Cheaper Medicines For Rare Genetic Disorders And Cancer

Department of Health

Adults with hereditary transthyretin-mediated amyloidosis and with certain cancers, will now have access to cheaper medicines under the Pharmaceutical Benefits Scheme (PBS) thanks to the Australian Government.

Selumetinib (Koselugo®) will list for the first time to treat symptomatic, inoperable benign nerve tumours in children two years old and over with neurofibromatosis type 1 (NF1).

NF1 is a rare genetic disorder affecting about one in 3,000 Australians.

Individuals with NF1 commonly have tumours that cause symptoms such as headaches, pain and disfigurement and lead to other complications, significantly impacting quality of life.

Koselugo works by blocking certain proteins involved in the growth of tumour cells.

Around 170 children are expected to benefit from this listing each year. Without subsidy, families might pay around $146,000 per year of treatment.

Patisiran (Onpattro®) will list for the first time to treat hereditary transthyretin-mediated (ATTR) amyloidosis that has affected many parts of the nervous system.

Hereditary ATTR amyloidosis is a rare and rapidly progressive disease that prevents the nerves, heart and other parts of the body from working normally.

If left untreated, the disease can progress to potentially life-threatening complications and death within a few years after symptom onset.

Onpattro works by lowering the amount of abnormal protein easing the effects of this debilitating disease.

This listing is expected to benefit around 55 patients each year. Without subsidy, they might pay around $647,500 per year of treatment.

Tecentriq® SC (atezolizumab) will list in a new subcutaneous form, which means it can be given as an injection under the skin.

The Albanese Government will fund the first immunotherapy cancer jab, allowing thousands of patients to bypass lengthy intravenous infusion and receive a seven-minute injection of a medicine that activates the immune system to identify and kill cancer cells.

More than 2,700 Australians with lung and liver cancer will now have affordable access to Tecentriq SC injection via the PBS as a new treatment option, significantly reducing their current treatment time and freeing up essential healthcare resources.

This new treatment approach has the potential to save patients hours of hospital time every year.

Without a PBS listing, the medicine would cost $120,000 per year.

Vitrakvi® (larotrectinib) will also be expanded to include treatment of adults with locally advanced or metastatic non-small cell lung cancer or soft tissue sarcoma solid tumours that have a specific gene fusion known as neurotrophic tropomyosin receptor kinase (NTRK).

NTRK gene fusions lead to abnormal proteins which may cause cancer cells to grow.

Vitrakvi works by stopping the fusion proteins working, helping to slow or stop the cancer growing.

Around 10 patients are expected to benefit from this listing each year. Without the PBS subsidy, they could pay over $300,000 per course of treatment.

Zyamis® (midazolam) will be listed to provide Australians living with epilepsy with a solution delivered via pre-filled syringes for the emergency treatment of potentially life-threatening seizures.

Zyamis will be listed on the PBS to treat a form of epilepsy known as Generalised Convulsive Status Epilepticus (SE).

SE is a prolonged or continuous seizure lasting more than 5 minutes, or a cluster of repeated seizures without full recovery in-between, and can be life-threatening or lead to long term consequences, such as permanent brain damage if not treated quickly.

More than 10,000 patients each year are expected to benefit from the PBS listing.

Without subsidy, each prescription would cost $270.

The PBS listings of these medicines means eligible patients will pay a maximum of $31.60 per script, or just $7.70 with a concession card.

Since July 2022, the Australian Government has approved extra funding for 227 new and amended listings on the PBS.

Quotes attributable to Minister Butler:

"Providing cheaper medicines to Australians is central to the Albanese Government.

"These PBS listings are yet another example - providing new help and new hope to Australian kids and adults - help and hope they would otherwise miss out on because of the exorbitant cost.

"The PBS, introduced by a Labor Government, sits alongside our other proud social reforms in the health space, notably, of course, Medibank and then Medicare, 40 years old this year.

"These listings are further demonstration of our commitment to ensuring all Australians can get affordable access to the medicines they need, when they need them."

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