22 May 2013

Rare disorders group questions consultation over drug

1:46 pm on 22 May 2013

A group representing patients with rare disorders says consultation by Pharmac on a funding decision may be window-dressing.

The drug-buying agency on Tuesday said it planned to refuse to fund a highly expensive medicine for patients with a rare blood disorder.

In January this year, eight patients with the rare PNH disorder urged Pharmac to fund the drug eculizumab which they view as a life-saving medicine.

Pharmac says there is some evidence the eculizumab is effective but it could cost up to $12 million a year.

It is consulting over its proposal to decline funding.

The executive director of the Organisation for Rare Disorders, John Forman, says he wonders what the purpose of the consultation is, given Pharmac has carried out a review and come to a conclusion.

"It seems to me like they now have a pre-determined position and that any consultation is likely to be perhaps a window-dressing exercise."

Mr Forman says Pharmac's processes put tiny numbers of patients with rare conditions at a disadvantage.