Proposal to list oral betaine for rare disorder
16 November 2016
PHARMAC is seeking feedback on a proposal to list betaine (Cystadane) resulting from a provisional agreement between Emerge Health Pty Ltd and PHARMAC.
The provisional agreement is the seventh that PHARMAC has reached with a bidder in a Request for Proposals we ran in 2014, related to the supply of medicines for rare disorders.
In summary, this proposal would result in betaine (Cystadane) being 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 would occur after any Medsafe approval of the pharmaceutical.
PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 5pm Wednesday, 30 November 2016 to:
Caroline De Luca
Senior Therapeutic Group Manager / Team Leader
PO Box 10 254
Fax: 04 460 4995
All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) prior to making a decision on this proposal.
Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.
We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.
Details of the proposal
Following Medsafe approval, betaine (Cystadane) would 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 would be listed as soon as practicable following Emerge Health’s notification to PHARMAC that Medsafe has granted registration.
- Cystadane would 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 paediatrician or metabolic physician. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- The patient has a confirmed diagnosis of homocystinuria; and
- Any of the following:
2.1 a cystathionine beta-synthase (CBS) deficiency; or
2.2 a 5,10-methylene-tetrahydrofolate reductase (MTHFR) deficiency; or
2.3 a defect in cobalamin cofactor metabolism; and
- An appropriate homocysteine level has not been achieved despite a sufficient trial of a low methionine diet and appropriate vitamin supplementation.
Renewal only from a paediatrician or 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 would apply to any individual patient funding applications approved via the Named Patient Pharmaceutical Assessment (NPPA) Policy.
Betaine (Cystadane) is an oral treatment used to treat homocystinuria resulting from a dysfunction in one of the metabolic pathways. Diagnosis is usually made following newborn screening, however it can occur later with the development of symptoms. The major clinical symptoms include developmental delay/intellectual disability, dislocation of the optic lens (ectopia lentis), myopia, skeletal abnormalities including scoliosis and chest deformities, a tendency to thromboembolic episodes, and osteoporosis.
Current funded treatment options include vitamin supplementation with vitamin B12, folate and vitamin B6, and diet restriction.
The proposal is to list betaine anhydrous powder, with Special Authority criteria restricted to patients diagnosed with homocystinuria who have not achieved an appropriate homocysteine level with low methionine diet and vitamin supplementation.
Cystadane is available as a 1 gram per gram powder for oral administration. Each bottle contains 180 g and the powder is measured using one of 3 measuring spoons (100 mg, 150 mg and 1 g) provided with the product. The powder should be mixed with water, juice, formula or food until completely dissolved and then ingested immediately after mixing.
PHARMAC estimates there would be between 22 to 45 patients (mainly children) who would be eligible for treatment with betaine under the proposed criteria. To date, PHARMAC has received 8 exceptional circumstances applications (via the NPPA policy, or the previous Community Exceptional Circumstances scheme) for the funding of betaine for individuals with homocystinuria.
Cystadane is available in Australia, the United Kingdom and Europe. It is registered with the Therapeutic Goods Administration (TGA), and Emerge Health intends to submit an application for registration of Cystadane to Medsafe as soon as possible.
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 here(external link).
This will be the last product to be considered as part of the Rare Disorders RFP. PHARMAC is looking to finalise the funding decisions arising out of the RFP by the end of 2016. Following the last decision, PHARMAC will begin an evaluation of the rare disorders funding pilot.
Last updated: 27 June 2018