About the Guidelines
Page last updated: September 2016
These Guidelines for preparing a submission to the Pharmaceutical Benefits Advisory Committee (PBAC Guidelines), version 5.0, assist applicants to prepare a submission to the PBAC for the public funding of a new medicine or medicinal product as part of the Pharmaceutical Benefits Scheme (PBS).
The guidelines reflect best practice as far as possible, and seek to maximise the confidence of the PBAC in accepting the many inferences necessarily made in submissions for public funding. They are designed to facilitate the evaluation and translation of the best available comparative clinical evidence for the requested PBS listing, followed by the most appropriate economic evaluation. They also ensure that the predicted use of the medicine in clinical practice is aligned with a standardised Excel workbook to allow these analyses to be presented consistently across submissions. However, although the guidelines present the currently preferred approach to the preparation of submissions to the PBAC, the approach is not prescriptive. Alternative approaches are permitted when adequately justified and supported by data.
These guidelines are available as an online resource at the PBAC Guidelines website. The site also includes additional information about the:
- role of the PBAC
- different types of submissions
- rationale and basis that the PBAC uses for an economic evaluation
- timeline for PBAC procedures
- PBAC process.
A submission template, Excel workbook, and other forms and checklists to help prepare a submission are all provided on the ‘Downloads’ section of this website.
The PBAC considers submissions from industry sponsors of medicines and medicinal products, medical bodies, health professionals, and private individuals and their representatives. However, for new products or new indications, it is normally the sponsor or manufacturer who holds the data required for such a submission. Sponsors usually engage public health and health economics experts to review the academic literature and help the company prepare a submission to the PBAC. These guidelines are primarily to assist these people in their task.
A submission to the PBAC for listing a proposed new medicine on the PBS consists of five sections:
- Section 1 – Context. Describes the proposed medicine, its intended use on the PBS and rationale for funding, and the therapy(ies) likely to be most replaced by prescribers in practice (the ‘main comparator’).
- Section 2 – Clinical evaluation. Provides the best available evidence comparing the clinical performance of the proposed medicine with that of the main comparator (preferably from direct randomised trials, or, if these are not available, from other suitable trials or studies). Concludes with a therapeutic conclusion stating whether the proposed medicine is superior, noninferior or inferior to the main comparator, taking account of any differences between the trial population and circumstances of use, and those proposed for the listing (applicability).
- Section 3 – Economic evaluation. Presents an economic evaluation of the consequences of substituting the proposed medicine for the main comparator in the context of the listing requested.
- Section 4 – Use of the medicine in practice. Includes the predicted extent of use of the medicine in the health system, and financial analyses for the PBS/Repatriation Pharmaceutical Benefits Scheme (RPBS) and the Australian Government health budget.
- Section 5 – Options to present additional relevant information (optional). Includes any other relevant information to support a submission.
All submissions should have an executive summary that clearly sets out the key aspects and issues presented in the main body of the submission. Additional information can be included as attachments or technical documents.
The guidelines are presented in two parts.
Part A provides all of the information requests, and further information on content and presentation for the majority of submissions. The information is arranged in exactly the same sections and order as is appropriate for a submission (Sections 1–5; see above).
Section 3 (Economic evaluation) has two alternative pathways:
- Section 3A – guidance for preparing Section 3 based on a cost-utility (preferred) or other cost-effectiveness analysis
- Section 3B – guidance for preparing Section 3 based on a cost-minimisation approach.
Part B provides additional information requests for submissions concerning the following product types:
- Product type 1 – fixed-dose combination products
- Product type 2 – nutritional products
- Product type 3 – vaccine products
- Product type 4 – codependent technologies.
Appendixes provide additional background about the guidelines, and further information on various aspects of the submission.
Section designations and cross-references within these guidelines
The following principles describe the scheme used for naming sections and subsections within the guidelines, and cross-referencing between parts and sections of the document:
- In Part A, the sections are labelled according to the main section to which they refer (ie 1–5). Each main section is made up of a series of subsections (eg Subsection 1.1, Subsection 2.1), which correspond to the subsections that should appear in a submission.
- In Part B, the product types are labelled P1, P2 etc, with subsections as Subsection P1.1, Subsection P1.2 etc.
- Cross-references to other sections and subsections within the same part are given as ‘see Section 3’ or ‘Subsection 3A.6’ etc. However, cross-references across parts are given as ‘see Part B, Section P1’ etc.
- Tables are numbered consecutively within each subsection – for example, Table 2.1.1, Table 2.1.2 etc in Subsection 2.1.
- Flowcharts are labelled by section as Flowchart 1.1, Flowchart 2.1 etc. Other figures are labelled consecutively within each main section of the guidelines, as for tables.
- Appendixes are labelled Appendix 1, Appendix 2 etc, with subsections as A1.1, A1.2 etc (but only as required for cross-referencing) and appendix tables are labelled consecutively within each appendix – for example, Table A2.1 in Appendix 2.
Writing and style conventions used in the guidelines
The PBAC Guidelines include a series of requests for specific types of information. The aim is to provide an ordered series of reference points (information requests) against which the specific information presented in a submission can be assessed to ensure that the submission is complete.
The ‘default’ writing style for requests for information uses the imperative voice, as follows:
‘Describe the proposed course of treatment.’ ‘Justify the exclusion of the study.’
Readers should interpret these imperative statements as indicating what, in general, should be done.
Within each section, the main requests for information are highlighted as a checklist of information requests in boxes. Further explanation for each request and any subsidiary requests are provided under the numbered subheadings following these boxes. These text headings therefore provide a template for the submission. Following these requests and heading template will improve the comparability of submissions considered by the PBAC and, hence, the consistency of decision making.
Resubmissions to the PBAC should address the concerns raised by the PBAC in response to the previous submission, as well as the information requests that are relevant to the inclusion of new information or revised analyses. Information presented in the submission that is not in dispute does not need to be repeated; however, the resubmission should stand alone and contain all the relevant supporting documentation required for PBAC to reach a decision. The aim of a resubmission should be to highlight and integrate new information into the body of the resubmission, and to discuss how new information addresses the main matters of concern to the PBAC.
Notes: If the submission is requesting listings for multiple patient indications, present separate Sections 1–4 in separate submissions or seek advice from the Pharmaceutical Evaluation Branch – see ‘Contact details’ below.
The PBAC is established under the National Health Act 1953. Its primary role is to recommend to the Minister for Health which medicines should be subsidised under the PBS. The PBAC is required, under the Act, to consider the effectiveness and cost of the proposed medicine compared with existing therapies. The functions of the PBAC are outlined in s101 of the Act.
In particular, the PBAC is required to consider the effectiveness and cost of the proposed medicine compared with alternative therapies. 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.
PBAC decision making is influenced by five quantitative factors:
- Comparative health gain. Assessed in terms of both the magnitude of effect and clinical importance of effect. Presented as both effectiveness and safety (discussed in Section 2), and the denominator of the incremental cost-effectiveness ratio or incremental cost-utility ratio (discussed in Section 3A).
- 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 health care resources (discussed in Section 3).
- 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 (discussed in Section 3A).
- Predicted use in practice and financial implications for the PBS. Presented as the projected annual net cost to the PBS/RPBS or the National Immunisation Program (discussed in Subsection 4.4).
- Predicted use in practice and financial implications for the Australian Government health budget. Presented as the projected annual net cost per year (discussed in Subsection 4.5).
Other less-readily quantifiable factors that also influence PBAC decision making include:
- Overall confidence in the evidence and assumptions relied on in the submission.
- 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 case by case.
- Presence of effective therapeutic alternatives. This helps to determine the clinical need for the proposed medicine.
- Severity of the medical condition treated. Relates to any restrictions requested in Subsection 1.4. The emphasis is on the nature and extent of disease as it is currently managed (see Subsection 1.2).
- 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 be greatest 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.
- Public health issues; for example, development of resistance (for antimicrobial agents; see Subsection 5.3).
- Any other relevant factor that may affect the suitability of the medicine for listing on the PBS.
- Submissions consist of an executive summary, the main text of the submission and additional information (attachments and technical documents).
- The preferred order for the presentation of information is the executive summary followed by five sections (1–5).
- Each section consists of subsections (1.1, 1.2 …; 2.1, 2.2 … etc), each of which has a series of information requests.
- The order of information requests in these guidelines forms a template for a submission. Presenting information in any other order will reduce the PBAC’s ability to evaluate the submission.
- Use frequent, accurate cross-referencing between the executive summary, main text and other technical documents.
- Use succinct, plain English wherever possible (while maintaining scientific rigour).
- Justify any variations to the requested information.
- The guidelines may state a preference for certain approaches or information to be presented as supplementary analyses. This preference enables the approach to the base case to be consistent across submissions. Ensure that supplementary analyses are well justified and clearly presented.
- Ensure that information presented in the submission is fit for purpose. When considering a more complex analysis, weigh the additional information requirements and evaluation burden against the additional confidence that such an analysis provides. Where complex methods reduce the confidence in estimates compared with simpler methods, they are unlikely to be preferred.
Attempt to address all of the relevant information requests in the PBAC Guidelines. Where an information request cannot be addressed, provide a clear explanation. The PBAC will find a submission difficult to assess when information requests are not addressed and no justification has been provided for the omission. Submissions should clearly explain when an information request is not relevant or the resulting evidence would not alter the certainty of the key factors influencing decision making by the PBAC.
Documents that should be read in conjunction with the PBAC Guidelines:
- Manual of resource items and their associated costs for use in submissions to the PBAC involving economic evaluation (Department of Health and Aged Care). This manual is revised periodically in the same way as the PBAC Guidelines.
- Glossary of terms: key terms for preparing submissions to a health technology assessment (HTA) advisory committee for funding of a medicine, medical service or prosthesis (PBAC, Medical Services Advisory Committee and Prostheses List Advisory Committee).
- Sources of data for use in generating utilisation estimates (Department of Health and Aged Care)
- Information on Section 100 – Highly Specialised Drugs Program criteria
- Standardised utilisation and cost model Excel workbook for PBAC submissions, which is available on the ‘Downloads’ section of the PBAC Guidelines website.
Procedures to support submissions are also available on the PBAC Guidelines website.
Enquiries about PBAC submissions can be mailed or emailed to the Pharmaceutical Evaluation Branch at the following address:
Pharmaceutical Evaluation Branch
Department of Health and Aged Care
GPO Box 9848
Canberra ACT 2601