21 April 2023 - Recommendations made by the PBAC in March 2023 relating to the listing of medicines on the Pharmaceutical Benefits Scheme and vaccines on the National Immunisation Program are now available.

In the end, the PBAC considered 59 submission requests. Three submission requests were withdrawn:

• Larotrectinib sulphate (Vitrakvi) - cancer
• Riociguat (Adempas) - pulmonary arterial hypertension
• Risdiplam (Evrysdi) - one of three patient populations (spinal muscular atrophy)

A further two submissions will be considered at a future meeting:

• Risperidone (Okedi) - schizophrenia
• Upadacitinib monohydrate (Rinvoq) - Crohn's disease

The PBAC considered a few submissions that did not appear in the most recent agenda (dated 7 March 2023):

• Deucravitinib (Sotyktu) - psoriasis
• Elexacaftor with tezacaftor and ivacaftor and ivacaftor (Trikafta) - cystic fibrosis
• Risedronate sodium (Actonel EC) - osteoporosis
• Trastuzumab deruxtecan (Enhertu) - breast cancer
• Upadacitinib monohydrate (Rinvoq) - axial spondyloarthritis

The 59 considered submission requests yielded 67 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
• Alirocumab (Praluent) - hypercholesterolaemia
• Daratumumab/daratumumab with hyaluronidase (Darzalex) - multiple myeloma
• Fosnetupitant chloride hydrochloride with palonosetron hydrochloride (Akynzeo) - nausea and vomiting
• Pemetrexed disodium heptahydrate (Pemetrexed Ever Pharma) - non-small-cell lung cancer/mesothelioma
• Pneumococcal polysaccharide conjugate vaccine (15 valent, adsorbed) - vaccination
• Risdiplam (Evrysdi) - spinal muscular atrophy
• Varicella zoster virus vaccine (adjuvanted) (Shingrix) - vaccination
• Zanubrutinib (Brukinsa) - chronic lymphocytic leukaemia/small lymphocytic leukaemia

The outcomes for the 59 considered submission requests are comprised of:

• 42 recommendations (63%)
• 20 rejections (30%)
• 5 deferrals (7%)

The recommendation rate for this meeting (63%) is comparable to the corresponding rate for the November 2022 meeting (58%). These rates are for considered submissions and thus do not include the outcomes for withdrawn submissions.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 = 12)
• A comparison against the lowest cost comparator (n = 11)
• A price reduction (either offered by the sponsor or sought by the PBAC) (n = 23). This includes comparisons against the lowest cost comparator.

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

• Abemaciclib (Verzenio) - price reduction, risk sharing agreement
• Deucravitinib (Sotyktu) - price reduction
• Elexacaftor with tezacaftor and ivacaftor and ivacaftor (Trikafta) - presumably a risk sharing agreement
  
• Enzalutamide (Xtandi) - price reduction, risk sharing agreement
• Finerenone (Kerendia) - price reduction offered in pre-PBAC response
• Pembrolizumab (Keytruda) - cost effective at the (reduced?) price proposed in the pre-PBAC response
• Romosozumab (Evenity) - price reduction, risk sharing agreement
• Trastuzumab deruxtecan (Enhertu) - price proposal, risk sharing agreement
• Varicella zoster virus vaccine (adjuvanted) (Shingrix)
  

At least 5 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.

Outcomes of note include:

• Apremilast - Amgen can still not crack it for a recommendation for the treatment of patients with psoriatic arthritis after the use of one or more conventional synthetic disease modifying anti-rheumatic drugs (csDMARDs) (i.e., methotrexate, sulfasalazine and/or leflunomide), but are ineligible or clinically inappropriate for treatment with a biologic or targeted synthetic disease modifying anti-rheumatic drug (bDMARD/tsDMARD).
• Enzalutamide - recommended for the treatment of patients with metastatic hormone sensitive prostate cancer irrespective of disease volume or suitability for docetaxel. The PBAC considered that enzalutamide, in combination with androgen deprivation therapy (ADT), provides a moderate clinical benefit compared to ADT alone. It should be priced no higher than apalutamide.
• Inclisiran - not recommended for the treatment of patients with hypercholesterolaemia and atherosclerotic cardiovascular disease. The PBAC considered the proposed positioning of inclisiran as a second-line alternative to ezetimibe was not justified. The PBAC considered inclisiran would be most appropriately positioned as an alternative to the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in the third-line setting. To address the uncertainty in the non-inferiority claim, an early reentry resubmission with a lower price than evolocumab would be required to support PBS listing in the same treatment setting as evolocumab for non-familial hypercholesterolaemia and heterozygous familial hypercholesterolaemia.
• Pembrolizumab - recommended for patients with unresectable/metastatic triple negative breast cancer but rejected for early satge triple negative breast cancer
• Pneumococcal polysaccharide conjugate vaccine (15 valent, adsorbed) (Vaxneuvance) - recommended for the prevention of pneumococcal disease in different paediatric patient populations and different dosing regimens for different states/territories.
• Romosozumab (Evenity) - recommended for the treatment of patients with severe osteoporosis in the first-line setting. The PBAC considered the clinical and cost effectiveness evidence for romosozumab was adequate to support listing in the first-line setting but not an expansion to the current second-line listing.
• Varicella zoster virus vaccine (adjuvanted) (Shingrix) - recommended for non-indigenous individuals aged 70 years, Aboriginal and Torres Strait Islander individuals aged ≥50 years and immunocompromised individuals aged ≥18 years with conditions at ‘high risk’ of herpes zoster infection [as advised by Australian Technical Advisory Group on Immunisation (ATAGI)]. The target patient population for the initial submission was adults 60-69 years of age.

Read PBAC outcomes