This is the text extract for Approval of proposal to widen access to ursodeoxycholic acid; Contact: Notifications, browse documents here.

16 February 2012

Approval of proposal to widen access to ursodeoxycholic acid

PHARMAC is pleased to announce the approval of a proposal to widen funded access to ursodeoxycholic acid for prevention of veno-occlusive disease in patients receiving high dose conditioning therapy prior to bone marrow or stem cell transplantation. This was the subject of a consultation letter dated 20 January 2012 (http://pharmac.govt.nz/2012/01/20 ). In summary, the effect of the decision is that additional criteria will be added to the Special Authority for ursodeoxycholic acid which will allow 13 weeks funded treatment for patients undergoing a haematological transplant as part of the conditioning regimen from 1 March 2012. Details of the proposal The Special Authority will be amended from 1 March 2012, as follows (additions in bold):

Initial application – (Pregnancy/Cirrhosis) - from any relevant practitioner. Approvals valid for 6 months for applications meeting the following criteria: Either: 1. Patient diagnosed with cholestasis of pregnancy; or 2. Both: 2.1. Primary biliary cirrhosis confirmed by antimitochondrial antibody titre (AMA) > 1:80, and raised cholestatic liver enzymes with or without raised serum IgM or, if AMA is negative, by liver biopsy; and 2.2. Patient not requiring a liver transplant (bilirubin > 170umol/l; decompensated cirrhosis) Note: Liver biopsy is not usually required for diagnosis but is helpful to stage the disease. Initial application – (Haematological Transplant) - from any relevant practitioner. Approvals valid for 6 months for applications meeting the following criteria: Both: 1. Patient at risk of veno-occlusive disease or has hepatic impairment and is undergoing conditioning treatment prior to allogenic stem cell or bone marrow transplantation, and 2. Treatment for up to 13 weeks. Renewal – (Pregnancy/Cirrhosis) - from any relevant practitioner. Approvals valid for 2 years where the treatment remains appropriate and the patient is benefiting from treatment. Note: Ursodeoxycholic acid is not an appropriate therapy for patients requiring a liver transplant (bilirubin > 170 micromol/l; decompensated cirrhosis). These patients should be referred to an appropriate transplant centre. Treatment failure – doubling of serum bilirubin levels, absence of a significant decrease in ALP or ALT and AST, development of varices, ascites or encephalopathy, marked worsening of pruritus or fatigue, histological progression by two stages, or to cirrhosis, need for transplantation.

The length of the approval has been extended from 4 months to 6 months to ensure that patients have ample time to initiate their prescription, but there has been no change to the length of treatment (i.e. it remains 13 weeks).

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Feedback received We appreciate all of the feedback that we received and acknowledge the time people took to respond. All consultation responses received by 7 February 2012 were considered in their entirety in making a decision on the proposed changes. All responses were supportive of the proposal, and the following issues were raised in relation to specific aspects of the proposal: Theme Is there a requirement for renewal criteria for this indication? Comment The advice received to date is that it is very unlikely that patients would require a second course of ursodeoxycholic acid for this indication. We would reconsider adding renewal criteria in the future if a need arises.

More information If you have any questions about this decision, you can call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.

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