“No country can claim to have achieved universal healthcare if it has not adequately and equitably met the needs of those with rare diseases.”
Helen Clark, United Nations
[Statement from UN Development Programme Administrator Helen Clark to the International Conference on Rare Diseases & Orphan Drugs, Cape Town, 20 October 2016.]
A rare disease is defined as a health condition that affects 1 in 2000 people or less. There are more than 7,000 rare disorders which affect around 300,000 New Zealanders – half of whom are children.
People living with rare disease often have complex needs and face unique challenges. Clinicians also face the challenge of limited awareness, information or connections to specialists with knowledge on rare diseases.
CALL FOR ACTION
Rare Disorders NZ, and the support groups we represent, are calling for acknowledgement and awareness of the common challenges faced by people living with a rare disease, along with a commitment to address these challenges through the development of a New Zealand National Rare Disorder Framework. We want inclusion of patients and clinicians in decision-making processes, consideration of a wider definition of patient quality of life, and impact of rare disorders for carers.
In New Zealand a shift in mindset is needed for rare disorders to stop being considered in isolation, and instead to be regarded as a significant factor within health policy frameworks. This is in alignment with the global rare disease movement headed by Rare Disease International in its quest for universal health coverage.
New Zealand lags far behind most OECD countries in supporting people living with rare disorders and their families to access the best healthcare. Australia announced support of a national plan for rare disease patients in late 2018, ensuring that no one is left behind. New Zealand needs inclusive policies which acknowledge barriers for the thousands of vulnerable children, adults and their caregivers who continue to fall through the cracks. We need to end the “postcode lottery” to ensure all people living with rare disorders have access to the best healthcare.
People living with a rare disorder will have improved healthcare and wellbeing through access to diagnosis, medicines and services.
Rare Disorders NZ has identified seven strategic priorities and is leading the call for the development of a New Zealand National Rare Disorder Framework to ensure equitable healthcare for all people living with a rare disorder.
SEVEN STRATEGIC PRIORITIES
Early and accurate diagnosis of rare disease to improve health outcomes for patients and long-term benefits to the New Zealand economy.
- ACCESS TO MEDICINES
Equitable access with an appropriate assessment pathway and criteria for rare disorders which takes into consideration their small patient populations, progressive disease and limited data.
- COORDINATED CARE
A coordinated ‘holistic’ approach to care is essential for integrated and cohesive healthcare, this would enable inter-disciplinary services and effective collaboration between all health professionals, community service providers, Ministry of Social Development and Ministry of Health.
- ACCESS TO WIDER SERVICES
Access based on need not on a set condition or disease type. Simplified process to enable access to health and disability services that is person centred and holistic so that no-one is left behind because they don’t tick a box.
A national coordinated programme of research on rare diseases that includes active participation by patients, carers and patient advocacy groups. Improved government commitment and associated funding for rare disease research.
- NATIONAL RARE DISEASE REGISTRY
Improved data to ensure accurate awareness of the demographics of rare disorders and to allow responsive access to each other and to clinical trials that may be available.
- WORKFORCE DEVELOPMENT
Clear planning for sustainable training to ensure workforce are ready and available – including genetic counsellors, genetic biostatistician, trained specialists, allied health professionals and lab workers.