Scoop has an Ethical Paywall
Work smarter with a Pro licence Learn More

Video | Agriculture | Confidence | Economy | Energy | Employment | Finance | Media | Property | RBNZ | Science | SOEs | Tax | Technology | Telecoms | Tourism | Transport | Search


Pharmac Shares 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 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 (Zelenska). 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.

Advertisement - scroll to continue reading

Are you getting our free newsletter?

Subscribe to Scoop’s 'The Catch Up' our free weekly newsletter sent to your inbox every Monday with stories from across our network.

“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 and 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 minutes of the meeting released later in the year.

© Scoop Media

Business Headlines | Sci-Tech Headlines


Join Our Free Newsletter

Subscribe to Scoop’s 'The Catch Up' our free weekly newsletter sent to your inbox every Monday with stories from across our network.