28 Sep 2022

Pharmac agrees to fund drug for those with spinal muscular atrophy after years of lobbying

9:24 am on 28 September 2022
ILLUSTRATION - 03 February 2020, Baden-Wuerttemberg, Backnang: A child suffering from spinal muscular atrophy (SMA) is sitting in a therapy chair. In the foreground is a box of the drug Spinraza next to an angel sculpture with the word "Hope". Children in Germany now have a chance to win a gene therapy for terminally ill babies.
The Swiss pharmaceutical company Novartis started the raffle of a treatment with Zolgensma for 100 children on 03.02.2020. It is the most expensive drug in the world, with approximately two million euros for one dose. The therapy is for children under the age of two who suffer from SMA, which causes muscle atrophy and can lead to death in severe cases if left untreated. Photo: Sebastian Gollnow/dpa (Photo by Sebastian Gollnow / DPA / dpa Picture-Alliance via AFP)

Nusinersen (branded as Spinraza) would be the first medicine publicly funded for people who have spinal muscular atrophy. Photo: AFP

New medicines for those with a rare spinal disease and people at risk of anaphylaxis are set to be publicly funded.

Pharmac is looking at funding adrenaline auto-injectors, such as EpiPens, for people who are at risk of anaphylaxis.

There have been calls for the funding since 1997 and it is expected up to 17,000 people would benefit from the injectors.

The drug-buying agency is also considering funding nusinersen which would be the first medicine publicly funded for people with spinal muscular atrophy.

It costs about $400,000 a year if bought privately.

Director of operations Lisa Williams told Morning Report nusinersen (branded as Spinraza) has been on the options investment list for two years and with the large Budget increase for Pharmac announced earlier this year it was now able to be funded.

It would be used for all forms of spinal muscular atrophy for those under 18 as well as babies who were symptomatic.

Asked about relief for those aged over 18 who have had to travel to Australia to access the drug, she said if treatment had already started and they met the criteria they would be considered for a waiver.

Negotiations had gone well with the drug company, Biogen, which was also offering a discount on some of its other medicines, Williams said.

Asked why the decision hasn't been made sooner to alleviate the suffering of many, she said: "I do want to acknowledge the time that many people have put into advocating for those who live with spinal muscular atrophy."

She said it was exciting to share the news of the decision to one advocate yesterday.

However, the drug was not being funded because of effective lobbying - Pharmac looked at the impact of the disease on those who had spinal muscular atrophy.

As for the auto-injectors for those at risk of anaphylaxis, Williams said adrenaline has been available for those needing it via syringes, however, the Budget funding had prompted the decision to look at the more accessible auto-injectors.

"The key thing that people have been telling us over the years is that having an auto-injector is far more suitable."

She agreed 25 years had been a long time for New Zealanders to wait but Pharmac had got a great deal and with the extra Budget funding it was in a position to go ahead with funding.

Feedback on the proposed funding of nusinersen can be submitted until 24 October. If approved, funding would start from 1 January 2023.

Feedback on the proposed funding of adrenaline auto-injectors is due by 11 October.

Allergy NZ Chief Executive Mark Dixon said he was 'thrilled' by today's annoucement.

He said it was "the biggest thing to happen" for New Zealand's allergy community in 25 years.

Dixon commended Pharmac's decision and said he hoped it would be seen though to the proposed full funding.

EpiPens were a "massive" expense, with the cheapest around $120, hse said. They had to be replaced yearly and may not even get used.

'It may have taken a while'

Asked about the fact it has taken 25 years to fund the adrenaline auto-injector pens, Health Minister Andrew Little said Pharmac had to make independent clinical decisions on many drugs and could not afford to pay for every treatment.

"It may have taken a while. I think what it really highlights is the need to ensure with Pharmac funding we are keeping ahead of what is needed."

A review has been undertaken of Pharmac's operations which concluded that it needed to do better with regard to helping people with rare disorders.

Little said the decision to fund nusinersen was a response to the review as well as the extra funding the government had provided in the Budget.

"There's still work to do. I think Pharmac will be working closely with the Ministry of Health now to work up a proper coherent rare disorders strategy and engaging with the relevant community as well. But obviously the additional funding helps as well."

Spinraza response

SMA advocate Fiona Tolich said spinal muscular atrophy was a devastating condition of which the most common type was also the most severe form.

Pre-treatment, babies died at an average age of 13 months old "from a very treatable condition", she said.

The application Pharmac was currently looking at was only for those under 18.

Tolich said New Zealand was behind the eight ball and other countries were now also funding the treatment for adults.

She said Pharmac needed to quickly recognise that data and get access for adults as well.

Tolich also said it was "crucial" to get SMA on the newborn screening program.

"Ultimately we want access to treatment before kids experience that decline" she said.

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