Public funding for CAR-T therapy ciltacabtagene autoleucel for refractory or relapsed multiple myeloma (MM) has moved one step closer with a positive recommendation from the Medical Services Advisory Committee, although price negotiations with the sponsor face ongoing disagreements and delays.
In its third application, sponsor Janssen-Cilag is seeking ciltacabtagene autoleucel (cilta-cel, CARVYKTI) be funded to treat 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.
Already, MSAC has twice deferred its support for the proposal, including last November due to what it described as an unacceptably high and underestimated incremental cost-effectiveness ratio among other “highly uncertain” estimates.
It had also wanted the company to offer a significant price reduction for the treatment that is reported to cost about $500,000.
Alternative way forward
In the latest meeting in April [link here], MSAC supported public funding of the therapy but only via an alternative model to that proposed by Janssen-Cilag that “appropriately managed the clinical, economic and financial uncertainty associated with the public funding” – together with a Risk Sharing Arrangement that “appropriately managed the risk of the applicant’s financial estimates being exceeded”.
In its advice to the Minister for Health Mark Butler, the committee said the sponsor’s proposal limited the number of patients who could receive treatment with cilta-cel in a given funding year to compensate for the high cost of treatment and provide certainty on financial estimates, which “was inequitable and unethical”.
“MSAC noted that if implemented, the proposal would mean that a patient’s eligibility to receive cilta-cel would be influenced by the time of year they were deemed eligible for treatment, meaning that an eligible patient presenting at the beginning of the year had a higher likelihood of receiving the treatment than an eligible patient presenting at the end of the year,” the committee wrote.
“MSAC noted Janssen-Cilag presented the proposal as a Risk Sharing Arrangement, however MSAC rejected this characterisation, noting [such arrangements]… require the treatment provider to bear the financial risk associated with the financial estimates being exceeded and, in no circumstances, the patient.”
The committee also accused the company of delaying publicly funded access to Australian patients by not attempting to adequately address its concerns and remaining steadfast on the unit price since an initial application in July 2022.
MSAC’s “alternative way forward” suggested that payment at the requested price should be made to the company in instalments over a four-year period, where there was evidence that the patient remained with no signs of disease progression.
This was appropriate considering evidence that suggested some patients had recurrence within four years of cilta-cel treatment, the committee added.
In response, Janssen-Cilag parent company, Johnson & Johnson, welcomed the positive recommendation from MSAC to publicly fund the CAR T-cell therapy.
“Johnson & Johnson will work through the further negotiations with Federal and State Governments which are necessary to navigate the complexities of bringing this highly specialised therapy to Australian patients,” it said in its official response.
Myeloma Australia said the positive recommendation was a welcome step for patients with MM but it was still unable to say how long it will be until cilta-cel will be available.
“This is fantastic news for the myeloma community but there is still significant work to be done before Carvykti will be available in hospitals. The manufacturer will now work with State, Territory and Federal Government representatives to arrange the necessary agreements and prepare hospitals to be able to deliver Carvykti safely,” it said in a statement (link).
Tim Murphy, the Leukaemia Foundation’s head of blood cancer partnerships, told the ABC the verdict was a “huge step” for patients with no other treatment option, but stressed there was an urgency to make CAR-T available as soon as possible.
“We implore every single party involved in these negotiations to do it quickly. Diligently, thoroughly – but quickly – because people’s lives are at stake,” he said.
Minister Butler said the government was “working methodically” to make the therapy “a reality” for eligible patients, but added it “doesn’t happen overnight”.