Abstract

Advanced therapy medicinal products (ATMPs) have already played a transformational role in the lives of patients with rare diseases. However, questions do remain with regards to longevity of product effect, repeatability of dosing and manufacturing scalability. An additional challenge is the high upfront cost of ATMPs and how conventional reimbursement schemes affect ability to pay. There is a need to focus on solutions to these challenges, through ongoing studies, new gene therapy platforms, technology advances to support ATMPs manufacturing at scale, as well as alternative drug pricing models. At the end of the day, ATMPs have saved and changed lives. Taking positive steps must remain a priority so that greater numbers of innovative products can reach more patients.