A wide range of issues impact development, availability, and accessibility of treatment and services for genetic and rare diseases. Policy is one tool that can be used to address the system for development of treatments and help ensure access to treatments when available; however, are current policies adequate to create a sustainable future for orphan treatments? How can disease characteristics and disease registries provide evidence to inform delivery of services and public policy? Is policy in the form of insurance mandates enough to ensure access to treatment, or are there other significant barriers? This workshop will engage participants in thinking about these important questions through presentations from the industry perspective, using the example of lysosomal storage diseases, and from the public health perspective, using the example of metabolic diseases identified through newborn screening.

Presenters:
Sarah Scollon — Genetic Counselor, Project Specialist, Hawaii Department of Health & Hawaii Community Genetics
John Yee — Vice President, Global Medical Affairs, Genzyme Corporation