New Zealand’s Pharmaceutical Management Agency (PHARMAC) is requesting feedback on a proposed change in the enzyme replacement therapy (ERT) available for Gaucher disease types 1 and 3 under the country’s public health plan.
The change involves moving to Elelyso (taliglucerase alfa) as the publicly funded ERT for these Gaucher patients, instead of the currently funded Cerezyme (imiglucerase). The change would begin in August, with Elelyso becoming the only funded ERT after a seven-month transition period.
“The proposal would improve access to a high cost treatment for a rare condition, while managing pharmaceutical costs,” officials wrote in the proposal.
Gaucher disease (GD) is a rare disease resulting from deficits in the glucocerebrosidase enzyme, leading to the accumulation of a type of fat molecule. This molecule builds up in macrophages – which are called Gaucher cells when this happens – which deposit in many parts of the body, primarily the bone marrow, liver, and spleen.
Elelyso, by Pfizer, corrects glucocerebrosidase deficiencies with a modified version of the enzyme. The infused enzyme breaks down the excess fat accumulated in Gaucher cells, alleviating the symptoms of the disease.
The medication is indicated in New Zealand for long-term treatment of adults and children with GD type 1, but unlike Cerezyme (marketed by Sanofi), it is not approved for GD type 3.
All 20 patients identified as now receiving Cerezyme have through February 2019 to change their ERT. A patient who cannot tolerate Elelyso may, upon a request filed by a treating physician and reviewed by an expert panel, have treatment with Cerezyme reinstated.
Transition to Elelyso would be supported by the treating clinician in collaboration with PHARMAC, support services, and the country’s district health boards. Officials also noted that patients who have not had a magnetic resonance imaging scan in the past year may require one before switching therapies. Patients now taking Cerezyme at home would have to go to hospital outpatient clinics for the first two to four infusions of Elelyso. Subsequent treatments at home would be assisted by a trained nurse.
A small number of community pharmacies currently dispensing Cerezyme would need to work with treating clinicians and patients in this switch of ERT.
Hospital pharmacies will be assisted by PHARMAC during the treatment transition. Elelyso would be dispensed to patients with a valid Special Authority approved by the expert panel, called the Gaucher Panel. Reimbursement claims could be made by pharmacies with a community pharmacy contract.
Regarding hospital restrictions, they would be similar to the currently applied for Cerezyme, except a change of higher maximum dose to 30 units/kg every other week, or approximately 60 units/kg/month, if approved. This change could provide clinical benefits to some patients, the authors mentioned.
The proposal indicates the Gaucher Panel would remain in place and consider both new applications for Elelyso treatment as well as renewals for existing patients.