POGO-FUNDED RESEARCH: An Economic Evaluation of CAR-T Therapy

Acute lymphoblastic leukemia (ALL) is the most common form of childhood cancer and for most patients, the standard chemotherapy protocol works very well with an overall cure rate of 90%. But, there is a subset of this population that is treatment resistant. For these young patients, a relatively new therapy called chimeric antigen receptor T-cells (CAR-T) therapy may be the answer.

CAR-T therapy uses the patient’s own immune system to do the work of destroying the ALL cancer cells. It starts with harvesting immune cells called T-cells from a patient’s blood. Millions of copies of these cells are grown in a lab—engineered to recognize, target and destroy the cancer cells—then infused into the patient’s bloodstream. Once back in the patient’s body, the cells divide and increase in number, creating a massive army against leukemia cells. Importantly, unlike an infusion of a drug, these cells can remain in the body indefinitely, providing constant surveillance against ALL’s return. The results to date are promising, but not without significant risk.

“Like most cancer therapies, CAR-T cell treatment can have severe and potentially fatal side effects,” says Dr. Alexandra Zorzi, pediatric oncologist at Children’s Hospital, London Health Sciences Centre. “The ‘activation’ of your own immune system can lead to the release of too many cytokines, which can result in laboured breathing, high fevers, and potentially life-threatening decrease of blood flow to internal organs.”

But when it works, it works miracles. At least that is what we know in the short term. “CAR-T cell therapy has the potential to be a major game changer in childhood ALL,” says Dr. Paul Gibson, medical officer with POGO. The early results have shown remarkable response rates, even in children who have been heavily treated for ALL previously. Not only is the response rate impressive, but so are the sustained remissions many patients are experiencing. “This is the first true gene therapy in childhood cancer therapy,” says Dr. Gibson. “While very expensive upfront, it may not only save lives, but save children from needing to be treated many more times in the future.”

Currently, this therapy is only offered at the Children’s Hospital of Philadelphia. The cost to send an Ontario patient for treatment is close to $500,000, not including the cost to families who often have to leave work and home for several months.

In 2016, POGO awarded Dr. Petros Pechlivanoglou with a seed grant for his project “Economic Evaluation of CAR-T Therapy for Children with High Risk Relapsed ALL.” Dr. Pechlivanoglou and his co-investigators—Drs. Sumit Gupta, Jason Pole, Paul Nathan, Tal Schechter-Finkelstein and Wendy Ungar, together with PhD student Jill Furzer—are using statistical and mathematical modelling to determine the value of CAR-T therapy from a clinical and economic perspective. What is the trade-off between the treatment’s effectiveness and its cost to the Canadian healthcare system and society overall? Where should we focus our efforts to collect more evidence in the future? And, how will this information be used to inform policy decisions?

“This economic evaluation of CAR-T therapy is only one example of new therapies,” says Dr. Petros Pechlivanoglou. “As new cancer innovations emerge, both in the pediatric and the adult world, we are going to have this challenge of understanding the ‘value-for-money’ proposition again and again. This grant will help us tease out the methods needed to find timely answers to such questions and make prioritization decisions about future clinical, economic and policy research.”
– Petros Pechlivanoglou, PhD

Dr. Petros Pechlivanoglou is a scientist at The Hospital for Sick Children Research Institute and assistant professor at the University of Toronto. His research focuses on methods around the use of health decision analysis, administrative data and prediction modelling in economic evaluation, and the health economics of pediatric oncology and pre-term birth.