and discuss best practices used that may improve efficiency and effectiveness. Areas that will be discussed include:
  • Criteria used to determine whether there is sufficient evidence to justify moving into translational research – both translational research from basic to clinical and from clinical research toward practice recommendations for the development of a novel drug and/or biomarker/diagnostic;
  • Organizational structure, financial models, legal issues;
  • Partnerships, data sharing, and IP;
  • Communication strategies.

 Workshop Scenario:

     Session specific scenarios were developed to help guide remarks and frame the discussions and goals for the workshop. At the heart is a hypothetical patient organization, The Colten Foundation. An individual from this foundation is seeking advice on establishing a translational research program for the development of a novel drug and/or biomarker/diagnostic. The foundation is trying to plan for and think about the challenges they may encounter and is interested to learn more about how your organization tackled them (where applicable); other best practices, general advice, and lessons learned would also be extremely helpful.
     To the best of your knowledge there is no resource currently available that provides advice or “best practices,” so it is important that you convey the most critical information that can assist this individual in their series of decision matrices. The following questions help frame the context:

1) What advice would you want to provide to this individual and their organization?

2) What do you wish you had known then, that you know now?

3)  If I had only known X, I could have avoided Y.

8:30 a.m.          Welcome Introductions and Workshop Objectives

Timothy Coetzee, Chair
Executive Director
Fast Forward/ National Multiple Sclerosis Society

8:40 a.m.          Opening Remarks: Opportunities for Foundation Support

Joyce Nelson
President and Chief Executive Officer
National Multiple Sclerosis Society

9:05 a.m.          Challenges and Opportunities for Neurological and Mental Health Translational Research: Basic to Clinical Research and Clinical toward Practice 

Dennis Choi
Executive Director, Strategic Neurosciences Initiative
Director, Comprehensive Neuroscience Center
Emory University

Session I: Preparing for a translational Research program

Session Objective:  Discuss the challenges and issues in the development of a translational research program for the development of either a novel drug or biomarker.

Session I Scenario

 An individual from The Colten Foundation, a patient organization that has sufficient resources, has left you a voice mail asking for advice about how they should go about establishing and maintaining a productive and efficient translational research program. They are particularly interested in the criteria considered and the decision matrix you and your organization used to determine whether or not your organization would establish a formal translational research program for the development of a novel drug and/or biomarker/diagnostic.  

9:20 a.m.          Introduction to the Session

Maria Carrillo, Session Chair
Director, Medical Scientific Relations
Alzheimer's Association

9:25 a.m.          Panelist Remarks

Richard Insel
Executive Vice President, Research
Juvenile Diabetes Research Foundation 

Katie Hood
President and Chief Executive Officer
Michael J. Fox Parkinson’s Research Foundation

Celia Dominguez
Vice President, Chemistry
CHDI Management, Inc.

10:05 a.m.        Respondent Panel Discussion

Louis DeGennaro
Chief Scientific Officer
The Leukemia & Lymphoma Society

Cynthia Joyce
Executive Director
Spinal Muscular Atrophy Foundation

Lucie Bruijn
Vice President of Research
Amyotrophic Lateral Sclerosis Association

10:20 a.m.        Discussion with Attendees

Maria Carrillo, Moderator
Director, Medical Scientific Relations
Alzheimer's Association

10:40 a.m.        BREAK

Session II: Orgranizational Structure, Leadership, and Financial Models        

Session Objective:  Explore several elements of how a program should be organized to ensure appropriate expertise and leadership is available. Examine a variety of organizational and financial models that may provide optimal support of translational research programs. 

Session II Scenario 

The individual from The Colten Foundation who previously spoke to you has used your decision matrix and decided to move ahead with a formal research program. Given how helpful you originally were, they are now asking for more information. In particular they are interested in hearing about models of organizational and leadership structure, and various financial models that you have found to be effective and efficient. 

10:55 a.m.        Introduction to the Session 

Timothy Coetzee, Session Chair
Executive Director
Fast Forward of the National Multiple Sclerosis Society

11:00 a.m.        Panelist Remarks

Jonathan Simons
President and Chief Executive Officer
Prostate Cancer Foundation 

Cynthia Joyce
Executive Director
Spinal Muscular Atrophy Foundation

11:40 a.m.        Respondent Panel Discussion

Jonathan Jacoby
Chief Operating Officer
CollabRx, Inc.

Linda Van Eldik
Associate Director, Northwestern Alzheimer's Disease Core Center
Northwestern University

11:50 a.m.        Discussion with Attendees

Timothy Coetzee, Moderator
Executive Director
Fast Forward of the National Multiple Sclerosis Society

12:15 p.m.        LUNCH

Session III: LEGAL, accounting, and process ISSUES

Session Objective: Explore the challenges and opportunities in legal, accounting, and associated process issues that a foundation would need to consider in conducting venture philanthropy translational research programs.

Session III Scenario

You are now very popular with The Colten Foundation! They just sat down with their start-up team and are trying to think about some of the new challenges they may face in terms of appropriate accounting and legal practices, and other process-related issues. They have once again called you to learn more about how you and your organization have navigated these issues.

1:00 p.m.          Introduction to the Session

Timothy Coetzee, Chair
Executive Director
Fast Forward/ National Multiple Sclerosis Society

1:05 p.m.          Panelist Remarks

Andrea Tobias
Portfolio Advisor
Fast Forward, LLC                         

Kenneth Schaner
General Counsel
Kenneth Schaner, PC

Peter Heinecke
Chief Business Officer
Experimed Bioscience, Inc.

1:45 p.m.          Respondent Panel Discussion

Carol Mimura
Assistant Vice Chancellor 
Intellectual Property & Industry Research Alliances (IPIRA)         
University of California, Berkeley               

Jennifer Taylor
Associate Director, Program Management
Head of External Alliances
Genomics Institute of the Novartis Research Foundation

1:55 p.m.          Discussion with Attendees

Timothy Coetzee, Chair
Executive Director
Fast Forward/ National Multiple Sclerosis Society                      

Session IV: partnerships, Data Sharing, and IP 

Session Objective:  Examine a variety of strategies for partnering with non-profit organizations, the NIH, and industry. Discuss what policies that have been effective in facilitating the efficient management of data sharing and IP. 

Session IV Scenario

The Colten Foundation has previously been working with just a couple other researchers or companies, but realizes that in order to develop an effective research program they will need to expand their portfolio of collaborators (e.g., non-profit organizations, the NIH, therapeutic/diagnostic industry). Of course, this results in more questions than answers, especially around issues of data sharing and IP. They again turn to you to help answer their questions. Are there any common resources available? What are some challenges they may encounter – and how your organization tackled them? How and when should strategic collaborations be established or expanded?

2:10 p.m.          Introduction to the Session

Judy Illes, Session Chair
Professor
Neurology
University of British Columbia

2:15 p.m.          Panelist Remarks

Margaret Anderson
Chief Operating Officer
Faster Cures

Sharon Hesterlee
Vice President, Translational Research
Muscular Dystrophy Association

Tricia Brooks
Managing Director, Alliance Development
Biotechnology Industry Organization

2:55 p.m.          Respondent Panel Discussion

Susan Wolf
McKnight Presidential Professor of Law, Medicine & Public Policy
University of Minnesota School of Law

3:05 p.m.          Discussion with Attendees

Judy Illes, Moderator
Professor
Neurology
University of British Columbia

3:20 p.m.          BREAK

Session V: Communications

Session Objective: Explore effective communication strategies that can be used to disseminate information on research and other scientific progress to potential and current funders as well as to constituents, while addressing the challenges of funder and constituent expectations.

Session V Scenario

The individual from The Colten Foundation is calling again, and has promised that this will be the last time she will call, but she has one last set of very important questions. Based on everything they have already learned from you, their translational research program is making great headway. They are very excited to share the progress with current and potential funders, as well as their constituents. But, she also recognizes the delicate balance that is needed between demonstrating progress and limiting false expectations. Therefore, she is very interested in discussing effective communication strategies that you have used for disseminating information on research and other progress to potential funders and constituents.

3:30 p.m.          Introduction to the Session

Jonathan Jacoby, Session Chair
Chief Operating Officer
CollabRx, Inc.

3:35 p.m.          Panelist Remarks

Sharon Terry
President and Chief Executive Officer
Genetic Alliance

Dan Zenka
Vice President, Communications
Prostate Cancer Foundation

Cathy Carlson
Senior Director, Research Information
Research and Clinical Programs Department
National Multiple Sclerosis Society

Sophia Colamarino
Vice President, Research
Autism Speaks

Randall Carpenter
Co-Founder, President and Chief Executive Officer
Seaside Therapeutics

4:25 p.m.          Discussion with Attendees

Jonathan Jacoby, Moderator
Chief Operating Officer
CollabRx, Inc.

Session VI: Wrap-up Discussion

Session Objective: In addition to the areas that have already been discussed, are there additional topics that require exploration? What “next steps” should the IOM’s Neuroscience Forum consider as it moves ahead in this area? How can we capture the innovation and ideas discussed during the meeting to assist you and new colleagues working in these areas?

4:40 p.m.          Discussion with Attendees

Timothy Coetzee, Chair
Executive Director
Fast Forward/ National Multiple Sclerosis Society

5:00 p.m.          ADJOURN

Back to Meeting

Study Staff

Activity Contact Information

For More Information Contact

Board on Health Sciences Policy
Phone:
202-334-1888
Fax:
202-334-1329
E-mail:
neuroforum@nas.edu

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