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Day Two In-Conference Tracks (IV-VI)

Thursday, October 22, 2015

Track IV-Current Topics in Rare Diseases-FDA

9:00

Track Chairperson’s Welcome

John Whyte

Director, Professional Affairs and Stakeholder Engagement, CDER, FDA

9:15

Patient-Focused Drug Development-Next Steps

Theres Mullin, Ph.D.

Director, Office of Strategic Programs, CDER, FDA

9:45

Clinical Outcome Assessments-Patient Reported Outcomes

Elektra Papadopoulos

Acting Associate Director, Clinical Outcome Assessments Staff, Office of New Drugs, CDER, FDA

10:15

Networking Break

Sponsored by:

TMS Health

10:45

Medical Devices

Jeffrey Shuren, M.D., J.D.

Director, Center for Devices and Radiological Health (CDRH), FDA

 

11:15

Professional Affairs of Stakeholder Engagement 

Naomi Lowy, M.D., 

Senior Medical Officer, Professional Affairs and Stakeholder Engagement, CDER, FDA

 

11:45

Pediatrics Rare Disease Priority Review Voucher 

Jonathan Goldsmith

Rare Diseases Program, Office of New Drugs, CDER, FDA

12:15

An Office of New Drugs Update on Rare Diseases

John Jenkins, M.D.

Director, Office of New Drugs, FDA

12:45

Close of Tracks and Networking Luncheon

In-Conference Tracks

I

Expanded Access

II

Facilitate Early Diagnosis and Treatment

III

Be Visible, Be Active, Be Heard

IV

Current Topics in Rare Diseases-FDA

V

Fueling Progress and Access

VI

Patient-Centered Outcomes Research

Track V-Fueling Progress and Access

9:00

Track Chairperson’s Welcome

Valerie Sullivan

EVP and Managing Director, Patient Access Solutions, TMS Health, A Xerox Company

9:15

Coordinate the Channels to Ensure Coverage-Specialty Pharmacy, Hub and Distribution Strategies to Facilitate Reimbursement 

Pamela Gavin

Chief Operating Officer, NORD

Jessica Black

Director, Xcenda

Nicole Hebert

Vice President, Commercial Services Operations, UBC

 Mark Vineis

Vice President, Cardinal Health Specialty Pharmacy

Dave Penake

Vice President, Commerical Operations, Corcept Terapeutics

 

10:15

Networking Break

Sponsored by:

TMS Health

10:45

Facilitate the Progress-Investing in Innovation and Research in Rare Diseases

James Geraghty

Entrepreneur in Residence, Third Rock Ventures

Peter Day

Managing Director, Healthcare Investment Banking, Piper Jaffray

Nicholas Galakatos

Managing Director, Clarus Ventures

George Milstein

Co-Head of Healthcare Investment Banking, Cowen Group

Daniel Krizek, CFA, CAIA

Director, Brookside Capital

Ann DeWitt, Ph.D., MBA

Senior Director of Investments, Sanofi-Genzyme BioVentures

 

11:45

Personalized and Precision Medicine-What Does This Mean for People with Rare Diseases?

Barbara Conley, M.D.

Associate Director, Cancer Diagnosis Program, National Cancer Center

Ricki Lewis, Ph.D.

Geneticist, DNA Science Blog

Joshua Denny, M.D., M.S., F.A.C.M.I.

Associate Professor of Bioinformatics and Medicine, Vanderbilt University Medical Center

Member,NIH Precision Medicine Initiative Working Group

12:45

Close of Tracks and Networking Lunch

Track VI-Patient Centered Outcomes Research

9:00

Track Chairperson’s Welcome

Eleanor M. Perfeto, Ph.D., MS

Professor, University of Maryland School of Pharmacy; Senior Vice President of Strategic Initiatives, National Health Council 

Jacque Kraska

Research Programs Manager, NORD

9:15

Patient-Centered Outcomes Research-What it Means to the Rare Disease Community

Christine Brown, MS

Executive Director, National PKU Alliance 

Jacque Kraska

esearch Programs Manager, NORD

10:15

Networking Break

Sponsored by:

TMS Health

10:45

Reorienting the Research Environment-Advocating for the Value of the Patient’s Voice

Marc Bloutin, JD

Chief Executive Officer, National Health Council 

Ron Bartek

President and Co-Founder, Friedreich’s Ataxia Research Alliance (FARA), NORD, Board of Directors 

Pat Furlong

Founding President and CEO, Parent Project Muscular Dystrophy

 

11:30

Partnerships and Funding for PCOR Projects

Eleanor M. Perfetto Ph.D., MS

Professor, University of Maryland School of Pharmacy; Senior Vice President of Strategic Initiatives, National Health Council

Stephanie Okey, MS 

Consultant, Former Genzyme

Theresa V. Strong

Grants Program Director, Foundation for Prader-Willi Research

12:15

Patient-Centered Outcomes Research and Policy-Panel Discussion

Eleanor M. Perfetto Ph.D., MS

Professor, University of Maryland School of Pharmacy; Senior Vice President of Strategic Initiatives, National Health Council

Danielle Whicher

CER Program Officer, PCOR

Marshall L. Summar, M.D.

Division Chief, Genetics and Metabolism, Children’s National Health System

Laurie Burke, B.S. Pharm, M.P.H. 

Founder and President, The Lora Group 

 

1:00

Close of Tracks and Networking Luncheon

Key Contacts

Program Development
Sharon Langan
sharon.langan@cbinet.com
(339) 298-2115

Registration
Roberts Apse
roberts.apse@cbinet.com
(339) 298-2290

Sponsorship & Exhibit Opportunities
Alexa Moore
alexa.moore@cbinet.com
(339) 298-2107

Speaking & Poster Submissions
Curry Wilson
curry.wilson@cbinet.com
(339) 298-2105