Editorial: Why Pharmac should fund SMA drug

EDITORIAL:

How must it feel to make decisions upon which lives depend?

That is often the role in which Pharmac finds itself — the Government agency that decides which drugs should be publicly funded. It is a job few of us would envy.

Pharmac balances a series of factors to make its decisions — but in essence it must weigh up a limited budget against which pharmaceuticals have the best chance of success.

This is an objective and rational process, as it should be. Emotion can have no place.

Yet who can fail to be moved by the case of 2-year-old Charlotte Bond, who has spinal muscular atrophy. It's a degenerative disease that affects the control of voluntary muscle movement. If untreated, the disease makes its sufferer weaker over time before causing an early death.

While SMA is rare, it is a genetic condition described as one of the more common rare diseases.

Tama Geddes, 2, is another sufferer whose family have been devastated by Pharmac's deferral on a decision to fund Spinraza — the only approved treatment for SMA which, while not a cure, has been proven to slow or block the effects of the disease.

That has left Charlotte's family gathering a petition to demand funding as they fight to prolong her life.

We've been here before. Notably with the battle to have cancer drugs Herceptin and Keytruda funded.

The human stories behind the battles are always heartbreaking.

Experts must put that aside and weigh up the evidence. In the case of Spinraza, Pharmac says it wants to wait for trials to show more evidence of long-term benefits. But this is a drug already funded in Australia and, very recently, approved for funding in Scotland. That suggests a level of international confidence that our authority should pay heed to.

The children suffering SMA may not have time to wait for trials to conclude.

Even with emotion cast aside, the better decision in this case is to fund the drug. The standard of proof of the drug's success can surely be lower when we are juggling the lives of young children.