The clock is ticking for patients with rare diseases – whose lives are at risk as they call for the Government to help fund their medication.

PHARMAC announced a $5 million fund for rare diseases nearly three years ago but patients say the fund failed to deliver to adult patients.

Campaigner Jenny Noble said there is not enough money in the budget to fund the medication they need.

She said it’s forcing Kiwis suffering from diseases, such as Pompe, to find alternative means or go without medication altogether.

There are 10 patients suffering from rare diseases.

Four of them are on clinical trials with an Australian company while another four are being treated by an American company.

The rest remain untreated and spend much of their lives in and out of Hospital, where DHBs do what they can to clinically treat those suffering, said Ms Noble.

The situation is becoming desperate – with one man dying last year because he could not access the medication he needed, she said.

"The drug didn’t come in time for him and that’s our reality. Without treatment these patients are going to pass away.”

Two of the adult patients affected are Ms Noble's own children, the only two people in New Zealand suffering from Mucolipidosis.

“There’s no therapy, no treatment for them whatsoever. It’s horrific . . . they often get side-stepped within the health system,” Ms Noble said.

“It’s devastating for all the families living with rare diseases because it doesn’t come easy,” she said.

She said her message to the Government is clear.

“Stop playing games. Stop stripping PHARMAC of its money. Give PHARMAC a proper budget that allows it to do its job.”

PHARMAC is not likely to fund medication for rare diseases anytime soon.

In a statement, PHARMAC said it has not received any information that indicates it should fund Myozyme, a medication for late-onset Pompe disease.

It said it has to stick within its budget.

Director of Operations Sarah Fitt said “A decision to fund this medicine over others, would mean a potential loss of health outcomes for other New Zealanders seeking access to medicines.”

The Ministry of Health declined to comment.

Ms Noble said their petition is now with the Health Select Comity after being submitted to Parliament in November last year.