Decision to move from Panel application to Special Authority process for enzyme replacement therapy for Gaucher disease

15 June 2022

What we’re doing

We are pleased to announce a decision to transition the process for managing funded access to enzyme replacement therapy (ERT) from the Gaucher Panel application process to a standard Special Authority from 1 July 2022. We anticipate that this change will provide a more streamlined way for clinicians to apply for funded access to ERT for their patients.

What this decision means

The changes to managing access to ERT for Gaucher disease will provide a more streamlined way for clinicians to apply for funding for their patients. Key benefits include:

Patients with a current approval for either taliglucerase alfa or imiglucerase will continue to receive funded access to ERT.
Reducing barriers by moving to a standard Special Authority, providing a simpler way for clinicians to apply for funded ERT for their patients without the need for Gaucher Panel assessment.
Increasing the renewal period from 12 months to three years to reduce the administrative burden on clinicians and provide greater certainty for patients.
Dosing equity with harmonised dosing regimens between patients on taliglucerase alfa and those who have remained on imiglucerase.

Taliglucerase alfa (Elelyso)

From 1 July 2022, funded access to taliglucerase alfa (Elelyso) will be managed through standard Special Authority and hospital restriction processes. Metabolic physicians can submit a Special Authority application for new patients without a requirement for review by the Gaucher Panel.

Patients with a current approval for taliglucerase alfa will be automatically issued a new initial approval on 1 July 2022. This will allow treating clinicians to apply for a renewal approval from 1 July 2022 without the requirement for assessment by the Gaucher Panel.

We have also made changes to the eligibility criteria for taliglucerase alfa, including extending the renewal period to three years and removing some detailed clinical information.

Imiglucerase (Cerezyme)

From 1 July 2022, funded access to imiglucerase will be transitioned from the Gaucher Panel to the Exceptional Circumstances Framework. At the same time, patients will be able to receive imiglucerase at a higher maximum dose of 30 units/kg each fortnight (ie every other week), as determined by their prescribing clinician.

Existing patients

For the small group of patients who are currently receiving funded access to imiglucerase, we will create a simple pathway for renewal applications via the Exceptional Circumstances Framework. This will enable approval if patients meet renewal criteria that are consistent with taliglucerase alfa. Similar pathways have been used for other therapeutics.

New patients

Any new initial applications for imiglucerase will be assessed using the Named Patient Pharmaceutical Assessment (NPPA) Policy. Applications will require supporting clinical information to demonstrate why the patient is unable to receive treatment with taliglucerase alfa.

Who we think will be interested

People currently receiving ERT for the treatment of Gaucher disease, and their whānau
Rare disorder 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

Any changes to the proposal

This decision was subject to a consultation letter dated 23 May 2022. We’re really grateful for the time people took to respond to this consultation. There have been no changes as a result of consultation feedback.

A summary of the main themes raised in feedback and our responses to them are summarised below.

Details about the decision

Taliglucerase alfa (Elelyso)

From 1 July 2022, applications for taliglucerase alfa will be made by standard Special Authority. Initial applications for taliglucerase alfa will be limited to metabolic physicians. Renewal applications can made by relevant practitioners working within their scope and on the recommendation of a metabolic physician.

Taliglucerase alfa access criteria will be amended in Section B and Part II of Section H of the Pharmaceutical Schedule as follows (additions in bold, deletions in strikethrough):

Special Authority for Subsidy

Special Authority approved by the Gaucher Treatment Panel Notes: Application details may be obtained from Pharmac's website schedule.pharmac.govt.nz/SAForms or: The Co-ordinator, Gaucher Treatment Panel Phone: 04 460 4990 Pharmac PO Box 10 254 Facsimile: 04 916 7571 Wellington Email: gaucherpanel@pharmac.govt.nz Completed application forms must be sent to the coordinator for the Gaucher Treatment Panel and will be considered by the Gaucher Treatment Panel at the next practicable opportunity. Notification of the 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 only from a metabolic physician. 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 enzyme replacement therapy (ERT) or the disease might be reasonably expected to compromise a response to ERT; and
Any of the following:
- Patient has haematological complications of Gaucher disease such as haemoglobin less than 95 g/l, symptomatic anaemia, thrombocytopenia; at least two episodes of severely symptomatic splenic infarcts confirmed with imagery; or
- Patient has skeletal complications of Gaucher disease 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; 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

Note: Indication marked with * is an unapproved indication.

Renewal – from a metabolic physician or any relevant practitioner on the recommendation of a metabolic physician. Approvals valid for 12 months 3 years for applications meeting the following criteria:

All of the following:

Patient has demonstrated a symptomatic improvement and has maintained improvements in the main symptom or symptoms for which therapy was started; 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 two years since initiation of treatment, and five yearly thereafter, demonstrate no deterioration shown by the MRI, compared with MRI taken immediately prior to commencement of therapy or adjusted dose; and
Patient has not had severe infusion-related adverse reactions that 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 adherent 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).

Following an agreement with Pfizer, the Sole Subsidised Supply status in the community for taliglucerase alfa (Elelyso) will be extended until 30 June 2025. This means that taliglucerase alfa (Elelyso) will be the only ERT for Gaucher disease Iisted on the Pharmaceutical Schedule for this period. An alternative brand allowance will remain in place. The mechanism for DHB hospitals to access ERT for patients with Gaucher disease will remain unchanged.

Imiglucerase (Cerezyme)

Imiglucerase (Cerezyme) will be funded through Pharmac’s Exceptional Circumstances Framework for patients who are unable to tolerate taliglucerase alfa due to clinical reasons at the following price and subsidy (ex-manufacturer, excluding-GST) from 1 July 2022:

Chemical	Formulation	Strength	Brand	Pack size	Price and subsidy
Imiglucerase	Powder for infusion	40U/ml 400U/vial	Cerezyme	1 vial	$2144.00

Imiglucerase will not be listed in the Pharmaceutical Schedule. As a part of an agreement with Sanofi-Genzyme, the net price of imiglucerase will be reduced via a confidential rebate from 1 July 2022. There will be protection against subsidy reduction until 1 July 2025.

Existing patients will be able to apply for renewal against criteria that have been developed in consultation with the Gaucher Panel and that are consistent with taliglucerase alfa (above). We will develop a simple renewal form and make this available on the Pharmac website(external link) from July 2022. New applications will be assessed on a case-by-case basis under the NPPA policy.

From 1 July, there will be an increase to the maximum funded dose of imiglucerase to 30 units/kg each fortnight (ie. every other week).

Our response to what you told us

We’re really grateful for the time people took to respond to consultation. A summary of the main themes raised in feedback and our responses to the feedback received are set out below.

Theme	Pharmac Comment
Strong support for the proposed increase to the maximum funded dose of imiglucerase to 30 units/kg every other week so that patients who were unable to transition to taliglucerase alfa have access to an equitable dosing regimen. Patient and whānau members shared their experiences and view that current dosing is inequitable.	We are pleased to receive supportive feedback for increasing the maximum funded dose of imiglucerase so that it is consistent with funded taliglucerase alfa dosing and international guidelines.
Pharmac should retain the discretion to approve a funded dose above 30 units/kg every other week and that this should continue to be reflected in the Special Authority criteria.	We appreciate that there may be cases where patients could benefit from a higher dose of taliglucerase alfa. Pharmac will continue to have the discretion to fund dosing regimens above the maximum funded dose via the Exceptional Circumstances Framework. The change to the criteria reflects that this will be considered via a different process to standard Special Authority applications.
The dosing restrictions for both imiglucerase and taliglucerase alfa should be increased further or removed altogether.	We include a maximum funded dose to help manage the costs associated with the use of these medicines within a fixed budget. The funded dose aligns with Medsafe approved dosing for taliglucerase alfa and imiglucerase. We acknowledge that there may be some clinical circumstances where an individual may benefit from a higher dose of enzyme replacement therapy, and we would continue to consider any applications for increased dosing via the Exceptional Circumstances Framework. Any proposal to further increase the maximum funded dose of both therapies would need to be considered separately.
Enable patients experiencing side-effects from taliglucerase alfa to transition to imiglucerase.	Taliglucerase alfa will continue to have sole subsidised supply status until 30 June 2025. However, we understand that this treatment may not be appropriate for all patients. We will continue to consider applications for funded access to imiglucerase via our Exceptional Circumstances Framework for patients who are unable to tolerate taliglucerase for clinical reasons. An application from the prescribing clinician will need to include information to demonstrate why the patient is unable to clinically tolerate taliglucerase, and any attempts at desensitisation.

Theme

Pharmac Comment

Strong support for the proposed increase to the maximum funded dose of imiglucerase to 30 units/kg every other week so that patients who were unable to transition to taliglucerase alfa have access to an equitable dosing regimen.

Patient and whānau members shared their experiences and view that current dosing is inequitable.

We are pleased to receive supportive feedback for increasing the maximum funded dose of imiglucerase so that it is consistent with funded taliglucerase alfa dosing and international guidelines.

Pharmac should retain the discretion to approve a funded dose above 30 units/kg every other week and that this should continue to be reflected in the Special Authority criteria.

We appreciate that there may be cases where patients could benefit from a higher dose of taliglucerase alfa.

Pharmac will continue to have the discretion to fund dosing regimens above the maximum funded dose via the Exceptional Circumstances Framework. The change to the criteria reflects that this will be considered via a different process to standard Special Authority applications.

The dosing restrictions for both imiglucerase and taliglucerase alfa should be increased further or removed altogether.

We include a maximum funded dose to help manage the costs associated with the use of these medicines within a fixed budget. The funded dose aligns with Medsafe approved dosing for taliglucerase alfa and imiglucerase.

We acknowledge that there may be some clinical circumstances where an individual may benefit from a higher dose of enzyme replacement therapy, and we would continue to consider any applications for increased dosing via the Exceptional Circumstances Framework.

Any proposal to further increase the maximum funded dose of both therapies would need to be considered separately.

Enable patients experiencing side-effects from taliglucerase alfa to transition to imiglucerase.

Taliglucerase alfa will continue to have sole subsidised supply status until 30 June 2025. However, we understand that this treatment may not be appropriate for all patients. We will continue to consider applications for funded access to imiglucerase via our Exceptional Circumstances Framework for patients who are unable to tolerate taliglucerase for clinical reasons.

An application from the prescribing clinician will need to include information to demonstrate why the patient is unable to clinically tolerate taliglucerase, and any attempts at desensitisation.

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.