Pharmac calls for applications for medicines for rare disorders

Te Pātaka Whaioranga – Pharmac is calling for applications for medicines to treat rare disorders. These will be considered at the next meeting of the Rare Disorders Advisory Committee meeting in May 2024.

“We are encouraging funding applications from pharmaceutical suppliers, clinicians as well as consumers, as we understand people living with rare disorders face many challenges including access to suitable health care and effective medicines” says Pharmac’s Director Advice and Assessment/Chief Medical officer, Dr David Hughes.  

“We also want to hear from the rare disorders community about the medicines we’ll be considering for funding for the treatment of rare disorders. We’ll be sharing the agenda of the Rare Disorders Advisory Committee meeting in April 2024 so those in the rare disorder’s community can see which medicines we will be considering.”

“It’s really important to us to hear from people with lived experience of the disorders we’ll be discussing, and those people will be welcome to engage with us at any time throughout this funding assessment process,” says Dr Hughes.

The Rare Disorders Advisory Committee last met in March 2023 at a joint meeting with members from Pharmac’s Gastrointestinal and Neurological Advisory Committees to discuss treatments for spinal muscular atrophy, Fabry disease, mucopolysaccharidosis, and short bowel syndrome. Records of that meeting [PDF, 525 KB] and the Committee’s previous meetings are available on the Pharmac website.

The agenda for the Rare Disorders Advisory Committee May 2024 meeting will include the consideration of new funding applications and review of existing funded medicines for rare disorders, including medicines considered via our Named Patient Pharmaceutical Assessment (NPPA) process for individual patients.

Funding applications need to meet the principles of our Rare Disorders policy and will be assessed in line with Pharmac’s usual processes.