Being diagnosed with a chronic illness is a life-changing event and can often require significant changes to a person’s lifestyle. While this is challenging enough on its own, a lack of public funding on treatments for the illness due to its rarity can make adjusting to the diagnosis even more daunting.

Rare diseases affect approximately 8% (400 million) of people globally, with around two million Australians living with a rare disease.1However, for New Zealand, the quantity of data on rare disorders in general is strikingly low, making it impossible to accurately estimate the prevalence of rare disorders in the country.2

This lack of data highlights the challenges for improving diagnoses, treatment and research for those living with rare disorders in New Zealand. In fact, New Zealand ranked last out of 20 OECD countries for the number of publicly funded modern medicines registered and launched between 2011 and 2020 (34 medicines).3

Rare disease advocator, Samantha Lenik, was diagnosed with the rare muscle-wasting disorder Pompe disease 10 years ago. Pompe disease is a rare, inherited and often fatal disorder that disables the heart and muscles. Mrs Lenik says a Pompe disease diagnosis, much like other rare diseases and disorders, is a life changing experience for the individual and their family.

“Being given any form of progressive, declining disease diagnosis is shocking, but in New Zealand, it is all the more terrifying when you are told there is a medicine that can slow down the progression and give you more time with your loved ones, but you can’t access it because the country doesn’t fund it,” says Mrs Lenik.

The lack of access to modern treatments in the country continues to plague New Zealanders living with rare diseases. In fact, less than a third (30%) of the modern medicines registered in 20 comparable OECD countries between 2011 and 2020 were registered in New Zealand – 131 out of 441 medicines – and only 26% of the medicines which were registered in New Zealand were then publicly funded.3

With a lack of access in New Zealand preventing Mrs Lenik from receiving treatment for her disease, she was lucky to be given access to a clinical trial in Australia.

A substantial portion of modern medicines are accessible through public funding channels in Australia compared to New Zealand, contributing to improved affordability and access for patients. Almost half (46.4%) of these medicines registered in Australia ultimately receive public funding, and on average, it takes approximately 481 days for modern medicines to receive public funding in Australia, a process that takes significantly longer in New Zealand, averaging at 798 days3.

“I never thought I’d have to experience the terror of having to decide whether to re-mortgage my home to pay for treatment, move overseas to access medicine, or worst of all, having to battle with the idea of not getting better due to not being able to afford treatment at all,” explains Mrs Lenik.

Following her treatment in Australia, Mrs Lenik was fortunate to continue her part in the clinical trial in New Zealand, through Auckland Hospital researchers collaborating with P3 Research at its Kapiti site. P3 Research has since merged with Holdsworth House and AusTrials to form Momentum Clinical Research.

Collectively the team has conducted more than 200 Phase Ib-IV clinical trials across a multitude of conditions, including rare diseases, and is encouraging overseas sponsors to host their clinical trials across their Trans-Tasman sites, with streamlined ethics processes.

Momentum Clinical Research Chief Medical Officer, Dr Richard Stubbs, emphasises how the entity works as a one stop solution for pharma companies to conduct clinical trials in Australia and New Zealand and for patients looking to participate in clinical trials at one of Momentum Clinical Research’s 13 sites across the Trans-Tasman.

“Rare disorder patients represent a significant minority group in any country’s health system; however, they face more barriers, and often suffer more from their condition than other patients. With access to treatments being limited for such patients, access to medications through involvement in clinical trials can become an important way in which their health outcomes can be improved,” says Dr Stubbs.

Mrs Lenik continues, “Participating in this trial has seen my muscle strength drastically improve and enabled me to live a full life for the past seven years. It shouldn’t be the case that the only way Kiwis can access life-saving medicines is through international clinical trials, self-funding, moving overseas, or compassionate access programmes.”

“Accelerating medical research through the introduction of a more streamlined and efficient approach to clinical trials is our goal. Identification of participants for clinical trials and their retention is an ongoing challenge for trial sponsors. Our streamlined approach to identifying participants and our presence in multiple locations throughout NZ and Australia allow us to meet and often exceed recruitment targets for the majority of our trials,” explains Dr Stubbs.

With rare disorder patients facing more barriers to treatment, the Trans-Tasman scale of Momentum Clinical Research is helping bring more clinical trials to New Zealand and Australia giving wider access to investigational medication. 

Allied Magazine