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Wednesday, June 26 • 8:00am - 9:15am
#319: How to Solve the Problem of Access for Rare Diseases

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-663-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

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.


Sissi Pham, PharmD


Kari Luther Rosbeck

Nicole Boice

Scott Demarest, MD

Sarah Pitluck, MSc

Sean Tunis, MD, MSc

avatar for Sissi Pham

Sissi Pham

Chief Executive Officer, AESARA
Sissi Pham is a founder and CEO of AESARA, Inc. and AESARA Foundation, both established in 2016. AESARA, Inc. was established with the commitment to absorb the administrative cost of the AESARA Foundation to enable the charity to give 100% of the donations to directly to humanitarian... Read More →
avatar for Kari Rosbeck

Kari Rosbeck

President and Chief Executive Officer, Tuberous Sclerosis Alliance
Kari joined the TS Alliance in June 2001 and became President and CEO in November 2007. Kari has been involved in nonprofit fundraising and volunteer management for nearly 30 years. During Kari’s tenure as President and CEO, the TS Alliance established a comprehensive research program... Read More →
avatar for Sarah Pitluck

Sarah Pitluck

Head, Global Pricing and Reimbursement, Spark Therapeutics
Ms. Pitluck is the Head of Global Pricing & Reimbursement (P&R) at Spark Therapeutics. In her current role, Ms. Pitluck priced the first FDA-approved gene therapy for a genetic disease, LUXTURNA®. She is now working to ensure market access for all US patients in need of LUXTURNA... Read More →

Nicole Boice

Founder, Global Genes Project

Scott Demarest

Assistant Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine; Children's Hospital Colorado
Scott Demarest received a Bachelor of Science in biology from the University of Texas at Austin before going on to medical school at the University of Texas Health Science Center in San Antonio. He completed his residency in Pediatrics and child neurology at Children's National Health... Read More →

Sean Tunis

Senior Strategic Advisor, Center For Medical Technology Policy (CMPT)

Wednesday June 26, 2019 8:00am - 9:15am PDT
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  12: Value-Access, Forum |   04: MedAffairs-SciComm, Forum |   05: Patient Engagement, Forum
  • Level Beginner
  • Featured Topics Patient Focused,Rare Disease
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum