Obeticholic AcidOCALIVA
Primary biliary cholangitis (previously known as primary biliary cirrhosis) in combination with ursodeoxycholic acid in UDCA inadequate responders and as monotherapy in patients intolerant to UDCA.
Decisions on record
- Meeting Mar 2021 Recommended Primary biliary cholangitis
- Meeting Nov 2020 Deferred Primary biliary cholangitis
- Meeting Jul 2019 Not recommended Obeticholic acid is indicated for the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid
- Meeting Nov 2018 Not recommended Primary biliary cholangitis
Access path
- TGA registered · OCALIVA
TGA label narrower than the PBS population
- Nov 2018Not recommended
vs UDCA monotherapy (for inadequate responders); placebo (for…
- Jul 2019Not recommended
Comparator changed: UDCA monotherapy (for inadequate responders); placebo (for…
- Nov 2020Deferred
Comparator changed: UDCA plus placebo (for UDCA-inadequate responders); placebo (for…
- Mar 2021Recommended · restricted
Comparator changed: UDCA monotherapy for UDCA-inadequate responders; placebo for…
- PBS listing · Restricted
From the public summary
6.1 The PBAC recommended the listing of obeticholic acid (OCA) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in UDCA inadequate responders and as monotherapy in patients intolerant to UDCA. The PBAC was satisfied that OCA provides, for some patients, an improvement in efficacy compared to UDCA monotherapy in UDCA inadequate responders and placebo in UDCA intolerant patients.PSD · Mar 2021
6.3 The PBAC noted that an updated economic model was presented for the UDCA inadequate responders (i.e. those receiving OCA + UDCA) that included: changes to the disutility value of pruritus (0.03 in Year 1 and 0.015 in Years 2 to 30 compared to 0.03 in Year 1 only in November 2020).PSD · Mar 2021
5.3 Table 1 outlines the changes to the economic model which were recommended by the PBAC in November 2020 (paragraph 2.2) and how and/or if the recommendations have been addressed in the March 2021 revised economic model. 6PSD · Mar 2021
ICER $'''''''''''''''2/QALY in submission ICER should be < ICER = $''''''''''''''''1/QALY $''''''''''''''''''1/QALY with the three The reduced effective DPMQ, in $''''''''''''''''3/QALY in pre-PBAC (which changes outlined above only combination with the other 2 incorporated the RSA) changes outlined in the rows above, did not result in an ICER of < $'''''''''''''''1.PSD · Mar 2021
The resubmission described OCA 5-10 mg titration dosing as superior in terms of effectiveness compared with best supportive care when used as combination therapy 15PSD · Nov 2020
The resubmission described OCA 5-10 mg titration dosing as superior in terms of effectiveness compared with placebo when used as monotherapy for patients who are intolerant to UDCA. This claim is unchanged from the previous submission. The PBAC previously considered that the clinical claim was not adequately supported by the data (paragraph 7.8, obeticholic acid PSD, July 2019).PSD · Nov 2020
6.2 The PBAC noted and welcomed the input from individuals (31) and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with obeticholic acid including providing a treatment option once UDCA ceases to work, improved symptom control and improved quality of life.PSD · Jul 2019
6.3 The PBAC noted the advice received from the PBC Foundation (UK) which advised that the use of obeticholic acid reduces symptom burden, improves quality of life, slows disease progression and improves biochemical markers.PSD · Jul 2019
Cost-effectiveness
The Sponsor indicated that it was unable to reduce the ICER to less than $55,000 to <$75,000 per QALY through a reduction in the DPMQ of OCA only. PSD · 2021
Decision context
PopulationAdults with primary biliary cholangitis inadequately responding to ursodeoxycholic acid treatment for at least 12 months, or adults unable to tolerate UDCA due to severe side effects.
Risk sharingRisk sharing arrangement with financial caps approximately 29% below revised financial estimates, beyond which percentage rebates would be applied.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2021 | Recommended · restricted | ursodeoxycholic acid (UDCA) | $55k–75k | Cost-utility · QoL |
| Nov 2020 | Deferred | UDCA monotherapy for UDCA-inadequate responders; placebo for UDCA-intolerant patients | — | RCT · Surrogate |
| Jul 2019 | Not recommended | UDCA plus placebo (for UDCA-inadequate responders); placebo (for UDCA-intolerant patients) | — | RCT · Surrogate |
| Nov 2018 | Not recommended | UDCA monotherapy (for inadequate responders); placebo (for UDCA-intolerant patients) | — | RCT · Surrogate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| POISE | Ph 3 | 217 | DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Ver… | completed |
Consumer voice
Consumers reported that obeticholic acid provides a treatment option when UDCA fails, improves symptom control and quality of life, and slows disease progression with improved biochemical markers.
providing a treatment option once UDCA ceases to work, improved symptom control and improved quality of life Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC requires 12+ months of UDCA treatment failure or documented severe intolerance; TGA label does not specify these criteria.