Pharmac shares the agenda items for the upcoming Rare Disorders Committee meeting
Te Pātaka Whaioranga – Pharmac is creating visibility of what medicines are being considered that could impact the rare disorders community by sharing the agenda items for the upcoming Rare Disorders Advisory Committee(external link) meeting.
Pharmac’s Chief Medical Officer, Dr David Hughes is pleased to have a number of applications for the clinical advisors to consider. “Three of the proposals are for spinal muscular atrophy (SMA) – two are for wider eligibility groups for nusinersen and risdiplam at other ages and stages of the condition.
“The other treatment for SMA is a new proposal for a gene therapy, called onasemnogene abeparvovec (Zolgensma). This is the first of its kind to be considered for the condition and has the benefit of being a single treatment delivered by infusion.
“We will also be reconsidering a handful of applications because we have received new information. These include treatments for Fabry disease, short bowel syndrome, and Morquio syndrome (type A),” says Dr Hughes.
“In addition to considering the applications, for the first time we have invited people from the community to join to share their lived experience of Fabry disease and SMA. This will allow our expert advisors to hear directly from people who are affected by these conditions and by our funding decisions.”
Pharmac is working with Rare Disorders New Zealand to include the voices of people who are best placed to represent their communities in these discussions.
“We understand that people living with rare disorders face many challenges, including access to suitable health care and effective treatment,” says Dr Hughes. “We hope by sharing the agenda items today, people can see what medicines we are assessing see that we are committed to work in this therapeutic group area.”
The Rare Disorders Advisory Committee meeting will take place Tuesday 7 March, with the records of the meeting released later in the year. Funding applications being considered meet the principles of our Rare Disorders policy and are going through assessment in line with Pharmac’s usual processes. Find out more about Pharmac's funding for rare disorders.
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)
- P-001028: Agalsidase alfa (Enzyme Replacement Therapy)(external link)
- P-000444: Migalastat (external link)
MPS (a metabolic condition that primarily affects the skeleton)
- P-000341: Elosulfase alfa(external link) Application previously declined 2018 meeting(external link).
Short bowel syndrome (intestinal failure caused from congenital diseases or extensive surgical resection)
- P-001020: Teduglutide(external link)
Spinal muscular atrophy (SMA) (a disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement)
- P-001865: Onasemnogene abeparvovec(external link) (Zolgensma) – gene therapy
- P-001819: Risdiplam(external link) – pre-symptomatic SMA
- Risdiplam and nusinersen – people not currently eligible
- SMA types IIIb and IIIc (symptomatic after 3 years of age)
- People with any type who are aged 19 years and over
- SMA type IV (adult-onset) aged 19 years and over at symptom onset