CAR-T Therapy

Certain cells from a person's body are collected, modified in a lab, multiplied thousands of times, and injected back into the body—what for?

This is a simplified version of how CAR-T cell therapy works to treat cancer. CAR-T cells have achieved remarkable complete response rates (no signs of cancer post treatment) of over 90% in certain tumor types, like Acute Lymphoblastic Leukemia. This therapy is also convenient, requiring only a single infusion.

Despite this promise, there are only six FDA-approved CAR-T products in the market: two from Gilead, two from BMS, one from J&J, and one from Novartis. These drugs have been available for the past 3 to 6 years. We might expect these treatments to have become blockbuster successes, given the deadly nature of cancer and the universal desire for a cure. But surprisingly, that hasn’t been the case.

For example, Yescarta from Gilead had the highest sales among these therapies in the first nine months of 2024, reaching $1.1B, while the rest generated less than $650M each. Why is this happening?

Scientific and Market Factors

• Solid vs. Liquid Tumors
  CAR-T cell therapy is currently effective only against liquid tumors. Solid tumors often prevent modified CAR-T cells from entering the Tumor MicroEnvironment (TME), which is necessary for them to kill tumor cells. Additionally, solid tumors have multiple tumor-specific antigens, making the creation of CARs more complex.

• Market Share
  Liquid tumors account for only 10% to 15% of adult cancers. CAR-T therapies are indicated only for a subset of these, typically in late stages when other treatments have failed.

• High Cost
  The cost of these therapies ranges from $350K to $500K, reflecting high process costs due to personalization, labor, and materials. These factors significantly limit the number of patients who can benefit.

Cost Front Improvements

Encouragingly, progress is being made on the cost front. For instance, NexCAR19, a drug approved in India, competes in the same space as Yescarta and Breyanzi but is priced at just $30K to $40K, about a tenth of the cost. This is possible due to lower process and labor costs in India, among other factors.

Moreover, there are about 200 clinical trials investigating CAR-T for solid tumors, reflecting the significant investments in this space. With advancements in cost reduction and breakthroughs in solid tumor treatments, the future for CAR-T cell therapy looks promising.

Key Takeaway for Pharma Executives

For pharma executives, success in CAR-T cell therapies lies in rethinking traditional commercial operations: shifting focus from duration and dosage metrics to building robust systems for patient eligibility analysis, holistic patient support, and digital interventions for continuous post-treatment monitoring.