Pharmac seeking feedback on funding a second SMA treatment

Te Pātaka Whaioranga - Pharmac is initiating a consultation today, seeking feedback on a proposal to fund of risdiplam (branded as Evrysdi) for people with the rare genetic disorder spinal muscular atrophy, who meet the eligibility criteria.

Pharmac’s director of operations Lisa Williams says Pharmac is thrilled to be taking the next step towards progressing a second treatment for those with spinal muscular atrophy for funding, with nusinersen (branded as Spinraza) funded from 1 January 2023.

“Risdiplam has the potential to make a substantial difference to the lives of young people in New Zealand living with spinal muscular atrophy and for their whānau and support networks too,” says Ms Williams. “Having a treatment available that can be taken orally, from home, and once a day has major benefits to improve access for those who need it.”

“We have received clinical advice from our Rare Disorders and Neurological Advisory Committees along with our Pharmacology and Therapeutics Advisory Committee (PTAC) recommending that we fund risdiplam. The eligibility criteria we are proposing are based on the expert advice we have received and clinical data available, similar to the criteria for nusinersen,” says Ms Williams.

The consultation is available on the Pharmac website for anyone wanting to have their say. “Consultation is a very important step in our process,” says Ms Williams. “It’s how we check that the people who will get the most benefit from the medicines will be able to access them.

“As well as seeking feedback on the funding of risdiplam, we are also interested in feedback on how community distribution of risdiplam could be managed. If the decision is approved, we will be working closely with the supplier, Roche and our colleagues across the health sector to plan for the implementation of risdiplam.”

If approved, funding would start from 1 May 2023.