18 August 2023 - Recommendations made by the PBAC in July 2023 relating to the listing of medicines on the PBS and vaccines on the NIP are now available.

In the end, the PBAC considered 66 submission requests. Two submission requests were withdrawn:

• Daprodustat (Jesduvroq) - anaemia
• Foslevodopa with foscarbidopa (Vyalev) - Parkinson's disease

The PBAC considered a few submissions that did not appear in the most recent agenda (dated 18 July 2023):

• Nivolumab (Opdivo) - oesophageal cancer
• Nivolumab with relatlimab (Opdualag) - malignant melanoma
• Trastuzumab deruxtecan (Enhertu) - breast cancer
• Upadacitinib monohydrate (Rinvoq) - Crohn's disease
• Zoledronic acid (APO-Zoledronic Acid) - breast cancer

The 48 considered submission requests yielded 64 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):

• Acalabrutinib (Calquence) - chronic lymphocytic leukaemia/small lymphocytic leukaemia
• Adalimumab (Ardalicip) - multiple diseases
• Avacopan (Tavneos) - granulomatosis with polyangiitis, microscopic polyangiitis
• Cytosine arabinoside with daunorubicin (nanoparticle, liposomal) (Vyxeos) - acute myeloid leukaemia
• Enoxaparin sodium (Exarane, Exarane Forte) - venous thromboembolism
• Olipudase (Xenpozyme) - Niemann-Pick disease
• Varicella zoster virus vaccine (adjuvanted) (Shingrix) - vaccination

The outcomes for the 48 considered submission requests are comprised of:

• 44 recommendations (67%)
• 18 rejections (27%)
• 2 deferrals (3%)

The recommendation rate for this meeting (67%) is comparable to the corresponding rate for the November 2022 meeting (63%). These rates are for considered submissions and thus do not include the outcomes for withdrawn submissions.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 = 10)
• A comparison against the lowest cost comparator (n = 14)
• A price reduction (either offered by the sponsor or sought by the PBAC) (n = 17). This includes comparisons against the lowest cost comparator.

The following medicines/vaccines were recommended on a CEA basis:

• Durvalumab (Imfinzi)
• Lumacaftor with ivacaftor (Orkambi) - risk sharing agreement
• Mobocertinib succinate (Exkivity) - price reduction offered
• Nivolumab (Opdivo) - risk sharing agreement
• Olaparib (Lynparza) - price reduction offered
• Onasemnogene abeparvovec (Zolgensma) - price reduction required
• Pembrolizumab (Keytruda) - price proposed, risk sharing agreement
• Tafamidis (Vyndamax) - price reduction offered, risk sharing agreement
• Trastuzumab deruxtecan (Enhertu)
• Varicella zoster virus vaccine (adjuvanted) (Shingrix) - price reduction offered

Insights

• AstraZeneca is the biggest winner for this meeting with recommendations for acalabrutinib (Calquence), dapagliflozin propanediol monohydrate (Forzxiga), durvalumab (Imfinzi) and trastuzumab deruxtecan (Enhertu)
• 15 of the 20 submission requests for a cancer medicine were recommended; the remaining 5 were rejected
• 11 of the 24 submission requests associated with a CEA were recommended; the rest were rejected
• The PBAC considered two medicines associated with a co-dependent technology (niraparib tosylate monohydrate (Zejula) and olaparib (Lynparza). Both are associated with the same MSAC application (1658/1658.1) and both medicines were recommended. Interestingly, the PBAC outcome for each medicine makes reference to an MSAC outcome (recommendation) from March 2023. The outcome for application 1658.1 has not been published on the MSAC website.
• There seems to be an increasing trend for recommended medicines requiring another submission. The PBAC considered a resubmission for ixekizumab (Taltz) for patients with non-radiographic axial spondyloarthritis following its recommendation in November 2021; the resubmission was rejected. The PBAC considered a resubmission for nivolumab with relatlimab (Opdualag) for patients with malignant melanoma following its recommendation in March 2023; the resubmission was recommended.
• The PBAC rejected the submission for olipudase (Xenpozyme) for patients with Niemann-Pick disease. It is unclear if it will head straight to the LSDP.
• The third submission for esketamine hydrochloride (Spravato) was rejected; the fourth submission for tafamidis (Vyndamax) was recommended
• Tirzepatide (Mounjaro) will only be recommended for certain patients with type 2 diabetes mellitus following a substantial price reduction

Read PBS News