Terminally ill patients despair amid price war over CAR-T cancer therapy

Australia risks ‘being pushed to the back of the queue’ of countries in gaining access to a revolutionary cancer treatment that can put those facing imminent death into remission.

A chimeric antigen receptor (CAR) T-cell engineered to recognise and attack cancer cells.

Australia risks “being pushed to the back of the queue” of countries to gain access to a revolutionary cancer treatment that can put those facing imminent death into remission amid a standoff between medical technology advisers and a pharmaceutical company.

The Medical Services Advisory Committee has deferred a decision to approve public funding for a type of CAR-T cell therapy – dubbed “the future of cancer treatment – for the rare blood cancer multiple myeloma, leaving hundreds of terminally ill patients in limbo.

The MSAC, responsible for recommending public funding for new medical services, acknowledged the significant benefits the drug in question, CARVYKTI, would bring to myeloma patients but demanded that pharmaceutical company Janssen reduce the price before it would grant a positive recommendation for funding.

Scientists say CAR-T cell therapy, a cutting-edge treatment that re-engineers a patient’s own T-cells to enable them to fight cancer, represents a “total paradigm shift” in cancer care for the sickest patients who have failed to respond to conventional remedies. About 300 patients currently live with terminal myeloma and their lives depend on the therapy being publicly funded.

Patients who receive CAR T-cell therapy have their T-cells re-engineered in a laboratory to carry special structures called chimeric antigen receptors (CARs) on their surface. When these CAR T-cells are reinjected into the patient, they multiply rapidly, and the engineered receptors help the patient’s T-cells identify and attack cancer cells throughout the body.

CARVYKTI, also known as cilta-cel, has been developed by global pharmaceutical company Janssen and was approved by the Therapeutic Goods Administration last year to treat people with multiple myeloma who have not been able to combat their cancer with multiple previous lines of treatment. 

It has already been knocked back once for public funding. 

Miles Prince, Director of Molecular Oncology at the Epworth Hospital in Melbourne.

MSAC said in its latest statement deferring a funding decision that it considered the treatment was effective and safe enough for people as a fifth-line therapy after other treatments had failed, but was concerned the price was too high for it to be cost-effective. MSAC advised the government to negotiate with Janssen on the cost of the treatment so that it was good value for money.

MSAC noted that state jurisdictions considered substantial price reduction was required for public funding to be supported.

Australian cancer researchers said the therapy was so extraordinarily effective – it has been shown in almost all cases to put terminally ill patients into remission after a one-off treatment – that Janssen would be unlikely to be of a mind to negotiate on the price, and would instead prioritise other markets.

The pharmaceutical company has offered to reimburse taxpayers in any case in which its treatment does not work.

“This is probably the greatest breakthrough we’ve ever had with multiple myeloma treatment,” said Miles Prince, director of Molecular Oncology at the Epworth Hospital in Melbourne.

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

'Multiple myeloma patient Helen Buckingham

“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 CARVYKTI for countries where the novel treatment is funded. A one-off course of treatment of CARVYKTI costs $500,000 and involves significant manufacturing costs for what is a highly specialised and personalised treatment.