14 Dec 2016

Patients call for funding of drugs for rare disorders

9:25 am on 14 December 2016

People with rare health disorders are urging MPs to consider whether extra funding is needed for potentially costly medicines.

Samantha Lenik presents petition to MP Mark Mitchell seeking more funding for medicines for rare diseases.

Samantha Lenik presents petition to MP Mark Mitchell seeking more funding for medicines for rare diseases. Photo: RNZ / Karen Brown

Whangaparaoa woman Samantha Lenik yesterday presented a petition at Parliament urging MPs to look at the funding and resources for those with rare diseases.

She's one of 11 adults in New Zealand with Pompe disease, which she found out she had three years ago.

"I'm 42, I'm a mum with two young kids. I want to be able to live a long and full life to be there for them as long as possible, and currently Pharmac will not fund the treatment, Myozyme, that I need.

"This treatment is funded in 76 countries around the world but not currently in New Zealand."

Mrs Lenik acknowledges that the enzyme replacement therapy she needs, Myozyme, is highly expensive, at between $200,000 and $1 million a year per person, but said Pharmac could negotiate a better price.

"We fund, you know, drugs that cost less money but for large groups of people, so just because it's an expensive drug, why should we be excluded?"

Mrs Lenik travels to Adelaide fortnightly to receive a similar drug as part of a clinical trial.

She said while that was helping her, it was no good for others who may be worse off and needed help to breathe with the disorder.

"A lot of people are on crutches, wheelchairs or mobility scooters because they are unable to walk.

"I've got a 9-year-old son and a 7-year-old daughter and I would love to be able to spend many more Christmases with them."

At Parliament with Mrs Lenik yesterday was the chairman of Lysosomal Diseases New Zealand, John Forman.

He said Pharmac had made a start with a dedicated fund for those with rare disorders, but there was not enough in the kitty.

"The $5 million fund is almost all spent and when we started out this process there were an estimated about 100 patients who were in need of treatments. We estimate that fewer than five have got access to an orphan drug that was not previously funded in any other way."

He said decisions made about funding for rare disorders were tortuously slow.

An application that had been before Pharmac's pharmacology and therapeutics advisory committee (PTAC) For a couple of months took almost a year to reach that point, he said.

"Pharmac advise us that it won't be until possibly February or even March, when PTAC has its next meeting, that they will confirm the minutes to determine whether or not they made a favourable recommendation about it getting across the line. We think that's completely unacceptable."

The petition, signed by almost 3000 people, was received by Rodney MP Mark Mitchell.

"I'm a firm believer that actually politicians shouldn't interfere with Pharmac and the decisions they make, but I'm very happy to support them [patients] in [the] sense of accepting the petition and lodging it with the table office," he said.

Pharmac wouldn't be interviewed but said it has made provisions to spend $25 million over five years under a pilot commercial process which had improved access to nine medicines.

A decision regarding one more medicine was pending.