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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
President and Chief Executive Officer, Tuberous Sclerosis Alliance, United States
Panelist
Founder, Global Genes Project , United States
Panelist
Assistant Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine; Children's Hospital Colorado, United States
Panelist
Head, Global Pricing and Reimbursement, Spark Therapeutics, United States
Panelist
Founder and Strategic Advisor, CTMP; Founder, Rubix Health LLC, United States
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