Government’s prolonged negotiations delay access to CAR-T therapy for multiple myeloma

The CAR-T therapy Carvykti (cilta-cel) was recommended by the Medical Services Advisory Committee (MSAC) last year as a fifth-line treatment for relapsed or refractory multiple myeloma. However, negotiations over funding have remained contentious, with ongoing discussions centred on an updated economic model and financial estimates.

Johnson & Johnson Innovative Medicine (formerly Janssen) has been working to secure funding for Carvykti, following its fourth submission to MSAC.

“Johnson & Johnson continues to work with Federal and State governments and departments of health to navigate the complexities of bringing this highly specialised therapy to Australian patients. A fourth submission to MSAC was necessary to reduce the administrative workload on CAR-T treatment facilities and physicians and to find agreement on the payment schedule for Carvykti,” the company said in a statement.

The push for Carvykti’s availability has gained strong clinical and consumer backing, with advocacy from The Leukaemia Foundation, Myeloma Australia, Haematology Society of Australia and New Zealand (HSANZ), Australasian Leukaemia and Lymphoma Group (ALLG), and the Peter MacCallum Cancer Centre. Patients and healthcare professionals have highlighted the shortcomings of existing treatment options, citing limited efficacy, the burden of frequent hospital visits, procedural discomfort, ongoing side effects, multidrug resistance, and the substantial financial strain.

Adding weight to the debate, J&J announced last year that its phase 3 CARTITUDE-4 trial had delivered “statistically significant and clinically meaningful” overall survival results, comparing Carvykti to standard combination therapies in multiple myeloma patients who had undergone one to three prior lines of treatment.

Despite the widespread support from patient groups and clinicians, States and Territories – acting as joint funders through the National Health Reform Agreement (NHRA) – have resisted unless a significant reduction in Carvykti’s price was achieved. J&J has now lowered the pricing from its April 2024 proposal and has also removed its previous proposal to cap the number of patients eligible for treatment each year.

A key sticking point in negotiations has been the Pay-for-Performance (PfP) model. MSAC rejected J&J’s proposal to use overall survival (OS) as the primary outcome measure. Instead it maintained that stringent complete response (sCR) with no subsequent therapies would be a more appropriate metric and proposed a four-year PfP arrangement, reflecting available clinical evidence on the treatment’s diminishing effectiveness over time.

However, MSAC signalled it would consider a shorter PfP arrangement – measuring stringent complete response (sCR) at 12 months and progression-free survival (PFS) without subsequent treatments – if the price and incremental cost-effectiveness ratio (ICER) were deemed acceptable.

MSAC also discussed an alternative funding model, suggesting a single payment upon successful infusion, provided it accounted for the treatment’s declining effectiveness over time. This approach, however, would necessitate a further price reduction.

As negotiations continue, the future of Carvykti’s availability in Australia remains uncertain, with pricing and performance measures at the heart of the ongoing funding impasse. MSAC reiterated that a review post-funding remains essential.

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