Rare Disorders Month: We need action to support the 300,000 Kiwis seeking better healthcare – Opinion

THREE KEY FACTS

• 300,000 New Zealanders are living with a rare disorder.
• In July last year, the country’s Rare Disorders Strategy was released by the Ministry of Health.
• It acknowledges that people with rare disorders and their families often face barriers in getting timely, effective and equitable healthcare.

For 25 years, Rare Disorders NZ has fought for 300,000 Kiwis living with rare conditions, a group long overlooked by our healthcare system.

As we reflect on our anniversary and the progress made during Rare Disorders Month in March, it’s clear that while we’ve made strides, the work is far from done. Rare disorders continue to be neglected – and those affected face barriers to the care and resources they need.

This year, our focus has been on pressing the Government to take urgent action on implementing the national Rare Disorders Strategy, a plan that has been agreed upon but delayed for far too long. Until it is fully implemented, people living with rare disorders will continue to struggle in a healthcare system that isn’t designed to meet their unique needs.

Living with a rare disorder is often a lifelong challenge. These conditions are frequently complex and invisible, leaving patients vulnerable to misdiagnoses, delays in care and inadequate treatment.

Despite affecting 6% of the population, rare and undiagnosed disorders remain underfunded and undervalued within the healthcare system, with little awareness or support.

However, Rare Disorders Month also brought hope. One of the most significant developments was the formation of New Zealand’s first-ever cross-party parliamentary group on rare and undiagnosed disorders. MPs from across the political spectrum have come together to advocate for those living with rare conditions. This is a powerful message that rare disorders are no longer being ignored and are deserving of urgent action and attention.

This month also showcased the innovative research taking place in Aotearoa. The groundbreaking work being done by New Zealand-based researchers offers a glimmer of hope for faster diagnoses and better treatments for rare disorders. With more progress being made, we’re closer than ever to providing patients with better care and outcomes.

The potential of gene technology was another highlight. With the Gene Technology Bill currently before the health select committee, we heard first-hand from the principal investigator of the CRISPR/Cas9 clinical trial for hereditary angioedema (painful swelling in the subcutaneous tissue, triggered by an allergic reaction or medication).

Gene-editing technologies such as CRISPR have the potential to revolutionise the treatment of rare disorders. If we update regulatory frameworks to make these technologies more accessible, we could see life-changing treatments available to many rare disorder patients in the near future.

Despite these developments, there is still much work to do.

The New Zealand healthcare system is under strain. Resources are limited and healthcare workers are stretched thin.

Those with rare disorders face additional complexities that make accessing care even more difficult. While healthcare needs are growing across the country, those with rare conditions are especially vulnerable and need targeted support.

This raises a key question: how can we build on the momentum created by the cross-party parliamentary group to drive systemic change in the broader health sector? The cross-party support for rare disorders should serve as a model for addressing other areas of the health system, such as mental health, long-term care and preventative services. If we can work together to improve the lives of those with rare disorders, we can make the healthcare system stronger for everyone.

We’ve been invited to meet with new Health Minister Simeon Brown in May and we’ll use this time to press the urgency of implementing the Rare Disorders Strategy. This strategy is not just about improving care for people with rare disorders, it’s about creating a healthcare system that is responsive and accessible for all New Zealanders. By ensuring that 300,000 Kiwis living with rare conditions get the care they need, we strengthen the system for everyone.

The cross-party parliamentary group is an encouraging step, but we must ensure that it leads to tangible action. The Government must act swiftly to implement the Rare Disorders Strategy and provide the care and resources that 300,000 Kiwis need and deserve.

Rare Disorders Month has been a time to celebrate the progress we’ve made, but it also serves as a reminder that there’s much work left to do. The journey is not over, but with continued advocacy and determination, we will ensure that no one is left behind.

Making New Zealand a better place for those living with rare disorders will ultimately make this country a better place for all.