You can search all of our articles using this search block.

Listing Yorvipath: why a third PBAC submission must succeed
John Forbes John Forbes

Listing Yorvipath: why a third PBAC submission must succeed

Palopegteriparatide (Yorvipath) is the first medicine that physiologically replaces the missing hormone in chronic hypoparathyroidism, and Australia's TGA was the first regulator worldwide to approve it after the FDA — yet two PBAC rejections in 2025 have left roughly 6,400–9,800 Australian adults tethered to a seventy-year-old calcium-and-vitamin-D regimen that controls a blood test while driving the kidney damage, neuropsychiatric symptoms and quality-of-life impairment documented in every major international cohort. The PaTHway trial now has three-year data showing 96% of patients remain independent of conventional therapy and mean eGFR has risen by 8.8 mL/min/1.73 m², with the largest gains in those with the worst baseline kidney function; Germany, France, the UK, Japan and the US have all moved to reimbursement while Australia has not. The PBAC's concerns were legitimate — ICER, surrogate endpoints, time horizon and limited Australian data — but each is answerable through instruments the Committee has itself built for rare-disease decisions: a confidential Special Pricing Arrangement, a Risk-Sharing Arrangement with expenditure caps, narrow initiation criteria aligned with the 2025 ESE guideline, explicit stopping rules, and a sponsor-funded Australian Hypoparathyroidism Registry. The article makes a respectful, specific case for a third submission framed as a Managed Access Program modelled on the Zolgensma and ivacaftor precedents, and calls on clinicians, patient organisations and affected Australians to coordinate their comment at the next consultation window.

Read More
The Clinical, Economic, and Ethical Imperative for Public Subsidy of Palopegteriparatide (Yorvipath) in Australia
John Forbes John Forbes

The Clinical, Economic, and Ethical Imperative for Public Subsidy of Palopegteriparatide (Yorvipath) in Australia

This report argues that the Australian Government’s refusal to subsidise Yorvipath (palopegteriparatide) represents a dangerous “false economy” that prioritises short-term pharmaceutical savings over the prevention of catastrophic long-term renal failure. By synthesizing clinical evidence from the Phase 3 PaTHway trial with Australian health expenditure data, the analysis demonstrates that the current “standard of care” is actively nephrotoxic, driving patients toward chronic kidney disease and dialysis costs exceeding $85,000 annually. The report concludes that the bureaucratic impasse cited by the PBAC—specifically regarding economic modeling horizons and local data gaps—is best resolved not by denial of care, but through a Managed Access Program (MAP) that grants immediate access to high-risk patients while collecting real-world data to validate the drug's long-term value.

Read More