Amar Kelkar, MD, on Assessing Price and Value for Cell and Gene Therapies


The stem cell transplantation physician at the Dana-Farber Cancer Institute discussed how to look at the value therapies provide and how to improve the cost-effectiveness of their use.

“The takeaway is that a lot of these therapies are overpriced. Tere's a lot of factors that go into that: There's scarce supply along with high demand, as well as manufacturing delay, and, of course, these are novel therapies on patent and so there's natural investment recoupment from these companies, as well. All these things factor into these higher prices. Our goal in doing this type of research is to try and push back on these higher prices to get them to a level that we consider to be cost-effective so that more patients can get these treatments...”

Over the past few years, novel cell therapies and gene therapies have provided transformative new treatment options for many patients with serious hematological diseases, such as leukemia and lymphoma, sickle cell disease, and thalassemia. Although these advanced therapies are typically one-time treatments that are expected to provide durable long-term efficacy, or in some cases even a cure, they often come at a very high financial cost in the realm of millions of dollars per patient. These high prices can limit access to treatment for some patients.

Amar Kelkar, MD, a stem cell transplantation physician at the Dana-Farber Cancer Institute, gave a talk on the topic of payment and value for cell therapies at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. After his presentation, CGTLive® interviewed Kelkar to learn more. Kelkar discussed the various ways of looking at value for therapeutic products, such as viewpoints that focus on clinical efficacy alone, viewpoints that focus purely on financial and other costs when comparing therapies that are considered equivalent, and viewpoints that take both efficacy and cost into account. He additionally discussed the importance of finding ways to improve the cost-effectiveness of cell and gene therapies, explaining the potential impact this could have on the healthcare system and how it could open the door to treating more patients. Kelkar also spoke about how specialized diagnostic testing could help identify which patients are most likely to benefit from pricey treatments before the decision to use them is made.

Click here for more coverage of Tandem 2024.

1. Kelkar A. The space between value and payment in cell therapies - sustainability, evaluation, and operational cost. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.
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