#WODC USA Session preview- Rare Diseases: From Endpoints to Endgame

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#WODC USA Session preview -Rare Diseases: From Endpoints to Endgame by Red Team Associates

Normally, there is a clinical signal that sets commercial planning and development going. In the rare disease world, however, the noise in the market goes ahead of the clinical signal. ‘Why can’t my child get the orphan drug you’re developing?’ is a classic indication of this situation. And it is problematic.

The logical, simple antidote? Get the commercial folks involved from the get-go.

 This 4-hour workshop will showcase a patient perspective to the above-described scenario in the rare disease world, along with how three orphan product developments manage the market noise way before they were commercialized.

1:00        Introduction to the workshop
Harris Kaplan, Managing Partner, Red Team Associates 

1:10        At home with Centronuclear and Myotubular Myopathies: a parent’s perspective
Paul Frase, Founder, Joshua Frase Foundation

1:30        Getting a new drug to a rare disease market
Chris Garabedian, Former CEO, Sarepta Corporation

1:40        Coordinating clinical and commercial views to develop a compelling value proposition
Harris Kaplan, Managing Partner, Red Team Associates

2:40        Coffee break

3:10        Endpoints that matter to patients and their families

Linda Deal, Senior Director of Patient Centered Outcomes Assessment, Pfizer 

3:20        Extensive orphan drug clinical development experience from Shire to Novartis
Lawrence Charnas, MD, PhD, Sr. Translational Medicine Expert, Musculoskeletal Diseases, Novartis Institutes for Biomedical Research

3:30        Roundtable discussions

Moderators:
Chris Garabedian, Former CEO, Sarepta Corporation
Linda Deal, Senior Director of Patient Centered Outcomes Assessment, Pfizer
Lawrence Charnas, MD, PhD, Sr. Translational Medicine Expert, Musculoskeletal Diseases, Novartis Institutes for Biomedical Research

4:30        Feedback from each group

4:55        Closing summary

Harris Kaplan, Managing Partner, Red Team Associates

5:00        End of workshop and Welcome Drinks Reception 

Sponsored by Red Team Associates 

Register your ticket now for you chance to get involved in this workshop!

World Orphan Drug Congress USA 2016

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