Although the concept of using genetically engineered T cells to fight cancer has been under study for more than 50 years, the most dramatic strides in designing new therapies have come in the past several years.
That is when reports started build- ing about patients with recurrent hematologic malignancies who had experienced long-standing remissions after treatment with chimeric antigen receptor (CAR) T-cell agents directed at CD19. Since then, study results with objective response and complete remission rates of greater than 70% have been disclosed in various lymphoma and leukemia trials.
Given such positive findings, experts feel it is highly likely that the first CARs will reach the US market this year and that more options will be available in the next several years. The FDA currently is reviewing applications for 2 drugs, and pharmaceutical companies have additional compounds in early-phase development.
There are, however, many complex considerations in bringing this new technology to patients, as we explore in our cover story in this issue of OncologyLive®
, “Pivotal Year Looms for CAR T-Cell Therapies.” One of the primary challenges will be identifying and managing the potentially life-threatening adverse events that patients treated with these therapies are at risk of experiencing.
Other challenges will be commercial; for example, the regimens have been designed for the sickest patients in comparatively less common cancers and may be difficult to use in broader populations or earlier in treatment timelines.
Then there is the question of cost both to individual patients and to the healthcare system. Since none of the drugs has reached the market yet, we don’t know the pricing but some analysts said 2 years ago that they expected CAR treatments to cost up to $300,000 per patient.
That brings us to another important point that Editor-in-Chief Maurie Markman, MD, raises in his insightful commentary, “Clinical Trial Reform Is Urgently Needed.” Although we routinely discuss the value of a novel anticancer drug in the context of what it will cost, the real question is how well a therapy works. How we pay for a clinical advance—particularly one as potentially remarkable as CARs—should be a separate issue.
Our dysfunctional healthcare payment systems have married these concepts. It would be quite a shame for a revolutionary advance such as the CAR agents to be underutilized because of cost concerns. Let’s hope we can figure this out so patients truly bene t from the many years of research that have gone into deliver- ing these therapies.