Decision to change Pharmac’s policy principles for funding rare disorder medicines
What we’re doing
Following our recent consultation to change Pharmac’s policy principles for funding rare disorder medicines, we received valuable feedback suggesting that the definition of rare disorders used by Pharmac be changed from 1 in 50,000 to 1 in 2,000.
As a result, Pharmac will update its rare disorders policy principles by broadening the definition of a rare disorder. From 1 November 2025, the definition of a rare disorder will read:
“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 change aligns Pharmac’s policy with the Aotearoa New Zealand Rare Disorders Strategy 2024 and reflects feedback we have received.
Why we are doing this
In July 2024, the Ministry of Health released the Rare Disorders Strategy, developed in response to long-standing advocacy and a recommendation from the Government’s 2022 review of Pharmac. Pharmac contributed to the development of the Strategy and is committed to supporting its implementation.
Aotearoa New Zealand Rare Disorders Strategy | Ministry of Health(external link)
The Strategy defines a rare disorder as affecting fewer than 1 in 2,000 people, whereas Pharmac’s previous policy principles used a threshold of 1 in 50,000. This misalignment has meant that some medicines which meet the Strategy’s definition are not eligible for consideration under Pharmac’s rare disorders pathway.
By updating our definition, we will:
- align with the Strategy and broader health system expectations
- enable more medicines to be assessed for funding earlier in the assessment process
- address known gaps where medicines have not been considered due to the current threshold
- demonstrate our commitment to improving access to treatments for people with rare disorders.
Any changes to the original proposal?
We received valuable feedback through our public consultation, which ran from Monday 11 August until Friday 29 August.
People recommended that the definition of a rare disorder should align with the Aotearoa New Zealand Rare Disorders Strategy. Specifically, they suggested changing the proposed wording from ‘a clinically defined disorder that affects an identifiable and measurable patient population’ to ‘a medical condition with a specific pattern of clinical signs, symptoms and finding’.
People thought that ‘identifiable and measurable’ could be too specific or set unrealistic expectations for evidence. After carefully considering this feedback, we have made further changes to the definition.
The revised definition now reads that a rare disorder 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 change better aligns our policy settings with the Strategy.
Feedback also highlighted requests for clearer differentiation between the rare disorders pathway and the Named Patient Pharmaceutical Assessment (NPPA) process. To address this feedback, we will publish additional information online to clarify the differences between the processes.
We also received feedback that wasn't related to this change regarding the funding of Rare Disorders medicines more generally. This feedback is being considered internally. We thank you for your input.
Who we think will be most interested
- People with rare disorders, their whānau, and caregivers
- Health professionals who work with patients with rare disorders
- Pharmaceutical providers
- Medsafe, Health NZ, the Ministry of Health
- Support and advocacy groups
Detail about this decision
From 1 November 2025 Pharmac’s rare disorders policy settings will be amended (additions in bold, deletions in strikethrough).
Funding applications for rare disorder medicines need to meet these three policy principles.
- Medsafe, or an approved international regulatory authority, has approved the medicine for the specific indication or condition.
- The disorder is a medical condition with a specific pattern of clinical signs, symptoms and findings clinically defined disorder that affects an identifiable and measurable patient population of less than that affects fewer than or equal to 1 in 2,000 people in Aotearoa New Zealand.
- The medicine is only regulated to treat the rare disorder. If it is registered for other disorders (or is part of phase three clinical trials for other disorders), those other disorders must meet principle 2.
If the intent of all three principles is met, Pharmac will accept an application for a rare disorders medicine and progress it. From there, we seek clinical advice from our expert advisory committees.
More information
If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.