Submission on Proposal to change Pharmac’s policy principles for funding rare disorder medicines

Rare Disorders NZ supports changing Pharmac’s definition of a rare disorder from 1 in 50,000 to 1 in 2,000. This will bring Pharmac’s definition in line with the Rare Disorders Strategy published by the Ministry of Health in 2024, as well as international definitions. 

The proposed change brings all rare disorder medicine applications under the process already in place for what we define as ultra-rare disorder medicines, giving Pharmac a mechanism to consider a funding application before an application is lodged with Medsafe if other criteria are met.

We hope this proposed change will encourage more applications for rare disorder medicines as it removes a key barrier that prevents applications currently being made. For consistency and clarity, Rare Disorders NZ recommends adopting the definition of a rare disorder in the Rare Disorder Strategy which is “a medical condition with a specific pattern of clinical signs, symptoms and findings that affects fewer than or equal to 1 in 2,000 people in Aotearoa New Zealand”.

This is preferred over the currently proposed definition of “a clinically defined disorder that affects an identifiable and measurable patient population of less than 1:2,000”.

Read the full submission here.