EvolocumabRepatha
Treatment of homozygous familial hypercholesterolaemia (HoFH), heterozygous familial hypercholesterolaemia (HeFH) and non-familial hypercholesterolaemia (non-FH) to reduce LDL cholesterol.
Decisions on record
- Meeting Mar 2024 Recommended Hypercholesterolaemia
- Meeting Jul 2022 Recommended Hypercholesterolaemia with LDL-C 1.8-2.6 mmol/L despite optimised statin and ezetimibe treatment
- Meeting Mar 2021 Recommended Familial and non-familial hypercholesterolaemia
- Meeting Nov 2020 Not recommended Familial heterozygous hypercholesterolaemia; Non-familial hypercholesterolaemia; Familial homozygous hypercholesterolaemia
- Meeting Nov 2019 Recommended Hypercholesterolaemia
- Meeting Jul 2019 Deferred Hypercholesterolaemia
- Meeting Jul 2018 Not recommended Adults with heterozygous familial hypercholesterolaemia or clinical atherosclerotic cardiovascular disease (ASCVD) in combination with a statin or statin with other lipid lowering therapies, or in combination with other lipid-lowering therapies in patients who are statin- intolerant. Adults and adol
- Meeting Mar 2018 Recommended Familial hypercholesterolaemia (FH)
4 earlier decisions
- Nov 2017 Deferred Familial hypercholesterolaemia (FH)/ hypercholesterolaemia with symptomatic atherosclerotic cardiovascular disease (ASCVD) who do not have underlying FH
- Jul 2017 Recommended Familial homozygous hypercholesterolaemia
- Mar 2016 Recommended Familial hypercholesterolaemia
- Mar 2015 Not recommended Hypercholesterolaemia
Access path
- TGA registered · Repatha
TGA label narrower than the PBS population
- Mar 2015Not recommended
vs ezetimibe and placebo
- ↻ resubmittedMar 2016Recommended · restricted
- Jul 2017Recommended
Listing: Authority Required → Restricted
- Nov 2017Deferred
Listing: Restricted → Authority Required
- Mar 2018Recommended · restricted
Comparator changed: ezetimibe, placebo, alirocumab → ezetimibe
- Jul 2018Not recommended
Inadequately defined population and eligibility criteria, insufficiently defined atherosclerotic disease, inadequate…
- Jul 2019Deferred
Comparator changed: ezetimibe and placebo → placebo
- Nov 2019Recommended · restricted
Comparator changed: placebo → atorvastatin, rosuvastatin, ezetimibe
- Nov 2020Not recommended
Evolocumab is a relatively new drug with uncertain uptake and financial estimates; it is a novel third-line therapy…
- ↻ resubmittedMar 2021Recommended
Listing: Authority Required → Restricted
- Jul 2022Recommended · restricted
Evidence: Other → RCT
- Mar 2024Recommended · restricted
Evidence: RCT → Cost-minimisation
- PBS listing · Restricted
From the public summary
Requested change to the restriction level from Authority Required (telephone/online) to Authority Required (STREAMLINED) for initial treatmentPSD · Mar 2024
6.1 The PBAC recommended changing the restriction level from Authority Required (telephone/online) to Authority Required (STREAMLINED) for the initial treatment listings for evolocumab injection 140 mg in 1 mL single use pre-filled pen and evolocumab injection 420 mg in 3.5 mL single use pre-filled cartridge for the treatment of homozygous familial hypercholesterolaemia (HoFH), heterozygous familial hypercholesterolaemia (HeFH) and non-familial …PSD · Mar 2024
The submission presented a stepped economic evaluation of evolocumab for the treatment of non-familial hypercholesterolaemia patients with atherosclerotic disease and additional risk factors, who have an LDL-C level between 1.8 andPSD · Jul 2022
2.6 mmol/L despite optimised treatment with statins and ezetimibe. The economic evaluation was based on relative LDL reductions from the FOURIER trial and other modelled variables. The economic evaluation was presented as a cost- effectiveness/cost-utility analysis. 15PSD · Jul 2022
The submission claimed that evolocumab (plus optimised background treatment) is superior to ‘placebo’ (optimised background treatment) in terms of comparative efficacy and similar in terms of comparative safety in patients with familial hypercholesterolaemia or non-familial hypercholesterolaemia with ASCVD and risk factors, who have LDL-C levels between 1.8 and 2.6 mmol/L.PSD · Jul 2022
2.6 mmol/L, based on the same clinical data. The ESC noted that the same proportional benefit was expected to be achieved across different LDL-C percentile ranges (see Table 8 above), with lower absolute treatment benefit expected due to lower baseline risk in the proposed additional patient group.PSD · Jul 2022
5.2 The PBAC noted and welcomed the input from individuals (1), health care professionals (54) and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with evolocumab, including: 11PSD · Mar 2024
• its effectiveness in managing hypercholesterolaemia (and subsequently cardiovascular risk), which can be challenging to treat with other medications such as statins and ezetimibe in certain patients.PSD · Mar 2024
Cost-effectiveness
Not a full economic submission; Category 3 submission requesting restriction level changes and clinical criteria modifications only.
a ''''''% price reduction, which resulted in an incremental cost-effectiveness ratio (ICER) of less than $15,000 - $45,000 per QALY using the revised base case parameters requested by the July 2019 PBAC PSD · 2019
Decision context
PopulationAdults with HoFH, HeFH, or non-FH with symptomatic atherosclerotic cardiovascular disease or elevated LDL cholesterol despite optimised statin and ezetimibe therapy.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2024 | Recommended · restricted | — | — | Cost-minimisation |
| Jul 2022 | Recommended · restricted | placebo (optimised background treatment) | $25k–35k | RCT · Surrogate |
| Mar 2021 | Recommended | — | — | Other |
| Nov 2020 | Not recommended | — | — | Other |
| Nov 2019 | Recommended · restricted | atorvastatin, rosuvastatin, ezetimibe | $15k–45k | RCT · LDL cholesterol reduction |
| Jul 2019 | Deferred | placebo | — | RCT · Surrogate |
| Jul 2018 | Not recommended | ezetimibe and placebo | — | RCT · Surrogate |
| Mar 2018 | Recommended · restricted | ezetimibe | $15k–45k | RCT · Other |
| Nov 2017 | Deferred | ezetimibe, placebo, alirocumab | — | RCT · Surrogate |
| Jul 2017 | Recommended | — | — | RCT |
| Mar 2016 | Recommended · restricted | ezetimibe and placebo | $15k–45k | RCT · LDL cholesterol reduction |
| Mar 2015 | Not recommended | ezetimibe and placebo | — | RCT · LDL-C |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| FOURIER | Ph 3 | 27,564 | Time to Cardiovascular Death, Myocardial Infarction, Hospitalization for Unstable Angina, … | completed |
| MENDEL-2 | Ph 3 | 615 | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12 | completed |
| LAPLACE-2 | Ph 3 | 2,067 | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12 | completed |
| GAUSS-2 | Ph 3 | 307 | Percent Change From Baseline in LDL-C at Week 12 | completed |
| RUTHERFORD-2 | Ph 3 | 331 | Percent Change From Baseline in LDL-C at Week 12 | completed |
| TESLA | Ph 2, PHASE3 | 58 | Part A: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Wee… | completed |
| DESCARTES | Ph 3 | 905 | Percent Change From Baseline in LDL-C at Week 52 | completed |
| OSLER-1 | Ph 2 | 1,324 | Number of Participants With Adverse Events | completed |
Codependent tests
| Service | Biomarker | MSAC outcome | Year |
|---|---|---|---|
| Genetic testing for heritable mutations associated with familial hypercholesterolaemia | germline variants in LDLR, PCSK9, and APOB genes | supported | 2019 |
Consumer voice
Consumer input from 1 individual, 54 healthcare professionals, and 1 organisation described benefits of evolocumab including effectiveness in managing hypercholesterolaemia, good safety and tolerability, easy administration, and improved quality of life. Comments expressed support for Authority Required (STREAMLINED) listing and raised concerns about access delays and the required duration of prio
its effectiveness in managing hypercholesterolaemia (and subsequently cardiovascular risk), which can be challenging to treat with other medications such as statins and ezetimibe in certain patients. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with symptomatic ASCVD or elevated LDL despite optimised statin-ezetimibe therapy; TGA label includes all primary hypercholesterolaemia.