31 October 2018 - A thorough examination of the Public Summary Documents from the July 2018 PBAC meeting has identified some important information not provided in the official PBAC outcomes.

Additional submissions

The PBAC considered a small number of minor submissions not cited in the published agenda:

• Correspondence from the RMSANZ regarding the PBS listings of botulinum toxin type A
  
• Correspondence from a clinician requesting that the PBAC consider changing the number of repeats and authority level of imatinib continuing treatment for rare diseases, to be consistent with imatinib continuing treatment for other indications such as CML in the chronic phase
  
• A request from an undisclosed party to permit the administration of nusinersen sodium (Spinraza) in a private hospital setting to a patient with SMA undergoing a separate procedure under general anaesthetic.
  

Additional outcomes

• Contrary to the PBAC official outcomes, the submission for lumacaftor with Ivacaftor (Orkambi) for children aged 6-11 years was deferred; the official PBAC outcomes states that it was recommended.  The submission was recommended at a later date at an out-of-session meeting.
  
• Lenvatinib mesylate (Lenvima) has since been recommended by the PBAC for the first-line treatment of patients with unresectable hepatocellular carcinoma on a cost minimisation basis with sorafenib tosylate

Out of session outcomes

The following medicines/combination products were in fact recommended at an out-of-session meeting:

• Lumacaftor with ivacaftor (Orkambi)
  
• Lenvatinib (Lenvima)

Medicines recommended with a RSA or MAA

The following medicines/combination products were recommended with a risk share agreement (RSA) or a managed access program (MAP):

• Avelumab (RSA)
  
• Golimumab (RSA)
  
• Guanfacine hydrochloride (RSA)
  
• Lanreotide acetate (RSA)
  
• Ivacaftor with lumacaftor (MAP)
  
• Ivacaftor with lumacaftor (MAP)
  
• Midostaurin (RSA)
  
• Nivolumab (malignant melanoma) (RSA)
  
• Pembrolizumab (NSCLC) (RSA)
  
• Tolvaptan (RSA)
  
• Trifluridine with tipiracil hydrochloride (RSA)
  

Inconsistencies

The first tranche of Public Summary Documents (PSDs) from the July 2018 PBAC meeting includes seven previously published PSDs that have been updated based on the outcomes from the July 2018 meeting. These medicines/medicinal preparations have been considered at previous PBAC meetings.  This appears to be a new way of publishing the PSDs. This is helpful; however:

• If the DoH now plans to produce one PSD for a medicine/medicinal preparation/vaccine that results in multiple submissions (and thus outcomes), then why are there multiple PSDs for midostaurin (2 submissions), tolvaptan (4 submissions) and trifluridine with tipiracil hydrochloride (6 submissions)?
  
• The PSD for lumacaftor with ivacaftor for patients aged 12 years and older makes reference to the third and fourth submissions but not the first and second submissions
  
• The header of the PSD for guselkumab (Tremfya) is 'Public Summary Document - March 2018 PBAC meeting.' The document starts with a discussion of the submission/outcome from the March 2018 meeting and ends with an addendum that discusses the submission/outcome from the July 2018 PBAC meeting. The header of the PSD for lumacaftor with ivacaftor (Orkambi) for patients aged 12 years and older is 'Public Summary Document - July 2018 PBAC meeting.' The document starts with a discussion of the submission/outcome from the July 2018 PBAC meeting and ends with an appendix that discussses the submission/outcome from the July 2017 PBAC meeting. If one's usual practice is to scroll to the end of the document to reveal the PBAC's outcome and related discussion, one could easily assume that the fourth submission for Orkambi was rejected.