PHARMAC call for applications for rare disorder medicines

2 July 2018

Clinicians, patients and support groups for rare disorders have all been encouraged to be involved in this funding process. A standing Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee is being established to provide advice on applications.

PHARMAC established a rare disorder pilot in 2016 and approved ten medicines, although NZORD points out that two of the ten medicines have no patients currently in New Zealand and one of the ten approved medicines is used to treat tuberculosis – which may occur rarely in NZ but is not a rare disorder.

NZORD is cautiously optimistic about PHARMAC’s current time frames and their recognition of the need for specific processes around rare disorder medicine funding decisions. We do, however, see the following issues:

1. The regularity of this call for funding applications has not been established and transparency around this process is essential

2. The prevalence of 1:50,000 PHARMAC is using is extremely low, and specifically excludes several disorders such as Cystic Fibrosis and some forms of Muscular Dystrophy, which are treated as rare disorders by Australia, Scotland, UK, USA and others

NZORD is calling on PHARMAC to carefully apply their policy to ensure that the funded medicines in this round do not include antimicrobials or antivirals for infectious diseases (however rare), or any rare disorders that are not currently able to be diagnosed in New Zealand – as happened in the 2016 funding process.

“We want to make sure that PHARMAC’s focus is on funding medicines for those New Zealanders who are currently living with a rare disorder, and those newborn babies who can be identified and treated early,” says NZORD Chief Executive Dr Collette Bromhead.

Applications close on Monday 3 September and will be considered by the Subcommittee in November.

PHARMAC call for applications