The development of the Rare Disorders Strategy

How the Rare Disorders Strategy came to be

For over 20 years, Rare Disorders NZ along with other patient groups and stakeholders has been calling for action to improve services for people living with a rare disorder.

The call was amplified through Rare Disorders NZ's Fair for Rare campaign in 2020 along with a petition to parliament in 2021 led by Sue Haldane (a parent of a child with a rare disorder) and supported by Rare Disorders NZ. The petition resulted in oral submissions to the Health Select Committee and a meeting with then Minister of Health Andrew Little.

The independent Pharmac Review, released in 2022, heeded the call from Rare Disorders NZ's submission for a national Rare Disorders Strategy, and included this in one of its recommendations.

On the day the Pharmac Review was released, the Minister of Health announced that he had directed Manatū Hauora | Ministry of Health to develop a strategy for rare disorders.

Rare Disorders NZ worked with Manatū Hauora |The Ministry of Health during the development of the Strategy to ensure that the Strategy was informed by lived experience. The Aotearoa New Zealand Rare Disorders Strategy was released in July 2024.

Reflections on the Strategy

The Aotearoa New Zealand Rare Disorders Strategy (the Strategy) is an important first step for the Government to recognise the unique needs and challenges of people living with a rare disorder, who make up 6% of the population.

Rare Disorders NZ was actively involved in the development of the Strategy and provided input and feedback throughout the process. We pushed for the inclusion of four key implementable actions in the Strategy:

It was pleasing to see that we succeeded in getting three out of the four actions included in the Strategy:

While not explicitly mentioned, the final Strategy includes the building blocks for establishing a Rare and Undiagnosed Centre of Expertise;
The Strategy stipulates the intention to incorporate coding for rare disorders in the national data collection classification system;
Rare Disorders NZ has been identified as having an important role to play in actioning the Strategy and we have been told that we will be included in the implementation process.

We were also pleased that our proposal for New Zealand’s official definition of a rare disorder was accepted, ensuring the definition aligns with the widely accepted international definitions of a rare and ultra rare disorder.

We strongly advocated for both Manatū Hauora | Ministry of Health and Te Aka Whai Ora | Māori Health Authority to engage directly with the rare community to ensure the Strategy would be informed by lived experience. The engagement approach Te Aka Whai Ora | Māori Health Authority took was exemplary for the way in which whānau Māori were approached and able to provide their reflections and input. Unfortunately, a similar process for the wider rare community did not happen, though through our insistence Manatū Hauora | Ministry of Health actuated webinars with the patient community, support group leads and experts as a way to collect their input to feed into the Strategy.

One of the most significant outcomes we achieved in the Strategy was that people and their whānau living with a rare disorder will be one of the population groups routinely considered by decision makers when designing health system improvements. The rare voice will be sought for input in major system reviews, service evaluations, business cases and investment decision-making through consumer forums, advisory boards and committees.

Where the Strategy falls short

A glaring omission from the Strategy was regarding access to rare disorder medicines. We wanted to see intention to establish a single barrier-free pathway to rare disorder medicines.

Unfortunately, medicines were only very briefly referred to in the Strategy, which is particularly disappointing given the independent Pharmac Review specifically called for the development of a Rare Disorders Strategy.

We will continue to work with Pharmac on how to improve medicine access for the rare disorder community.

We would also like to have seen the Strategy acknowledge the impact rare disorders have on every aspect of life, not just on health. Living with a rare disorder impacts finances, education, workforce participation, as well as everyday life tasks.

We will continue to work with respective government agencies to ensure that people living with a rare disorder are taken into consideration in system investments and policy development, and that consideration is given to establishing pathways to recognise the unique needs and challenges of the rare community.

We expressed our concerns regarding these omissions in meetings and the following correspondence with the Ministry and the Minister’s office during the development of the Strategy.