Decision to list galsulfase (Naglazyme) for mucopolysaccharidosis (MPS) VI

Medicines

Decision

PHARMAC is pleased to announce the approval of an agreement with BioMarin Pharmaceutical Australia Pty Ltd to list galsulfase (Naglazyme) for people with MPS VI in the Pharmaceutical Schedule.

This was the subject of a consultation letter dated 3 February 2016, available on PHARMAC’s website.

No changes were made to the proposal following consultation. In summary, the effect of the decision is that galsulfase (Naglazyme) will be funded in the community under Special Authority criteria and in DHB hospitals subject to restrictions. Listing in the Pharmaceutical Schedule would occur subject to Medsafe approval of the pharmaceutical.

Galsulfase is the second medicine funded as a result of the Medicines for Rare Disorders Request for Proposals process.

Details of the decision

Following Medsafe approval, galsulfase (Naglazyme) 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 and subsidy

Galsulfase

Inj 1 mg per ml, 5 ml vial

Naglazyme

1

$2,234.00

  • A confidential rebate will apply to Naglazyme, which will reduce the net price of the treatment to the Funder.
  • Naglazyme will be listed as soon as practicable following BioMarin’s notification to PHARMAC that Medsafe has granted registration.
  • Naglazyme will be listed subject to the following Special Authority criteria in the community and equivalent restrictions in Section H (the Hospital Medicines List):

Special Authority for Subsidy

Initial application only from a metabolic physician.  Approvals valid for 12 months for patients meeting the following criteria:

Both:

Renewal only from a metabolic physician. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. The treatment remains appropriate for the patient and the patient is benefiting from treatment; and
  2. Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication and/or adjustment of infusion rates; and
  3. Patient has not developed another life threatening or severe disease where the long term prognosis is unlikely to be influenced by Enzyme Replacement Therapy (ERT); and
  4. Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT.
  • Naglazyme will have Sole Supply Status in the community, Hospital Supply Status in DHB hospitals and subsidy and delisting protection until 30 June 2018.
  • Prior to a listing on the Pharmaceutical Schedule (i.e. until Medsafe approval is granted) net pricing will apply to any individual patient funding applications approved via the Named Patient Pharmaceutical Assessment (NPPA) Policy.

Feedback received

We appreciate all of the feedback that we received and acknowledge the time people took to respond. All consultation responses received by 18 February 2016 were considered in their entirety in making a decision on the proposed changes. Most responses were supportive of the proposal, and the following issues were raised in relation to specific aspects of the proposal:

Theme Comment
Supportive of the proposal. Galsulfase should be listed on the Pharmaceutical Schedule without delay. Galsulfase will be listed as soon as practicable following notification to PHARMAC that the product has been approved by Medsafe.
Concerns that galsulfase is very expensive and patient numbers are very low. PHARMAC is comfortable that the commercial arrangements for galsulfase mitigate any fiscal risks from this high cost treatment.
Concerns that galsulfase compares poorly with other treatments due to high cost and this proposal would set a precedent for other similar treatments for other rare metabolic disorders. PHARMAC notes this decision results from the Rare Disorders RFP and has been assessed following the process outlined in Schedule 2 of the RFP.It’s a trial process, which aims to improve access to medicines for people with rare disorders by encouraging competition among suppliers. We will be evaluating the effectiveness of this process before any decisions are made about future commercial processes for medicines for rare disorders. You will find more information about the RFP here.

Concerns regarding the Special Authority criteria

  • some considered the criteria were too open
  • one respondent considered the Special Authority should be amended to allow for longer initial approvals as initial benefits may be difficult to measure and renewal criteria be amended.
We acknowledge these concerns.  While commercial arrangements make it possible to fund galsulfase for all patients with MPS VI; we note that, given the rarity of this condition, the clinical appropriateness of treatment will be a matter for individual patients to discuss with their metabolic physician.PHARMAC notes the renewal criteria do not include specific measures of benefit, so it will be open to treating clinicians to identify whether a benefit has been achieved.
Interested to understand how many patients would access medicines that may be funded from the Medicines for Rare Disorders RFP.Concerned that, for small patient groups, there is a risk of clustering, particularly for small DHBs. Could DHBs consider a risk sharing arrangement to minimise the impact on small DHBs?   At this stage, we are not able to provide an estimate of patient numbers for all proposals resulting from the Rare Disorders RFP, as some proposals are still in progress. PHARMAC estimates there are less than five patients in NZ with MPS VI. Icatibant was listed on 1 January 2016 and PHARMAC estimated this would be accessed by up to 25 patients.DHBs are able to establish and manage their own risk-pooling approaches (such as the one in place for haemophilia treatments).
Supportive of the proposal. MPS VI has significant long-term debilitating effects on patients, leading to significant compromise in health, community participation and quality of life.Alternative treatment strategies for MPS VI are limited and include bone marrow transplant. Noted.

More information

Naglazyme is approved in Europe, US, Australia and other countries. BioMarin has submitted an application for registration to Medsafe, and this is currently under assessment (see http://www.medsafe.govt.nz(external link)).

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz.