Decision to widen access to medicines for severe haemophilia A and multiple sclerosis
On this page
Educational resources
Emicizumab (Hemlibra) - Roche NZ's dedicated website(external link)
Ocrevus for PPMS – Your Questions Answered - Multiple Sclerosis New Zealand's website(external link)
(Resources added 4 October 2023)
What we’re doing
We're pleased to announce that we have made a decision to widen access to two treatments (emicizumab and ocrelizumab) from 1 October 2023:
- Access to emicizumab (branded as Hemlibra) will be widened to include people with severe haemophilia A without FVIII inhibitors.
- Access to ocrelizumab (branded as Ocrevus) will be widened to include people with primary progressive multiple sclerosis.
In addition, from 1 October 2023 applications for Special Authority for all funded multiple sclerosis (MS) treatments will be able to be made by any relevant practitioner (currently only a neurologist or general physician can apply for a Special Authority).
These medicines will be funded through an 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. These contractual amendments will not change access to pertuzumab, however we have made changes to allow for ‘treatment holidays’. More details can be found through our recent decision on intravenous trastuzumab.
Emicizumab for severe haemophilia A
What does this mean for people
Emicizumab is currently funded as preventative (prophylactic) treatment for people with haemophilia A who have developed inhibitors to Factor VIII. From 1 October 2023, access will be widened to all people with severe haemophilia A.
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. Access to emicizumab will support them to live a higher quality of life.
Emicizumab is also simpler to administer and requires less health resources to manage compared to current treatment options. We received consultation feedback from people with haemophilia A and their whānau, describing the difference this change would make to their life.
Any changes to the original proposal?
This decision was subject to a consultation letter dated 22 June 2023.
We received supportive feedback from consumers, clinicians, advocacy groups, and industry. We would like to thank everyone that took the time to respond. We have not made any changes to the proposal.
A summary of the feedback received and our responses to this is included below.
Who we think will be most 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
Detail about this decision
Emicizumab will 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 will apply that will reduce the net price to the Funder.
Hemlibra will have subsidy and delisting protection until 1 October 2027.
Emicizumab will continue to be listed with “Xpharm” restriction. An Xpharm listing means that community pharmacies cannot claim subsidy because Pharmac has made alternate distribution arrangements.
People who are prescribed emicizumab will need to confirm with their Haemophilia Treatment Centre (HTC) their preferred nominated pharmacy where they choose to collect their emicizumab from. The HTC will then liaise with the distributor of emicizumab to confirm the designated pharmacy for each person prescribed emicizumab.
When the prescription for emicizumab is ready for collection, the designated pharmacy will contact individuals directly to inform them that their medicine is available to collect.
This process is the same way in which emicizumab is currently distributed to eligible people with severe haemophilia A with inhibitors.
The following changes will occur in Section B and Part II of Section H of the Pharmaceutical Schedule. The eligibility for emicizumab will be amended from 1 October 2023 to the following:
Initial application – (Severe Haemophilia A with or without FVIII inhibitors) only from a haematologist. Approvals valid 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.
Our response to what you told us
We’re really grateful for the time people took to respond to this consultation. Responses were largely supportive of the proposal. A summary of the main themes raised in the feedback and our responses are available below.
| Theme | Pharmac staff comment |
|---|---|
| Support for the proposal. We received a number of personal stories, including those from whānau of children with haemophilia, telling us how emicizumab would substantially improve their quality of life. | We are grateful to all the people who wrote in to tell us about their experiences with haemophilia and what treatment with emicizumab would mean to them and their whānau. Hearing how medicines impact the lives of New Zealanders is really important in helping us understand the value of the medicines we are proposing to fund. |
| Requests for additional funding to pharmacies for the workload associated with distribution of emicizumab. | The distribution mechanism is different to how other medicines are managed and is intended to support pharmacies in managing the risks involved in the supply of emicizumab. As the supplier of emicizumab, Roche, manages distribution of the treatment, we have passed this feedback onto them for consideration. |
Ocrelizumab for primary progressive multiple sclerosis
What does this mean for people?
Ocrelizumab is currently funded and available for the treatment of relapsing remitting multiple sclerosis (RRMS). From 1 October 2023, access will be widened to include primary progressive multiple sclerosis (PPMS). This will be the first funded treatment for people with PPMS.
From 1 October 2023 applications for Special Authority for any funded MS treatments will be able to be made by any relevant practitioner (currently only a neurologist or general physician can apply for a Special Authority).
Funding ocrelizumab for PPMS will mean everyone who currently uses ocrelizumab for RRMS will be automatically issued with a new Special Authority number by 1 October. If a person needs to change to a different treatment for RRMS following this, their prescriber will need to apply for a new initial Special Authority number under the Multiple Sclerosis treatment form(external link).
We would like to reassure people with RRMS that their access to treatment will not be impacted as a result of this proposal.
Any changes to the original proposal?
This decision was subject to a consultation letter dated 22 June 2023.
We received feedback from consumers, clinicians, advocacy groups, and industry, and want to thank everyone for their feedback.
Responses were overall supportive of the proposal. Following the consultation period, we have aligned the prescriber restrictions across all multiple sclerosis treatments to allow any relevant prescriber to apply. No other changes were made to the proposal.
We also received requests for changes to the Special Authority criteria for ocrelizumab for primary progressive multiple sclerosis (PPMS).
A summary of the feedback received and our responses to this is included below.
Who we think will be most 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
Detail about this decision
Ocrelizumab will 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 will apply that will reduce the net price to the Funder.
The Ocrevus brand will have subsidy and delisting protection until 1 October 2026.
The following changes will occur in Section B and Part II of Section H of the Pharmaceutical Schedule.
A new Therapeutic Group will be created in the Pharmaceutical Schedule under Multiple Sclerosis Treatments from 1 October 2023 called Multiple Sclerosis Treatments – Other.
Ocrelizumab will be moved to Multiple Sclerosis Treatments – Other from 1 October 2023. The same eligibility criteria will apply as for Multiple Sclerosis treatments (dimethyl fumarate, fingolimod, glatiramer acetate, interferon beta-1-alpha, interferon beta-1-beta, natalizumab and teriflunomide) above. This change will allow for people on ocrelizumab to change to these treatments.
Criteria for people with PPMS will be added from 1 October 2023:
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; 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 – (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 (ie patient has walked 20 metres with bilateral assistance/aids, without rest in the last six months)
The eligibility criteria for all other Multiple Sclerosis treatments (ie dimethyl fumarate, fingolimod, glatiramer acetate, interferon beta-1-alpha, interferon beta-1-beta, natalizumab and teriflunomide) funded for the treatment of RRMS will be amended from 1 October 2023 to allow:
- applications from any relevant practitioner
- people without PPMS to change from other multiple sclerosis treatments to ocrelizumab.
Our response to what you told us
We’re really grateful for the time people took to respond to this consultation. Responses were largely supportive of the proposal. A summary of the main themes raised in the feedback, and our responses are available below.
|
Theme |
Pharmac staff comment |
|---|---|
|
Supportive of the proposal. Many responders commented that this proposal would mean there would be a treatment available that could slow the progression of their disease, allow them to retain independence for longer and live well for longer. They shared personal stories explaining their experience or that of their whānau with PPMS and what the availability of a treatment would mean for people with PPMS and their whānau. |
We are grateful to all the people who shared their experiences with PPMS with us, and what treatment would mean to them and their whānau. Hearing how medicines impact the lives of New Zealanders is really important in helping us understand the value of the medicines we are proposing to fund. |
|
Requested removal or amendment of the upper EDSS limit. |
The EDSS limit of 6.5 is in line with the clinical advice we have received from our expert advisors, the Neurological Advisory Committee. In summary, our experts considered that the EDSS limit reflects the population included in the trials, and targets treatment to those most likely to benefit. Our expert advisors considered a higher EDSS would be inappropriate due to a current lack of evidence supporting clinical benefit. More details about the clinical advice we received is available on the application tracker(external link). We would welcome a funding application for widened access should new evidence supporting clinical benefit at higher EDSS become available. |
|
Requested implementation activity to support GPs and neurologists to identify eligible patients in the absence of a PPMS registry |
We appreciate the importance of identifying people with PPMS. We have passed this feedback onto the supplier, Roche. Roche is working on educational materials to support healthcare professionals identify people with PPMS who may be eligible for treatment, and to raise awareness among the MS community that there is treatment available. This includes a FAQ codesigned with Multiple Sclerosis NZ. Pharmac will support these endeavours through our partnership with He Ako Hiringa(external link) to raise awareness of the availability of ocrelizumab for PPMS. |
|
Requested that the 20-metre walk required for renewal can be sighted and approved by a health professional other than a neurologist such as a GP or a physiotherapist |
There are no requirements for a neurologist to sight the 20-metre walk requirement. Applications for renewal can be made by any relevant practitioner, working within their scope of practice (for example a person’s GP), who has confirmed the person with PPMS has walked 20 meters with bilateral assistance/aids, without rest in the last six months. |
|
Allow for renewal of people who cannot walk more than 20 metres for non-MS related issues |
We would consider a Special Authority waiver application for people with PPMS who are unable to walk due to non-MS related issues and have other clinical information to suggest the intent of the renewal criteria would otherwise be met. |
|
Requested that people who do not meet criteria due to a significant spinal cord MS lesion be eligible for treatment. |
The funding criteria are in line with the clinical advice we have received from our expert advisors, the Neurological Advisory Committee, and target treatment to those most likely to benefit. In summary our expert advisors considered the evidence supported that only those with EDSS scores between 2.0 and 6.5 would benefit from treatment and be eligible. Those with higher EDSS scores as a result of a significant spinal cord MS lesion would be outside this target group. More details about the clinical advice we received is available on the application tracker(external link). We would welcome a funding application for wider access should new evidence supporting clinical benefit at higher EDSS, including where that score is as a result of a significant MS lesion, become available. |
|
Requested that those who miss SA renewal timelines can continue to access treatment |
Special Authority renewal applications can now be submitted by ‘any relevant practitioner’, for example a person’s GP. We would be happy to consider a Special Authority waiver application for an extension of funding, if a person was unable to be seen by their prescriber before their Special Authority approval expired. |
|
Requested that the administration required for access to ocrelizumab and other MS treatments be simple so as to not present access barriers for patients |
Our intent is to ensure access is as simple as possible. Everyone who is currently taking ocrelizumab for RRMS who has an active Special Authority for Multiple Sclerosis Treatments(external link) would be issued a new Special Authority approval number for ocrelizumab on 1 October 2023. From 1 October 2023 anybody switching to ocrelizumab from another Multiple Sclerosis Treatment, or vice versa, would require a new Special Authority approval. |
|
Concerns regarding the criteria prohibiting those with a history of RRMS from accessing ocrelizumab for PPMS |
Ocrelizumab is funded for both RRMS and PPMS. The funding criteria for each are different and are based on the clinical advice we have received from our expert advisors, the Neurological Advisory Committee, to target treatment to those most likely to benefit. We would consider a Special Authority waiver application for people with PPMS where there has been uncertainty with diagnosis and other clinical information is provided to suggest the intent of the criteria would be met. |
|
Requested reconsideration of funding for fampridine |
We have assessed a proposal to fund fampridine for symptomatic walking improvement for MS. PTAC recommended that the application be declined. Full details about the advice we received is available from the Application Tracker.(external link) We would welcome any updated information that would support reconsideration of this application. |
If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.