Rare disorders call for applications

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 Wednesday 11 June 2025.

Applications need to be submitted through PharmConnect by 5pm Monday 24 March 2025.

Along with asking for new funding applications we want to hear from people living with rare disorders, their whānau, and health care professionals supporting them. Hearing about your experiences helps us to better understand what we should be funding.

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 the principles of our Rare Disorders policy:

1. The medicine has been approved by Medsafe, or an approved international regulatory authority[1], for the identified indication[2].
2. The disorder is a clinically defined disorder affecting an identifiable and measurable patient population with a prevalence of less than 1:50,000 in New Zealand.[3]
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.

If the application doesn’t meet these principles, we might decide there is a different Advisory Committee that is more appropriate to take it to. We might 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.

All applications need to meet the requirements of a full funding application addressing our Factors for Consideration as best they can.

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. Medsafe approval would still be required before a pharmaceutical could be listed on the Pharmaceutical Schedule.

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 Monday 24 March.

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	Detail
24 March 2025	Closing date for applications to be considered at the June meeting
April 2025	Agenda for the June meeting published
11 June 2025	Rare Disorders Advisory Committee meeting
September 2025	Record of meeting published

Applications received after 24 March 2025 may be considered for future Advisory Committee meetings. 

What’s considered at the meeting?

The Rare Disorders Advisory Committee meeting in June 2025 will include discussion on new and existing applications and horizon scanning for medicines in the development pipeline. It will also look at the medicines we have considered through our Named Patient Pharmaceutical Assessment (NPPA) pathway.

Seeking clinical advice informs our assessment of medicines. If we receive a positive funding recommendation from the Committee we rank the medicine on our options for investment list. This list includes medicines that we would like to fund if our fixed budget allowed it.

You can view all of the applications we’re currently considering on the application tracker(external link).

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 April 2025.

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

The Rare Disorders Advisory Committee last met in May and July 2024. At this time, it considered treatments for Gaucher disease, Fabry disease, hereditary angioedema, Pompe disease and von Hippel-Lindau disease. The records of these meetings will be available on our website soon on our Specialist advisory committees webpage.

We are prioritising engagement with our Rare Disorders Advisory Committee and have been supporting Ministry of Health – Manatū Hauora in its important work to develop the rare disorders strategy. The strategy was published July 2024: Aotearoa New Zealand Rare Disorders Strategy | Ministry of Health(external link).

Pharmac acknowledges that the Aotearoa New Zealand Rare Disorders Strategy defines a rare disorder as a medical condition affecting fewer than or equal to 1 in 2,000 people in New Zealand.

Currently, our Rare Disorders Policy aligns with the Strategy’s definition of an ultra-rare disorder, which affects less than 1 in 50,000 people in New Zealand. This helps remove market entry barriers to New Zealand for medicines used in a small group of people. We are working to update our policy to ensure alignment with the Strategy. This will have no practical implications for this process.

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] We are aware of, and support, the Aotearoa New Zealand Rare Disorders Strategy Te Rautaki o Aotearoa e Pā ana ki ngā Mate Mokorea. This Strategy defines ‘rare disorder’ as a condition that affects fewer than or equal to 1 in 2,000 people in Aotearoa New Zealand, and ‘ultra-rare’ as one that affects fewer than or equal to 1 in 50,000 people in Aotearoa New Zealand. Pharmac is currently working to align definitions. This will have no practical implications for this process.