CannabidiolEPIDYOLEX
Severe myoclonic epilepsy in infancy (Dravet syndrome) — treatment of generalised tonic-clonic seizures or generalised clonic seizures that are not adequately controlled with at least two other antiseizure medications, as adjunctive therapy.
Decisions on record
- Meeting Mar 2026 Recommended Severe myoclonic epilepsy in infancy (Dravet syndrome)
- Meeting Jul 2025 Recommended Seizures associated with Lennox-Gastaut syndrome
- Meeting Sep 2022 Recommended Seizures associated with Lennox-Gastaut syndrome in patients aged 2 years and older inadequately controlled by at least two anti-epileptic drugs no PSD
- Meeting Jul 2022 Not recommended Lennox-Gastaut syndrome in patients aged 2 years and older not achieving adequate seizure control with at least two anti-epileptic drugs
- Meeting Mar 2022 Not recommended Lennox-Gastaut syndrome
- Meeting Nov 2020 Recommended Lennox-Gastaut syndrome; Dravet syndrome no PSD
- Meeting Jul 2020 Deferred Lennox-Gastaut syndrome (LGS) Dravet syndrome (DS)
Access path
- TGA registered · EPIDYOLEX
TGA label narrower than the PBS population
- Jul 2020Recommended · restricted
vs placebo plus standard care
- Mar 2022Not recommended
Comparator changed: placebo plus standard care → standard care (oral anti-epileptic drugs…
- Jul 2022Not recommended
ICER remained high and above acceptable threshold ($75,000–<$95,000 per QALY vs PBAC's requested <$45,000–<$55,000 per…
- ↻ resubmittedJul 2022Recommended · restricted
Comparator changed: standard care (oral anti-epileptic drugs and non-pharmacotherapy…
- Jul 2025Recommended · restricted
Evidence: RCT → Other
- Mar 2026Recommended · restricted
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended amending the restriction level of cannabidiol for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), from Authority Required (Telephone/online PBS Authorities system) to Authority Required (STREAMLINED).PSD · Jul 2025
5.2 The PBAC also recommended the following amendments to the current PBS criteria for cannabidiol in the LGS indication, as proposed in Option 2 of the submission:PSD · Jul 2025
4.21 No economic analysis was provided, as the submission stated that the proposed changes to the PBS restrictions would not impact the cost-effectiveness of cannabidiol.PSD · Jul 2025
4.4 The PBAC recalled its previously expressed view that the claim that cannabidiol was of superior comparative effectiveness and inferior comparative safety compared to the nominated comparator (standard care) was reasonable (paragraph 6.19, cannabidiol PSD, March 2022 PBAC meeting).PSD · Jul 2022
4.3 Epilepsy Action Australia and the Epilepsy Foundation provided input supporting amendments to the PBS restrictions for cannabidiol. Their comments highlighted barriers to accessing EEGs and specialist care, particularly in rural and remote areas, and emphasised the need to allow paediatricians to prescribe cannabidiol and to amend EEG requirements where testing is not feasible, to improve equitable access, reduce financial burden, and ensure timely …PSD · Jul 2025
4.4 The National Paediatric Medicines Forum (NPMF) provided input in support of amending the PBS restrictions for cannabidiol, including lowering the restriction level to Authority Required (STREAMLINED), amending clinical criteria to ease EEG requirements, and allowing paediatricians to prescribe the medication for continuing treatment.PSD · Jul 2025
4.17 The PBAC previously advised that a RSA would be required to manage the outstanding uncertainty regarding the dose likely to be used in clinical practice (paragraph 7.10, cannabidiol Minutes, March 2022 PBAC Meeting).PSD · Jul 2022
4.18 The resubmission proposed a % rebate on PBS expenditure that is in excess of the financial estimates outlined in Table 3. The resubmission proposed a % rebate (rather than %) as the resubmission considered there was uncertainty around patient numbers and the potential for use outside the requested indication, ‘given that different definitions of LGS can be applied in clinical practice (e.g., where ‘LGS-like’ conditions are labelled LGS)’.PSD · Jul 2022
Cost-effectiveness
No economic analysis was provided. The submission stated that proposed changes to PBS restrictions would not impact cost-effectiveness of cannabidiol, as eligible patient population would likely remain the same.
The PBAC considered this was appropriate as eligible patient population would likely remain the same. PBAC · 2026
Decision context
PopulationPatients with severe myoclonic epilepsy in infancy (Dravet syndrome) aged 2 years or older, with generalised tonic-clonic seizures or generalised clonic seizures not adequately controlled with at least two other antiseizure medications, to receive cannabidiol as adjunctive therapy.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | — | — | Other |
| Jul 2025 | Recommended · restricted | — | — | Other |
| Jul 2022 | Not recommended | standard care | $75k–95k | RCT · Seizure reduction |
| Jul 2022 | Recommended · restricted | standard care | $75k–95k | RCT |
| Mar 2022 | Not recommended | standard care (oral anti-epileptic drugs and non-pharmacotherapy interventions) | — | RCT · drop seizure reduction |
| Jul 2020 | Recommended · restricted | placebo plus standard care | — | RCT · Reduction in seizure frequency |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| GWPCARE2 | Ph 3 | 199 | Change In Convulsive Seizures During The Treatment Period Compared To Baseline | completed |
| GWPCARE1 Part B | Ph 3 | 120 | Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Perio… | completed |
| GWPCARE3 | Ph 3 | 225 | Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period | completed |
| GWPCARE4 | Ph 3 | 171 | Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period | completed |
| GWPCARE5 | Ph 3 | 681 | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Occurring in ≥5% o… | completed |
Consumer voice
Health care professionals, consumer groups (Epilepsy Action Australia, Epilepsy Foundation), and the National Paediatric Medicines Forum provided input supporting cannabidiol access for LGS patients, highlighting improved seizure control and quality of life, while raising concerns about EEG access barriers in rural and remote areas and advocating for streamlined authority processes and expanded pr
Cannabidiol is generally considered to be well tolerated, with fewer side effects and drug interactions compared to other AEDs. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to Lennox-Gastaut syndrome only, excluding Dravet syndrome covered by TGA label.