Tobias Polak

Results from expanded access programs: a review of academic literature 151 7♦ Comparison with other data When comparing the expanded access program with clinical trials, the authors note that both safety and efficacy are in line with previously reported results: ‘Results from this ongoing expanded access program support previous observational and clinical trial data showing that add-on CBD may be an efficacious long-term treatment option for TRE.’ ‘AE rates were similar to those reported in the initial analysis of the expanded access program, as well as to those reported in randomized controlled trials.’ Several limitations to the study are mentioned. Besides the fact that the expanded access program was not placebo-controlled – a limitation that applies to all expanded access programs – ‘there was inter-site variability in reporting methods, and information such as the reasons for AED dose reductions was not captured. Although parents/caregivers reported only the specific seizures that were countable, some seizure types that can be difficult to count (eg, absence) were included in the total seizure frequency data. In some of the site protocols, enrolment was dependent on predetermined seizure frequency, which was known to possible participants; hence, baseline overreporting cannot be completely excluded in this prospective data collection.’ Conclusion The authors conclude that: ‘The pooled data across the expanded access program provides initial insights on the long-term treatment effect of CBD that support the recent evidence from rigorous, double-blind, placebo-controlled trials showing meaningful reductions in seizure frequency for patients who received add-on CBD vs placebo’. No recommendations for future studies are given.

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