Public cash sought for $1b drug
A drug offers hope for free – for now, write Jacques Steenkamp and Imogen Neale.
A drug company demanding $1 million taxpayer funding to treat New Zealand children with a rare genetic disorder has already been rebuffed by overseas authorities, which accused the company of unethical pricing.
American company 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 tries to get its drug Spinraza publicly funded. That free medication 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 free trials after being refused NHS funding. The Pharmac subcommittee met this week to consider 13 funding applications, including Spinraza.
Biogen, which made US$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 the NHS declined funding for the drug. An EAP, 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 for free. It’s estimated that the retail cost is US$750,000 (NZ$1.14 million) for the first year followed by fourmonthly injections priced at NZ$135,000 per injection.
In October 2017, the authorities in Denmark refused some Spinraza treatment because of its ‘‘unreasonably high price’’ compared to the clinical effect, and the same month Norwegian authorities rejected the funding because the price of the medicine was ‘‘unethically high’’.
Starship Hospital spokesperson Sarah McMahon said the hospital was treating a small number of children who 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 needs. To be told he was declined for it was just devastating.’’
Biogen’s Fiona Tigar has confirmed the company is providing Spinraza ‘‘free-of-charge’’ to Starship Hospital. However, she said it was up to Starship’s clinicians to decide whether both newly diagnosed patients and those living with the disease were eligible for the treatment. ‘‘Biogen has not made any changes to the criteria for the program.’’
Tigar said the pricing of Spinraza was complex and needed to reflect the ‘‘highly specialised nature of the product and the enormous investment Biogen has made ... during product development’’.
‘‘We have made a submission to Pharmac for reimbursement and will work closely with them to determine an appropriate ... We are committed to tackling the challenges of neuroscience medicine but need to recoup our investment,’’ Tigar said.
However, US charity Cure SMA provided the initial seed money for the drug in 2003 before Biogen’s involvement in 2012.
It was reported in October that global sales of Spinraza surpassed US$1 billion in annual revenue.
Meanwhile, questions have also been asked about the relationship between MDANZ and Biogen because of MDANZ’s repeated calls for Pharmac to fund Spinraza. MDANZ chief executive Ronelle Baker said the relationship with Biogen is ‘‘one of many that we have with commercial or non-profit groups’’.
‘‘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.
‘‘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.’’
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.
This was the hope that we were holding on to and that Tama – as well as so many – desperately needs.
Lisa Geddes and her two-year-old son Tama Hubbard who has SMA type 1.