Listed medicines are rarely assessed for efficacy

BACKGROUND: Listed medicines are added to the Australian Register of Therapeutic Goods conditional upon a declaration that information regarding their quality, safety and efficacy is held by the sponsor. AIM: To assess both the extent to which targeted reviews by the Therapeutic Goods Administration (TGA) as reported in compliance review reports published in 2022 and 2023 appraised the efficacy of those medicines and to document the extent of random compliance reviews from 2016. METHODS: The outcomes recorded in the 'Issues related to efficacy' section of individual compliance review reports were documented. Other matters of regulatory importance noted in the reports, such as the use of indications not permitted for these medicines, were also recorded. All random reviews in the Listed Medicine Compliance Review Reports database were documented. RESULTS: Of 215 targeted reviews, 62 recorded some degree of assessment of issues related to efficacy by the TGA. Only in six reports were there details of a critical appraisal of the sponsor's efficacy information. Further, 33 of the 62 medicines had used indications not permitted under Australian regulations. Thirty-seven random reviews between 2016 and December 2018 were identified. CONCLUSIONS: Reliance in recent years on targeted reviews has resulted in only about 2% of medicines added to the Australian Register of Therapeutic Goods having their evidence of efficacy reviewed. Use of indications not permitted for these medicines is common. Contrary to current TGA documents, no random review has been conducted since December 2018.