RDNZ's feedback on proposal to fund Nusinersen (Spinraza) for Spinal Muscular Atrophy

Rare Disorders NZ have been long-time advocates for access to medicines for Rare Disorders including Spinal Muscular Atrophy (SMA). For the purpose of this submission, we will keep it to SMA, an area of significant unmet need.

It is important to reference the advocacy as it has taken a significant toll on patients, their loved ones, and the rare disorder community in the time that it has taken to get to this point.  This is important to note as we would like this consultation process to consider the unmet need of many in the community that this proposal leaves behind.  We would like to see changes to include the full community so the relentless advocacy efforts can be focused on other areas of unmet need within the rare disorders community as opposed to access to medicine.

Rare Disorders NZ fully support the consultation and funding of this drug/medicine as outlined in the consultation proposal. However, we do ask that Pharmac consider our following recommendations in addition.

What Rare Disorders NZ would like to propose: