Early Recommendations for Promoting Future Cell and Gene Therapy Competition Through Biosimilars

Introduction

Transformative cell and gene therapies (CGT) provide new and better treatment options for patients with conditions including genetic disorders and cancers by stopping or slowing disease progression. However, the introduction of CGTs has been complicated by high prices and other financial barriers, posing challenges to payers’ budgets. Encouraging competition from biosimilars in the field of CGT in anticipation of the expiration of patents and other exclusivity rights holds promise for reducing the cost of these treatments, enhancing patient access, and ultimately improving outcomes. However, because CGTs are highly complex biologics, they may present considerable hurdles to effective competition through the process of “biosimilarization.” This raises doubts about the practicality of fostering competition from CGT biosimilars. These challenges necessitate the development of strategies that would facilitate such competition in the CGT market through a supportive policy and regulatory environment.

Building on our detailed findings published here, in this document we describe the timely development of a set of policies to achieve such a competitive market. The policies will need to address a set of issues, including regulatory pathways, advanced manufacturing, and intellectual property (IP) protections. Ensuring robust competition, to the extent possible, will be critical to fulfilling CGTs’ potential to improve the lives of patients living with serious conditions who have few or no other treatment options.

This project was supported by a grant from Arnold Ventures.

Read the full issue brief here.