26 April 2024 - Recommendations made by the PBAC in March 2024 relating to the listing of medicines on the PBS and vaccines on the NIP are now available.

In the end, the PBAC considered 59 submissions. 

Four submissions were withdrawn:

• Cytosine arabinoside with daunorubicin (nanoparticle, liposomal) (Vyxeos) - acute myeloid leukaemia
• Glycomacropeptide with docosahexaenoic acid and low phenylalanine (PKU GMPro ULTRA) - phenylketonuria
• Pegcetacoplan (Syfovre) - age-related macular degeneration
• Venetoclax (Venclexta) - chronic lymphocytic leukaemia

Three submissions will be considered at a future PBAC meeting:

• Aflibercept (Eylea) - age-related macular degeneration
• Aflibercept (Eylea) - macular oedema
• Ravulizumab (Ultomiris) - myasthenia gravis

The 59 submissions yielded 93 submission requests and 93 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 (Yuflyma) - multiple diseases
• Atezolizumab (Tecentriq) - multiple diseases
• Avacopan (Tavneos) - granulomatosis with polyangiitis/microscopic polyangiitis
• Bevacizumab (Vegzelma) - multiple diseases
• Dabrafenib maleate/Trametinib dimethyl sulphoxide - brain cancer
• Evolocumab (Repatha) - Hypercholesterolaemia
• Ibrutinib (Imbruvica) - chronic lymphocytic leukaemia/non-Hodgkin's lymphoma
• Larotrectinib sulphate (Vitrakvi) - non-small cell lung cancer/sarcoma
• Tofacitinib citrate (Xeljanz) - rheumatoid arthritis/psoriatic arthritis
• Ustekinumab (Stelara) - multiple diseases
• Ustekinumab (Wezlana) - multiple diseases

The outcomes for the 93 considered submission requests are comprised of:

• 81 recommendations (87%)
• 11 rejections (12%)
• 1 deferral (1%)

The outcome for one submission (bulevirtide (Hepcludex)) cannot be made public until the TGA outcome is known.

The recommendation rate for this meeting (87%) is the highest recommendation rate in years. This rate is for considered submissions and thus does not include the 'outcomes' for withdrawn submissions. This result depends at least in part on whether one included the outcomes (expressed as other matters) as actual outcomes. This rate will change (albeit slightly) once the outcome for bulevirtide is known/made public.

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 submissions' than '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 = 9)
• A comparison against the lowest cost comparator (n = 17)
• A price reduction (either offered by the sponsor or sought by the PBAC) (n = 26). This includes comparisons against the lowest cost comparator.

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

• Anifrolumab (Saphnelo) - systemic lupus erythematosus
• Avacopan (Tavneos) - granulomatosis with polyangiitis/microscopic polyangiitis
• Cabozantinib maleate (Cabometyx) - Thyroid cancer
• Dabrafenib maleate/Trametinib dimethyl sulphoxide - brain cancer
• Edavarone (Radicava) - Amyotrophic lateral sclerosis
• Icosapent ethyl (Vazkepa) - hypertriglyceridaemia
• Larotrectinib sulphate (Vitrakvi) - non-small cell lung cancer/sarcoma
• Migalastat hydrochloride (Galafold) - Fabry disease
• Nivolumab (Opdivo) - bladder cancer
• Influenza vaccine (quadrivalent) (Flucelvax Quad) - vaccination
• Romosozumab (Evenity) - osteoporosis
• Selumetinib sulphate (Saphnelo) - neurofibroma
• Trastuzumab deruxtecan (Enhertu) - breast cancer

Insights:

• There were no surprise late agenda items (liraglutide for obesity, nivolumab/ipilimumab and pembrolizumab for multiple indications)
• With the high recommendation rate, most applicant companies fared well. AstraZeneca fared well with 4 recommendations for 4 submissions/submissions requests. Pfizer had a mixed bag with recommendations for Etrasimod arginine (Velsipity) and tofacitinib citrate (Xeljanz) but rejections for respiratory syncytial virus vaccine (bivalent) (Abrysvo) and rimegepant sulphate (Nurtec ODT).
• With the high recommendation rate, most submissions for a new medicine were recommended. Of the 13 submission requests for a new medicine, only 2 were rejected: osilodrostat phosphate (Isturisa) and rimegepant sulphate (Nurtec ODT).
• With the high recommendation rate, most submissions for a cancer medicine were recommended.  4 submissions for cancer medicine were rejected: brentuximab vedotin (Adcetris), irinotecan hydrochloride trihydrate (liposomal) (Onivyde), nivolumab (non-small cell lung cancer) and talazoparib tosylate (Talzenna)
  
• No medicine appears to have been considered by the PBAC for listing with a Managed Access Program
• No medicine appears to have been recommended with a price premium; two medicines were recommended on the basis of there being an additional price reduction (selumetinib sulphate (Koselugo) and trastuzumab deruxtecan (Enhertu)
• With the high recommendation rate, most submissions lodged under parallel process were recommended. On the limited available information, it is understood the following submissions lodged under the parallel process were rejected: brentuximab vedotin (Adcetris), irinotecan hydrochloride trihydrate (liposomal) (Onivyde), talazoparib tosylate (Talzenna)
• With the high recommendation rate, most resubmissions were recommended; the following resubmissions were rejected: nivolumab (Opdivo), osilodrostat phosphate (Isturisa) and rimegepant sulphate (Nurtec ODT).
• Of the 11 rejections one was due to an incorrect proposed main comparator, four due to uncertain clinical benefit and six due to uncertain cost effectiveness
• There were 3 submissions/submission requests for an NIP vaccine; two were recommended and the other was rejected
• There wasn't a submission for a LSDP medicine
• There does not appear to have been a submission for a medicine with a co-dependent technology 
• The outcomes provide no insights on acceptable/unacceptable ICERs
• No submission request appears to have been associated with real world evidence
• Two submissions/submissions requests have not be considered by other HTA agencies: adalimumab (Humira) for immune-mediated inflammatory disease and sitagliptin phosphate monohydrate with dapagliflozin propanediol monohydrate (Sidapvia) for type 2 diabetes mellitus
• The PBAC considered 2 submissions for a new biosimilar medicine: bevacizumab (Vegzelma) and ustekinumab (Wezlana); both were recommended