Subject: Ocaliva 2029-A SGM P2023

Drug

OCALIVA  (obeticholic acid)

Policy:

      I.  INDICATIONS

          The indications below including FDA-approved indications and
          compendial uses are considered a covered benefit provided that
          all the approval criteria are met and the member has no
          exclusions to the prescribed therapy.

          FDA-Approved Indication
          Ocaliva is indicated for the treatment of adult patients with
          primary biliary cholangitis (PBC):
          •    without cirrhosis or
          •    with compensated cirrhosis who do not have evidence of
                portal hypertension, either in combination with
                ursodeoxycholic acid (UDCA) with an inadequate response to
                UDCA, or as monotherapy in patients unable to tolerate
                UDCA.

          This indication is approved under accelerated approval based on
          a reduction in alkaline phosphatase (ALP). An improvement in
          survival or disease-related symptoms has not been established.
          Continued approval for this indication may be contingent upon
          verification and description of clinical benefit in confirmatory
          trials.

          All other indications are considered experimental/investigational
          and not medically necessary.


    II.  DOCUMENTATION

          Submission of the following information is necessary to initiate
          the prior authorization review:
          A.  For initial requests: Pretreatment serum alkaline
               phosphatase (ALP) level
          B.  For continuation of therapy: Current serum alkaline
               phosphatase (ALP) and/or current total bilirubin level


  III.  EXCLUSIONS

          Coverage will not be provided for members with any of the
          following exclusions:
          A.  Member has decompensated cirrhosis (e.g., Child-Pugh Class
               B or C) or a prior decompensation event
          B.  Member has compensated cirrhosis with evidence of portal
               hypertension (e.g., ascites, gastroesophageal varices,
               persistent thrombocytopenia).


   IV.  CRITERIA FOR INITIAL APPROVAL

          Primary biliary cholangitis (PBC) (previously known as
          primary biliary cirrhosis)
          Authorization of 6 months may be granted for treatment of PBC
          in members 18 years of age or older when all of the following
          criteria are met:
          A.  Diagnosis of PBC is confirmed by at least two of the following
               three criteria:
               1.  Biochemical evidence of cholestasis with elevation of
                    alkaline phosphatase (ALP) level for at least 6 months
                    duration
               2.  Presence of antimitochondrial antibodies (AMA) (titer
                    >1:40 by immunofluorescence or immunoenzymatic
                    reactivity) or PBC-specific antinuclear antibodies (ANA)
                    (e.g., anti-gp210, anti-sp100)
               3.  Histologic evidence of PBC on liver biopsy (e.g., non-
                    suppurative inflammation and destruction of interlobular
                    and septal bile ducts)
          B.  Member has an elevated serum ALP level prior to initiation of
               therapy with the requested drug
          C.  Member meets at least one of the following requirements:
               1.  Inadequate response to at least 12 months of prior
                    therapy with ursodeoxycholic acid (UDCA)/ursodiol and
                    the member will continue concomitant therapy with
                    UDCA/ursodiol, or
               2.  Intolerance to UDCA/ursodiol


     V.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for members who
          have achieved or maintained a clinical benefit from Ocaliva
          therapy (i.e., at least a 15% reduction in ALP level, ALP level
          less than 1.67-times upper limit of normal [ULN], or total
          bilirubin less than or equal to ULN).

Place of Service:

Outpatient

The above policy is based on the following references:

1. Ocaliva [package insert]. Morristown, NJ: Intercept Pharmaceuticals, Inc.; May 2022.
2. Lindor KD, Gershwin E, Poupon R, et al. Primary biliary cirrhosis. 2009;50:291-308.
3. European Association for the Study of the Liver. EASL clinical practice guidelines: Management of cholestatic liver diseases. J Hepatol. 2017;67:145-172.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

August 13, 2023