Ombudsman criticises Pharmac's approach to rare diseases

NZORD - the New Zealand Organisation for Rare Disorders

The Ombudsman has criticised a Pharmac decision from September 2012 on an exceptional circumstances application for a specialised medicine to treat Freda Evans who has Pompe disease. He has suggested Pharmac consider any further comments that Ms Evans wishes to make and give further specific consideration to items they have glossed over in their decision.

In the opinion released to the patient on 31 May 2013 and published 12 July 2013 Ombudsman Dr David McGee, who has since retired, raised a number of concerns about Pharmac’s policies and procedures. Specifically in the opinion:

He mentioned Pharmac’s documentation describing the decision criteria was “unduly complicated”.
He noted that Pharmac has a general discretion to consider applications despite the tight eligibility criteria in the Named Patient Pharmaceutical Assessment (NPPA) policy, but noted that the information on the policy did not explain this.
Dr McGee was concerned that Pharmac’s NPPA policy does not record the decision criteria to be used under that policy. He also considered the decision criteria under the NPPA policy ought to be clearly differentiated from those under the Pharmaceutical Schedule.
He was concerned that Pharmac didn’t specifically consider the patient’s suggestion for a trial period of the medicine. The Ombudsman said they should revisit this and consider any further comments on this suggestion the patient wishes to make.
He noted the lack of evidence in regard to rare diseases and treatments inevitably puts their assessment at a potential disadvantage.
Dr McGee specifically noted that the principle of states not abandoning their citizens is of obvious relevance where there is no known alternative method of treatment.
He noted a contestable legal argument about whether Pharmac appropriately excludes social and ethical factors from their consideration, though he said it was not his role to make a definitive decision on that legal dispute.
He considered that Pharmac should allow submissions to be made by Ms Evans and her supporters about questions of fairness, equity and community values, and consider these alongside submissions made by her clinician when the application is reconsidered.

· He identified “matters of high policy to be addressed by the Government and Parliament”.

Ms Evans’ complaint to the Ombudsman has been supported by the New Zealand Organisation for Rare disorders (NZORD), Lysosomal Diseases New Zealand (LDNZ), and the Muscular Dystrophy Association of New Zealand (MDANZ). “Our groups are delighted that our concerns have been heard in the system and responses provided that recognise the weakness of individuals against the enormous power of the bureaucracy,” said John Forman, NZORD’s executive director.

The groups have been seeking better accountability and governance oversight of Pharmac for many years. “We were frustrated that approaches to Ministers, the Health select committee, and other officials over the past two years since the development of the NPPA policy, had not resulted in any external scrutiny of Pharmac or response to our concerns,”, says Forman. “At last we have found a part of the system willing to investigate Pharmac and hold them to account, and Pharmac has been found seriously wanting.”

The Ombudsman’s report includes criticism of aspects of the relationship between Pharmac and District Health Boards, and concern was expressed by Dr McGee at Pharmac’s apparent power to bind DHB’s medicine purchasing decisions through a provision it has put in the schedule. Dr McGee said, “I have seen no statutory authority which allows Pharmac to give directions to DHBs, whether through the schedule or otherwise.”

Forman concluded by saying that the report vindicates many years of detailed work the groups have invested in scrutinising Pharmac’s operations. “Pharmac should be commended for the significant achievements it has made with a Pharmaceutical Schedule that provide good savings and good pharmaceutical outcomes for a great number of New Zealanders. But it is time for Pharmac to acknowledge that a strict health economics focus without a moral compass is abandoning patients at the margins. We hope that this opinion will cause Pharmac, government, Ministers, the Health select committee and other officials, to respond with serious scrutiny and review of Pharmac’s policies regarding specialised medicines for rare diseases.”

NZORD will assist with a further application for funding of the treatment (Myozyme) that Ms Evans needs, as soon as discussions with clinicians and other advisors are completed.

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