A decision on whether to fund CAR T-cell therapy ciltacabtagene autoleucel for adult patients with RRMM has again been deferred by the Medical Services Advisory Committee, despite it acknowledging the proposed use was reasonable.

The Medicare gatekeeper committee said a substantial price reduction was required for public funding to be supported for the therapy, which is reported to have a list price of US$465,000 (A$716,000) for a one-time infusion in the US.

It marks the second setback for drug company Jannsen-Cilag in its attempts to gain public funding for its therapy, known as cilta-cel (Carvykti).

The firm sought funding via the National Health Reform Agreement for the treatment of adult patients with RRMM, who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.

In its assessment, MSAC said the application had outlined a “reasonable” clinical place for cilta-cel in the context of RRMM for patients who have a high unmet clinical need.

It also accepted the clinical claim that the therapy had superior effectiveness in terms of durable survival outcomes and a different safety profile compared with standard care.

In its evaluation, the committee acknowledged submissions from the Medical and Scientific Advisory group of Myeloma Australia which stated that “Cilta-cel is game changing therapy for those patients with late-stage disease. Current available therapies for patients on 5th line treatment have survival in the range of 3-6 months compared to 3-4 years with Cilta-cel.”

However, the incremental cost-effectiveness was both “unacceptably high and underestimated”, the committee said (link here), adding state and territory health departments had argued substantial price reductions would be needed for public funding to be supported.

“MSAC considered that the estimated costs associated with cilta-cel therapy were highly uncertain and underestimated,” it said.

“MSAC deferred its advice to see whether a lower incremental cost-effectiveness ration (ICER) could be achieved through adjusting inputs in the economic model (particularly hospital costs and health benefits gained – to be verified by jurisdictions) and for a significant price reduction to be offered by the company.”

An ICER in the range of those for other treatments in later line RRMM recommended by PBAC would be more likely to be acceptable, it added.

Furthermore, MSAC requested that the Department of Health negotiate a pay-for-performance arrangement to incentivise payment on the performance of the deeper level of clinical response achieved at 12-months after cilta-cel infusion.

The decision has made some waves in the mainstream media, with The Australian newspaper running an article last week warning Australia risked “being pushed to the back of the queue” of treatment access as a result.

“This is probably the greatest breakthrough we’ve ever had with multiple myeloma treatment,” Melbourne haematologist Professor Miles Prince told the media outlet (link here).

“This therapy is really paving the way for the future of cancer treatment,” Professor Prince said.

“These repeated rejections are hugely disappointing. It is not only a sad day for patients with myeloma, it’s a sad day for the way Australia is approaching what I would say is a climate change moment in treatment of patients with cancer.”

“We’ve been lucky enough to be chosen to be one of the first countries in the league ladder to be offered this product because we have been seen as the leading country which has contributed to research and therefore our patients are deserving of getting access to this treatment.”

“If we reject it, they’ll go to other countries to get approval, and we’ll end up falling to the bottom of the queue. People will lose their lives because of this decision; hundreds of people will die. What I don’t understand is the government’s priorities.”

Janssen responded to the decision by saying it was “unfortunate that the MSAC assessment … does not recognise (the drug’s) full value to patients and clinicians”.

The company said it was prioritising supply of the therapy for countries where the novel treatment is funded.