Pharmaceutical Law, Reform and RegulationGuidelines for Submissions to the PBAC

V5: Translating Legislation and Policy into HTA Guidance

Professor Andrew WilsonChair, PBAC

Australian Government Department of Health

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Themes

• Context of PBAC Decision Making

• Current Issues and Challenges

• Key Factors Influencing the Decision Making

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Context of PBAC Decision making• Established under the National Health Act 1953. • Recommends to the Minister for Health which

medicines should be subsidised under the PBS. • Required, under the Act, to consider the effectiveness

and cost of the proposed medicine compared with existing (alternative) therapies (s101).

• It cannot make a positive recommendation for a medicine that is substantially more costly than an alternative medicine unless it is satisfied that the proposed medicine also provides a significant improvement in health.

• Unchanged since V4

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Changing EnvironmentIt’s an Exciting Time

• New drugs.

– More effective drugs in new classes

– Drugs where no previous pharmaceutical option

• New approaches.

– Many drugs need to be taken for indefinite period or for indefinite number of cycles

• A changing industry.

• Growing community expectations.

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Some Major Issues• Quality and quantity of evidence of effectiveness

and harm particularly with pressure to bring to market earlier.

• Place in therapy especially for new therapeutic approaches rather new generation within class.

• Targeting of therapy – molecular markers

• Biosimilars

• Price expectations and global price setting.

• Cost of incremental increases.

• Patient and clinician inputs.5

Key factors influencing decision making by the PBAC

• Comparative health gain.

– Assessed in terms of both the magnitude of effect and clinical importance of effect.

– Relevance and validity of measured outcome

– Minimum clinical important differences

– Maturity of the trial data

– Cross-overs

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Key factors influencing decision making by the PBAC

• Comparative cost-effectiveness. – Presented as incremental cost-effectiveness ratios

(including incremental cost-utility ratios) or a cost-minimisation approach.

– Includes a consideration of comparative costs, including the full spectrum of cost offsets.

– Comparators• Therapy most likely to be replaced in clinical practice.

• Clinical practice effects

• Pricing effects

• Recency effects

• Treatment positioning effects

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Key factors influencing decision making by the PBAC

• Patient affordability in the absence of PBS subsidy.

– Presented as cost per patient per course for acute or self-limited therapy, or cost per patient per year for chronic or continuing therapy.

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Key factors influencing decision making by the PBAC

• Predicted use in practice and financial implications for the PBS.

– Presented as the projected annual net cost to the PBS/RPBS or the NIP.

– Use beyond restrictions

– Use beyond indications

– Ongoing vs intermittant therapy

– Predictability of market (share)

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Key factors influencing decision making by the PBAC

• Predicted use in practice and financial implications for the Australian Government health budget.

– Presented as the projected annual net cost per year.

– Implications for forward budget

– Other costs to government

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Other less-readily quantifiable but influential factors

• Overall confidence in the evidence and assumptions relied on in the submission. – Increases the likelihood that a risk management

approach will be required.

– Risk Share vs Managed Access

• Equity. – Implicit equity and ethical assumptions, such as

age, or socioeconomic and geographical status, may vary for different submissions and need to be re-evaluated on a case-by-case basis.

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Other less-readily quantifiable but influential factors

• Presence of effective alternatives.

– Help to determine the clinical need for the proposed medicine.

– Is there a potential opportunity cost consequence?

• Severity of the medical condition treated.

– The emphasis is on the nature and extent of disease as it is currently managed.

– Relates to any restrictions.

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Other less-readily quantifiable but influential factors

• Ability to target therapy with the proposed medicine precisely and effectively to patients likely to benefit most.

– The cost-effectiveness of the proposed medicine may not be in patients likely to benefit the most.

– Claims of benefits that are greater than the average result from an intention-to-treat analysis should be supported by appropriate trial evidence.

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Other less-readily quantifiable but influential factors

• Other public health considerations.

– Eg Development of resistance. The PBAC supports the prudent use principles for antimicrobials, and may consider specific advice on the likely extent of the development of resistance to a new antimicrobial agent and appropriate management strategies that might be applied through a PBS listing.

– Eg Impacts of new vaccines on immunisation schedules

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In Conclusion

• The PBAC guidelines and processes continue to evolve.

• More to come (patient/consumer inputs, biosimilars).

• The decisions are getting harder because of greater uncertainty in the evidence of benefit and harm and the size of benefits relative to cost.

• The total cost consequences are becoming greater.

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