Today is Rare Disease Day
Pharmac understands that people living with rare disorders face many challenges, including access to health care and effective medicines.
We have been working to improve funded access to medicines for rare disorders and since 2017, we have:
- Established a Rare Disorders Advisory Committee, made up of experts in the management of rare disorders.
- Invited pharmaceutical suppliers to submit funding applications for treatments for rare disorders.
- Adjusted our policy settings for rare disorders treatments.
- Regularly reviewed the portfolio of medicines for rare disorders and funded new medicines.
- Invited and heard from people with rare disorders at Rare Disorders Advisory Committee meetings.
- Funded rare disorders medicines like nusinersen, risdiplam, and emicizumab used in the treatment of spinal muscular atrophy and haemophilia A.
- Changed our processes so we can assess a funding application at the same time Medsafe is assessing the application for regulatory approval. This means that medicines will be able to be considered for funding sooner in New Zealand.
But we understand there is still more to do.
Improved quality of life and better health for people with haemophilia A
We spoke to Deon York and Karl Archibald of Haemophilia New Zealand, on the treatment of haemophilia A without factor VIII inhibitors.