PHARMAC releases new Gaucher disease treatment proposal

30 May 2018

NZORD is keen to hear from patients who are currently on therapy and from any others who aren't to get their views on this process and the proposed change.

Gaucher disease is an autosomal recessive disease of lipid storage which results from the deficiency of a lysosomal enzyme. The severity varies widely from patient to patient and can present in childhood or later in life.

The basic goals of treatment are elimination or improvement of symptoms, prevention of irreversible complications and improvement in overall health and quality of life. In children, an additional goal is the optimisation of growth.

In summary:

  • The funded ERT for the treatment of Gaucher disease would change from imiglucerase (Cerezyme), supplied by Sanofi, to taliglucerase alfa (Elelyso), supplied by Pfizer.
  • Taliglucerase alfa would be funded from 1 August 2018 and would become the only funded ERT for the treatment of Gaucher disease in the community after a seven month transition period.
  • Special Authority and Hospital Restrictions would apply to taliglucerase alfa. These would be similar to the restrictions that currently apply to imiglucerase, except that, if approved by the Gaucher Panel, the criteria would allow a higher maximum dose (30 units/kg every other week, approx. 60 units/kg/month) where clinically appropriate, which could provide clinical benefits for some patients.

This proposal results from a Request for Proposals (RFP) for the supply of a first line ERT for the treatment of Gaucher disease.  Consultation closes at 4 pm on 11 June 2018 and can be emailed to consult@pharmac.govt.nz.

The consultation document is available on the PHARMAC website and they will be communicating with the eight prescribers that look after the 20 patients that would be affected by this proposal.  PHARMAC has pre-engaged with those prescribers and they will be making contact with their patients to share information about the proposal with them.