Proposal to widen access to medicines for haemophilia A and multiple sclerosis
Pharmac is seeking feedback on a proposal to widen access to medicines for haemophilia A and multiple sclerosis.
On this page
A decision has been made
We have published our decision following this consultation.
What we’re proposing
We are seeking feedback on a proposal to:
- widen access to emicizumab, brand name Hemlibra, for the treatment of severe haemophilia A without FVIII inhibitors
- widen access to ocrelizumab, brand name Ocrevus, for the treatment of primary progressive multiple sclerosis.
These medicines would be funded through a provisional agreement with the supplier, Roche Products (NZ) Ltd. The agreement includes amendments to the contractual terms for pertuzumab (branded as Perjeta), funded for the treatment of HER2 positive metastatic breast cancer in combination with trastuzumab.
We welcome your feedback on this proposal. Consultation closes at 5pm Friday 7 July 2023 and feedback can be emailed to consult@pharmac.govt.nz.
Emicizumab for severe haemophilia A
What would the effect be?
From 1 October 2023, access to emicizumab would be widened to people with severe haemophilia A without inhibitors, subject to eligibility criteria. This would mean that any person with severe haemophilia A, defined as endogenous factor VIII (FVIII) activity less than or equal to 2%, would be able to access funded emicizumab, regardless of FVIII inhibitors titres.
We anticipate that around 115 additional people with severe haemophilia A would access emicizumab in the first year, increasing to 140 people after 5 years. There is not enough information to understand if haemophilia A disproportionately affects Māori, Pacific peoples or other populations experiencing health inequities.
Emicizumab would continue to be distributed directly to an individuals nominated collection point, such as a community pharmacy (see distribution section below). People could self-administer emicizumab via subcutaneous injection.
People with haemophilia A treated with FVIII require intensive specialist care and management. We expect that the suitability and efficacy of emicizumab would reduce the need for specialist resources, which would benefit people who experience barriers accessing these services.
Who we think will be interested
- People with severe haemophilia A, their whānau, friends and caregivers
- Healthcare professionals involved in the care of people with haemophilia A
- Te Whatu Ora – Health New Zealand hospitals and other organisations who deliver services and support for people, and their whānau who are affected by haemophilia A
- People or groups with an interest in treatments for haemophilia
- Pharmacies and wholesalers
- Pharmaceutical suppliers of haemophilia A products
About haemophilia A and emicizumab
Haemophilia A is a hereditary, life-long bleeding disorder caused by a deficiency of factor VIII, which results in prolonged spontaneous and injury-related bleeding, joint damage, and in some cases can be life threatening. There are about 500 people in New Zealand with haemophilia A. The disorder is severe for around 150 people who are more likely to have serious bleeds. Most of these people are managed appropriately with currently funded treatments.
Haemophilia A can have a high impact on the everyday life of people with the disorder and their whānau especially if they are a young child.
Emicizumab is currently funded as preventative (prophylactic) treatment for people with haemophilia A who have developed inhibitors to FVIII. Most people do not develop inhibitors and are treated with intravenous (IV) FVIII, which helps the blood to clot and stop bleeding. FVIII is used as both prophylaxis and to manage active bleeding episodes. FVIII is typically administered two to three times a week. Managing the frequency of Factor VIII treatment can be challenging and demanding for both the person, their family/whānau and the health system.
Emicizumab is a protein that helps the blood to clot. It works by replacing the function of factor VIII. It is highly effective in preventing bleeding and reduces the frequency of traumatic bleeding episodes in people with haemophilia A.
Emicizumab is simpler to administer and requires less health resources to manage compared to current treatment options.
Emicizumab is administered subcutaneously at a recommended dose for routine prophylaxis of 3 mg/kg once weekly for the first 4 weeks, followed by a maintenance dose of 1.5 mg/kg weekly.
Why we’re proposing this
PTAC recommended the application to be funded with a high priority, due to the high health need of people with this condition, the suitability compared to current treatment and the benefits to the person, their family/whānau, and the health system through a reduction in bleeding events.
The Committee recommended changes to the eligibility criteria PTAC recommended to appropriately target funding to those who would benefit from treatment and provide lifelong funding after initial approval, without the need for periodic renewal.
The application has been ranked on the Options for Investment list since March 2022. We have now reached a commercial agreement with the supplier that enables us to progress it.
Details about our proposal
Emicizumab would continue to be listed in Section B and Part II of Section H of the Pharmaceutical Schedule at the current list price (ex-manufacturer, excluding GST). A confidential rebate would apply that would reduce the net price to the Funder.
The Hemlibra brand would have subsidy and delisting protection until 1 October 2027.
Emicizumab would continue to be listed with “Xpharm” restriction. An Xpharm listing means that community pharmacies cannot claim subsidy because Pharmac has made alternate distribution arrangements.
The eligibility for emicizumab would be amended in Section B as follows (deletions in strikethrough, additions in bold):
Initial application – (Severe Haemophilia A with or without FVIII inhibitors) only from a haematologist. Approvals valid for 6 months without further renewal unless notified for applications meeting the following criteria:
Both:
- Patient has severe congenital haemophilia A with a severe bleeding phenotype (endogenous factor VIII activity less than or equal to 2%); and
- Emicizumab is to be administered at a dose of no greater than 3 mg/kg weekly for 4 weeks followed by the equivalent of 1.5 mg/kg weekly.
All of the following:
- Patient has severe congenital haemophilia A and history of bleeding and bypassing agent usage within the last six months; and
- Either:
- Patient has had greater than or equal to 6 documented and treated spontaneous bleeds within the last 6 months if on an on-demand bypassing agent regimen; or
- Patient has had greater than or equal to 2 documented and treated spontaneous bleeds within the last 6 months if on a bypassing agent prophylaxis regimen; and
- Patient has a high-titre inhibitor to Factor VIII (greater than or equal to 5 Bethesda units per ml) which has persisted for six months or more; and
- There is no immediate plan for major surgery within the next 12 months; and
- Either:
- Patient has failed immune tolerance induction (ITI) after an initial period of 12 months; or
- The Haemophilia Treaters Group considers the patient is not a suitable candidate for ITI; and
- Treatment is to be administered at a maximum dose of 3 mg/kg weekly for 4 weeks followed by the equivalent of 1.5 mg/kg weekly.
Renewal only from a haematologist. Approvals valid for 6 months for applications meeting the following criteria:
Both:
- Patient has had no more than two spontaneous and clinically significant treated bleeds after the end of the loading dose period (i.e. after the first four weeks of treatment until the end of the 24-week treatment period); and
- The treatment remains appropriate and the patient is benefiting from treatment.
A similar restriction would apply in Part II of Section H of the Pharmaceutical Schedule.
Distribution of emicizumab
To access supply of emicizumab people would enrol with a pharmacy of their choice and collect the medicine as required. These distribution arrangements are currently in place for emicizumab, however, would need substantial expansion to meet the larger group of people who would access the medicine.
We are seeking specific feedback on this part of the proposal to ensure the process would work for both the individual and their family/whānau. We are also interested to hear from distributors, wholesalers and pharmacies to understand the feasibility and suitability of expanding the distribution system.
Ocrelizumab for primary progressive multiple sclerosis
What would the effect be?
From 1 October 2023, access to ocrelizumab would be widened to people with primary-progressive multiple sclerosis (PPMS), subject to Special Authority criteria.
There are currently no funded pharmaceutical treatments for primary progressive multiple sclerosis. This proposal would mean that ocrelizumab would be the first funded targeted medicine for this type of multiple sclerosis.
We anticipate that around 85 additional people with multiple sclerosis would access ocrelizumab in the first year, increasing to 210 after 5 years. We anticipate the uptake would likely be everyone with PPMS who meets the eligibility criteria, as there are no alternative targeted treatments. There is a significantly lower incidence of PPMS in Māori compared with non-Māori.
Change for people with relapsing-remitting multiple sclerosis (RRMS) who use ocrelizumab
Ocrelizumab, is currently funded for most people with the relapsing-remitting type of multiple sclerosis (RRMS) alongside other MS treatments. We would need to make some changes to the Special Authority forms for all RRMS treatments if we include ocrelizumab eligibility criteria for PPMS, so the software that generates Special authority applications could manage this change. This would mean that people with RRMS would need new Special Authority numbers if they want to change between funded multiple sclerosis treatments (additional details are below).
Who we think will be interested
- People with multiple sclerosis, their whānau, friends and caregivers
- Healthcare professionals involved in the care of people with multiple sclerosis
- Te Whatu Ora – Health New Zealand hospitals and other organisations who deliver services and support for people, and their whānau who are affected by multiple sclerosis
- People or groups with an interest in treatments for multiple sclerosis
- Pharmacies and wholesalers
- Pharmaceutical suppliers
About PPMS and ocrelizumab
Multiple sclerosis (MS) is an autoimmune condition in which the immune system attacks the central nervous system, leading to demyelination. It may cause numerous sensory and physical symptoms, and often progresses to physical and cognitive disability. PPMS is a type of MS that causes symptoms to get gradually and consistently worse. This is different to RRMS with relapses of symptoms with a later recovery (ie. a health decline that eases, recovers, then later relapses again). Approximately 10 to 15 percent of people with MS have the PPMS type.
Ocrelizumab is a recombinant humanised monoclonal antibody (IgG1 subtype) that selectively targets CD20-expressing B-cells and is used for the treatment of people with PPMS and RRMS.
Ocrelizumab would allow people with PPMS to have an improved quality of life, as it would slow progression to more severe illness and enable people to live more independently. We expect this would also relieve some carer burden for those who care for people with PPMS.
Ocrelizumab is administered by intravenous (IV) infusion. The first dose is followed by another dose after 2 weeks; then maintenance dosing occurs every 6 months. We expect this proposal would create a small increase on demand for infusion services, however would reduce health system resource by delaying the progression of disability for people with PPMS.
Why we’re proposing this
PTAC initially recommended this application be declined. However in November 2020, following updated evidence, PTAC recommended that ocrelizumab be funded for the treatment of PPMS with a low priority.
Our expert clinical advisors told us that there is a high health need for people with PPMS and a lack of funded treatment options. Ocrelizumab would be the first targeted treatment funded for PPMS and is expected to improve the quality of life of people living with PPMS.
The application has been ranked on our Options for Investment list since March 2022. We are pleased to have now reached a provisional commercial agreement with the supplier to progress it.
Details about our proposal
Ocrelizumab would continue to be listed in Section B and Part II of Section H of the Pharmaceutical Schedule at the current price (ex-manufacturer, excluding GST). A confidential rebate would apply that would reduce the net price to the Funder.
The Ocrevus brand would have subsidy and delisting protection until 1 October 2026.
Eligibility criteria for PPMS would be added to ocrelizumab in Section B of the Pharmaceutical Schedule as follows:
Initial application — (Primary progressive multiple sclerosis) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- Diagnosis of primary progressive multiple sclerosis (PPMS) meets the 2017 McDonald criteria and has been confirmed by a neurologist;(external link)and
- Patient has an EDSS 2.0 (score less than or equal to 2 on pyramidal functions) to EDSS 6.5; and
- Patient has no history of relapsing remitting multiple sclerosis.
Renewal application – (Primary progressive multiple sclerosis) from any relevant practitioner. Approvals valid for 12 months for applications where the patient has had an EDSS score of 2.0 to 6.5 (inclusive) at any time in the last six months (i.e. patient has walked 20 metres with bilateral assistance/aids, without rest in the last six months)
A similar restriction would apply in Part II of Section H of the Pharmaceutical Schedule.
Change for people with RRMS who use ocrelizumab
The eligibility criteria for people with RRMS using ocrelizumab would not change as part of this proposal. To ensure the software that generates Special Authority applications can manage this change we would need to amend the forms for this treatment. Ocrelizumab would also move to its own therapeutic subgroup within the Pharmaceutical Schedule to enable the Special Authority forms to work.
We would like to reassure people with RRMS using ocrelizumab (about 390 people in the last year) that there would be no changes in access. Everyone who currently uses ocrelizumab for RRMS would be automatically issued with a new Special Authority number. If a person needed to change to a different treatment for RRMS in the future, their prescriber would need to apply for a new initial Special Authority number under the Multiple Sclerosis treatment form(external link).
Everyone who currently takes any of the other funded MS treatments (fingolimod, teriflunomide, natalizumab, dimethyl fumarate, glatiramer acetate, interferon alpha-1-alpha or interferon beta-1-beta) would not need new Special Authority numbers. Changing between these multiple sclerosis treatments could continue to occur without further application as it does now. If a person changes to ocrelizumab for RRMS, their prescriber would then need to apply for a new initial Special Authority number using the new ocrelizumab Special Authority application form.
Pertuzumab contractual changes
This proposal would also result in updated pricing for pertuzumab (brand name Perjeta) through a confidential rebate. No change to the current list price of pertuzumab is proposed.
While the eligibility criteria would remain unchanged, we are separately consulting on a proposal that includes changes to the eligibility criteria for ‘treatment holidays’.
To provide feedback
Send us an email: consult@pharmac.govt.nz by 5pm Friday 7 July 2023.
All feedback received before the closing date will be considered by Pharmac’s Board (or its delegate) prior to making a decision on this proposal.
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