IMG 6004
IMG 6004
7 Jul

Advocacy Update - July 2022

7 July 2022

Funding for Medicines for Rare Disorders

The Health Select Committee’s hearing into Carmen Shank’s petition to fund Trikafta on 8 June provided the opportunity for CFNZ both to support Carmen’s call for funding and raise the dire situation facing those with rare disorders in Aotearoa New Zealand.

The Pharmac Review Panel’s Final Report laid bare how desperate the situation is for those seeking access to medicines for rare disorders, with only 0.8% of Pharmac’s budget being spent on such medicines.

While the Review Panel’s report provides a set of recommendations for change, CFNZ is concerned that these changes will take significant time to implement and deliver meaningful outcomes. Action to improve the current situation is needed now.

While the Minister of Heath cannot direct Pharmac to fund specific medicines, he can advise Pharmac of his expectations. He did so after the May Budget announcement, indicating that he expected Pharmac to focus its new funding on addressing treatment gaps, especially for cancer.

CFNZ has therefore written to the Minister of Health asking him to advise Pharmac that he also expects priority to be given to funding medicines for rare disorders. By rare disorders we have made clear that we mean the internationally accepted definition that includes conditions such as CF, rather than the current narrow and restrictive definition used by Pharmac which does not.

Rare Disorders NZ has also written to the Minister of Health making the same request, and we understand that at least other 12 rare disorder organisations have also written.

The expectation that progress will be made to improve the situation of those with rare disorders is now not only a domestic issue but also an international commitment.  In December 2021, New Zealand supported the United Nations resolution to promote and protect the human rights of those living with rare disorders, including provision of equitable access to health care and medicines.

We have yet to receive a response from the Minister.


Trikafta Application to Pharmac

CFNZ understands that Vertex’s application for funding of Trikafta was considered again at the Respiratory Advisory Committee on 27 April and at the Pharmacology and Therapeutics Committee (PTAC) on 19 May. We have yet to be advised of the outcomes of those two meetings, with the formal records expected to be publicly released by Pharmac in late July and early August respectively.

We understand that Pharmac also intends to release its Technology Assessment report (TAR). A TAR contains Pharmac’s detailed analyses of new pharmaceuticals and is used to determine the cost-effectiveness of medicines that Pharmac is asked to fund.  Pharmac does not always release TARS but has indicated it intends to do so for Trikafta. The date of that release has yet to be advised.

CFNZ understands that the RAC and PTAC records together with the TAR will enable Vertex to review and reassess its commercial proposal to Pharmac, and for Pharmac to undertake a further ranking of Trikafta. CFNZ understands that this will potentially occur in September.

CFNZ continues to urge both Pharmac and Vertex to negotiate a fair and reasonable price for Trikafta for all those aged 6 years and over who would benefit.