Proposal to fund risdiplam (Evrysdi) for spinal muscular atrophy

Medicines Consultation Closes 24 Feb

What we’re proposing

We are seeking feedback on a proposal to fund risdiplam, branded as Evrysdi, for the treatment of spinal muscular atrophy (SMA) for people who meet the eligibility criteria, from 1 May 2023.

Risdiplam would be funded through a provisional agreement with the supplier, Roche.

In addition, we are interested in feedback on how community distribution of risdiplam could be managed. 

Consultation closes at 4pm, Friday 24 February 2023 and feedback can be emailed to consult@pharmac.govt.nz 

What would the effect be?

From 1 May 2023, risdiplam would be funded in New Zealand. Risdiplam would have the same access criteria as nusinersen (Spinraza) for the treatment of symptomatic type 1, 2 and 3a SMA for people who start treatment when they are 18 years or younger.

This would mean that there would be two funded options for the treatment of symptomatic SMA in New Zealand. Risdiplam would provide an oral treatment option alongside nusinersen, which has been funded since 1 January 2023 and is given as an intrathecal injection into the spinal canal in hospital.

SMA is a rare disorder. We estimate that in the first year, 30 to 50 people would be eligible for funded treatment with either nusinersen or risdiplam. We expect that each year up to four people may be diagnosed with SMA and be eligible for treatment. As a result, the number of people receiving treatment is anticipated to increase over time.

Risdiplam would not be funded in the pre-symptomatic setting at this time, because it is not approved by Medsafe for this use in New Zealand and we have not yet assessed it for funding. Under this proposal we would widen access to risdiplam to include pre-symptomatic use if risdiplam is granted Medsafe approval for this use in the future, and we receive a positive funding recommendation from our clinical advisors. Risdiplam in the pre-symptomatic setting will be considered by the Rare Disorders Advisory Committee in March 2023.

A direct delivery process would be set up to manage the supply of risdiplam to an individual’s nominated location. See the details about our proposal section for more details.

Pharmac recently funded nusinersen (Spinraza) for SMA from 1 January 2023. When we consulted on funding nusinersen, we heard that people would like wider access to SMA treatments, including for those who are aged over 18 years at the time they start treatment. We will be seeking expert clinical advice on the funding of both nusinersen and risdiplam for wider groups in March.

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
  • People or groups with an interest in treatments for rare disorders
  • Pharmacies and wholesalers
  • Pharmaceutical suppliers

About spinal muscular atrophy

Spinal muscular atrophy (SMA) is a rare genetic disorder. SMA impacts infants through to adults and can cause disability and early death.

SMA is a disorder that spans a wide spectrum of severity. There are different types of SMA, such as type 1 ,2 and 3a (also referred to as types 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.

Terminology

SMA Type

Age at Symptom Onset

Highest Motor Function
Achieved

Average Life Expectancy

Pre-natal

0

Prenatal

None – unable to sit
or roll

Weeks

Infantile-onset

I

< 6 months

None – unable to sit
or roll

Up to 2 years

Childhood-onset

II

6 – 18 months

Sitting. Unable to walk independently.

Adulthood

III

< 3 years (IIIa)

>3 years (IIIb)

> 12 to ≤18 years (IIIc)

Independently stand and walk. May lose ability to walk over time.

Normal lifespan

Adult-onset

IV

> 18 years

Mild motor impairment.

Normal lifespan

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 3a, 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.

Pharmac has recently announced the decision to fund nusinersen (Spinraza) from 1 January 2023 for people with SMA types 1, 2 and 3a and those with pre-symptomatic 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 until recently there was no funded treatment. Pharmac is in touch with the National Screening Unit and continues to work with Te Whatu Ora to support the consideration of this service.

About risdiplam (Evrysdi)

Risdiplam (brand name Evrysdi) is a medicine used to treat SMA. It is an oral liquid taken once every day. The recommended dose of risdiplam is determined by age and body weight. It is approved by Medsafe for treatment of SMA in people aged 2 months and older (see Medsafe Datasheet(external link) for details).

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. Risdiplam works by helping the body make more of this protein and maintain it throughout the body with continued use. 

Evidence shows that risdiplam, compared to placebo, improves survival and improves likelihood of achieving motor milestones for infants with SMA, and improves and maintains motor function and respiratory function in people with SMA. 

Risdiplam is available as a powder to be mixed with water to make an oral solution. This would be prepared by a health professional before it is dispensed to the individual. Reusable oral syringes are provided with the bottle to measure the right dose. Once the solution is made, it can be stored in the fridge for up to 64 days. If necessary, it may be stored at room temperature (below 40˚C) for no more than a total combined time of 5 days.

This means that risdiplam could be taken or given at home and would not require the specialist resources that are required to administer nusinersen, which is given by intrathecal injection. 

Why we’re proposing this

We received a funding application to fund risdiplam for symptomatic SMA types 1, 2 and 3 in December 2020. We sought advice from the Rare Disorders and Neurological Advisory Committees at a joint meeting in March 2021 and again in July 2021. Initially, the Committees recommended that risdiplam be funded for type 1 SMA with a high priority and deferred making a recommendation for type 2 and 3 SMA, pending more evidence.

In July 2021, the two Advisory Committees assessed new evidence and recommended that risdiplam should also be funded for type 2 and non-ambulant (not able to walk around) type 3 SMA, where people are less than 25 years at the time of treatment initiation.

The Pharmacology and Therapeutics Advisory Committee (PTAC) also assessed the funding application for risdiplam in August 2021. The Committee recommended that risdiplam be funded for type 1 SMA with a high priority, and a medium priority for type 2 and 3a SMA, where treatment is initiated at or before 18 years of age, regardless of ambulatory status. 

The funding criteria for risdiplam recommended by our Advisory Committees differ. The reasons for these differences are outlined in the records of the Committee meetings. These records can be read on the Application Tracker(external link).

Our clinical advisors made these funding recommendations based on the high health need of people with SMA and their family/whānau, the improved suitability of risdiplam as an oral treatment compared to the alternative treatment that is given by intrathecal injection, and the meaningful clinical benefit of risdiplam.

Clinical advice we have received indicates that risdiplam and nusinersen are likely to have a similar benefit for people with SMA, although there are currently no direct comparisons of the two medicines.

We are proposing to progress the funding of risdiplam for symptomatic SMA for the same groups as those eligible for nusinersen. This is based on the clinical advice and recommendations we have received, economic modelling and commercial arrangements. The treating clinician would determine the best treatment option for an individual in collaboration with the person and their whānau or caregiver.

Pharmac first prioritised risdiplam for the treatment of symptomatic SMA as an option for investment in June 2021. We have re-ranked the proposal several times as we received new information and/or pricing proposals.

We are proposing to fund risdiplam now as we have reached a provisional agreement with Roche. This in combination with our recent budget uplift, has enabled us to progress this proposal. 

Pre-symptomatic SMA

In August 2022, Pharmac received an application to fund risdiplam for the treatment of pre-symptomatic SMA. Risdiplam is not currently Medsafe approved in the pre-symptomatic setting. We understand the supplier of risdiplam, Roche, applied to Medsafe for this use in June 2022.

Risdiplam in the pre-symptomatic setting is also being considered by the Rare Disorders Advisory Committee in March 2023. If we receive a positive funding recommendation from our expert advisors and risdiplam is granted Medsafe approval in this population, risdiplam would also be funded for the treatment of pre-symptomatic SMA at a future date.

Pharmac also intends to seek further advice and assess the use of both risdiplam and nusinersen in wider populations (adults and additional subtypes) in March.

You can read more about the clinical advice we’ve received on the Application Tracker(external link)

Details about our proposal

Risdiplam (Evrysdi) would be listed in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2023 as follows:

Chemical

Formulation

Brand

Pack size

Proposed price
and subsidy

Risdiplam

Powder for oral solution, 750 mcg per ml, 
60 mg per bottle

Evrysdi

1 bottle OP

$14,100.00

A confidential rebate would apply that would reduce the net price of risdiplam to the Funder. The Evrysdi brand would have protection from delisting and subsidy reduction until 30 June 2026.

Risdiplam would be listed with a “Xpharm” restriction. An Xpharm listing means that community pharmacies cannot claim subsidy because Pharmac has made alternate distribution arrangements.

Risdiplam 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:

  1. Patient has genetic documentation of homozygous SMN1 gene deletion, homozygous SMN1 point mutation, or compound heterozygous mutation; and
  2. Patient is 18 years of age or under; and
  3. Patient has experienced the defined signs and symptoms of SMA type I, II or IIIa prior to three years of age. 

Renewal – (spinal muscular atrophy (SMA)) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. There has been demonstrated maintenance of motor milestone function since treatment initiation; and
  2. Patient does not require invasive permanent ventilation (at least 16 hours per day) in the absence of a potentially reversible cause while being treated with risdiplam; and
  3. Risdiplam not to be administered in combination other SMA disease modifying treatments or gene therapy.

Criteria would be updated at a future date if the conditions to widen access to pre-symptomatic use are met. 

Direct delivery of risdiplam

We are proposing that the supplier, Roche, would manage a direct delivery programme to get risdiplam to users. This would help remove access barriers for people receiving the treatment and their whānau, help manage the supply of a small volume product, reduce financial impact on community pharmacies and reduce the costs through the distribution chain. This process would include the reconstitution of the product so it is ready to use.

We seek feedback regarding on the proposed approach and how direct delivery  could best be managed.

  • The supplier would arrange for direct delivery of risdiplam to a nominated location. This could be a community pharmacy, a Te Whatu Ora hospital, or another location if necessary to provide access to treatment closer to home
  • Prescribers would send prescriptions to one or more central dispensing point(s) for processing along with a completed form to nominate the delivery location
  • The person or a caregiver or someone from their whānau would collect supply monthly from nominated location if necessary.
    • We would like to hear from people if there is a preference to have the option of direct to home delivery, or if people would be comfortable with a pharmacy or hospital as a collection point?

Further details on how people would be delivered their medicine would be confirmed and made available if this funding proposal is approved.   

To provide feedback

Send us an email: consult@pharmac.govt.nz by 4pm, Friday 24 February 2023.

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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