Call for funding applications for medicines for rare disorders
What we’re doing
Pharmac | Te Pātaka Whaioranga is asking for new funding applications for medicines for rare disorders.
We know that people with rare disorders face challenges including access to suitable health care and accessing effective medicines. We are committed to a regular call for applications for medicines for rare disorders ahead of our Rare Disorders Advisory Committee meetings.
We are asking for new applications from pharmaceutical suppliers so we can consider taking them to our next Rare Disorders Advisory Committee meeting on 29 July 2026.
Applications need to be submitted through PharmConnect by 5pm Friday 1 May 2026.
We also want to hear from people living with rare disorders, their whānau, and health care professionals, about new medicines that have not been previously considered for funding. Hearing about new medicines and how these could improve your daily life helps us to better understand what we should be considering, and what may be available for rare disorders in New Zealand in the future. Pharmac can then reach out to suppliers to check if these products could be available for the New Zealand market. This information can be shared with us via our rare disorders email address: raredisorders@pharmac.govt.nz.
Learn more about our work to fund medicines for rare disorders
Who we think will be interested
- Pharmaceutical suppliers of medicines for rare disorders
- People with rare disorders and their whānau, family and caregivers
- Healthcare professionals and support groups with an interest in medicines for rare disorders.
How does the call for applications work?
We are asking for funding applications for medicines for rare disorders that meet Pharmac’s Rare Disorders policy principles:
- The medicine has been approved by Medsafe, or an approved international regulatory authority[1], for the identified indication[2].
- 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[3]
- The medicine is only regulatory approved for the treatment of the rare disorder, or if it is approved for other disorders (or is part of phase three clinical trials for other disorders), the cumulative prevalence across all indications still meets principle 2.
All applications need to meet the requirements of a full funding application addressing our Factors for Consideration as best they can.
Pharmac has recently updated its rare disorders policy principles to better align with the Aotearoa New Zealand Rare Disorders Strategy(external link). This means that applications for medicines treating conditions affecting fewer or equal to 1 in 2,000 people in New Zealand will be able to be considered by Pharmac through this process.
There is some uncertainty about what this may mean for the number of applications we receive and capacity for applications to be considered by the Rare Disorders Advisory Committee. We’ll remain flexible about how we get timely advice. This may mean more meetings or using other Advisory Committees to help where appropriate. For each application we’ll make sure the relevant expertise is available at the Advisory Committee meeting when it’s considered.
A key difference for applications for medicines for rare disorders is that we can accept funding applications before the supplier applies to Medsafe for regulatory approval. We still need a supplier interested in supplying New Zealand, and Medsafe approval would still be required before a pharmaceutical could be funded by Pharmac.
If the application doesn’t meet the rare disorders principles, it may be referred to a different Advisory Committee that is more appropriate. We may also decide we can’t accept the application at this time – we would talk to you about this and might be able to reconsider it in the future.
Information to help you submit a funding application, including guidelines for applications is available on our website: Medicine funding application
To submit applications
Applications can be submitted through our application portal – PharmConnect by 5pm on Friday 1 May 2026.
Pharmac is available to meet with interested suppliers or other applicants prior to submission of any application. If you would like to discuss the application process, please contact us at raredisorders@pharmac.govt.nz
Next steps following the call for applications
| Date |
Action |
|---|---|
|
1 May 2026 |
Closing date for applications to be considered at the July 2026 meeting |
|
May 2026 |
Agenda for the July meeting published |
|
29 July 2026 |
Rare Disorders Advisory Committee meeting |
|
By 9 September 2026 |
Provisional recommendations released within 30 days of the meeting |
|
By October 2026 |
Record of meeting published on Pharmac’s website |
Applications received after 1 May 2026 may be considered for future Advisory Committee meetings.
What’s considered at the meeting?
The Rare Disorders Advisory Committee meeting in July 2026 will include discussion on new and existing applications for treatments for rare disorders and horizon scanning for medicines in the development pipeline. The Committee will also look at the medicines we have considered through our Named Patient Pharmaceutical Assessment (NPPA) pathway and whether these could be funded via the Pharmaceutical Schedule.
Seeking clinical advice informs our assessment of medicines. If we receive a positive funding recommendation from the Committee, we complete an assessment of the medicine considering its benefits and costs using the Factors for Consideration. We then rank the medicine on one of our priority lists for funding. This includes our options for investment list, which is a list of the medicines that we would like to fund if our budget allows it.
You can view all of the applications we’re currently considering on the application tracker(external link).
Rare Disorders Advisory Committee membership
The Rare Disorders Advisory Committee assesses applications within the context of rare disorders. Members are appointed for their expertise and understanding of evidence in rare disorders, including where only early or limited evidence is available. We won’t have every specialty represented on the Committee and can’t expect to.
Where more specialist knowledge is needed, we reach out to experts outside of the Committee. At the 2025 meeting, several Invited Experts attended to support the discussion and help answer questions for specific areas. Invited experts are not present and do not participate when the Committee votes on a recommendation.
This extra input can occur at any point in the process, before, during or after the clinical advice meeting.
Lived experience input from people with rare disorders
We want to hear from the rare disorders community about the medicines we are considering for funding for the treatment of rare disorders.
We will share the agenda of the Rare Disorders Advisory Committee meeting in May 2025.
Once the agenda is confirmed, we will seek patient lived experience submissions to support the applications being considered.
This approach has been trialled at previous meetings, with summary information (without personal details) included in the Committee records that are shared on our website. In 2025, we moved to written and video submissions of patient lived experience and shared these with the Committee before the meeting. We intend to follow this same approach for the 2026 meeting.
We are grateful for the patients and groups who shared their experiences with us and provided these submissions, it provided valuable support to the Committee’s consideration of the applications. We also thank Rare Disorders New Zealand for their support of this process.
We welcome feedback from people with rare disorders to support the Committees discussion and our on-going assessment of applications.
Queries and feedback about applications can be emailed to raredisorders@pharmac.govt.nz
About medicines for rare disorders
Previous Rare Disorder Advisory Committee meetings
The Rare Disorders Advisory Committee last met in June 2025. It considered treatments for paroxysmal nocturnal haemoglobinuria, hereditary angioedema, Fabry disease and spinal muscular atrophy. The full agenda for the meeting, provisional recommendations, and records of previous meetings are available on our Specialist advisory committees webpage.
Pharmac approach to Rare Disorders and updated policy principles
We are prioritising engagement with our Rare Disorders Advisory Committee and continue to support work on the rare disorders strategy. The strategy was published July 2024: Aotearoa New Zealand Rare Disorders Strategy. As noted, Pharmac has recently updated its rare disorders policy principals broadening the definition of a rare disorder (from affecting 1 in 50,000 to 1 in 2,000 people in Aotearoa New Zealand), which aligns with the Strategy, enabling more medicines to be considered.
Along with other key health agencies, Pharmac has recently committed to prioritise implementing the Rare Disorders Strategy.
Pharmac has also committed to a 12-month reset programme to become a more outward-focused and transparent organisation. More information can be found on the reset programme webpage.
Footnotes
[1] Refer to Medsafe website for guidance on recognised regulatory authorities(external link)
[2] Medsafe approval must still be gained before a pharmaceutical is listed on the Pharmaceutical Schedule.
[3] Updated to broaden the definition of a rare disorder (from affecting 1 in 50,000 to 1 in 2,000 people in Aotearoa New Zealand). This aligns with the 2024 Rare Disorders Strategy released by the Ministry of Health