positions available now

Public Submissions to the August 2018 PBAC Special Meeting – Part 2

In the second part of this two-part series (Read Part 1 about here) we bring you further insight into opinions from the public submissions to the August 2018 PBAC Special Meeting.

Opinions from the Public Submissions

28 public submissions were received regarding the PBAC considerations for PD-1 and PD-L1 checkpoint inhibitors for multiple cancer types.


Do you think that different evidentiary requirements are appropriate for rare cancers?


YES – 64%   NO – 4%

No answer provided – 14%
Not published – 14%
Other* – 4%


* ”There are no set evidentiary requirements for applications seeking PBS listing of a drug, other than that the available data be presented systematically, in an unbiased manner and present evidence of comparative efficacy, safety and cost-effectiveness…”


How do you think cost-effectiveness should be established in this case?

A number of submissions commented on managed entry agreements and evidence development. A shared funded access mechanism could be applied where, after commencement, fit-for-purpose data could be collected prospectively to confirm the biological rationale and effectiveness of the product.

Multiple submissions stressed the generation of real-world outcome data for populations with rare and less common cancers as critical.

Do you think it is possible for the PBAC to satisfy itself that treatment with a PD-1 or PD-L1 checkpoint inhibitor is cost-effective without an economic model that is specific to that kind of cancer? How?

A number of submissions commented on the difficulties and challenges of determining cost-effectiveness without an economic model, or assessment of multiple indications within a single economic funding model. In the absence of multi-tumour data, there is significant uncertainty, and a greater tolerance of uncertainty may be justified in situations of high unmet need.

Some submissions provided possible approaches to establishing cost-effectiveness across cancer types:

  • Develop a separate model for each cancer type included in the multi-tumour list and perform separate cost-effectiveness analyses to identify the tumour type(s) where the drug is cost-effective
  • Develop a single, comprehensive model to simultaneously inform cost-effectiveness in multiple cancer types with the possibility of clustering different but related tumours types (e.g. similar natural progression, treatment approach). This model could allow estimation of incremental cost-effectiveness ratios (ICERs) of individual tumour types based on the relative prevalence and utilisation of the proposed drug in each tumour type
  • In the absence of an economic model, establish scores of clinical value (e.g. ESMO Magnitude of Clinical Benefit Scale (MCBS), ASCO value framework) and link price to scoring systems
  • Follow-on indication pathway: resources are devoted at the start of the agreement to define the populations and clarify expectations of the evidence and cost-effectiveness. For follow-on indications in the 3-year risk-sharing deeds, cost-effectiveness is verified at the end of the agreement period and this could be accomplished using a multi-indication model using pre-agreed principles
  • Rare cancer pathway: a pay-for performance type approach to medicines access should ideally not require the construction of, or evaluation of, a traditional PBAC-level economic model. This approach, in this population, controls expenditure and maximises value for public money by ensuring funds are spent when response (as a measure of effectiveness) is confirmed

FDA – 3 submissions referenced the FDA’s decision to consider pan-tumour indications and the approval of Keytruda® (pembrolizumab).

MA OIT – 4 submissions referred to the Medicines Australia’s (MA) Oncology Industry Taskforce’s (OIT) preliminary research into innovative international models for subsidy consideration across multiple indications. This found that there are various initiatives underway in overseas health systems (Belgium, Denmark, Netherlands, Germany and Italy). As per Medicines Australia: “While many of these agreements would not fit within the Australian legislative requirements, there are elements (e.g. initial horizon scanning of upcoming indications) that could be quite useful locally.” For further information, please refer to the submission by Medicines Australia: http://www.pbs.gov.au/industry/listing/elements/pbac-meetings/agenda/pdf/august-2018-special-meeting/submission-27-medicines-australia.pdf

Outcomes of the PBAC’s considerations will be published through the PBS website scheduled for 28 September 2018.

Abbreviations: PBAC = Pharmaceutical Benefits Advisory Committee; PD-1 = programmed cell death-1; PD-L1 = ligand of PD-1 receptor; ICER = Incremental Cost-Effectiveness Ratio; ESMO = European Society for Medical Oncology; MCBS = Magnitude of Clinical Benefit Scale; ASCO = American Society of Clinical Oncology; FDA = Food and Drug Administration; MA = Medicines Australia; OIT = Oncology Industry Taskforce

Source: Pharmaceutical Benefits Scheme


The Commercial Eyes Market Access team has extensive experience in pricing and reimbursement and can help you navigate Australia and New Zealand’s sophisticated and mature systems of Health Technology Assessment. Contact us on (03) 9251 0777 to learn more and let us help you envisage, achieve and defend the optimum market access outcomes for your business.

This article was written by Michelle Yassa, Market Access Consultant.

This entry was posted in Life Sciences Industry, Market Access, PBAC, PBS and tagged , , , , .
Copyright © 2020 Commercial Eyes