How does the operation of PHARMAC's 'Community Exceptional ...

The discussion with ‘G’ traversed the ground of what PHARMAC defined as
exceptional and why the definition of rarity should be 10 cases or less in New
Zealand at any one time.
It is ludicrous…well they have this number [10] now, and I think the
number is completely arbitrary and some cases will be exceptional
and there will be less than three people in New Zealand with this very
rare disease and some cases will be exceptional for other reasons.
There might be a hundred people with the disease being exceptional.
One of our problems is that the conditions are for such few people
that double-blind trial studies are so small that they lose validity.
(Interviewee G)
‘G’ believes that PHARMAC would rather take the line that the treatments are
not clinically proven to cover for the real reason that the budget simply cannot
sustain the high costs involved.
I recall in some conditions Community Exceptional Circumstances
would be the right thing to do…in one case they would not agree to
fund a few children with a rare genetic disease. This would have
placed about $10 million a year on the books. They simply said…
“We agree that Community Exceptional Circumstances ought to be
approved in these cases, but we simply cannot fund that. There is
not enough money’. (Interviewee G)
Finally, ‘G’ raised the issue of the New Zealand Medicines Strategy of which
NZORD was one organisation that lobbied the government to develop. ‘G’ said
that if PHARMAC and the DHBs had adhered to the principles underlined in the
Medicine Strategy, the commitment to funding medicines for orphan diseases
would be met.
The Medicine Strategy provides a set of criteria to guide decision
making that includes the relative benefits that come from cost-utility
analysis, but also includes factors like ethics, community values,
affordability, fairness and the health needs. The strategy tries to
balance these factors with the cost-utility analysis approach and
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