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Pharmac funds first-of-its-kind drug to treat spinal muscular atrophy

9 November, 2022

Interview by Joe Wickins, adapted by Stella Huggins

Listen to the full interview

A first-of-its-kind medicine (SPINRAZA) that treats spinal muscular atrophy, was finally funded by Pharmac last week on 8 December. Spinal muscular atrophy is a rare genetic disease, often labelled 'baby motor neuron disease', as it is characterised by healthy muscles, but a deficit in ‘message sending’ in nerve cells. 

The disease is crippling- in Aotearoa, 50% of all kids with the condition are type 1 and the average age of death for type 1 is 13 months old. 

Three treatments exist internationally, including SPINRAZA, funded just last week. It operates by bolstering a ‘backup copy’ of the gene that controls the production of the protein needed to ensure the nerves don’t die, which is faulty in sufferers. Other treatments include oral and gene therapy.

Comparatively, Australia already has all three treatments available, despite Aotearoa applying for SPINRAZA at the same time in 2018.

Fiona Tolich, a long-term campaigner and advocate for Spinal Muscular Atrophy, and a Trustee of Patient Voice Aotearoa, told Joe on The Wire that SPINRAZA was something “that we should never have had to fight so hard to get, because it really is life-saving.” 

Tolich says in the time since the application went through, children have had to deal with horrific medical interventions and consequences. Experiences like having feeding tubes inserted, losing the ability to walk, roll, and crawl. Some have had spinal rods to stop spinal collapse.

She stresses that “it’s incredibly cruel, because cognitively these children are very sharp, very aware and they know what’s happening to their body... they know what the future holds. Which is incredibly scary when you consider that could be stopped.”

The community is pleased with the overdue allocation of funding for the medication, but Tolich is critical of Pharmac’s process, citing the amount of advocacy and time it takes for medications to be funded.

She also believes we need a societal rethink on how we evaluate drug funding, “if we actually just funded these medicines it would take so much pressure off the health system, it would take so much pressure off the social welfare system. Because you would actually keep people well and out of hospital and contributing to the economy, as opposed to spending more to keep them sick.”

Tolich says Pharmac needs to “follow Treasury guidelines- which they don’t”, and calculate the “true value” of medicines like SPINRAZA. She claims that 73 medicines still remain on Pharmac’s waiting list.

Public interest journalism funded by NZ On Air