Michael Rice, Sharon Hesterlee, Kristin Phillips, Nick Sireau

Presented by Muscular Dystrophy Association, Taysha Gene Therapies, AKU Society, and Cello Health BioConsulting, now part of Lumanity

This session, part of our 2021 Fall Pharma Progress event, looked at partnerships with patients and preparing for the commercialization of new, rare disease treatments.

Michael Rice, VP, Head of Advanced Therapeutics, spoke of the rich pipeline of treatments for rare diseases, especially as philanthropic venture capital firms are starting to fill the funding gap, and how approval timelines are slowly starting to catch up with the speed of development of new treatments. He argued that over the next few years, we will start to see more therapies derived from novel genetic and cellular
technology platforms emerge for currently unserved (or underserved)
rare diseases.

Sharon Hesterlee, Chief Research Officer at the Muscular Dystrophy Association, highlighted that the standardized trial design and knowledge sharing between national and international partners are also important to reduce inequalities in treatment of patients with rare diseases.

Stressing the need for early patient engagement, Kristin Phillips, Senior Director, Patient Experience at Taysha Gene Therapies, suggested that traditional key opinion leader (KOL)-centred patient journey mapping may miss important milestones since by the time a patient has been referred to a KOL, they have often already been living with their condition—with or without a definitive diagnosis—for years. For Kristin, earlier patient engagement by manufacturers is crucial to understanding the challenges of diagnosis, patients’ treatment needs, and the true impact on their lives.