Defining disruption: What are durable cell and gene therapies (CGTs)?

The development of transformative cell- and gene-based technologies has raised the possibility that patients with rare diseases could live without the need for ongoing treatments or the burden of daily disease management after a single course of treatment.

Known as durable cell and gene therapy (CGT), this new category of medical technology offers a variety of benefits. In some cases, cell and gene therapies can improve patient survival rates and/or survival times. In others they can offer individuals improved health and well-being during the course of their condition. Some gene therapies stop disease progression, preventing patients from becoming sicker. Others offer quality-of-life improvements, including improved functioning, less pain, and a greater sense of well-being. Many of the conditions addressed by cell and gene therapy are rare and may have limited alternative treatments—a situation that has brought communities of patients, providers, developers, and payers together for support and advocacy.

The FoCUS Project has developed this Toolkit as a resource discussing the financial challenges of delivering and financing of an advanced durable therapy. The sidebar provides alternative sites to learn more about the science and potential value of gene therapy.