Proposal to fund nusinersen (Spinraza) for spinal muscular atrophy
What we’re proposing
We are seeking feedback on a proposal to fund nusinersen, branded as Spinraza, for the treatment of spinal muscular atrophy (SMA) in New Zealand.
Nusinersen would be funded through a provisional agreement with the supplier, Biogen NZ Biopharma Ltd (Biogen). This agreement also includes price reductions for three currently funded multiple sclerosis treatments supplied by Biogen.
Consultation closes at 4pm, Monday 24 October 2022 and feedback can be emailed to email@example.com
What would the effect be?
From 1 January 2023, nusinersen would be funded in New Zealand. We are proposing that people aged 18 years and under with pre-symptomatic SMA or symptomatic type I, II or IIIa SMA would be eligible for funded treatment with nusinersen.
SMA is a rare disorder. New Zealand epidemiology data suggests that up to 50 people could be eligible for funded treatment with nusinersen. The number of people receiving treatment is anticipated to increase over time. We expect that each year up to four people may be diagnosed with SMA and be eligible for treatment.
Nusinersen would be funded subject to a provisional agreement (contract) with the supplier, Biogen. This agreement also includes proposed reductions in the net price of three funded multiple sclerosis treatments, natalizumab, dimethyl fumarate and interferon beta-1-alpha. There would be no changes to the eligibility criteria for these multiple sclerosis treatments for patients.
Who we think will be interested
- People with SMA, their whānau, friends, and caregivers
- Healthcare professionals involved in the care of people with SMA
- Te Whatu Ora – Health New Zealand hospitals and other organisations who deliver services and support for people, and their whānau who are affected by SMA
- Pharmacies and wholesalers
- Pharmaceutical suppliers
About spinal muscular atrophy (SMA)
Spinal muscular atrophy (SMA) is a rare genetic disorder. SMA impacts infants through to adults and can cause disability and early death. There are currently no funded treatments in New Zealand.
SMA is a disorder that spans a wide spectrum of severity. There are different types of SMA, such as type I, II or IIIa. These types of SMA are generally diagnosed when symptoms of SMA occur before three years of age. SMA affects the control of muscle movement and people with different types of SMA generally experience different levels of symptom severity. The table below shows a general overview of the different types of SMA.
|Age at Symptom Onset
|Highest Motor Function
|Average Life Expectancy
|None – unable to sit
|< 6 months
|None – unable to sit
|Up to 2 years
|6 - 18 months
|Sitting. Unable to walk independently.
|< 3 years (IIIa)
>3 years (IIIb)
> 12 to ≤18 years (IIIc)
|Independently stand and walk. May lose ability to walk over time.
|> 18 years
|Mild motor impairment.
In severe cases SMA can impact a baby’s ability to swallow, hold their head up, sit and roll over. Sadly, infants with the most severe types of SMA often pass away in their first few months or years of life. In later onset SMA, such as type IIIa, people can experience significant muscle weakness and disability, with some people losing the ability to walk over time. SMA also has a significant impact on whānau and caregivers of people with SMA.
SMA diagnosis is confirmed with genetic testing. SMA can also be diagnosed before symptoms start (‘pre-symptomatic SMA’), however this testing isn’t widely available in New Zealand yet because there isn’t a funded treatment (like nusinersen). Pharmac is in touch with the National Screening Unit, and we understand that if a treatment was funded, a national screening programme would likely be rolled out. We understand that this may take up to two years.
Nusinersen (brand name Spinraza) is a medicine used to treat SMA. It can be used before or after symptoms of SMA begin.
Motor neurons play an important role in the body, sending messages from the brain to muscles and telling them what to do. People with SMA can’t make enough of a particular protein (called SMN) which is needed for motor neurons to function well. Nusinersen works by increasing the production of this protein, helping the motor neurons to function.
From the data currently available, nusinersen has been shown to be an effective treatment for SMA. While nusinersen doesn’t cure SMA, it can reduce how severe a person’s condition is. For people treated symptomatically with nusinersen, clinical trials have shown that treatment can have a positive impact on motor function, need for ventilation and survival. Clinical trials have also shown that many infants treated with nusinersen pre-symptomatically meet expected motor developmental milestones, like sitting and rolling over, as well having improved survival.
Nusinersen is given as an injection into the spine by a trained healthcare professional in a hospital setting. When starting treatment, people have four injections within a couple of months, and from then on receive one injection every four months. Treatment is used long term.
Why we’re proposing this
We first received a funding application in 2018 to fund nusinersen for the treatment of paediatric patients (18 years of age or under) with infantile-onset spinal muscular atrophy (SMA) (Type I) or childhood-onset SMA (Types II and IIIa) with onset of symptoms prior to three years of age. At first our expert clinical advisors recommended that a funding recommendation be deferred until further evidence about the treatment was available.
In 2019 we received further information, as well as an application to fund nusinersen for pre-symptomatic SMA. Our Rare Disorders Subcommittee assessed the new evidence and recommended that nusinersen be funded:
- with a high priority, within the context of the rare disorders therapeutic area, for the treatment of pre-symptomatic individuals with spinal muscular atrophy and two or three SMN2 copies, subject to the Special Authority criteria; and
- with a medium priority, within the context of the rare disorders therapeutic area, for the treatment of symptomatic patients aged 18 years or under with type I, II, and IIIa spinal muscular atrophy who experience symptoms prior to three years of age, subject to the Special Authority criteria.
In 2020, our Pharmacology and Therapeutics Advisory Committee (PTAC) also reviewed the new evidence and recommended that nusinersen be funded with a high priority for the above indications.
Our clinical advisors made these funding recommendations based on the absence of funded alternatives, the high health need of people with SMA and their family/whānau, and the meaningful clinical benefit of nusinersen.
Pharmac first prioritised nusinersen as an option for investment in April 2020. We have re-ranked the proposal several times as we received new information and/or pricing proposals.
We are proposing to fund nusinersen now as we have reached a provisional agreement with Biogen, which includes price reductions on other medicines. This in combination with our recent budget uplift, has enabled us to progress this proposal.
You can see the history of the applications and the advice we’ve received here:
- Nusinersen for SMA type I, II and III(external link)
- Nusinersen for pre-symptomatic individuals with SMA(external link)
Details about our proposal
Nusinersen (Spinraza) would be listed in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 January 2023 as follows:
|Proposed price and subsidy
|Inj 12 mg per 5 ml vial
A confidential rebate would apply that would reduce the net price of nusinersen to the Funder. The Spinraza brand would have protection from delisting and subsidy reduction until 30 June 2026.
Nusinersen would be listed as a PCT only pharmaceutical in Section B of the Pharmaceutical Schedule, meaning that only Te Whatu Ora hospitals would be able to make a subsidy claim.
Nusinersen would be listed in Section B and Part II of Section H subject to the following eligibility criteria:
Special Authority for Subsidy
Initiation application – spinal muscular atrophy (SMA) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- Patient has genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygous mutation; and
- Patient is 18 years of age or under; and
- Patient has experienced the defined signs and symptoms of SMA type I, II or IIIa prior to three years of age; or
- Patient is pre-symptomatic; and
- Patient has only two or three copies of SMN2
Renewal – spinal muscular atrophy (SMA) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:
- There has been demonstrated maintenance of motor milestone function (as assessed using age-appropriate scales) since treatment initiation; and
- Patient does not require invasive permanent ventilation (≥16 hours per day), or non-invasive permanent (≥16 hours per day) assisted ventilation (breathing support administered via nasal cannula or face mask), in the absence of a potentially reversible cause while being treated with nusinersen.
The renewal requirement detailing non-invasive ventilation follows expert advice we received regarding current clinical practice in New Zealand. This outlined that currently in New Zealand, supportive care for people with SMA does not include invasive permanent ventilation. In addition, we received advice that (in the absence of a reversible cause) the requirement for non-invasive ventilation (as described in the criteria) would indicate that a person was beginning to respond less well to treatment and would therefore be an appropriate stopping criterion for continued funded access. You can read the advice we received here under "seeking clinical advice": 2020 PTAC record(external link).
We understand that due to the way nusinersen is given, the number and range of service providers who would administer nusinersen would likely be limited. We are proposing that nusinersen would be funded from 1 January 2023 but acknowledge that hospitals may need to establish/expand services to administer nusinersen to patients and that this may take some time. We acknowledge that due to this and the holiday season treatment might not be available for everyone on day one of funding, however we are proposing 1 January 2023 to ensure that patients have access to treatment at the earliest opportunity, as soon as hospitals are ready to deliver it. We would like to hear from the health sector about whether the proposed implementation date is reasonable.
Price changes to three currently funded treatments supplied by Biogen
The supplier of nusinersen, Biogen, also supplies three treatments that are currently funded for people with relapsing remitting multiple sclerosis: natalizumab (Tysabri), dimethyl fumarate (Tecfidera) and interferon beta-1-alpha (Avonex). As part of this proposal the net price for all three treatments would reduce by confidential rebate from 1 January 2023 and they would all have protection from delisting and subsidy reduction until 30 June 2026.
There would be no other changes to the current eligibility criteria or list price for natalizumab, dimethyl fumarate or interferon beta-1-alpha as part of this proposal.
To provide feedback
Send us an email: firstname.lastname@example.org by 4pm, Monday 24 October 2022
All feedback received before the closing date will be considered by Pharmac’s Board (or its delegate) prior to making a decision on this proposal.
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