Value and affordability of CAR T-cell therapy in the United States

Bone Marrow Transplant. 2020 Sep;55(9):1706-1715. doi: 10.1038/s41409-020-0956-8. Epub 2020 May 30.

Abstract

In the United States the increasing number of Food and Drug Administration (FDA)-approved, innovative, and potentially effective commercial cancer therapies pose a significant financial burden on public and private payers. Chimeric antigen receptor (CAR) T cells are prototypical of this challenge. In 2017 and 2018, tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Kite) were approved by the FDA for use after showing groundbreaking results in relapsed/refractory B-cell malignancies. In 2020 and 2021, four further submissions to the FDA are expected for CAR T-cell therapies for indolent and aggressive B-cell malignancies and plasma cell myeloma. Yet, with marketed prices of over $350,000 per infusion for the two FDA-approved therapies and similar price tags expected for the coming products, serious concerns are raised over value and affordability. In this review we summarize recent, peer-reviewed cost-effectiveness studies of tisagenlecleucel and axicabtagene ciloleucel in the United States; discuss key issues concerning the health plan budget impact of CAR T-cell therapy; and review policy, payment and scientific approaches that may improve the value and affordability of CAR T-cell therapy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Cost-Benefit Analysis
  • Humans
  • Immunotherapy, Adoptive*
  • Neoplasm Recurrence, Local*
  • Receptors, Antigen, T-Cell
  • United States
  • United States Food and Drug Administration

Substances

  • Receptors, Antigen, T-Cell