In the first of a two-part series, we provide insights into opinions from 28 public submissions to the August 2018 PBAC Special Meeting held on 17 August 2018 regarding considerations for PD-1 and PD-L1 checkpoint inhibitors for multiple cancer types
28 public submissions were received regarding the PBAC considerations for PD-1 and PD-L1 checkpoint inhibitors for multiple cancer types.
- Consumer organisation/research
- Government HTA agency
- Pharmaceutical industry
- Professional organisation
- Research and other – PBAC evaluation group
Opinions from the Public Submissions
Potential advantages of the PBAC’s consideration:
- Timely & equitable access
- Provides clinicians with flexible treatment options
- Efficiency gains in the assessment & evaluation of data
- Reduces PBAC workload
- Budget certainty for government & sponsors
- Opportunities for innovative pricing arrangements
- Maintain reputation as a world leader in HTA & delivery of innovative medicines to Australians
- Social justice
Potential disadvantages of the PBAC’s consideration:
- Uncertainty with efficacy & safety data, cost-effectiveness & budget impact
- Facilitating access to treatments that may be ineffective and/or harmful – opportunity cost
- Accessibility to biomarker testing
- Indication leakage
- Timing may be premature given the number of ongoing trials, putting further PBAC applications at risk of heavy scrutiny
- Equity across therapeutic areas
- Increasing financial burden & burden on evaluation of cost-effectiveness
- Limitations of current regulatory framework
What is the minimum level of evidence of effectiveness you think should be required before PD-1 and PD-L1 checkpoint inhibitors are considered for subsidy for a particular kind of cancer?
Aside from commenting on flexibility or responses not published/provided, five public submissions all stated that earlier phase, single-arm evidence should be made acceptable. Four submissions stressed the importance of subgroup analyses according to diagnostic assays such as biomarker expression.
The three government HTA agencies who provided public submissions had differing views: no minimum level of evidence should be required, single-arm evidence should be acceptable, or there should not be any difference in the level of effectiveness required for treatment subsidy in any therapeutic area.
Other views included evidence development and managed entry agreements rather than extrapolating evidence across tumours, with real-world data collected prospectively to confirm the biological rationale and effectiveness. A few submissions commented on a pay for performance model in the case of rare cancers, where effectiveness would be determined at the level of the individual.
Tune in next week for Part 2 of this series.
Abbreviations: PBAC = Pharmaceutical Benefits Advisory Committee; PD-1 = programmed cell death-1; PD-L1 = ligand of PD-1 receptor; HTA = Health Technology Assessment; MSAC = Medical Services Advisory Committee; IFNγ = Interferon gamma; TMB = Tumour Mutational Burden; dMMR = deficient mismatch repair; MSI-H = High Microsatellite Instability
Source: Pharmaceutical Benefits Scheme
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This article was written by Michelle Yassa, Market Access Consultant.