Easier access to funded enzyme replacement therapy

Media release Medicines

PHARMAC is providing greater funded access to the expensive enzyme replacement therapy imiglucerase (Cerezyme), mainly expanding its use in children.

Imiglucerase treats Gaucher’s disease, a rare, inherited enzyme deficiency disorder which causes harmful quantities of fats (lipids) to build up in the body. This can lead to an enlarged liver, low levels of red blood cells (anaemia), fatigue, bone pain, thinning of the bones and fractures.

Currently 19 New Zealand patients receive funded treatment with imiglucerase, through the Pharmaceutical Schedule. The decision to widen access from 1 September 2013 will allow a further 2-3 patients to be treated per year.

PHARMAC Chief Executive Steffan Crausaz says the latest decision will increase the funded dose available to treat more severe forms of Gaucher’s disease, and also make funding available for a further group of patients available through the Schedule for the first time.

Though classification can be difficult, there are three main types of Gaucher’s disease, known as Type 1, Type 2 and Type 3. Type 1 patients have been the only ones funded through the Pharmaceutical Schedule until now, although some Type 3 patients are being funded through PHARMAC’s exceptions policies.

“Changing the funding criteria in the Pharmaceutical Schedule will make it clear that these patients – often children – can now be considered for funding,” says Steffan Crausaz. “Funding patients through a Pharmaceutical Schedule listing provides greater certainty of access to patients, continuity of supply, and reduces the administrative burden for clinicians.”

“Until now, patients seeking this access needed to have applications individually considered through the Named Patient Pharmaceutical Assessment policy. Now, in line with the intent of that policy, we are moving to formalise that access and make it clear that these patients are eligible for funding.”

Steffan Crausaz says PHARMAC will continue to assess applications for funding through an expert panel of clinicians, who work with the patients’ treating doctors.

Though Gaucher’s disease is rare and few new patients present, PHARMAC expects expenditure to grow in coming years because treatment is based on the patient’s weight, and the longer patients remain on treatment, and grow, then their dosages will increase.

More information: 021 863 342