Proposal to widen access and change the funded enzyme replacement therapy for Gaucher disease
17 May 2018
What we’re proposing
PHARMAC is seeking feedback on proposed changes to the funding of enzyme replacement therapy (ERT) used in the treatment of Gaucher disease (a rare disorder):
- The funded ERT for the treatment of Gaucher disease would change from imiglucerase (Cerezyme), supplied by Sanofi, to taliglucerase alfa (Elelyso), supplied by Pfizer.
- Taliglucerase alfa would be funded from 1 August 2018 and would become the only funded ERT for the treatment of Gaucher disease in the community after a 7-month transition period.
- Special Authority and Hospital Restrictions would apply to taliglucerase alfa. These would be similar to the restrictions that currently apply to imiglucerase, except that, if approved by the Gaucher Panel (a panel of expert doctors), the criteria would allow a higher maximum dose (30 units/kg every other week, approx. 60 units/kg/month) where clinically appropriate, which could provide clinical benefits for some patients.
- Transition timeframes and further information are detailed below.
Consultation closes at 4 pm on Monday 11 June 2018 and can be emailed to email@example.com.
What would the effect be?
From 1 August 2018, eligible people with Gaucher disease (type 1 and 3) would have funded access to a new enzyme replacement therapy (ERT), taliglucerase alfa (Elelyso). Currently, 20 patients with type 1 or type 3 Gaucher disease access funded ERT with imiglucerase.
- Patients could access a higher maximum dose of ERT on switching to taliglucerase alfa (30 units/kg every other week, approx. 60 units/kg/month), where clinically appropriate and approved by the Gaucher Panel.
- During a transition period, from 1 August 2018 to 28 February 2019, all current patients receiving funded ERT for Gaucher disease would need to change to taliglucerase alfa to continue to receive a funded treatment. This change would be carefully supported by the treating clinician, working with the patient and family/caregivers. PHARMAC would directly communicate with relevant clinicians, support services and DHB services regarding the change process.
- Prior to changing treatments, a small number of patients may require an MRI scan if they have not received one in the previous 12 months.
- Patients who currently manage their infusions at home (~10) would need to attend hospital outpatient clinics for the first 2-4 infusions of the new treatment. This would be led and organised by the treating clinician. Following this, patients could continue to manage infusions of the new treatment at home. Support would be provided by a dedicated infusion nurse during the transition (provided by the new supplier).
- If a patient does not tolerate the new treatment for a clinical reason, PHARMAC would consider a Named Patient Pharmaceutical Assessment (NPPA) application from a clinician to reinstate imiglucerase for an individual patient, following advice from the Gaucher Panel.
- From 1 March 2019, imiglucerase (Cerezyme) for the treatment of Gaucher disease would be delisted, meaning that it would no longer be funded.
More information would be available during the transition period from pharmacists, prescribers and PHARMAC’s website.
For community pharmacies
From 1 August 2018, a small number of community pharmacies dispensing imiglucerase would need to liaise with treating clinicians and patients to support patients switching to taliglucerase alfa and manage stock of both products during the transition. PHARMAC would directly contact relevant pharmacies involved in dispensing ERT, to provide transition guidance, prior to August 2018.
For hospital pharmacies
From 1 August 2018, taliglucerase alfa would be funded, when dispensed to patients with a valid Special Authority approved by the Gaucher Panel, for use in hospital or the community. Reimbursement claims could be made (electronically or manually) by pharmacies with a community pharmacy contract. PHARMAC would directly contact hospital pharmacies with existing patients to provide transition guidance prior to August 2018.
Between 1 August 2018 and 1 March 2019, treating clinicians would need to assess and transition all existing patients receiving funded ERT for the treatment of Gaucher disease to taliglucerase alfa.
PHARMAC would directly communicate with relevant clinicians and support services involved in the care of these patients regarding the change process. We will be asking these clinicians what support and resources are required during the consultation process.
Guidelines to prescribers to support the change to taliglucerase alfa would be provided by the Gaucher Panel, as well as recommendations in relation to individual patients about the change, increased dosing (if applicable), and monitoring.
- Treating clinicians would need to organise patients who currently manage their infusions at home to attend hospital outpatient clinics, for the first 2-4 infusions of the new treatment. Pharmacies and community nursing support would also need to be aware of these arrangements.
- The Gaucher Panel would remain in place and consider both initial applications for new patients commencing taliglucerase treatment, and renewal applications for existing patients.
- Some patients may require additional assessment (including an MRI) prior to the proposed change.
- The Gaucher Panel patient application forms would be updated, to make it easier and clearer for clinicians to provide the information required.
- Some patients and their families may need extra support with this change. The supplier would provide a dedicated infusion nurse to provide personal support during the transition.
- As noted above, if a patient does not tolerate the new treatment for a clinical reason, PHARMAC would consider a NPPA application from a clinician to reinstate imiglucerase for an individual patient, following advice from the Gaucher Panel. Dosing would be advised by the Gaucher Panel.
The proposal would improve access to a high cost treatment for a rare condition, while managing pharmaceutical costs. We anticipate there would be some small additional DHB resource required to support the transition in treatment for the 20 patients receiving ERT. The supplier would provide a dedicated infusion nurse to provide support for healthcare professionals and patients during the transition.
Who we think will be interested
- People and their families currently receiving imiglucerase (Cerezyme) for the treatment of Gaucher disease.
- Rare disorder-related consumer support organisations.
- Clinicians and nurses involved in the management of Gaucher disease.
- Pharmacists, DHBs and suppliers of ERT for the treatment of Gaucher disease.
About Gaucher disease and taliglucerase alfa
Gaucher disease is a rare inherited lysosomal storage disorder (lipid storage disease) resulting from the shortage of an enzyme that leads to accumulation of a type of fat. Gaucher cells with fatty deposits typically build up in different parts of the body, primarily the liver, spleen and bone marrow. It can cause a wide variety of symptoms, relating to spleen and liver enlargement, anaemia, bone involvement, and many other signs and symptoms. These can include fatigue, easy bruising and a tendency to bleed, bone pain, degeneration and fractures. In rare cases, the brain and nervous system are affected.
The severity varies widely; some patients present in childhood with almost all the complications of Gaucher disease while others have no symptoms. Gaucher disease has traditionally been divided into 3 clinical subtypes; however, some cases do not fit precisely into one category.
- Type 1 – the most common, no neurologic involvement, primarily adults.
- Type 2 – the most severe neuronopathic type, generally fatal by age 2.
- Type 3 – affects children, characterised by sub-acute neurological symptoms such as seizures, cognitive impairment, progressive encephalopathy.
Treatment of Gaucher disease is tailored to the individual, because of how variably the disease manifests and progresses. Treatment goals are elimination or improvement of symptoms, prevention of irreversible complications and improvement in overall health and quality of life. In children, growth is an additional goal.
In New Zealand, 20 patients with type 1 or type 3 Gaucher disease currently receive funded ERT with imiglucerase. Funding applications are managed by the Gaucher Panel, which is a panel of expert doctors. Patients with type 2 Gaucher disease are not eligible for funded ERT, as ERT has little or no effect on neurological symptoms.
Currently, imiglucerase is funded for patients with Gaucher disease type 1 or Gaucher disease type 3* who the Gaucher Panel consider meet the eligibility criteria. It is given as an intravenous infusion usually every two weeks at the following doses:
- a maximum monthly dose of 15 international units (IU)/kg/month;
- a maximum monthly dose of 30 IU/kg/month for certain children; and
- PHARMAC Board (or delegated authority) approval is required for doses greater than 30 IU/kg/month.
All patients’ clinical information and doses are reviewed annually by the Gaucher Panel, with some reviewed six monthly. The Panel also determines the dose and the number of vials approved for each patient based on weight.
More background information on Gaucher disease treatment in New Zealand is provided in the RFP document discussed below.
Taliglucerase alfa – new proposed treatment
Taliglucerase alfa is indicated for long-term ERT for adults and children with a confirmed diagnosis of Gaucher disease type 1 associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopaenia (ie enlarged spleen or liver, insufficient red blood cells or platelets). Use in type 3 Gaucher disease is not an approved indication in New Zealand, as is the case for the current funded product imiglucerase.
Taliglucerase alfa dosing in adult and paediatric patients ranges from 30 units/kg to 60 units/kg of body weight every other week (every 2 weeks). For more information see the Medsafe datasheet(external link).
Taliglucerase alfa is available as a 200 unit vial of powder, requiring reconstitution with sterile water for injection and dilution with sodium chloride. This smaller vial size may allow for more efficient dose rounding for patients.
Taliglucerase alfa is used in many countries for the treatment of Gaucher disease, including Australia, Canada, US and some European countries.
Why we’re proposing this
As a result of the RFP, PHARMAC has entered into a provisional agreement with Pfizer New Zealand Limited for the supply of taliglucerase alfa, and feedback to this consultation will help us to decide if this agreement should be confirmed.
Reasons for running the RFP
New suppliers of ERT with increased interest in the New Zealand market created an opportunity for PHARMAC achieve savings and also consider widening access to allow increased doses of ERT for patients with Gaucher disease.
In November 2017 both the Gaucher Panel and PTAC (PHARMAC’s clinical advisory body, the Pharmacology and Therapeutics Advisory Committee) reviewed a funding application for taliglucerase alfa. Minutes of the November PTAC meeting are available here. [PDF, 764 KB]
PTAC and the Gaucher Panel considered that taliglucerase alfa would be a suitable alternative ERT for all Gaucher patients currently on treatment in New Zealand, and endorsed the proposed transition approach. PTAC recommended that, based on the proposed commercial arrangements, the Gaucher Panel should be able to determine the dosing of all Gaucher patients up to a maximum of 30 U/kg/every other week (or 60 U/kg/month) of taliglucerase alfa.
There is clinical evidence and also local data from Australia to support switching to taliglucerase alfa from imiglucerase. Evidence signals efficacy, safety and tolerability of taliglucerase alfa in ERT-naïve patients and in patients switched from imiglucerase. Refer to the PTAC minutes for more detail.
This proposal would increase the funded dose of ERT available for patients with Gaucher disease which could provide some additional clinical benefit, through a commercially favourable agreement for this high cost treatment for a rare disorder.
Details about the our proposal
Listing of taliglucerase alfa (Elelyso)
From 1 August 2018, Pfizer’s brand of taliglucerase alfa (Elelyso) would be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List) of the Pharmaceutical Schedule as follows:
|Taliglucerase alfa||Inj 200 units vial||Elelyso||1|
Special Authority restrictions (applied by the Gaucher Panel) for taliglucerase alfa would be similar to current criteria for imiglucerase, except for the following:
- The maximum dose of ERT for all patients (adults and children, type 1 and 3) would be amended to allow, if approved by the Gaucher Panel, an increase to a maximum dose of 30 units/kg every other week (approx. 60 units/kg/month) where clinically appropriate.
- Hospital restrictions would limit use to patients with a Special Authority approved by the Gaucher Panel.
- The eligibility criteria and new application forms would be updated in consultation with the Gaucher Panel and prescribers, to reflect the proposed changes in treatment, dosing and access criteria and to make it easier for clinicians to apply. A new Special Authority approval number for taliglucerase alfa would be issued for approved patients. The criteria would be:
Special Authority – Retail pharmacy
Initial application from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- The patient has a diagnosis of symptomatic type 1 or type 3* Gaucher disease confirmed by the demonstration of specific deficiency of glucocerebrosidase in leukocytes or cultured skin fibroblasts, and genotypic analysis; and
- Patient does not have another life-threatening or severe disease where the prognosis is unlikely to be influenced by taliglcuerase alfa or might be reasonably expected to compromise a response to therapy with taliglcuerase alfa; and
- Taliglucerase alfa is to be administered at a dose no greater than 30 unit/kg every other week rounded to the nearest whole vial (200 units), unless otherwise agreed by PHARMAC; and
- Supporting clinical information including test reports, MRI whole body STIR, serum glucosylsphingosine, haematological data, and other relevant investigations, are submitted to the Gaucher Panel for assessment; and
- Any of the following:
5.1 Patient has haematological complications such as haemoglobin less than 95 g/l, symptomatic anaemia, thrombocytopenia; at least two episodes of severely symptomatic splenic infarcts confirmed with imagery; or massive symptomatic splenomegaly; or
5.2 Patient has skeletal complications such as acute bone crisis requiring hospitalisation or major pain management strategies; radiological MRI Evidence of incipient destruction of any major joint (e.g. hips or shoulder); spontaneous fractures or vertebral collapse; chronic bone pain not controlled by other pharmaceuticals; or
5.3 Patient has significant liver dysfunction or hepatomegaly attributable to Gaucher disease; or
5.4 Patient has reduced vital capacity from clinically significant or progressive pulmonary disease due to Gaucher disease; or
5.5 Patient is a child and has experienced growth failure with significant decrease in percentile linear growth over a 6-12 month period.
Renewal from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- Patient has demonstrated a symptomatic improvement or no deterioration in the main symptom for which therapy was initiated; and
- Patient has demonstrated a clinically objective improvement or no deterioration in haemoglobin levels, platelet counts and liver and spleen size; and
- Radiological (MRI) signs of bone activity performed at one year and two years since initiation of treatment begins, and two to three yearly thereafter, demonstrate no deterioration shown by the MRI, compared with MRI taken immediately prior to commencement of therapy or adjusted dose; and
- Serum glucosylsphingosine levels taken at least 6 to 12 monthly show a decrease compared with baseline; and
- Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication and/or adjustment of infusion rates; and
- Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT; and
- Patient is compliant with regular treatment and taliglucerase alfa is to be administered at a dose no greater than 30 unit/kg every other week rounded to the nearest whole vial (200 units), unless otherwise agreed by PHARMAC; and
- Supporting clinical information including test reports, MRI whole body STIR, serum glucosylsphingosine, haematological data, and other relevant investigations are submitted to the Gaucher Panel for assessment as required.
The Gaucher Panel would remain in place, at least for the next few years. PHARMAC may consider moving the application process to a standard Special Authority in the future.
DHB hospitals would be able to claim use of taliglucerase alfa via the Combined Pharmaceutical Budget, as is currently the case for imiglucerase.
The price and subsidy would be notified should PHARMAC decide to progress the proposal following consideration of consultation feedback. A confidential rebate would apply which would reduce the net price to the Funder.
Taliglucerase alfa Sole Supply
From 1 March 2019 until 30 June 2023, taliglucerase alfa (Elelyso) would be awarded Sole Supply Status in the community for ERT for the treatment of Gaucher disease in the community. This would result in taliglucerase alfa (Elelyso) being the only funded ERT for Gaucher disease in the community during this time. PHARMAC also reserves the right to extend the sole supply period for a further two years.
From 1 March 2019, the other funded ERT for Gaucher disease (imiglucerase, Cerezyme) would be delisted from Section B of the Pharmaceutical Schedule.
A Brand Switch Fee would be able to be claimed on the first taliglucerase alfa dispensing by community pharmacies, per patient, between 1 March 2019 to 31 May 2019 (i.e. after the delisting of imiglucerase).
To provide feedback
Send us an email: firstname.lastname@example.org by 4pm on Monday 11 June 2018.
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.
Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.
We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.
Last updated: 31 October 2019