Melatonin Prolonged Release
Treatment of insomnia in children aged 2-18 years with Autism Spectrum Disorder (ASD) and/or Smith-Magenis Syndrome (SMS), where sleep hygiene measures have been insufficient.
From the public summary
The PBAC did not recommend the listing of melatonin for the treatment of insomnia in children aged 2-18 years with Autism Spectrum Disorder (ASD) and/or Smith- Magenis Syndrome (SMS) where sleep hygiene measures have been insufficient.PSD · Mar 2021
The PBAC acknowledged there was a clinical need for effective treatments for insomnia in patients with ASD and a very high clinical need in the SMS population. The PBAC also noted the input from SMS Australia which highlighted the impact insomnia and night time waking has on people with the condition and their carers and noted the input that melatonin is widely used in the population but is often purchased from overseas in jurisdictions where melatonin is …PSD · Mar 2021
The submission presented a trial-based economic evaluation based on the NEU_CH_7911 randomised trial, and a modelled economic evaluation based on cost per year of response, extrapolated over a 9-year time horizon. The type of economic evaluation presented was a cost-effectiveness analysis, reported as a cost per responder and cost per additional responder year.PSD · Mar 2021
Table 8 summarises the key components of the economic evaluation.PSD · Mar 2021
The submission described prolonged release melatonin (2, 5, or 10 mg) as superior in terms of efficacy compared with placebo. No claim was made as to comparative safety.PSD · Mar 2021
Whilst a statistically significant treatment difference was observed in favour of prolonged release melatonin in terms of total sleep time, the clinical relevance of the treatment difference was unclear (32 minutes per night). Only 48/61 (79%) patients in the placebo arm, and 52/58 (90%) patients in the melatonin arm, were included in the calculation of the primary outcome.PSD · Mar 2021
The trial enrolled children 2 to 17.5 years with a confirmed history of either ASD according to the Diagnostic and Statistical Manual of Mental Disorders (DSM-5/4) criteria or International Classification of Diseases 10th Revision (ICD-10), or neurodevelopmental disabilities caused by neurogenetic diseases (including SMS); and sleep problems including: a minimum of 3 months of impaired sleep defined as ≤6 hours of continuous sleep and/or ≥ 0.5 hour sleep …PSD · Mar 2021
Cost-effectiveness
Economic analysis based on cost per responder and cost per responder year; ICER value not stated in public text
Decision context
PopulationChildren aged 2 to 18 years with Autism Spectrum Disorder (ASD) and/or Smith-Magenis Syndrome (SMS) with insomnia who have failed to achieve adequate response to sleep hygiene measures.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2021 | Not recommended | placebo (representing standard supportive care including behavioural therapy, sleep hygiene, environmental, dietary, and | — | RCT · Total sleep time; sleep latency |
Consumer voice
Smith-Magenis Syndrome Australia reported that individuals with SMS experience chronic sleep problems, particularly night-time waking, which significantly impacts daytime mood and learning and creates supervision challenges. The organisation indicated that prolonged release melatonin is beneficial for reducing night-time waking, though families remain concerned about the lack of regulation around
almost all individuals with SMS have chronic sleep problems; with night-time waking being the most concerning for parents. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both TGA and PBAC indications specify identical populations: children aged 2-18 with ASD and/or SMS with insomnia after failed sleep hygiene.