It is now over 2 years since Pharmac announced its special fund for rare diseases, and progress has been excruciatingly slow. The first few decisions under this fund were simply to list on the schedule medicines which were previously accessed through the exceptional circumstances scheme, and it was frustrating to see no progress towards funding of drugs for rare diseases, for which no previous access had been gained in NZ. Finally, in March this year, this changed when funding on Naglazyme for Maroteaux-Lamy syndrome was approved.
This was a small step but an important one. It confirmed the long awaited change in Pharmac policy that I and many other rare disease advocates had work on for more than a decade, It opened up the prospect that more of the treatments for extremely rare metabolic diseases could be approved for funding in the near future.
This document LDNZ- to Pharmac – funding of Naglazyme – Feb-March 2016 provides a brief summary and includes the submission made by LDNZ, spelling out the brief history of the issues and putting the decision in context of other medicine funding policy decisions, including the stripping of purchasing power from Pharmac in recent years by government and District Health Boards.