26 April 2022 - Yesterday, we noted a recommendation rate of 78% which the highest value we have recorded since the November 2019 meeting. Is this a remarkable result or just an aberration?

Recommendation rates are highly variable and are driven in part by the mix of submissions considered by the PBAC. A high recommendation rate may be due to a high proportion of 'minor' submissions that are generally more successful than 'major' submissions. Higher recommendation rates may be due to more CMA type submissions that CEA submissions or fewer submissions for (new) cancer medicines which are generally more challenging.

Can we test any of these hypotheses with the available data?

Submissions are no longer classified as 'major' or 'minor' so one cannot readily test this hypothesis. One could perhaps rate category 1 & 2 submissions are majors and the rest as minors but this might be a little too simplistic.

There is not enough data at present to test the second hypothesis as the outcome statement for many medicine is very limited and one cannot readily the type of economic evaluation in the submission (if one was included/required). One only has to read the outcome statement for tepotinib hydrochloride monohydrate (Tepmetko) to realise that.

That brings us to the third hypothesis. The PBAC considered 12 submissions for a new cancer medicine. That is a high number of submissions for a new cancer medicine to be considered by the PBAC at a given time. The 12 submissions (11 medicines) were for:

• Cemiplimab
• Dostarlimab
• Enfortumab vedotin
• Gilteritinib fumarate
• Ixazomib
• Larotrectinib sulphate
• Niraparib tosylate monohydrate
• Sacituzumab govitecan
• Selinexor - combination with dexamethasone
• Selinexor - combination with bortezomib and dexamethasone
• Sotorasib
• Tepotinib hydrochloride monohydrate

Seven of the 12 were recommended; the rejected medicines are in italics.  A PBAC meeting where there were more recommendations for a new cancer medicine that there were rejections is most unusual.

That is not to say all even were recommended first up; it took Bayer four attempts to secure a recommendation for larotrectinib sulphate.

We will have more to say on the rejections for dostarlimab and sotorasib in a future issue.

The PBAC also considered a number of submissions for a new indication for a listed cancer medicine.  Those not recommended are in italics.

• Avelumab
• Carfilzomib
• Enzalutamide
• Nivolumab - gastric cancer/oesophageal cancer
• Nivolumab - oesophageal cancer (squamous cell)
• Palbociclib
• Pembrolizumab - renal cell carcinoma
• Pembrolizumab - head and neck cancer
• Pembrolizumab - oesophageal cancer
• Pembrolizumab - uterine cancer (combination with lenvatinib mesylate)
• Pembrolizumab - uterine cancer
• Zanubrutinib - Waldenström's macroglobulinaemia (first-line and later-line)

Once again, more submissions (submission requests) were recommended than rejected.