Pharmac widens access to treatment for life-long bleeding disorder, severe haemophilia A

Te Pātaka Whaioranga – Pharmac has confirmed it is widening access to emicizumab (branded as Hemlibra) for the treatment of people with severe haemophilia A without factor VIII inhibitors.

“Widening access to this treatment will help people manage a life-long bleeding disorder” Pharmac’s Director, Pharmaceuticals Geraldine MacGibbon says.  “We expect that 115 people will access this treatment during the first year of funding, increasing to 140 people as they transition from other treatments. There are many children in this group, so it is positive that they’ll have a life-long treatment available to them.”

The Chair of Haemophilia New Zealand, Deon York applauds the announcement. “This represents the most significant change to care and treatment for people with severe haemophilia A for nearly 25 years. We are elated by the decision and convey our heartfelt appreciation”.

Haemophilia A is a hereditary, life-long bleeding disorder. It is caused by a deficiency of factor VIII, which impairs the body's ability to make blood clots. This causes prolonged spontaneous and injury-related bleeding, which can be traumatic for those affected. Without treatment, people with severe haemophilia A can experience negative long-term outcomes like severe joint damage. 

“Previously, people with severe haemophilia A have needed regular intravenous infusions of factor VIII, often multiple times per week. Emicizumab is given as a subcutaneous injection which means that people, or their caregivers, will be able to administer the treatment themselves” Geraldine MacGibbon says.

"This decision has the potential to make a big difference to their lives. Through our consultation, we heard from a number of people living with severe haemophilia A and their whānau, who shared information about the difference this change will make for them.” 

“Access to emicizumab is also likely to reduce the burden on the wider health sector as a result of reduced need for specialist consultations and a reduction in the number of bleed events requiring hospitalisation.”

In addition to the widened access to emicizumab for the treatment of severe haemophilia A, Pharmac is also widening access to ocrelizumab for primary progressive multiple sclerosis. Widened access for both treatments will start from 1 October 2023. 

Emicizumab and ocrelizumab are both supplied by Roche Products (New Zealand) Ltd. Roche’s General Manager, Alex Muelhaupt, says it's wonderful news that a wider group of New Zealanders can now access these treatments. “We want to acknowledge the work done by the multiple sclerosis and haemophilia communities over the years, leading to this agreement.”

MacGibbon says that this funding proposal shows the Pharmac model of negotiation and maximising the medicines budget in action. “Our team is determined to fund as many products as possible through our fixed budget that is provided to us by the government. We spend every cent we get on medicines, vaccines and devices, and we will continue to work to get the best health outcomes for people living in New Zealand.”