Biogen has been supporting the Muscular Dystrophy Association of New Zealand (MDANZ) and is offering free medication to children with spinal muscular atrophy (SMA) as it attempts to win support to get its drug Spinraza publicly-funded.
That free treatment is likely to last only until Pharmac’s rare disorders subcommittee decides whether they will fund Spinraza in New Zealand; in the UK, Biogen pulled its free trials after being refused NHS funding.
The Pharmac subcommittee held its first ever meeting this week to consider 13 funding applications, including Spinraza.
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Biogen, which made $12.3 billion in revenues last year, recently closed its Expanded Access Programme (EAP) to treat SMA type 1 patients in England, Wales and Northern Ireland after it was decided the NHS would not fund the drug.
An Expanded Access Programme, similar to the one that Biogen has dropped in the UK, has been running at Starship Hospital in Auckland this year. A handful of children have been receiving Spinraza treatment for free.
It’s estimated that the treatment’s retail cost is US$750,000 (NZ$1.14 million) for the first year followed by four-monthly injections for the rest of the patient’s life priced at NZ$135,000 per injection.
In October 2017, the authorities in Denmark recommended Spinraza for use only on young babies with SMA type 1 and refused treatment for the rest of the population quoting an “unreasonably high price” compared to the clinical effect. Norwegian authorities rejected the funding around the same time because the price of the medicine was “unethically high”.
Starship Hospital spokesperson Sarah McMahon said they were treating a small number of children that were diagnosed before the EAP was established but “since we began the EAP earlier this year, we have not seen any newly diagnosed cases of type 1 SMA”.
This news came as a shock to Lisa Geddes from Taupo whose two-year-old son Tama was diagnosed with type 1 SMA several months ago and was told he wasn’t eligible for treatment.
“Everything sounded really promising. And then we got told, not so long ago, that because he has sat up unsupported, in his life, that he was denied the treatment,” she said.
“I just lost it. I just broke down and just said, this was the hope that we were holding on to and that Tama, as well as so many desperately need. To be told he was declined for it was just devastating.”
Biogen’s Fiona Tigar has confirmed that they are providing Spinraza “free-of-charge” to Starship Hospital on “compassionate grounds”. However, she said it was up to Starship’s clinicians to decide whether both newly diagnosed patients and those living with the disease are eligible for the treatment.
“Biogen has not made any changes to the criteria for the program,” Tigar said.
She said the pricing of Spinraza was very complex and needed to reflect the “highly specialised nature of the product and the enormous investment Biogen has made into clinical trials, clinician involvement and patient access during product development”.
“We have made a submission to Pharmac for reimbursement and will work closely with them to determine an appropriate price for Spinraza in New Zealand.
“It has taken almost fifteen years and an enormous investment from Biogen to bring this product to the SMA community. We are committed to tackling the challenges of neuroscience medicine but need to recoup our investment to be able to do so and continue bringing innovative products like Spinraza to patients who need them,” Tigar said.
However, US charity Cure SMA provided the initial seed money for the drug back in 2003 before Biogen’s involvement in 2012.
It was reported in October that global sales of Biogen’s Spinraza surpassed US$1 billion in annual revenue.
Meanwhile, questions have also been asked about the relationship between the Muscular Dystrophy Association of New Zealand (MDANZ) and Biogen as there have been repeated calls for Pharmac to fund Spinraza coming from MDANZ in the past year.
MDANZ chief executive Ronelle Baker said their relationship with Biogen is “one of many that we have with commercial or non-profit groups that are working in the same field as us”.
“We have a positive relationship with Biogen, and currently a shared interest in advancing the well-being of people with SMA – at present, this happens to involve access to the first ever, established treatment for SMA (Nusinersen/Spinraza).”
She confirmed that their SMA member’s reference group has determined access to Spinraza as a priority.
“We have therefore been supporting a community-driven campaign to raise awareness of SMA and Spinraza.
“Our members are fully aware of the application made by Biogen for funding reimbursement … However, we don’t control individuals and what they decide to do is completely up to them,” Baker said.
Biogen did not respond to questions on whether they believe it’s ethical to donate money to an organisation (MDANZ) whose members are publicly campaigning for their drug to be funded in New Zealand.
Sunday Star Times