1.1 Clinical issue addressed by the submission
Page last updated: September 2016
- Summarise the rationale for listing the medicine and tabulate the key components of the clinical claim (Subsection 1.1.1)
- Describe the target population and disease or condition in the Australian setting (Subsection 1.1.2)
- Describe the proposed intervention, justify the main comparator and identify key differences between them (Subsection 1.1.3)
- Provide the dates of previous PBAC submissions for the medicine; for resubmissions, describe how the current resubmission addresses the main PBAC concerns (Subsection 1.1.4)
1.1.1 Rationale for listing
Outline the expected impact of the proposed medicine in terms of patients’ health, health-related costs or cost offsets, and the impact on issues such as access or equity. Limit your response to less than half a page.
Under the National Health Act 1953, the primary objective of the PBS is to improve health, so the PBAC primarily focuses on health outcomes. The details of nonhealth-related impacts of the proposed medicine should be presented as supplementary analyses in Section 2 and/or Section 3, or discussed in Section 5 as other relevant factors.
Tabulate the proposed population, intervention, comparator, key effectiveness and safety outcome(s), and the overall clinical claim for the proposed medicine in Table 1.1.1.
Briefly state the target disease or condition and population to be treated
Briefly describe the intervention
Briefly describe the comparator
Briefly state the patient-relevant clinical effectiveness and safety outcomesa
State the clinical claim that the submission presents as follows: ‘In [population and health issue], [proposed medicine] is no worse than/as effective as/more effective than [main comparator] at improving/reducing [outcome(s)]’
a Outcomes should be directly related to the quality and/or length of a patient’s life.
1.1.2 Target population and disease or condition
Provide an overview of the disease or condition that can be treated by the proposed medicine. Include enough detail of diagnosis, symptoms, prognosis and other related issues to assist the assessment of the submission.
If the medicine is proposed for use in a subgroup(s) of the Australian population with the disease or condition, indicate whether the usual course of the disease or condition – or the available treatment options for that subgroup(s) – differs from that of the whole population.
Describe the Australian population who would be treated with the proposed medicine, such as their age, sex, important comorbidities, and disease- or condition-related characteristics. Provide data (preferably include Australian datasets or studies involving Australian participants). Summarise the incidence and prevalence of the disease or condition in Australia using data from a reputable source, such as those listed in ‘Sources of data for use in generating utilisation estimates’ (see also Section 4).
Where data sources involving Australian participants are not available, discuss whether population characteristics presented here are likely to be representative of the Australian setting. Include percentages and means with estimates of uncertainty (eg interquartile range, standard deviation and ranges) for these data, where possible.
Pharmacological action and therapeutic class of the proposed medicine
Present the therapeutic class, Anatomical Therapeutic Chemical classification and a description of the pharmacological action of the proposed medicine. Provide enough detail to support the proposed target Australian population described in Subsection 1.1.2 and the proposed listing. Tabulate this information to allow an easy comparison of the proposed medicine with the comparator(s).
Selection of the comparator(s)
Select the comparator(s) in the context of the targeted Australian population, the current alternative therapies in Australia, and the therapies most likely to be replaced in clinical practice. A single comparator will be appropriate in most circumstances.
Most comparators will be one of the following:
- A current PBS listed medicine. If the proposed medicine is likely to replace listed PBS medicines, the relevant comparator would be a medicine prescribed on the PBS to treat that target population.
- Standard medical management. If the proposed medicine is for a target population for which there are no currently listed PBS medicines, or the proposed medicine will be used in addition to – rather than replace – a medicine, the comparator would usually be standard medical management. Standard medical management would need to be clearly defined and could include a non-listed medicine, a surgical procedure, best supportive care or conservative management. In the absence of a PBS-listed medicine, standard medical management may be to use a medicine that is not PBS listed. In this circumstance, this medicine may be the appropriate comparator.
Choosing the medicine most likely to be replaced
Where there is more than one comparator, the main comparator should be the therapy that prescribers would most replace with the proposed medicine. The PBAC bases its judgment about the main comparator on what would be likely to happen, rather than what should happen, in keeping with the above approach to the main comparator.
The following general hierarchy is intended to assist in selecting the appropriate main comparator:
(a) An existing pharmacological analogue. If the proposed medicine is in a therapeutic
class for which pharmacological analogues are already listed, the main comparator
would usually be the analogue that is prescribed on the PBS for the largest number of
patients in the target population. Reference to the TGA-approved indications, to trial
evidence, or to any other authority, would not usually constitute reasonable grounds to
exclude an unrestricted pharmacological analogue as a main comparator.
(b) New therapeutic class. If the proposed medicine is in a new therapeutic class
would be used for a target population for which there are other, widely used, listed
medicines, the main comparator would usually be the medicine that is prescribed on the
PBS for the largest number of patients in the target population.
(c) Manner of administration. A particular manner of administration of the proposed
medicine (for example, injected or instilled as an eye drop rather than taken orally) is
also a significant consideration.
Near market comparator
If there is a reasonable expectation that another medicine will enter the Australian market for the targeted Australian population, and that it might be considered at the same or an adjacent PBAC meeting, then it would be prudent to regard this other medicine as an additional contingency comparator to inform a PBAC consideration across the new competing medicines.
Different comparators for different subpopulations or different target populations
It may be appropriate to use different comparators for different subpopulations where the overall target population for the proposed medicine includes one or more sub-populations and:
- the proposed medicine is claimed to be significantly more effective or significantly less toxic than the main alternative comparator therapy in the subpopulation(s) (but not in the remainder of the target population), or,
- where the main comparator therapy used to treat the overall target population cannot be used. That therapy is, therefore, not an alternative therapy for that subpopulation.
In these circumstances, it may be useful also to provide information permitting a comparison with other alternative therapies for the subpopulation(s). The PBAC will usually assess the overall cost and effectiveness of the proposed medicine, considering:
- the evidence justifying the claim for the difference in responses for different populations and therefore for the alternative comparator
- whether the price of the proposed medicine reflects the comparisons for the particular subpopulations
- the size of the subpopulations as a proportion of the overall target population.
Similar considerations are likely to apply where listing of the proposed medicine is sought for more than one target population, and the alternative therapies are different for some of the populations.
Tabulate the dates of previous PBAC considerations (and the listing dates, where applicable) for all indications for which the proposed medicine sought listing in Table 1.1.2.
PBAC meeting date(s)
Listing date (if applicable)
For resubmissions, present a table with a summary of the issues raised by the PBAC (cross-reference to the PBAC minutes), and show how the current resubmission addresses the issues, with cross-referencing to the relevant sections of the current resubmission.