Oct 19, 2022, 10:21 PM

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:

  1. Broadening of the criteria to include SMA Type 3B, 3C and adults.  We do not see a reason why children who became symptomatic after 3 years of age should not be included.  The disease still has a devastating impact on them and can be treated, so should be. Whilst we appreciate the initial application was for 18-year-olds and under, countries like Australia are now funding adults, after their initial applications were for the same initial age gap.  Both Australia and New Zealand had applications submitted by Biogen in 2018, Australia had access that year and we are yet to have this confirmed.  As Australia now fund the treatment of adults, I would like to see some proactive consideration here.  We do not believe a separate application needs to be waited for, resulting in significant time lost.  This would also allow for ease of transition of patients from paediatric hospital care to adult services. We have also already seen adults leave New Zealand to shift abroad to get access.  There is a need, and we should be broadening this to include all patients.  This would also avoid unnecessary applications via the Waiver process and more unneeded stress on the community.
  2. Inclusion of pre-symptomatic children with 1 copy of SMN2.  We think there is an ethical question here of screening someone and then not offering them access to treatment that could see them live and live better.  There are cases of children who have 1 copy of SMN2 and a milder form of SMA. Treatment would absolutely be of significant benefit to them.
  3. The funding of Evrysdi (Risdiplam).  This would allow clinicians to make the right treatment choice for their patient. Due to the administration of Nusinersen, for some patients, spinal fusions or severe spinal deformity make the administration via lumbar puncture technically challenging.  This treatment is already recommended for funding and it would be a case of either as opposed to both.  This might also allow for more remote families to avoid any interruptions to their income (which is already severely impacted) in order to access clinical and procedural needs in major cities.  We do want to be very clear though, We absolutely want to see funding go through for Nusinersen.  This is paramount.  We are wanting to specifically highlight the need for patient centred options with a clinician’s expert advice for their individual and unique situation.

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