Decision to list cholic acid (Cholebiol) for rare disorder

Medicines

Decision

PHARMAC is pleased to announce the approval of an agreement with Max Health Limited for the listing of oral cholic acid capsules (Cholebiol). This is the sixth product to be listed following a Request for Proposals (RFP) we ran in 2014, related to the supply of medicines for rare disorders.

This was the subject of a consultation letter dated 28 June 2016.

In summary, the effect of the decision is that cholic acid (Cholebiol) will be funded in the community under Special Authority criteria and in DHB hospitals subject to restrictions for the treatment of bile acid synthesis disorders due to single enzyme defects with neonatal/infant-onset liver disease.

Listing in the Pharmaceutical Schedule will occur subject to Medsafe approval of the pharmaceutical. The decision is as consulted on, with the exception of the following:

  • Original pack (OP) dispensing has been added to the listing; and
  • An additional applicant type – paediatric gastroenterologists – has been added to the Special Authority.

Details of the decision

Following Medsafe approval, cholic acid (Cholebiol) 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

Cholic acid

Cap 50 mg

Cholebiol

90 OP

$8,438.00

Cholic acid

Cap 250 mg

Cholebiol

90 OP

$42,691.00

  • Cholebiol will be listed as soon as reasonably practicable following Max Health Limited’s notification to PHARMAC that Medsafe has granted registration.
  • Cholebiol 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 hepatologist, paediatric hepatologist or paediatric gastroenterologist. Approvals valid for 12 months for applications meeting the following criteria:

Both:

  1. The patient has neonatal/infantile onset (under 12 months of age) metabolic liver disease; and
  2. The patient has a confirmed single enzyme deficiency resulting from an inborn error in primary bile acid synthesis.

Feedback received

We appreciate all of the feedback that we received and acknowledge the time people took to respond. All consultation responses received by 12 July 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

Concerns regarding the availability of an oral liquid formulation of cholic acid. Questioned the suitability of capsules being listed when a newly diagnosed patient would very likely to be newborn or infant.

Doses required are unlikely to coincide with strengths of capsules available.

The manufacturer advises the cholic acid capsules can be opened and the contents mixed with formula, expressed breast milk, or juice for older infants. The manufacturer also advises dosing should be rounded to the nearest 50 mg increment, preventing the need for a part dose. This information will be available on the product datasheet and patient information when Medsafe approved.  

Requested paediatric gastrotenterologist/ hepatologist be added as a more appropriate term in the criteria. Some clinicians are called gastroenterologists but practice hepatology.

The Special Authority criteria have been amended to include paediatric gastroenterologists as an applicant type.

Supportive of the proposal. Noted there are no current patients with inherited bile acid synthesis, but if a new patient did present then would want to use cholic acid.

Noted.

Concerns regarding proposed pack size given small number of patients and potential for significant wastage to occur and cost to pharmacies.

PHARMAC considers the pack size of cholic acid of 90 capsules is appropriate for the likely dosing schedule. For a neonate, the appropriate dose would be 50 mg, therefore one pack would provide at most 90 days treatment. See below re wastage.

Concerns regarding the high price and request that cholic acid be listed as an original pack (OP) or the wastage rule applied to avoid the significant negative financial impact if pharmacists are left with part packs of the product.

Original pack dispensing has been added to the listing for Cholebiol capsules. This would mitigate the financial risk to pharmacies.

More information

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.