Our progress in the funding of medicines for rare disorders

Releasing the agenda offers more transparency to those affected by rare disorders, their whānau, and communities, and gives more visibility to those looking for updates on specific medicines.

“Medicines being considered by the Committee include treatments for Fabry disease and spinal muscular atrophy (SMA), and Pharmac staff and the Committee are looking forward to hearing directly from people who are affected by these rare disorders,” says Dr David Hughes, Pharmac’s Chief Medical Officer.

“This means Pharmac’s expert advisors, and our own staff will gain a deeper understanding of the experiences of those affected by these conditions. We’re making sure the right voices are heard by working with the consumer group Rare Disorders NZ(external link) – we’d like to offer them a big thank you for their time and expertise.”

“People living with rare disorders face many challenges, including access to suitable healthcare and effective treatment, and we acknowledge the difficulties they experience, as well as by their whānau and communities,” says Dr Hughes.

There are a number of medicines being considered in the March meeting. This includes three to treat spinal muscular atrophy, one of which is a new proposal for a gene therapy, onasemnogene abeparvovec (Zolgensma). The Committee will also consider proposals to widen eligibility for nusinursen and risdiplam, as well as considering new information received on treatments for Fabry disease, short bowel syndrome, and Morquio syndrome (Type A).