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How to Solve the Problem of Access for Rare Diseases
Session Chair(s)
Sissi Pham, PharmD
Chief Executive Officer
AESARA, United States
This panel will share learnings and build on outcomes from the recent Rare Access to Critical Therapies (ACT) Stakeholder Summit convened by Global Genes and the Child Neurology Foundation, two patient advocacy organizations which brought together more than 200 individuals representing key stakeholders in the rare disease drug development space to build awareness and understanding about access to and pricing of rare disease therapies.
Learning Objective : Discuss critical access issues related to rare disease drug development; Describe lessons learned from recent experiences to improve communication and collaboration among stakeholders and ensure the patient and caregiver needs are truly understood, considered and incorporated into access and pricing decision-making.
Speaker(s)
Panelist
Kari Luther Rosbeck
Tuberous Sclerosis Alliance, United States
President and Chief Executive Officer
Panelist
Nicole Boice
Global Genes Project , United States
Founder
Panelist
Scott Demarest, MD
University of Colorado School of Medicine; Children's Hospital Colorado, United States
Assistant Professor, Departments of Pediatrics and Neurology
Panelist
Sarah Pitluck, MSc
Spark Therapeutics, United States
Head, Global Pricing and Reimbursement
Panelist
Sean Tunis, MD, MSc
Rubix Health LLC, United States
Founder and Strategic Advisor, CTMP; Founder
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