Executive Summary 

This report serves as an urgent call to action to enhance patient access to CAR T-cell therapy –an innovative, individualized and potentially curative treatment for certain blood cancers. While progress has been made in expanding availability of CAR T-cell therapy, many patients still face significant barriers that prevent them from receiving this life-changing treatment when they need it most. 

CAR T-cell therapy involves the engineering of a person’s own immune cells to target and treat cancer.²

These individualized medicines offer a different approach from conventional therapies, involving a one-time treatment that is intended to provide long-lasting remission. For some patients with certain forms of aggressive and difficult-to-treat blood cancers, CAR T-cell therapies have enabled them to remain cancer free for more than five years^3,4,5,6 – a significant milestone.

Despite its curative potential in some diseases, many patients who could benefit from CAR T-cell therapy and want to access this treatment, do not get the opportunity. In the US, as well as in European countries where CAR T-cell therapy is available to treat patients with large B-cell lymphoma (LBCL), only about two out of 10 eligible patients receive access to CAR T-cell therapy.^6,7 In the US, despite CAR T-cell therapy being available for nearly seven years in LBCL, potential patients are severely underserved and only about two out of 10 eligible patients receive access to CAR T-cell therapy.⁸

There are several reasons why patients do not get access to CAR T-cell therapy in the crucial treatment window when they are eligible. Limited knowledge of and capacity for CART-cell therapy outside specialized treatment centers,⁸ limited capacity at treatment centers,⁹ delays in referral,⁹ long journeys for treatment,⁹ financial and logistical burden on patients and caregivers,¹⁰ significant upfront investment and total cost of treatment and care,¹¹ and funding and reimbursement challenges⁸ are just some of the barriers that either prevent people from accessing treatment altogether or cause delays that risk a patient’s cancer progressing before they access treatment. As experience with CAR T-cell therapies has grown, so too have the strategies and initiatives aimed at addressing access hurdles. In the future, as more therapies are approved across broader indications, there will be more need as well as more opportunities to address access challenges, including the cost of care, through innovation and system-level efficiencies. But patients needing treatment today cannot wait.

Time is of the essence and urgent action is needed. This is why a group of clinical, patient support, provider and health system experts came together to develop the Vision for CAR T-cell therapy with recommendations for interventions that, if adopted and scaled, will help many more patients get the opportunity of treatment. This expert Steering Committee has set an ambitious goal: to double the proportion of eligible patients who are treated with CAR T-cell therapy by 2030. The aim is to unite diverse stakeholders behind this Vision and the meaningful changes that are needed to support more eligible patients in reaching the outcomes that CAR T-cell therapy can offer.

*We define eligible patient as a person who meets the regulatory criteria for treatment with CAR T-cell therapy – this may vary by country or region and the Vision is meant to be adaptable to local contexts.