Rare Disorders NZ Chief Executive Chris Higgins and Board Chair James McGoram had a constructive meeting with Minister of Health Hon. Dr Ayesha Verrall in June; the first between Rare Disorders NZ and the new health minister. The meeting provided an important opportunity to discuss the progress of the Rare Disorders Strategy currently being developed by Manatū Hauora and to seek the Minister’s assurance that funding would be allocated in the 2024/25 budget for its implementation.
The Minister indicated that along with the Pae Ora Strategies, the Rare Disorders Strategy would be resourced through the overall allocations to Te Whatu Ora and Te Aka Whai Ora.
Also discussed at the meeting was the urgent need to include SMA testing in the newborn screening heel prick test now that pre-symptomatic treatment of SMA is funded by Pharmac. It was explained by a Te Whatu Ora official present at the meeting that implementation would take some time as it was not a straightforward process. Rare Disorders NZ continues to have concerns about this given the risk of potential rapid deterioration of babies born with SMA without diagnosis, and will continue to explore possibilities for having things fast-tracked.
Finally, concerns around compassionate access to unfunded medicines under the new Therapeutic Products legislation were also discussed with the Minister. The Minister indicated that it was her intention that the compassionate access arrangements would continue under the new legislation and undertook to follow up further.
Rare Disorders NZ will continue to follow up on these issues with Te Whatu Ora to ensure they are not sidelined and action is taken to address them.