The recent rescindment of the Center for Medicare and Medicaid Innovation’s Cell and Gene Therapy Access Model has complicated the search for sustainable funding solutions in the healthcare sector.

At the 2025 ASTCT and CIBMTR Tandem Meeting in Honolulu, Hawaii, Corey Cutler highlighted the rising costs of cell and gene therapies, which pose significant challenges for affordability and access.

Currently, the two approved gene therapies in hematology have acquisition costs ranging from $2 million to $3 million, placing a heavy financial burden on patients with sickle cell disease, many of whom depend on Medicaid.

While some gene therapies are considered appropriately priced, others, such as lovotibeglogene autotemcel, are viewed as overpriced due to their uncertain clinical benefits.

David Rind emphasized the necessity of establishing value thresholds in drug pricing, with the Institute for Clinical and Economic Review (ICER) typically applying a threshold of $100,000 to $150,000 per quality-adjusted life year (QALY) to guide reimbursement decisions.

Rind also stressed the importance of cost-effectiveness analysis to prevent overpayment for mediocre treatments and to ensure access to innovative therapies.

He pointed out the disconnect in healthcare pricing, where the recipient of a drug often does not bear its cost, complicating fair pricing determinations.

Although ICER reports influence market dynamics, the ongoing challenge is to balance the need for innovation with affordability in the healthcare system.

ICER employs a framework that assesses the cost per QALY to determine reasonable pricing, aiming to balance affordability with health maximization.

Innovative financing solutions, such as outcome-based rebates and installment payments, are being discussed to tackle payment challenges, but recent policy shifts have hindered progress.

Looking ahead, the global market for chimeric antigen receptor (CAR) T-cell therapies is projected to reach $108 billion by 2033, with the United States accounting for about $25 billion of that market.

Real-world examples of drug pricing discrepancies were discussed, including Tafamidis and ensifentrine, where actual prices significantly exceed their assessed value by ICER.