CAR-T Therapy: What Real-World Clinical Practice Data Shows

The Austrian Institute for Health Technology Assessment (AIHTA) (https://aihta.at/page/homepage/en) has published a systematic review of real-world data (April 2022 – July 2024) on CAR-T therapy. 

The analysis included 26 studies (2716 patients) on six EMA-approved drugs: Kymriah®, Yescarta®, Tecartus®, Breyanzi®, Abecma® and Carvykti®.

Efficacy

In real-world clinical practice, the results are generally comparable with the registration studies. For new indications, including multiple myeloma, higher response rates have been noted, and indirect comparisons show the advantage of CAR-T over standard therapy. With long-term follow-up, OS and PFS decrease, which requires subsequent monitoring.

Safety

The main complications are cytokine storm and neurotoxicity, more often recorded with Yescarta® and Tecartus®.

Economics

• In Europe (Germany), the cost of CAR-T infusion is €200-250 thousand, excluding hospitalization and complications.

• In the US, the total annual cost of treating myeloma is about $625,000, including the drug ($465,000) and treatment of complications (over $110,000).

• For DLBCL in the US, the average 3-month cost of a CAR-T course is $380,000–526,000.

• In Germany, in the third line of DLBCL therapy, the median costs were: €310,000–340,000 for CAR-T, €74,000 for allogeneic transplantation, and €27,000 for maintenance therapy.

• According to pharmacoeconomic models, ICERs range from $9,000 to $4.1 million/QALY in adults and from $21,000 to $243,000/QALY in children. This range is due to differences in models and uncertainty in long-term survival, making economic evaluations highly unstable.

Conclusion: Real-world data support the efficacy of CAR-T, but high costs and uncertainty in long-term outcomes remain a major challenge for healthcare.

Source (https://eprints.aihta.at/1562/)