Decision to list oral betaine for rare disorder

Medicines

Decision

PHARMAC is pleased to announce the approval of an agreement with Emerge Health Pty Ltd to list oral betaine powder (Cystadane).

PHARMAC is pleased to announce the approval of an agreement with Emerge Health Pty Ltd to list oral betaine powder (Cystadane). This is the tenth and final product to be listed following a Request for Proposals we ran in 2014, related to the supply of medicines for rare disorders. This was the subject of a consultation letter dated 16 November 2016.

The decision means that betaine (Cystadane) will be funded in the community under Special Authority criteria, and in DHB hospitals subject to restrictions, for the treatment of homocystinuria.

Listing in the Pharmaceutical Schedule will occur after any Medsafe approval of the medicine. The decision is as consulted on, with the exception of some changes to the Special Authority criteria. Detail of the decision is set out on the following pages.

Details of the decision

Following Medsafe approval, betaine (Cystadane) will be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List, HML) of the Pharmaceutical Schedule as follows:

Chemical Presentation Brand Pack size Price and subsidy
Betaine Powder for oral soln Cystadane 180 g OP $575.00
  • Cystadane will be listed as soon as practicable following Emerge Health’s notification to PHARMAC that Medsafe has granted registration.
  • Cystadane will be listed subject to the following Special Authority criteria in the community and equivalent restrictions in the HML:

Special Authority for subsidy

Initial application 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 confirmed diagnosis of homocystinuria; and
  2. Any of the following:
    1. a cystathionine beta-synthase (CBS) deficiency; or
    2. a 5,10-methylene-tetrahydrofolate reductase (MTHFR) deficiency; or
    3. a disorder of intracellular cobalamin metabolism; and
  3. An appropriate homocysteine level has not been achieved despite a sufficient trial of appropriate vitamin supplementation.

Renewal only from a metabolic physician. Approvals valid for 12 months where the treatment remains appropriate and the patient is benefiting from treatment.

  • Prior to any listing on the Pharmaceutical Schedule (ie. if and until Medsafe approval is granted), the proposed list price 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 30 November 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 proposal. Suggested changes to the Special Authority criteria:

  • Defect in cobalamin cofactor metabolism: should read disorders of intracellular cobalamin metabolism.
  • Restriction to people without adequate homocysteine levels on vitamins or diet: should be vitamins only. Considered diet is difficult to define, administer and comply with.

The Special Authority and HML criterion has been amended in light of the feedback as follows:

  • Criterion 2.3 amended to state “a disorder of intracellular cobalamin metabolism”
  • Criterion 3 amended to remove the requirement of a low methionine diet prior to betaine treatment.

We note that dietary measures would be used in conjunction with vitamin supplementation where appropriate, but that all patients with homocystinuria would receive vitamin supplementation prior to considering betaine treatment.

Considered initial and renewal applications for betaine should be from metabolic physicians.

PHARMAC considers that Metabolic Physicians from the National Metabolic Service (NMS) are best placed to initiate treatment with betaine and the Special Authority and HML restrictions have been amended accordingly.

The NZ packaging for Cystadane will include a 1 g measuring spoon (1.7 mL spoon is equal to 1 g of betaine anhydrous powder). Noted the consultation document referred to 3 x measuring spoons (100 mg, 150 mg and 1 g) being provided as per the European packaging.

Noted. PHARMAC consider a 1 g measuring spoon would be suitable for most patients.

The consultation document noted that Cystadane powder should be mixed with water, juice, formula or food until completely dissolved and then ingested immediately after mixing.

The Australian/New Zealand product information states that the powder should be mixed with 120-180 mL of water for immediate ingestion.

Noted. PHARMAC acknowledges the information in the consultation letter was taken from the European product information available at the time for the same product and that this has now been superseded by the NZ product information. This updated administration information would be included in the NZ datasheet for the product if Medsafe approved.

More information

PHARMAC’s evaluation of Cystadane followed the process described in Schedule 2 of the Request for Proposals (RFP) for the supply of medicines for rare disorders. The RFP can be found on the PHARMAC website.

This is the last product to be considered as part of the Rare Disorders RFP. PHARMAC will begin an evaluation of the rare disorders funding pilot in early 2017.

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 66 00 50.