New PBS listings to expand access in rare disease, ophthalmology and women’s health

A wave of new PBS listings is set to transform care for Australians, spanning rare diseases, ophthalmology, and contraceptive options.

Medison Pharma’s RNAi therapy Oxlumo (lumasiran) has become the first therapy to target primary hyperoxaluria (PH) type 1 – a rare, potentially fatal genetic disease. PH1 causes elevated oxalate levels, leading to kidney stones, nephrocalcinosis, progressive renal failure, and systemic oxalosis, which can damage multiple organs.

With this PBS listing, around 50 patients per year are expected to benefit. PH1 is present from birth but often remains undiagnosed until severe symptoms emerge. Approximately 90% of patients experience symptoms during childhood or adolescence.

In ophthalmology, Novartis’ anti-VEGF intraocular injection Lucentis (ranibizumab) is now PBS listed for diabetic macular oedema (DME), positioning it as a competitor to Bayer’s Eylea (aflibercept), which gained reimbursement for the same indication in October 2024. This expansion could benefit around 500 patients annually.

Real-world evidence from the Fight Retinal Blindness! registry indicates that both anti-VEGFs are safe and effective over three years. While Eylea demonstrated superior anatomical outcomes and faster, larger visual gains at one year in eyes with moderate visual impairment (visual acuity 20/50), these differences were no longer significant by 18 months. The authors noted that the medium-term real-world treatment outcomes of both anti-VEGFs for diabetic macular oedema “seemed to be somewhat inferior to those reported in RCTs.”

Expanding contraceptive options, Mayne Pharma’s Nextstellis (estetrol with drospirenone) is now PBS listed. The therapy was TGA registered in November 2021 and reflects recent efforts to improve access to a broader range of hormonal contraceptives.

This development aligns with recommendations from the Senate inquiry ‘Ending the postcode lottery: Addressing barriers to sexual, maternity, and reproductive healthcare in Australia’, which urged the Department of Health and the Pharmaceutical Benefits Advisory Committee (PBAC) to collaborate with industry to expand access to non-subsidised hormonal contraceptives. The recommendation also complements the National Women’s Health Strategy 2020–2030, which prioritises equitable access to timely, appropriate, and affordable care for all women.

Minister for Health, Ageing and Disability, Mark Butler MP, said “We know these new and expanded listings will be lifechanging for thousands of Australians.”

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