16 December 2022 - The outcomes from the November 2022 PBAC meeting are now available.

In the end, the PBAC considered 62 submission requests. Seven submission requests were withdrawn:

• Acalabrutinib - chronic lymphocytic leukaemia and non-Hodgkin's lymphoma
• Alirocumab - hypercholesterolaemia
• Ibrutinib - chronic lymphocytic leukaemia and non-Hodgkin's lymphoma
• Ranibizumab - age-related macular degeneration
• Sotorasib - non-small-cell lung cancer

The Committee considered four submissions that were not noted in the most recent version of the agenda for the November 2022 meeting:

• Risankizumab - Crohn's disease
• Ruxolitinib phosphate - graft versus host disease
• Upadacitinib monohydrate - ulcerative colitis
• Vosoritide - achondroplasia

The 43 considered submissions yielded 62 outcomes. The outcome count could increase in time as more information become available with the publication of the Public Summary Documents.

Some submissions are associated with multiple patient populations (multiple requests):

• Adalimumab (Humira) - multiple diseases
• Asciminib hydrochloride (Scemblix) - multiple patient populations for chronic myeloid leukaemia
• Etanercept (Nepexto) - multiple diseases
• Lanreotide acetate (Mytolac) - multiple diseases

The 62 outcomes are comprised of:

• 36 recommendations (58%)
• 19 rejections (31%)
• 5 deferrals (8%)
• 2 no outcomes (3%)

The recommendation rate for this meeting (58%) is comparable to the corresponding rate for the July 2022 meeting (61%). These rates are for considered submissions and thus do not include the outcomes for withdrawn submissions.

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 cancer medicines which are generally more challenging.

Having said that, we also note that quite a few of the outcomes are associated with:

• A risk sharing agreement (n = 8)
• A comparison against the lowest cost comparator (n = 8)
• A price reduction (either offered by the sponsor or sought by the PBAC) (n = 14). This includes comparisons against the lowest cost comparator.

The following medicines were recommended on a CEA basis:

• Mepolizumab (Nucala) - risk sharing agreement
• Nivolumab (Opdivo) - further price reduction
• Ruxolitinib phosphate - risk sharing agreement
• Vosoritide - risk sharing agreement

At least 7 CEA submission requests were rejected or deferred. There may be more as the economic evaluation in some submissions cannot be determined from the information available.

Only three of the ten submission requests for a cancer medicine (new medicine, new indication or new combination product) were recommended.

Outcomes of note include:

• Asciminib hydrochloride (Scemblix) - recommended for four patient populations for chronic myeloid leukaemia; previously rejected for just one patient population
• Deucravacitinib (Sotyku) - The PBAC deferred making a recommendation in order to seek additional expert clinical advice on its appropriate and likely place in therapy.
• Dostarlimab (Jemperli) - rejected again. This one is proving to be difficult given the best available clinical evidence is a single-arm study.
• Elexacaftor with tezacaftor and ivacaftor and ivacaftor (Trikafta) - recommended for children aged 6-11 years with at least one F508del mutation
• Mavacamten (Camzyos) - The PBAC considered it is likely that mavacamten with or without beta-blocker/calcium channel blocker provided a short-term, moderate clinical benefit over standard of care, in terms of symptomatic improvement, but the longer-term clinical benefit and safety and the impact on other patient-relevant clinical outcomes such as hospitalisations or mortality is unknown.
• Mobocertinib succinate (Exkivity) - The PBAC considered the nominated comparator of standard care, comprising EGFR tyrosine kinase inhibitors, immune checkpoint inhibitor monotherapy, and chemotherapy was inappropriate as EGFR tyrosine kinase inhibitors and checkpoint inhibitors are generally not used in these patients.
• Olaparib (Lynparza) - The PBAC considered that a further price reduction would be required to ensure cost effectiveness given the remaining uncertainty in the modelled benefit of olaparib in the proposed population.
• Onasemnogene abeparvovec (Zolgensma) - The PBAC considered for onasemnogene abeparvovec to be similarly cost effective in the expanded population that the price would need to be substantially less than for the current listing.
• Patiromer sorbitex calcium (Veltassa) - The PBAC advised that it would be more appropriate for the price of patiromer sorbitex calcium to be based on a cost minimisation approach versus sodium polystyrene sulphonate or calcium polystyrene sulphonate resins given that these resins could be used to treat the overall target population.
  
• Pembrolizumab (Keytruda) - The PBAC considered that pembrolizumab would be cost effective for patients with cervical cancer with a price reduction.
• Selinexor (Xpovio) - The PBAC considered that selinexor could be considered cost effective if its price was determined on the basis of the cost minimisation of selinexor and bortezomib and dexamethasone versus carfilzomib and dexamethasone presented in the resubmission, after the inclusion of the confidential effective price for carfilzomib and an adjusted weighting for the use of carfilzomib once weekly and twice-weekly regimens.

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