An American drug company that calls for a $ 1 million taxpayer funding to treat New Zealand children with a rare genetic disorder has already been rejected by overseas authorities who have accused the company of unethical pricing.
Biogen supports the New Zealand Muscular Dystrophy Association (MDANZ) and offers free medicines to children with spinal muscular atrophy (SMA) while trying to gain support to publicly fund Spinraza.
This free treatment is likely to last only until Pharmac Subcommittee on Rare Diseases decides whether to fund Spinraza in New Zealand; in the UK, Biogen trained his free studies after refusing to fund the NHS.
The Pharmac Subcommittee held its first meeting this week to examine 13 funding applications, including Spinraza.
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Biogen, which earned $ 12.3 billion last year, has recently closed the Access Enhancement Program (EAP) to treat patients with Type 1 SMA in England, Wales and Northern Ireland after deciding that the NHS will not fund this drug.
An extended access program, similar to that Biogen took to the UK, was run at Starship Hospital in Auckland this year. A handful of children receive free treatment with Spinraza.
It is estimated that the retail cost of treatment is $ 750,000 (NZ $ 1.14 million) for the first year followed by four-month injections for the rest of the patient's life, at NZ $ 135,000 per injection.
In October 2017, the Danish authorities recommended the use of Spinraza only for infants with type 1 SMA and refused treatment for the rest of the population, mentioning an "unreasonably high price" compared to the clinical effect. The Norwegian authorities rejected funding at the same time because the drug price was "unethically high".
Spatial Hospital Spokeswoman Sarah McMahon said she was treating a small number of children who were diagnosed before the EAP, but "since we started the EAP earlier this year, we have not seen newly diagnosed cases of type 1 SMA" .
This news was a shock to Lisa Geddes of Taupo, whose 2-year-old son, Tama, was diagnosed with type 1 SMA a few months ago and was told he was not eligible for treatment.
"Everything sounded really promising, and then he told us, not long ago, that because he stood without support in his life, he was denied treatment," she said.
"I just lost it, I just broke up and I just said, that was the hope we held and that Tama and so many desperately need to say he was denied because it was just devastating.
Fiona Tigar from Biogen has confirmed that he offers Spinraza "free" to Starship Hospital on "compassionate reasons". However, she said it was up to Starship clinicians to decide whether both newly diagnosed patients and those living with the disease are eligible for treatment.
"Biogen did not make any changes to the program's criteria," Tigar said.
She said that Spinraza's charging was very complex and required reflection of "the highly specialized nature of the product and the enormous investment that Biogen did in clinical trials, clinician involvement and patient access during product development."
"We made a referral to Pharmac for reimbursement and we will work closely with them to establish a suitable price for Spinraza in New Zealand.
"It took nearly fifteen years and an enormous investment from Biogen to bring this product to the SMA community. We are committed to addressing the challenges of neuroscience, but we must recover the investment to do that and continue to bring products innovative Spinraza for patients who need them, "said Tigar.
However, the American Cure SMA company provided the initial money for this drug in 2003, before Biogen's involvement in 2012.
It was reported in October that Spinraza's global sales from Biogen exceeded the $ 1 billion annual revenue.
Meanwhile, questions have been asked about the relationship between the New Zealand Muscular Dystrophy Association (MDANZ) and Biogen, as Pharmac has been repeatedly asked to finance Spinraza from MDANZ over the past year.
MDANZ Executive Director Ronelle Baker said the relationship with Biogen is "one of the many we have with commercial or non-profit groups working in the same field as we do."
"We have a positive relationship with Biogen and now have a common interest in promoting the well-being of people with SMA – this now involves access to the first treatment set up for SMA (Nusinersen / Spinraza)."
She confirmed that the SMA member reference group has prioritized access to Spinraza.
That's why we supported a community-based campaign to raise awareness of SMA and Spinraza.
"Our members are fully aware of Biogen's demand for reimbursement financing … However, I do not control individuals, and what they decide to do is completely out of them," Baker said.
Biogen did not answer the question whether he believes it is ethical to donate money to an organization (MDANZ), whose members are in a public campaign to fund their drugs in New Zealand.