Stacey Kirk: Who's really driving the campaign to publicly fund a $1m drug for

The expensive drug debate is not just one of whether Pharmac will fund or not. Are we being fed the whole truth, when it comes to why these drugs are so expensive?
123RFThe expensive drug debate is not just one of whether Pharmac will fund or not. Are we being fed the whole truth, when it comes to why these drugs are so expensive?

You may not have even noticed it.

The latest campaign to fund the next “game-changer” miracle drug seems to have begun in New Zealand earlier this year, just before an application was lodged with Pharmac to fund spinal muscular atrophy (SMA) drug Spinraza – made by company Biogen. 

Remember that name – Biogen – it’s important. 

Drip fed throughout the year, in various publications including this one, have been different tales of heartbreak from a small handful of families whose children suffer some form of muscular atrophy, for which the prognosis is usually dire.

However, a detail that appears to have been largely glossed over in New Zealand, is that the early development of this drug at least, was largely paid for through university research grants from charity Cure SMA.  

What we’re dealing with is a drug which had much of its development bankrolled by the American taxpayer, its makers purchasing the US$75m license after phase three trials proved it was a likely winner, and then decided the price of life should be somewhere in the vicinity of US$750,000 (NZ $1.14m).

Now, here’s the kicker.

Flat Bush woman Fiona Tolich, 37, says SMA has been described as like being in the mafia: once you're in, there is no getting out. She is part of a campaing to call on Pharmac to fund the $1 million treatment Spinraza.
IMOGEN NEALE/STUFFFlat Bush woman Fiona Tolich, 37, says SMA has been described as like being in the mafia: once you’re in, there is no getting out. She is part of a campaing to call on Pharmac to fund the $1 million treatment Spinraza.

Muscular Dystrophy Association New Zealand does a brilliant job advocating for the families who have no other avenue to be heard and has played an important role in pushing these families to New Zealand news publications, as that may well be their only chance to apply pressure on Pharmac for funding. 

The organisation is a charity which draws its funding from a number of different places, including corporate sponsorship. At the top of its supporters list? 

Biogen. 

It’s among dozens of other donors, but it’s there on a webpage, transparent for anyone who cares to take the dive and look. That’s not a bad thing. But that it’s not been mentioned in a single interview or piece of reporting that advocates for this funding, is.

Because these stories are designed to subtly direct public anger at Pharmac for “denying” patients the drug. 

In fairness to the drug’s maker, this is what’s known as an “orphan disease”, which in the United States is defined as affecting fewer than 200,000 people. By definition, there is not a market large enough for a company to fully support further research and funding for new developments in treatment. 

In New Zealand, about 70 people are thought to have spinal muscular atrophy. Pharmac is unlikely to ever pay the list price, but in the absence of any other pricing data, funding here alone would earn the company up to $79.8m – nearly the equivalent of its licensing  fee. 

Funding has been granted in a number of markets far larger than New Zealand’s, while a number of countries declined initial funding citing the price as “unethically high”. 

Globally, Biogen cleared $12.2 billion in revenue last year. 

Most recently, the UK’s version of Pharmac declined to fund the drug, acknowledging that while evidence showed “significant benefits” to patients there were still “significant uncertainties” around long-term benefits. Coupled with Spinraza’s “extremely high” cost, it could not be recommended as a cost-effective treatment option.

Pharmac will be considering the drug for funding, this month, under its rare disorders fund. Spinraza’s application will be considered alongside funding for 13 other new medicines, for diseases like Fabry disease, Pompe disease, cystic fibrosis and Gaucher disease. 

Every reasonable New Zealander understands why they can’t all be funded. 

So which would you choose? 

Sunday Star Times