Oncologists now have greater discretion to use the immunotherapy in advanced cancers, ending what advocates have described as a ‘cancer lottery’ for patients with rare disease.
Cancer patient advocacy groups will have even bigger reason to celebrate spring – it also marks a significant expansion of the PBS listing for pembrolizumab.
The move was a shift away from the traditional approach of funding the immunotherapy cancer-by-cancer, giving clinicians scope to prescribe it for additional advanced or metastatic cancers where there was evidence the disease was sensitive to immunotherapy.
The change was expected to give hundreds of Australians new access to pembrolizumab (Keytruda, MSD) each year, according to the federal government.
MSD estimated more than 10,000 Australians would become eligible over the next four years under the broader funding arrangement, including patients with rare and less common cancers who previously had limited treatment options.
Eligible patients would pay the PBS co-payment of $7.70 for concession card holders or $25 for general patients per infusion.
Until now, PBS access to pembrolizumab depended largely on separate assessments for individual cancer indications.
The new approach allowed clinicians to use their clinical judgement, guided by the best available evidence, to determine whether treatment was appropriate for patients with immunotherapy-sensitive advanced or metastatic disease.
Federal health Minister Mark Butler described the change as “ground-breaking”, saying access would no longer simply depend on a patient having a particular cancer type.
“Availability for this drug will depend upon a doctor’s decision. The clinical judgement that Keytruda will help a patient with advanced or metastatic cancer fight back disease,” he said.
Mr Butler said the approach represented a major step towards greater equity in cancer treatment, particularly for people with rare cancers that may never have generated sufficient numbers for individual PBS submissions.
Pembrolizumab is a PD-1 checkpoint inhibitor that works by helping restore the immune system’s ability to recognise and attack cancer cells. It was funded on the PBS for more than a decade, and its indications progressively expanded.
The government said the drug was already PBS-listed for 17 cancer indications, with the latest expansion opening access for additional advanced cancers, including cholangiocarcinoma, ovarian cancer, and Merkel cell carcinoma.
The broader listing also coincided with the registration of pembrolizumab for certain patients with chemotherapy-resistant ovarian cancer, allowing immediate funded access for an estimated 80 eligible women with advanced or metastatic disease.
Associate Professor Sumitra Ananda, from the Peter MacCallum Cancer Centre and Epworth HealthCare, said oncologists and patients had advocated for a different approach to immunotherapy funding for years.
“This is an important moment that may expand immunotherapy access for thousands of Australians and give doctors the discretion to make appropriate evidence-based treatment decisions,” she said.
“Today’s announcement will help make this immunotherapy treatment option more affordable and potentially remove the financial burden that patients with advanced or metastatic cancer may face.”
Oncologist Associate Professor Amitesh Roy said the change would allow clinicians to use immunotherapy according to clinical evidence and their assessment of whether a patient’s advanced cancer was likely to be immune-sensitive.
“It is an important step in terms of improving access to our patients,” he said.
The affordability implications could be substantial. Mr Butler said patients benefiting from the expanded listing could otherwise face costs of thousands of dollars for each prescription.
Rare Cancers Australia CEO Christine Cockburn said the existing indication-by-indication system had created what amounted to a “cancer lottery”.
“If you get the wrong cancer, you’re going to get the short end of the stick when it comes [to] cancer care,” she said.
“Two people can be sitting in a chemo ward receiving the same medicine. One of them is paying $25 copay on the PBS and the other one’s fundraising, remortgaging, taking out loans to pay thousands of dollars.”
Ms Cockburn said the traditional system was particularly poorly suited to rare cancers because obtaining separate PBS listings for hundreds of small patient populations was often impractical.
Rare and less common cancers encompassed more than 250 cancer types, including cancers of the brain, eye, mouth, bone, thymus, and stomach, as well as sarcomas, she said.
An estimated 42,000 Australians were diagnosed with a rare or less common cancer each year.
Ms Cockburn said clinicians wanting to prescribe pembrolizumab would not have the added burden of a conversation around cost at a difficult time for patients.
“This listing on the PBS is an extraordinary shift towards equity, particularly for Australians with rare and less common cancers.”
The government also announced a separate, new PBS listing of pembrolizumab in combination with enfortumab vedotin (Padcev, Astellas Pharma Australia) for patients with stage 3 or 4 urothelial cancer.
More than 490 Australians were expected to benefit from the combination each year. Without the PBS subsidy, the government estimated patients might pay up to $4800 per script.
In addition, the PBS listing coincided with the registration of pembrolizumab in certain patients with chemotherapy-resistant ovarian cancer.
This allowed immediate funded access for approximately 80 eligible women with advanced or metastatic ovarian cancer – without the typical delay for medicines between a ARTG registration and PBS listing of 466 days.
Chifumi Umeda, managing director, MSD Australia & New Zealand, welcomed the PBS decision and the government support of cancer treatment funding reform.
“MSD has spent a decade working with the cancer community to arrive at this important day,” he said.
“It has been a long and winding road, but the result is affordable and equitable access to this immunotherapy for eligible Australians. We are thankful to Minister Butler for his support and stewardship in this funding in Australia.”
