Decision to list sodium phenylbutyrate (Pheburane) and sodium benzoate for urea cycle disorders

sodium phenylbutyrate

Grans 483 mg per g

Pheburane

174 g OP

$1,920.00

sodium benzoate

Soln 100 mg per ml

Amzoate

100 ml

CBS

Sodium benzoate is the preferred agent for patients with mild to moderate urea cycle disorders in New Zealand. 

PHARMAC has also decided to list sodium benzoate (Amzoate) in the Pharmaceutical Schedule, with similar criteria to Pheburane. We note sodium benzoate is unregistered. PHARMAC does not have a supply agreement for this product.

Suggested several changes to the proposed Special Authority criteria for Pheburane:

• Widen criteria to include two additional UCDs arginase deficiency, and hyperornithinemia-hyperamoonaemia-homocitrullinemia syndrome based on published guidelines (Orphanet J Rare Dis. 2012;7:32.)
• Allow nominated paediatrician for a child (or physician for an adult), or a metabolic nurse to make repeat prescriptions in consultation with metabolic physicians.
• Concerns that 3 year listing is not long enough considering rare nature of disease.

The Special Authority criteria are based on the international approved indications for Pheburane and are consistent with the Medsafe datasheet. PHARMAC notes if there were patients with other UCDs not included in the proposed criteria, that funding could be considered via NPPA. We also note that sodium benzoate will be listed with Special Authority criteria enabling use for any UCD.

Any prescriber could write repeat prescriptions for sodium phenylbutyrate; however a Metabolic Physician would need to apply for a Special Authority renewal annually. We note these applications can be made electronically. Clinician feedback was supportive of this requirement and considered it was appropriate.

The agreement with the supplier provides delisting and subsidy protection until 1 July 2018, the maximum time allowable under the conditions of the Rare disorders RFP. The supply agreement is evergreen and therefore the listing would not terminate when the protection ends.

Supportive of proposal, agree with proposed prescriber restriction to metabolic physicians. Very pleasing to see medicines for rare diseases previously accessed via NPPA becoming available via Special Authority.

Sodium phenylbutyrate would only be used by a very small number of patients and these patients are already catered for with access via NPPA if needed.

The proposal would not lead to improved patient care in New Zealand and main justification of proposal is a cost saving exercise for PHARMAC. Appreciate the benefits that may come with improved taste, likely compliance, and shelf life.

Noted.

PHARMAC considers that there are some gains other than palatability to be made for patients with UCDs and their clinicians from the proposed listing for Pheburane. These include:

• Pheburane is a registered product giving assurance of its quality, consistency and safety;
• having an agreement in place with the supplier for Pheburane gives greater certainty of continuity supply;
• reduced administrative burden for clinicians (no need for NPPA applications for either product);
• access to funded treatment through community pharmacies.

We note the cost of Pheburane is greater than the unregistered sodium phenylbutyrate used in DHB hospitals.

Supports the listing of sodium phenylbutyrate under the proposed Special Authority criteria and sees the advantage is that it will be easier to have timely prescribing of an appropriate product and approval for the paediatric population.

Noted

Supports the listing of a registered brand of sodium phenylbutyrate but concerns regarding the high price of the pharmaceutical.

Requested that Pheburane be listed as an original pack (OP) or the wastage rule applied to avoid pharmacists being left with part bottles of the product.

Noted.

Original pack dispensing has been added to the proposal for Pheburane.

Concerns that the proposal does not address any of the unmet pharmaceutical needs of patients with rare diseases who are hoping new medicines will be approved.

This decision results from the Rare Disorders RFP and has been assessed following the process outlined in the RFP.

The Rare Disorders RFP is a trial, 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 making any decisions about future commercial processes for medicines for rare disorders.

PHARMAC notes the aim of the Rare Disorders RFP was to seek proposals for funding of medicines and their listing on the Pharmaceutical Schedule. The proposal for Pheburane met the pre-requisite requirements of the RFP as follows:-

• The medicine is not registered for the treatment of another, non-rare disorder, or if it is, the cumulative prevalence across all the indications still falls within the definition of rare.
• There is no suitable comparable alternative treatment on the Pharmaceutical Schedule.
• There is no suitable funded alternative non-drug therapeutic modality for the rare disorder.

A prerequisite of the RFP refers to a listing on the Schedule and not funding via PHARMAC’s exceptional circumstances mechanism. If a medicine is listed through the contestable fund, all eligible patients could receive it without needing to make individual applications with uncertainty of outcome.

For more information about the RFP process.

Comment that it has insufficient information on numbers of patients to consider cost-benefit but these numbers are expected to be low.

Noted

Considers the potential benefits are in the availability of this formulation that may aid compliance.

Noted