The Medical Services Advisory Committee has reaffirmed its support for publicly funding the CAR-T therapy ciltacabtagene autoleucel for refractory or relapsed multiple myeloma, but says a new pricing proposal still falls short of expectations.

Sponsor Janssen-Cilag has submitted multiple applications for ciltacabtagene autoleucel (cilta-cel, CARVYKTI) to be funded for adults with myeloma who have accessed at least four prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody (5L+ RRMM).

Under its proposal, the B-cell maturation antigen-directed CAR-T cell therapy would be jointly funded as a Highly Specialised Therapy through the National Health Reform Agreement by the federal, state and territory governments.

MSAC twice deferred its support due to what it described as an unacceptably high and underestimated incremental cost-effectiveness ratio among other “highly uncertain” estimates, before making a positive recommendation last April [link here], but only via an alternative model to that proposed by Janssen-Cilag.

In its latest meeting in November 2024 [link here], the advisory committee considered an updated funding proposal submitted by Janssen-Cilag in October, which was accompanied by an updated economic model and financial estimates.

It said the sponsor had reduced its price since its April 2024 pricing proposal, along with dropping the suggestion to place caps on the number of patients treated with cilta-cel per year, which the committee had described “inequitable and unethical”.

However, despite these improvements, MSAC said the alternative funding still did not appropriately address the clinical, economic and financial uncertainty associated with public funding. The committee was also unhappy with Janssen-Cilag’s proposed pay-for-performance outcome measure of overall survival.

“MSAC considered the pay-for-performance [arrangement] should be based on an outcome that could be attributed directly to the use of cilta-cel, such as stringent complete response with no use of subsequent therapies. MSAC maintained that a four-year pay-for-performance arrangement is most closely aligned with the available clinical evidence (to account for the diminishing effectiveness of the treatment over time),” the committee said in the latest public summary document.

However, the committee said a shorter pay-for-performance arrangement would be acceptable if it was based on stringent complete response at 12 months and progression free survival thereafter with no subsequent treatments that resulted in a similar price and incremental cost-effectiveness ratio to the latest proposal.

While the price in the proposal was redacted, the incremental cost-effectiveness was put forward as $95,000 to < $115,000 per quality-adjusted life year.

MSAC said with a price reduction, it would also consider a single payment on successful infusion that accounted for the diminishing effectiveness of the treatment over time as an alternative funding arrangement to pay-for-performance.

The committee maintained its requirement for a review post funding.

Janssen-Cilag’s parent company Johnson & Johnson said it welcomed MSAC’s recommendation to continue to support the public funding of ciltacabtagene autoleucel and would continue negotiations with Federal and state governments.