Pharmac considers widening access to treatments for multiple sclerosis and haemophilia A

Te Pātaka Whaioranga - Pharmac is consulting on widening access for two treatments; ocrelizumab (branded as Ocrevus) as the first funded treatment for primary progressive multiple sclerosis, and emicizumab (branded as Hemlibra) for the treatment of all people with severe haemophilia A.

“We understand people living with these conditions are currently experiencing considerable unmet health needs so we’re pleased to be able to consider funding medicines that could increase their quality of life, ” says Pharmac’s acting Director, Pharmaceuticals Geraldine MacGibbon. "We want to hear from people affected by these conditions, their whānau, and treating healthcare practitioners to ensure the outcomes of this process meet their needs.”

First funded treatment for primary progressive multiple sclerosis

People with multiple sclerosis (MS) experience numerous sensory and physical symptoms which impacts their abilities over time. The primary progressive type of MS affects 10-15% of people with the condition and causes symptoms to get gradually and consistently worse, rather than people experiencing relapses with a later recovery as with relapsing-remitting MS.

“Ocrelizumab, which slows the progression of symptoms, would be the first treatment funded for primary progressive multiple sclerosis. This would support improved quality of life for about 85 people in the first year of funding, increasing to approximately 210 people each year after five years,” says Ms MacGibbon.

National Manager of Multiple Sclerosis New Zealand, Amanda Rose, was delighted to hear about the consultation, sharing that it will providing the hope that their community has been searching for. “This is a momentous day for the treatment of the most disabling form of multiple sclerosis. If the proposal is approved, New Zealand would be the first country in the Asia-Pacific region to provide funded access to this medicine for primary progressive multiple sclerosis. We will be supporting Pharmac to share this consultation to ensure we have input into the decision.”

Ocrelizumab is administered by an intravenous (IV) infusion. The first two doses are given two weeks apart, followed by maintenance dosing every 6 months.

Widened access to a treatment for severe haemophilia A

Haemophilia is an inherited disorder that impairs the body's ability to make blood clots, causing prolonged spontaneous and injury-related bleeding. There are a few types of the disorder, with people with severe haemophilia A having the worst long-term outcomes, such as severe joint damage. 

“Emicizumab is a treatment that helps the body to form blood clots and reduces the frequency of traumatic bleeding,” says Ms MacGibbon. “It is currently only funded as a preventative treatment for people with severe haemophilia A who are unable to be treated with Factor VIII medicines. We are proposing to widen access to emicizumab so it would be funded for anyone with severe haemophilia A. This would mean that another 115 people could benefit from the treatment in the first year, increasing to 140 people after 5 years – many of whom would be children.”

The treatment is delivered by a subcutaneous injection which people can self-administer. “Most people with severe haemophilia A would no longer need regular, often three to four times weekly, IV infusions to manage their condition, and we believe that this would help reduce potential barriers to treatment. As part of this consultation, we are interested in hearing how we could support the distribution of this product to best suit the needs of the community.”

Chair of Haemophilia New Zealand, Deon York, believes the consultation would be game-changing for the quality of life for many of their community across Aotearoa. “Widening access to this therapy would represent a significant improvement for our community, taking us closer to narrowing the equity gap and reducing the burden on patients and whānau.”

Working with suppliers

“Ocrelizumab and emicizumab have been on our options for investment list as medicines we would like to fund for these conditions, so we are pleased we have secured the provisional agreement with their supplier, Roche,” concludes Ms MacGibbon.

General Manager of Roche Products (New Zealand) Ltd Alex Muelhaupt says, “It's wonderful news that a wider group of New Zealanders are now a step closer to accessing these treatments. We want to acknowledge the work done by the NZ multiple sclerosis and haemophilia communities over the years, leading to this consultation.”

The consultation for today’s proposal is available on the Pharmac website for anyone wanting to give us their feedback. If approved, funded access to ocrelizumab and emicizumab would be widened from 1 October 2023.