NZORD - the New Zealand Organisation for Rare Disorders

The government’s announcement of a $2 hike in prescription charges next year is more than enough to cover the expected cost of specialised medicines for rare “orphan” diseases, says the NZ Organisation for Rare Disorders.

The estimated current annual costs of funding all five of the new enzyme replacement therapies for several dozen patients needing treatment for rare Lysosomal diseases is around $10 Million per year. That is half of what the prescription charge hike will bring in, in the first financial year, and just one quarter of the savings in a full year.

NZORD executive director, John Forman, says the National Party pledged in the 2008 election campaign to find a solution for access to specialised medicines, but various reviews over the years have not delivered on this. The recent launch of Pharmac’s new exceptional circumstances scheme, named NPPA, has not address the problem.

“They told us in a briefing last year that it did not do so, and that was confirmed by our own analysis of the new scheme. We now have it in writing in one case, as well as in other communications regarding several others, that patients in need of these specialised therapies are not eligible for consideration under that scheme.”

NZORD, Lysosomal Diseases New Zealand, and the Muscular Dystrophy Association, featured adult patients with Pompe disease in their campaign for an orphan drugs access policy last year. Forman states that Health Minister Tony Ryall insisted during the election campaign that the patients could apply under the new scheme, and suggests “he now needs to ask Pharmac why they told us one thing, which is correct, and told him another, which is not.”

“Now is a perfect opportunity for the government to keep this money saved for reuse within the medicine funding system. Keeping true to their word from 2008 to address this problem would be an honourable use of the funds”, says Forman. “Failure of the government to do so will be abandoning these patients to die, when there is an effective medicine now available to treat them.”

Prime Minister John Key is quoted in Hansard (Prime Minister’s Statement p16498, 8 February 2011) “I say that one should judge a society by how it looks after the sick and the vulnerable”. He repeated this phrase the day after the 2011 election on TVNZ’s Sunday programme.

“NZORD, LDNZ, MDANZ and dozens of patients with Lysosomal diseases, wait for the Prime Minister, the Health Minister and the National Party to be true to their words,” says Forman, “but unfortunately some of the patients do not have a long time to wait before their degenerative, fatal disease overtakes them.”

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