Decision to widen access and change the funded enzyme replacement therapy for Gaucher disease
6 July 2018
What we’re doing
We’re pleased to announce the following changes to the funding of enzyme replacement therapy (ERT) used in the treatment of the rare disorder Gaucher disease:
- The funded ERT for the treatment of Gaucher disease will change from imiglucerase (Cerezyme), supplied by Sanofi, to taliglucerase alfa (Elelyso), supplied by Pfizer.
- Taliglucerase alfa will be funded from 1 August 2018 and will become the only funded ERT for the treatment of Gaucher disease after a 7-month transition period.
- Special Authority and Hospital Restrictions will apply to taliglucerase alfa. If approved by the Gaucher Panel, the criteria allow for a higher maximum dose of 30 units/kg every other week (~60 units/kg/month) where clinically appropriate. This may provide additional clinical benefits for some patients.
- Transition timeframes and further information are detailed below.
This decision results from a Request for Proposals (RFP) for the supply of a first line ERT for the treatment of Gaucher disease.
Any changes to the original proposal?
This decision was subject to a consultation letter dated 17 May 2018. No changes were made to the original proposal.
Who we think will be most interested
- People currently receiving imiglucerase (Cerezyme) for the treatment of Gaucher disease, and their families.
- 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.
Detail about this decision
Listing of taliglucerase alfa (Elelyso)
From 1 August 2018, Pfizer’s brand of taliglucerase alfa (Elelyso) will be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List) of the Pharmaceutical Schedule as follows:
|Chemical||Presentation||Brand||Pack size||Price & Subsidy|
|Taliglucerase alfa||Inj 200 units vial||Elelyso||1||$1,072.00|
A confidential rebate will apply which will reduce the net price to the Funder.
From 1 March 2019 until 30 June 2023, taliglucerase alfa (Elelyso) will have Sole Subsidised Supply status in the community for ERT for the treatment of Gaucher disease in the community. This means that taliglucerase alfa (Elelyso) will be 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.
The mechanism for DHB hospitals to access ERT for patients with Gaucher disease remains unchanged.
Access criteria & the Gaucher Treatment Panel
Special Authority restrictions (applied by the Gaucher Treatment Panel) for taliglucerase alfa will be similar to current criteria for imiglucerase, except for the following:
- If approved by the Gaucher Treatment Panel, the maximum dose of ERT for all patients (adults and children, type 1 and 3) will be 30 units/kg every other week (~60 units/kg/month) where clinically appropriate. We expect that all patients will receive this higher dose.
- Hospital restrictions will limit use to patients with a Special Authority approved by the Gaucher Treatment Panel.
- The eligibility criteria and new application forms have been updated in consultation with the Gaucher Treatment Panel and prescribers, to reflect the changes in treatment, dosing and access criteria and to make it easier for clinicians to apply. Updated forms will be on the PHARMAC website before 1 August 2018.
- The Gaucher Treatment Panel will remain in place for now. PHARMAC may consider moving the application process to a standard Special Authority in the future.
The criteria will be as follows:
Special Authority for subsidy
Special Authority approved by the Gaucher Treatment Panel
Application details may be obtained from PHARMAC’s website or:
The Co-ordinator, Gaucher Treatment Panel
PHARMAC, PO Box 10 254
Phone: (04) 460 4990
Facsimile: (04) 916 7571
Completed application forms must be sent to the coordinator for the Gaucher Treatment Panel and will be considered by Gaucher Treatment Panel at the next practicable opportunity.
Notification of Gaucher Treatment Panel’s decision will be sent to the patient, the applying clinician and the patient's GP (if specified).
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 taliglucerase alfa or might be reasonably expected to compromise a response to therapy with taliglucerase 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:
- 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
- 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
- Patient has significant liver dysfunction or hepatomegaly attributable to Gaucher disease; or
- Patient has reduced vital capacity from clinically significant or progressive pulmonary disease due to Gaucher disease; or
- 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.
- There will be a 7-month transition period from 1 August 2018 until 28 February 2019. This is longer than usual, to accommodate for the scheduling of patients to attend hospital clinics that may fall over the Christmas holiday period.
- A new Special Authority approval number for taliglucerase alfa will be issued for patients with an approval number for imiglucerase prior to 1 August 2018.
- Approvals for imiglucerase that are due to expire in November 2018 will be extended to the end of the transition period at the end of February 2018. Criteria that currently apply to imiglucerase will remain in place until this time.
- Renewal applications for patients will be required for taliglucerase alfa in early 2019 for assessment by the Gaucher Panel in February 2019.
- From 1 March 2019, imiglucerase (Cerezyme) inj 40 iu per ml, 200 iu and 400 iu vials will be delisted from Section B and Part II of Section H of the Pharmaceutical Schedule.
- A Brand Switch Fee will be able to be claimed on the first taliglucerase alfa dispensing by community pharmacies, one per patient, between 1 March 2019 to 31 May 2019 (i.e. after the delisting of imiglucerase).
What will the effect of this decision be?
From 1 August 2018, eligible people with Gaucher disease (type 1 and 3) will have funded access to a new enzyme replacement therapy (ERT), taliglucerase alfa (Elelyso).
- During a transition period, from 1 August 2018 to 28 February 2019, all current patients receiving funded ERT for Gaucher disease will need to change to taliglucerase alfa to continue to receive a funded treatment. This change will be carefully supported by the treating clinician, working with the patient and family/caregivers. PHARMAC will directly communicate with relevant clinicians, support services and DHB services regarding the change process.
- Patients will be able to 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. We expect all patients will receive this higher dose.
- Prior to changing treatments, a small number of patients may require an MRI scan if they have not received one in the previous 18 months.
- Patients who currently manage their infusions at home (~10 patients) will need to attend hospital outpatient clinics for the first 2-4 infusions of the new treatment. This will be led and organised by the treating clinician. Following this, patients can continue to manage infusions of the new treatment at home. Support will 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.
More information would be available during the transition period from pharmacists, prescribers and PHARMAC’s website.
Between 1 August 2018 and 1 March 2019, treating clinicians will need to assess and transition all existing patients receiving funded ERT for the treatment of Gaucher disease to taliglucerase alfa.
PHARMAC will directly communicate with relevant clinicians and support services involved in the care of these patients regarding the change process.
Guidelines to prescribers to support the change to taliglucerase alfa will be provided by the Gaucher Panel, as well as recommendations in relation to individual patients about the change, increased dosing where applicable, and monitoring. A dosing table will also be provided to assist clinicians with determining the number of vials required.
For community pharmacies
From 1 August 2018, a small number of community pharmacies dispensing imiglucerase will 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 will directly contact relevant pharmacies involved in dispensing ERT, to provide transition guidance prior to 1 August 2018.
For hospital pharmacies
From 1 August 2018, taliglucerase alfa will 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 can be made (electronically or manually) by pharmacies with a community pharmacy contract. PHARMAC will directly contact hospital pharmacies with existing patients to provide transition guidance prior to August 2018.
Our response to what you told us
We’re really grateful for the time people took to respond to this consultation. A summary of the main themes raised in feedback, our responses to the feedback received, and changes we have made after listening to you are available on our notification webpage.
If you have any questions about this decision, you can email us at email@example.com; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.
Responders were supportive of the proposal, including the proposed transition plans and the proposed Brand Switch Fee.
Information from an experienced Australian clinician noted that taliglucerase alfa is likely more immunogenic that the other ERTs and may have more bone disease efficacy.
Our clinical advice from the Gaucher Treatment Panel notes the potential for increased immunogenic reactions. Transition guidance will indicate what additional monitoring may be required. Overall, we consider that the information provided by the responder comparing the different treatments for Gaucher disease, and advice from our clinical advisors, supports the change to taliglucerase alfa.
Responders shared learnings from Australian switch in ERT for Gaucher disease and use of taliglucerase alfa, including the support provided to patients and clinicians. They noted that a standard letter to patients from the treating clinician was helpful, alleviated concerns of patients and families about changing treatment.
PHARMAC is providing resources to treating clinicians, including a letter to patients, to support the change process.
Impacts of the change on DHBs, including the potential increase in eligible patients and dose changes, are unclear.
The changes are unlikely to result in more patients being eligible for treatment with ERT.
As per Gaucher Panel advice, all patients are expected to receive the increased dose of taliglucerase alfa of 30 U/kg/every other week. This will be communicated to treating clinicians with additional advice for individual patients.
Some patients may require an MRI earlier than otherwise expected; clinicians were notified of MRI requirements for 2018, including those related to a potential change in ERT, in January this year.
Imiglucerase (Cerezyme) is now Medsafe approved for type 3 Gaucher Disease indication (updated 31 May 2018), whereas taliglucerase alfa is not indicated for type 3 Gaucher disease.
We acknowledge that the Medsafe approval status for imiglucerase (the currently funded ERT) has changed since consultation on the proposal was released on 17 May 2018.
However, we note that patients with type 3 Gaucher disease have been receiving funded ERT with imiglucerase for many years despite it then being an unregistered indication and this will also be possible for taliglucerase alfa regardless of registration status. Our clinical advice supports the use of taliglucerase alfa in type 1 and type 3 Gaucher disease.
Last updated: 7 January 2019