Pharmac shares the agenda items for the upcoming Rare Disorders Committee meeting

Update Medicines

Pharmac has shared the agenda for the upcoming Rare Disorders Advisory Committee meeting to be held on Wednesday 29 May.

What we're doing

Pharmac is sharing what medicine applications will be considered at the Rare Disorders Advisory Committee meeting on Wednesday 29 May.

The agenda for the meeting includes applications for treatments for Fabry disease, Gaucher disease, hereditary angioedema, Pompe disease and Von Hippel-Lindau (VHL) disease.

The records for the Rare Disorders Advisory Committee meeting will be released later in the year. Funding applications will be reviewed using the Rare Disorders policy principles and will be assessed in line with Pharmac’s usual processes

Why are we releasing the agenda

We understand that people living with rare disorders face many challenges, including access to health care and effective medicines.

Releasing the Rare Disorders Advisory Committee meeting agenda offers more transparency to those affected by rare disorders, their whānau, and their communities. It also gives more visibility to those looking for updates on specific medicines.

We are working with the consumer group Rare Disorders New Zealand(external link) to make sure the right voices are heard at this meeting – we thank them for their time and expertise.

We will continue to work on improving funded access to medicines for rare disorders as part of our work to enhance our assessment and decision-making processes.

Agenda items for the next Rare Disorders Advisory Committee meeting

Fabry disease - a neurological condition that can affect many parts of the body, including the kidneys, heart, and skin.

The Committee is considering another treatment option, in addition to agalsidase alfa and migalastat which were recommended for funding in March 2023. 

Gaucher disease - an inherited lysosomal storage disorder resulting in build-up of a type of fat which can affect many parts of the body, including the spleen, liver and bones.

Hereditary angioedema - an inherited disorder that results in recurrent episodes of severe swelling (angioedema).

Pompe disease - an inherited lysosomal storage disorder which damages muscle and nerve cells throughout the body.

Von Hippel-Lindau (VHL) disease - a genetic multi-system disorder in which tumours grow in certain parts of the body and may become cancerous.