Proposal to list cholic acid (Cholebiol) for rare disorder
PHARMAC is seeking feedback on a proposal to list cholic acid (Cholebiol) resulting from a provisional agreement formed between Max Health Limited and PHARMAC.
The provisional agreement is the fifth 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 cholic acid (Cholebiol) being 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 would occur after Medsafe approval of the pharmaceutical.
PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 5pm on Tuesday, 12 July 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, cholic acid (Cholebiol) 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:
Price and subsidy
Cap 50 mg
Cap 250 mg
- Cholebiol would be listed as soon as practicable following Max Health Limited’s notification to PHARMAC that Medsafe has granted registration.
- Cholebiol 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 hepatologist or paediatric hepatologist. Approvals valid for 12 months for applications meeting the following criteria:
- The patient has neonatal/infantile onset (under 12 months of age) metabolic liver disease; and
- The patient has a confirmed single enzyme deficiency resulting from an inborn error in primary bile acid synthesis.
Renewal only from a hepatologist or paediatric hepatologist. Approvals valid for 12 months where the treatment remains appropriate and the patient is benefiting from treatment.
- Cholebiol would have subsidy and delisting protection until 30 June 2018.
- Prior to a listing on the Pharmaceutical Schedule (ie 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.
Cholic acid is an oral treatment for inborn errors of primary bile acid synthesis in the liver that can lead to progressive liver disease and death. Inborn errors of bile acid synthesis are rare genetic disorders that can present as neonatal cholestasis, neurologic disease or fat-soluble-vitamin deficiencies. The clinical presentation of these disorders is variable. Severe forms usually present within the first year of life. Milder forms may present in older children or adults.
Cholic acid is one of the primary bile acids that would normally be produced by the body. It is considered that taking cholic acid orally can mitigate the effects of the disorder. The proposal is to list cholic acid with Special Authority criteria restricting funding to patients with bile acid synthesis disorders due to single enzyme defects with neonatal/infant onset liver disease. Evidence suggests cholic acid would both extend life and improve quality of life.
PHARMAC estimates there would be up to 2 infants per year that would be eligible for cholic acid under the proposed indications.
To date, PHARMAC has not received any NPPA applications for the funding of cholic acid for individuals with a single enzyme deficiency resulting from an inborn error in primary bile acid synthesis.
Various forms of cholic acid are approved in Europe and the United States. Max Health Limited intends to submit an application for registration of Cholebiol to Medsafe as soon as possible.
PHARMAC’s evaluation of Cholebiol followed the process described in Schedule 2 of the Request for Proposals (RFP) for the supply of medicines for rare disorders. The RFP document can be found on the PHARMAC website.