The complainant suffers from adult late-onset Pompe disease, a metabolic disorder which is one of a number of diseases collectively called lysosomal storage disorders. Myozyme is an enzyme replacement therapy used in the treatment of Pompe disease. There are no alternative medicines available to treat the form of Pompe disease from which the complainant suffers.
In 2011 and 2012, the complainant made applications for a subsidy for Myozyme in exceptional circumstances. Myozyme is not listed on the Pharmaceutical Schedule, and is therefore not publicly funded by Pharmac apart from in exceptional circumstances. Both applications were declined.
Following an investigation of Pharmac’s decisions, in this Opinion the Ombudsman notes that in considering the second application, Pharmac should have specifically responded to the suggestion made by a clinician, on behalf of the complainant, of funding for a trial period.
The Ombudsman also examines the criteria for decision making on:
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the Pharmaceutical Schedule, under which listed medicines are publicly funded for any New Zealand-eligible patient; and
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the Named Patient Pharmaceutical Assessment Policy (NPPA Policy), under which applications may be made by individual patients in exceptional circumstances for subsidised access to treatments which are not included on the Pharmaceutical Schedule.
If a treatment has been “prioritised” by Pharmac for consideration for listing on the Pharmaceutical Schedule, then this is a factor taken into account in considering applications by individual patients under the NPPA Policy. The Ombudsman comments that Pharmac should more clearly build an external element into its prioritisation decision‑making in relation to the Pharmaceutical Schedule, since prioritisation has consequences for funding eligibility under the current NPPA Policy that Pharmac operates.
The Ombudsman further observes that the NPPA Policy itself ought to provide that prioritisation will not be a pre-requisite if substantial new information of a clinical or commercial nature becomes available, and suggests that Pharmac reconsider its terminology of “prioritisation” to give a clearer indication of the consequences of an assessment of a medicine and its assignment of a priority for funding.
Finally, the Ombudsman notes that the criteria under the NPPA Policy ought to be clearly differentiated from those under the Pharmaceutical Schedule.
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