Hear the voice of those with rare disorders

Rare disease patients and their support groups are calling on the government to put in place an orphan drugs access programme, similar to those in place or being developed in most other OECD countries.

They say that Pharmac is unable to manage access to specialised medicines under its current framework, and are calling for separate management of these medicines in the Ministry of Health, just like the separate process in place now for very specialised and high cost medical and surgical treatments.

The omission of fairness and equity from Pharmac’s policy and decisions relating to specialised medicines is regarded by the groups as a serious conflict with the rights of the patients and at variance with health legislation, health strategies, and Ministry of Health guidelines for decision making, which all place equity, fairness and community values into decision criteria.

NZORD and the support groups endorse Pharmac’s core role of getting a good deal on medicines for the bulk of the population, but say that Pharmac’s track record regarding medicines for orphan diseases is an appalling failure. “We think they should be left to get on doing what they can do well, and have their failure remedied by removing it from their responsibility,” the groups say.

Thursday 28 February is Rare Disease Day and draws attention to the needs of those with rare disorders. NZORD, the NZ Organisation for Rare Disorders, is hosting a seminar today at which several patients will be talking about their difficulties getting access to new treatments for rare “orphan” diseases. The groups’ concerns are reinforced by presentations from health researcher Greg Coyle who will discuss the findings of his PhD thesis which examined Pharmac’s exceptional circumstances scheme, and lawyer Lucy Elwood will be discussing legal aspects of the issues.

John Forman, executive director of NZORD says “These patients are being abandoned by the health system. There is a systems failure in this area, as the moral factors of fairness, equity and community values are not being included in Pharmac’s decisions on specialised medicines.”

“The 2011 review of the exceptional circumstances scheme and the introduction of the new NPPA scheme, actually made it more difficult for rare disease patients to get a fair hearing,” says Forman. “The criteria changed so rarity was no longer a prerequisite, but they also made it practically an exclusion criteria”, he said.

The criteria reflect Pharmac’s narrow health economics and budget management approach, and exclude the very important moral factors of equity, fairness, and community values. It appears to be a deliberate exclusion of moral considerations from decision making.

In trying to find solutions and answers to the way in which access to “orphan” medicines is managed, NZORD and a number of associated support groups have been sent around in circles. After a test case under the new system, which was rejected by Pharmac as not eligible for consideration, they approached Ministers for an examination of the policy framework, but were ‘fobbed off’. They then approached the Health Select Committee who directed them to the Health Minister. The Minister initiated a report from the Ministry of Health into the groups’ concerns, but before briefing with the Ministry were even completed the Minister wrote to say he could not interfere with funding decisions by Pharmac, despite the request being about policy accountability, not a specific funding decision. The Minister then referred the matter to Pharmac which resulted in a bland avoidance letter from Pharmac.

Unable to get any traction from these approaches, the groups submitted a case to the Ombudsman in October 2012, claiming unfair application of the policy to an individual case, and suggesting that the policy itself was not fairly formed. The case is still being investigated by the Ombudsman and a response is expected soon.

Groups participating in today’s seminar include:
The Muscular Dystrophy Association of New Zealand www.mda.org.nz
Lysosomal Diseases New Zealand www.ldnz.org.nz
The PNH Support Association of New Zealand www.pnhsanz.org.nz
Hereditary Angioedema Australasia www.haeaustralia.org.au

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