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Hyatt Regency Bethesda

2015 年 10 月 07 日 7:00 上午 - 2015 年 10 月 07 日 4:30 下午

One Bethesda Metro Center, 7400 Wisconsin Avenue, Bethesda, MD 20814-5326

Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

A collaborative workshop designed to examine practical issues in the structuring of rare diseases therapeutic development programs.

Session Chair(s)

Shaghig  Palanjian, MBA

Shaghig Palanjian, MBA

Vice President, Global Head, R&D QA and Compliance

Shire, United States

Scott  Schliebner, MPH

Scott Schliebner, MPH

Senior Vice President, Clinical Development Services

TFS Health Science, United States

Speaker(s)

David  Whiteman, MD

Rare Disease Protocol Development

David Whiteman, MD

Takeda Pharmaceutical Company Limited, United States

VP Researhc & Development

Scott  Schliebner, MPH

Leveraging Informatics to Accelerate Rare Disease Clinical Development

Scott Schliebner, MPH

TFS Health Science, United States

Senior Vice President, Clinical Development Services

Larry  Blankstein, PhD

Clinical Logistics for Rare Disease Trials

Larry Blankstein, PhD

Synlogic, United States

Head of Clinical Operations

Jonathan C. Goldsmith, MD, FACP

Overview of FDA Draft Guidance for Industry: Rare Diseases - Common Issues in Drug Development

Jonathan C. Goldsmith, MD, FACP

FDA, United States

Associate Director for Rare Diseases, Office of New Drugs, CDER

Jonathan C. Goldsmith, MD, FACP

Panel Discussion

Jonathan C. Goldsmith, MD, FACP

FDA, United States

Associate Director for Rare Diseases, Office of New Drugs, CDER

David  Whiteman, MD

David Whiteman, MD

Takeda Pharmaceutical Company Limited, United States

VP Researhc & Development

Scott  Schliebner, MPH

Scott Schliebner, MPH

TFS Health Science, United States

Senior Vice President, Clinical Development Services

Larry  Blankstein, PhD

Larry Blankstein, PhD

Synlogic, United States

Head of Clinical Operations

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