19 August 2022 - The recommendations from the July 2022 PBAC meeting are now available.

In the end, the PBAC considered 51 submission requests. Five submission requests were either withdrawn or not considered:

• Cemiplimab (Libtayo) - withdrawal was previously noted/advised
• Cytosine arabinoside with daunorubicin (nanoparticle, liposomal) (Vyxeos) - withdrawal was previously noted/advised
• Risankizumab (Rinvoq) - the request for patients with for severe, refractory fistulating Crohn’s disease was withdrawn; the associated submission request for patients with severe Crohn’s disease was not
• Tezepelumab (Tezspire) - the submission for patients with severe uncontrolled allergic and/or eosinophilic asthma (two requests) will be considered at a future PBAC meeting. No further details are available.

The Committee did not any submissions that were not noted in the most recent version of the agenda for the July 2022 meeting.

The 39 submissions yielded 56 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
• Risankizumab - Crohn's disease - severe and severe, refractory fistulating
• Romosozumab (Evenity) - first-line and second-line use
• Ruxolitinib phosphate (Jakavi) - acute graft versus host disease and chronic graft versus host disease
• Tezepelumab (Tezspire) - allergic asthma and eosinophilic asthma
• Ustekinumab - Crohn's disease and psoriasis

The 56 outcomes are comprised of:

• 31 recommendations (55%)
• 18 rejections (32%)
• 2 deferrals (4%)
• 5 no outcomes (9%)

The recommendation rate for this meeting (55%) is much lower than the corresponding rate for the March 2022 meeting (78%). The recommendation rate rises to 61% if the five withdrawn submission requests are excluded.

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. We will have more to say on this in an coming issue of MAESTrO Daily.

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

• A risk sharing agreement (n = 5)
• A comparison against the lowest cost comparator (n = 6)
  
• A price reduction (either offered by the sponsor or sought by the PBAC) (n = 14). This includes comparisons against the lowest cost comparator.

We will examine this further at a later date.

The following medicines were recommended on a CEA basis:

• Apalutamide (Erlyand) - price reduction required and a risk sharing agreement 
• Atezolizumab (Tecentriq) - risk sharing agreement
• Ruxolitinib phosphate (Jakavi) - only acute graft versus host disease
• Sapropterin dihydrochloride (Kuvan) - substantial price reduction required and a risk sharing agreement
• Vericiguat (Verquvo) - risk sharing agreement

At least 12 CEA submission requests were rejected. There may be more as the economic evaluation in some submissions cannot be determined from the information available.

Only two of the nine submissions for a cancer medicine (new medicine, new indication or new combination product) were recommended.

Outcomes of note include:

• Atezolizumab (Tecentriq) - the first medicine to be recommended for patients with early (stage II to IIIA) non-small-cell lung cancer. Use following surgery and platinum-based adjuvant chemotherapy.
• Cannabidiol (Epidyolex) - the fourth submission was rejected. The base case ICER ($75,000 - $95,000/QALY gained) was too high and needs to be in the range of $45,000 - $55,000/QALY gained. There is no explicit mention of a (substantial) price reduction but if it looks like a duck....
• Esketamine hydrochloride (Spravato) - the second submission was rejected. The PBAC considered the cost effectiveness of esketamine hydrochloride "remained highly uncertain and not cost effective." A stakeholder meeting is looming.
• Nivolumab with relatlimab - the submission was rejected because the PBC did not accept nivolumab as monotherapy as the comparator. It is unclear why the PBAC has expressed Opdualag as 'relatlimab with nivolumab' as the former has been added to the latter rather than the other way around. The PBAC's preferred comparator for nivolumab with relatlimab is unknown.
• Ustekinumab (Stelara) - the cost should be no greater than the cost of the alternative therapies (excluding adalimumab)
• The submission for tezepelumab will "be considered at a future PBAC meeting" whatever that means. The same statement was made for a submission for another AstraZeneca medicine roxadustat (Evrenzo) back in March last year. It is yet to appear on an agenda since then, including the agenda for the November 2022 meeting.

 PBAC outcomes