PBAC rejects first submission supported by real-world evidence

MAESTrO Database

4 July 2018 - A retrospective chart review of 211 Australian patients with chronic migraine served as the key evidentiary basis of the submission.

The submission was lodged in late 2017 to provide support for Allergan's request to revise the risk sharing arrangement (RSA) relevant to the use of botulinum toxin type A (Botox) for the treatment of chronic migraine.

The focus of the submission was ‘real‐world’ evidence, specifically all evidence when Botox was used for the treatment of chronic migraine, consistent with TGA‐approved dosing recommendations, in real‐world clinical practice settings.

The PBAC rejected the request at its meeting in March 2018. The PBAC considered that the claim made by the submission that the RSA should be revised as the greater than predicted use of Botox was due to greater than expected efficacy, and hence was cost‐effective, was not adequately supported by the evidence presented.

The PBAC considered that the retrospective chart review had a high risk of bias as it was uncontrolled and unblinded, with less than half the participants utilising a headache diary in the assessment of the primary outcome.

Those who think real-world evidence will supplant robust, well designed and executed phase III clinical trial evidence as the key evidential basis in a submission for a new medicine to a health technology assessment agency/committee like the PBAC anytime soon after regulatory approval (and perhaps for years after that) are kidding themselves (and others).

Read PBAC Public Summary Document for botulinum toxin type A (Botox) 

Michael Wonder

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Michael Wonder