PRE-CONFERENCE FOCUS DAY

Tuesday, August 20 2019

Workshop A Tuesday, August 20

9:00 am - 12:00 pm

Understanding Immunogenicity in Next Generation Factor Replacement Prophylaxis

Workshop Leader: Grant Blouse, Vice President, Translational Research, Catalyst Biosciences Workshop Leader: Nisha Jain, Executive Medical Director, Bioverativ, a Sanofi Company

Novel therapeutic strategies have been able to gradually improve the standard of care of hemophilia treatments. The major barrier to the success of this is the development of inhibitor in the patients. Attending this workshop will help you understand how immunogenicity risk assessment studies and real world data can help you to understand the why certain factor replacement products are more immunogenic. Attendees will discuss in detail:
  • Immunogenicity in extended half-life products, non factor replacement products and gene therapy
  • Why antibodies form in response to the different treatments
  • Strategies in eliminating immunogenic response and re-instate immune tolerance

Grant Blouse, Vice President, Translational Research, Catalyst Biosciences

Nisha Jain, Executive Medical Director, Bioverativ, a Sanofi Company

Workshop B Tuesday, August 20

1:00 pm - 4:00 pm

Improving Patient Access to Gene Therapy Through Novel Payer and Reimbursement Strategies

Workshop Leader: Michelle Rice, Senior Vice President, External Affairs, National Hemophilia Foundation Workshop Leader: Brendan Hayes, Director of External Affairs, National Hemophilia Foundation Workshop Leader: Edmund Pezalla, CEO/Founder, Enlightenment Bioconsult, LLC

With the iminent approval of gene therapy, existing payment approaches need to be reassessed to ensure better patient access and better value for across the system. Engaging with payers and providers to evaluate and innovate more value-based payments will be key to the success of gene therapy in the real world. The National Hemophilia Foundation is actively working with patients, payers and providers to improve management of the costs of hemophilia treatments. In this workshop, learn about the work of the Comprehensive Care Sustainability Collaborative in improving payer and provider engagement.

Attendees will discuss:

  • Understanding what payers are looking for to evidence the curative or long-lasting benefit of gene therapy
  • Alternative payment strategies to address affordability of gene therapy
  • Challenges around implementation of new payment models

Michelle Rice, Senior Vice President, External Affairs, National Hemophilia Foundation

Brendan Hayes, Director of External Affairs, National Hemophilia Foundation

Edmund Pezalla, CEO/Founder, Enlightenment Bioconsult, LLC