Proposal to move funding for Gaucher disease treatments from Panel application to Special Authority

Medicines Consultation Closed

What we’re proposing

We are seeking feedback on a proposal to transition the process for managing funded access to enzyme replacement therapy (ERT) for the treatment of Gaucher disease. This proposal would mean a move from the Gaucher Panel application process to a standard Special Authority from 1 July 2022. We anticipate that this change would provide a more streamlined way for clinicians to apply for funded access to ERT for their patients. 

As a part of this proposal, we would also increase the funded maximum dose of imiglucerase (which will be accessed via the Exceptional Circumstances Framework) for a small group of patients with Gaucher disease who are not able to use taliglucerase alfa for clinical reasons.  

We welcome feedback on this proposal. Consultation closes at 5 pm Tuesday 7 June 2022 and feedback can be emailed to gaucher@pharmac.govt.nz. 

Further details on this proposal can be found below.

What would the effect be?

Patients with a current approval for either taliglucerase alfa or imiglucerase would continue to receive funded access to ERT, however, we consider that the proposed changes to managing access to ERT for Gaucher disease would provide a more streamlined way for clinicians to apply for funding for their patients.

Access to ERT for 21 patients is currently managed by the Gaucher Panel, a panel of expert clinicians who assess funding applications for ERT against a set of eligibility criteria(external link).   

Taliglucerase alfa (Elelyso)

From 1 July 2022, funded access to taliglucerase alfa (Elelyso) would be managed through standard Special Authority and hospital restriction processes. Metabolic physicians would be able to apply for initial applications for new patients through the online Special Authority system without the requirement for review by the Gaucher Panel. 

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

We are also proposing a few changes to the eligibility criteria in consultation with the Gaucher Panel. These include an extended three-year renewal period and the removal of detailed clinical information no longer considered necessary. 

Imiglucerase (Cerezyme)

From 1 July 2022, the management of funded access to imiglucerase would be transitioned from the Gaucher Panel to the Exceptional Circumstances Framework. At the same time, patients would be able to receive imiglucerase at a higher maximum dose of 30 units/kg 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 would create a simple pathway for renewal applications via exceptional circumstances that would enable streamlined approval should patients meet renewal criteria that are consistent with taliglucerase alfa. 

New patients

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

Who we think will be interested

  • People and their families currently receiving ERT for the treatment of Gaucher disease
  • 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 

About Gaucher disease

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, and bone pain, degeneration and fractures. In rare or severe 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 to eliminate or improve symptoms, prevent irreversible complications and to improve overall health and quality of life. In children, growth is an additional goal. 

Background to the proposal

ERT has been funded since 1997 with all applications for funding being assessed by the Gaucher Panel. This included assessment for dosing regimens. 

Following a Request for Proposals (RFP) process in 2018 for the sole subsidised supply of ERT, eligible patients with Gaucher disease (types 1 and 3*) were enabled funded access to taliglucerase alfa (Elelyso) at a higher maximum dose of 30 units/kg every other week, where clinically appropriate and approved by the Gaucher Panel. At that time, Pharmac indicated that the Gaucher Panel would remain in place for the next few years and that we may consider moving the application process to a standard Special Authority process in the future. 

There are currently 21 eligible patients for ERT. Most now receive taliglucerase alfa, while a small number have remained on imiglucerase due to clinical reasons, at their previous dosing levels. 

* Use in type 3 Gaucher disease is not an approved indication in New Zealand.

Why we’re proposing this

We are proposing to transition access to taliglucerase alfa to a standard Special Authority to provide a simpler, more streamlined way for clinicians to apply for funded ERT for their patients. We are also proposing to increase the renewal period from 12 months to three years to reduce the administrative burden on clinicians.

Furthermore, we consider that the small group of patients requiring treatment with imiglucerase would no longer require ongoing assessment through the Gaucher Panel and that access for existing patients could be managed via an exceptional circumstances pathway with renewal criteria consistent with taliglucerase alfa.   

Details about our proposal

Taliglucerase alfa (Elelyso)

From 1 July applications for taliglucerase alfa would be made by standard Special Authority. Initial applications for taliglucerase alfa would be limited to metabolic physicians, while renewal applications would be able to be made by relevant practitioners working within their vocational scope and on the recommendation of a metabolic physician. 

Taliglucerase alfa access criteria would 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:

  1. 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
  2. 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
  3. Any of the following:
    1. 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
    2. 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
    3. Patient has significant liver dysfunction or hepatomegaly attributable to Gaucher disease; or
    4. Patient has reduced vital capacity from clinically significant or progressive pulmonary disease due to Gaucher disease; or
    5. Patient is a child and has experienced growth failure with significant decrease in percentile linear growth over a 6-12 month period; and
  4. 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:

  1. Patient has demonstrated a symptomatic improvement and has maintained improvements in the main symptom or symptoms for which therapy was started; and
  2. Patient has demonstrated a clinically objective improvement or no deterioration in haemoglobin levels, platelet counts and liver and spleen size; and
  3. 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
  4. Patient has not had severe infusion-related adverse reactions that were not preventable by appropriate pre-medication and/or adjustment of infusion rates; and
  5. Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT; and
  6. 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).

Subject to a provisional agreement with Pfizer, the Sole Subsidised Supply status in the community for taliglucerase alfa (Elelyso) would 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 would remain in place. The mechanism for DHB hospitals to access ERT for patients with Gaucher disease would remain unchanged. 

Imiglucerase (Cerezyme)

We propose that imiglucerase (Cerezyme) would be funded for patients who are unable to tolerate taliglucerase alfa due to clinical reasons through Pharmac’s Exceptional Circumstances Framework 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 would not be listed in the Pharmaceutical Schedule. As a part of a provisional agreement with Sanofi-Genzyme, the net price of imiglucerase would be reduced via a confidential rebate from 1 July 2022 and there would be protection against subsidy reduction until 1 July 2025. 

Existing patients would be able to apply for renewal against criteria that have developed in consultation with the Gaucher Panel and are consistent with taliglucerase alfa (above). A simple renewal form would be developed and made available on the Pharmac website. New applications would be assessed on a case-by-case basis against the NPPA policy. 

As a part of this proposal, there would be an increase to the maximum funded dose of imiglucerase to 30 units/kg every other week, thereby enabling the harmonisation of dosing regimens between patients who were successfully transitioned to taliglucerase alfa and those who remain on imiglucerase. 

To provide feedback

Send us an email: gaucher@pharmac.govt.nz by 7 June 2022. 

All feedback received before the closing date will be considered by Pharmac Board (or its delegate) prior to making a decision on this proposal.

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