Section 1: Role of the Pharmaceutical Benefits Advisory Committee
The Pharmaceutical Benefits Advisory Committee (PBAC) is established under the National Health Act 1953 (the Act). Its primary role is to recommend to the Minister for Health which drugs and medicinal preparations should be subsidised by the Australian Government under the Pharmaceutical Benefits Scheme (PBS). In doing this, PBAC is required by the Act to consider both the effectiveness and cost of the proposed drugs and medicinal preparations.
Since the beginning of 2006, PBAC has also been required under the Act to recommend to the Minister for Health vaccines for funding under the National Immunisation Program (NIP). The principles described here for considering PBS listing of drug products in general also apply to the funding for vaccines by the NIP. Accordingly, in the remainder of these guidelines, unless otherwise indicated, references to the PBS should be taken to include the NIP, and the term 'drugs' to include vaccines. Following due process, PBAC regularly reviews the list of PBS items, including restrictions, maximum quantities and number of repeats. It also provides advice about any other matters relating to the PBS that are referred to it by the minister.
Box 1.1 lists the main roles of PBAC. Further details are given in the remainder of this section.
|Recommends drugs and medicinal preparations to the Minister for Health for funding under the Pharmaceutical Benefits Scheme (PBS).|
|Recommends vaccines for funding under the National Immunisation Program (since 2006).|
|Advises the minister and the Pharmaceutical Benefits Pricing Authority about cost-effectiveness ('value for money').|
|Recommends maximum quantities and repeats on the basis of community use, and any restrictions on the indications where PBS subsidy is available.|
|Regularly reviews the list of PBS items.|
|Advises the minister about any other matters relating to the PBS.|
The membership of PBAC is prescribed in the Act. The members, who are appointed by the Minister for Health, include medical practitioners (specialists, general practitioners and clinical pharmacologists), pharmacists, consumers and health economists. The membership is published in the Government Gazette and details are available at Participants in the listing process.
Under the Act, PBAC may also establish subcommittees, comprising members with appropriate expertise, to help it perform its functions. There are currently two subcommittees:
- Drug Utilisation Sub-Committee (DUSC) - which monitors the patterns and trends of drug use and makes such data available publicly. DUSC evaluates use and financial forecasts of selected major submissions to PBAC. DUSC was formed by PBAC in 1988. The members have a broad range of relevant expertise and mainly come from organisations interested in the evaluation of drug utilisation.
- Economics Sub-Committee (ESC) - which advises on cost-effectiveness policies and evaluates cost-effectiveness aspects of major submissions to PBAC by reviewing and interpreting economic analyses and assessing their quality, validity and relevance. After a preliminary period as a working party, ESC was formed by PBAC at the beginning of 1994. The members include clinicians, clinical epidemiologists, health economists, biostatisticians and clinical pharmacologists. As part of its terms of reference, ESC is also responsible for revisions of the guidelines (see Subsection 2.1 ).
The primary objective of the PBS is to improve health. The range of drugs and forms available under the PBS provides a formulary of drugs to meet the health needs of the majority of the Australian community. The role of a drug product in meeting the health needs of the Australian community is therefore a primary consideration. Thus, PBAC focuses on health outcomes.
PBAC may also consider nonhealth outcomes, including aspects of the delivery of a health care intervention beyond the health gain obtained; for example, greater convenience or production gains to society beyond those valued by the population benefiting with improved health. However, the valuation of nonhealth outcomes is not straightforward and those outcomes might not be as influential in decision making as health outcomes.
Similarly, PBAC mainly considers the costs of providing health care resources. These extend beyond the costs of the drug to include possible cost offsets of reduced provision of health care resources as a result of listing a drug. PBAC may also consider costs and cost offsets of nonhealth care resources, but these might not be as influential in decision making as health care resources.
All new pharmaceutical products must be registered on the Australian Register of Therapeutic Goods (ARTG) by the Therapeutic Goods Administration (TGA) before being generally marketed in Australia. Registration is based on assessment of quality, safety and efficacy, a process that usually involves the Australian Drug Evaluation Committee (ADEC). PBAC therefore accepts that products included on the ARTG have established safety and efficacy adequate to allow marketing in Australia. Products are registered for specific therapeutic indications and, in general, PBAC does not recommend a product to be listed in the PBS for indications beyond those registered.
Listed drugs are classified either as pharmaceutical benefits (drugs listed under section 85 of the Act), special pharmaceutical products (drugs listed under section 100 of the Act as requiring special distribution arrangements, such as highly specialised drugs; see Subsection 1.3.6), or vaccines (listed in the NIP schedule under section 9C of the Act). However, the requirements of the PBAC under the Act, and thus the considerations for listing and submission requirements, are the same for each type of listing.
PBAC encourages the quality use of medicines (QUM) through the inclusion of cautions and notes in the PBS Schedule, the wording of PBS restrictions and the provision and publication of Australian drug utilisation data. It supports the educational activities promoting the appropriate use of pharmaceutical benefits by the National Prescribing Service (NPS), particularly its Rational Assessment of Drugs and Research (RADAR) program. In making a submission, sponsors should be aware of the possibility that PBAC could refer matters for inclusion in this program alongside a recommendation to list a proposed drug. Further information on QUM is provided in Part II, Subsection F.1 .
Under the Act, PBAC is required to consider the effectiveness and cost of a proposed PBS listing compared with other therapies. Therefore, when recommending listings to the Minister for Health, the committee also advises the Pharmaceutical Benefits Pricing Authority (PBPA) about how the new listing compares with alternative drugs and/or current standard care in terms of cost-effectiveness ('value for money'). The general guidelines followed by PBAC are shown in Box 1.2.
To assess value for money, PBAC considers the clinical place, overall effectiveness, cost and cost-effectiveness of a proposed drug compared with other drugs already listed in the PBS for the same, or similar, indications. Where there is no listed alternative, PBAC considers the clinical place, overall effectiveness, cost and cost-effectiveness of the proposed drug compared with standard medical care. On the basis of its community usage, PBAC recommends maximum quantities and repeats and may also recommend restrictions as to the indications where PBS subsidy is available.
|A new drug may be recommended for listing if:|
|- it is needed for the prevention or treatment of significant medical conditions not already covered, or inadequately covered, by drugs in the existing list and is of acceptable cost?effectiveness|
|- it is more effective or less toxic (or both) than a drug already listed for the same indications and is of acceptable cost-effectiveness|
|- it is at least as effective and safe as a drug already listed for the same indications and is of similar or better cost-effectiveness.|
|A new drug that is less effective and/or more toxic than a drug already listed for the same indications might be considered for listing. In such a circumstance, other supportive factors would be needed to justify a recommendation, for example, if the new drug would decrease the overall costs of therapy and/or if it were restricted to a subsequent line of therapy after the more effective or less toxic therapy.|
|Recommendation to list a new drug is unlikely if:|
|- its use might increase problems of abuse or dependence|
|- its sole use would be to treat an individual patient whose response to, or need for, a drug is unique.|
|Removal of a drug from the list may occur if:|
|- a more effective or equally effective but less toxic drug becomes available|
|- evidence becomes available that the effectiveness of the drug is unsatisfactory|
|- evidence becomes available that the toxicity or abuse potential of the drug outweighs its therapeutic value|
|- the drug has fallen into disuse or is no longer available|
|- treatment with a drug is no longer deemed cost-effective compared with other therapies.|
|PBAC follows due process in considering the removal of a drug, including consulting with affected stakeholders.|
|At the direction of the Minister for Health, PBAC:|
|- takes into account the community need or benefit, particularly for additional forms of an already-listed drug where proliferation of products might cause confusion|
|- gives a lower priority for listing to a drug intended specifically for in-hospital use, since the PBS is primarily for community-based patients|
|- gives a low priority for listing to a drug for the treatment of clinically minor or trivial conditions.|
PBAC makes recommendations about the maximum quantity and the number of repeat prescriptions that should be available for each form of a drug. For acute medical conditions, the maximum quantity is usually sufficient for a normal single course of treatment (bearing in mind the size of the manufacturer's pack). For chronic medical conditions, the maximum quantity and repeats usually provide up to six months' therapy, depending on the need for clinical review of the condition to be treated. For patients requiring higher than average doses, increases in the listed maximum quantities and repeats are generally available through the authority system.
Drugs and medicinal products can be listed on the PBS as:
- unrestricted benefits, which have no restrictions on their therapeutic uses for the purposes of subsidy
- restricted benefits, which can only be prescribed for specific therapeutic uses
- authority required benefits, which are restricted and can only be prescribed with previous approval from Medicare Australia or the Australian Government Department of Veterans' Affairs.
A drug or drug form is considered for restricted benefit or authority required listing for the following reasons:
- to limit PBS usage so that this is in accordance with the approval and registration granted by the TGA
- to allow the controlled introduction of a drug in a new therapeutic class
- to limit PBS usage to the indications, conditions or settings seen as being appropriate for clinical, cost-effectiveness, or other reasons
- to alleviate concerns about adverse reactions, possible misuse, overuse or abuse.
Following an agreement between federal and state and territory health ministers, the Highly Specialised Drugs Working Party was established in 1991. The Highly Specialised Drug Program operates under section 100 of the Act and subsidises the use of highly specialised drugs through hospital outpatient departments for community patients whose treatment is not appropriate for a community medical practice setting.
PBAC considers submissions from industry sponsors of drug products, medical bodies, health professionals, 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.
PBAC is conscious of the need to be as open as possible in its proceedings, consistent with the secrecy provisions of the Act. It therefore provides to sponsors all relevant documents and evaluations considered by the committee. It also allows up to two sets of written pre-PBAC consultation documents from each sponsor in relation to its submission for a product, as well as a hearing before the committee when it is considering advice from its subcommittees.
Although marketing approval and registration on the ARTG are prerequisites for PBS listing (see Subsection 1.3.1), PBAC accepts submissions before finalisation of marketing approval, provided that the TGA delegate has recommended the drug for registration in his or her overview (advice to ADEC).
In formulating its conclusions, PBAC may seek expert opinion from relevant professional bodies and/or appropriate specialists, and may meet with representatives of relevant medical professional organisations and colleges. PBAC may also seek input from appropriate consumer bodies. As a routine, PBAC seeks advice from the Australian Technical Advisory Group on Immunisation in relation to vaccines, the Nutritional Products Working Party in relation to nutritional products and the Expert Advisory Group on Antibiotic Resistance in relation to the development of resistance to new antimicrobial agents. Where advice is obtained, due process is followed, in which the relevant sponsor is informed and given an opportunity to reply.
PBAC is conscious of the need to avoid unnecessary delays between marketing approval and subsidised listing where the latter is appropriate. To this end, all submissions received by a reasonable cut-off date are considered at the next PBAC meeting. These cut-off dates are provided to the pharmaceutical industry well in advance of meetings.
The meeting dates for the following year, and the associated cut-off dates, are advised to industry following each July PBAC meeting and are posted in the Pharmaceutical Benefits Scheme (PBS) Calendar on the PBS website.1 The cut-off date for major submissions is generally 17 weeks before the PBAC meeting (18 weeks over the Christmas to New Year period). Minor submissions may be accepted up to 6 weeks later (11 weeks before the PBAC meeting). Contact should be made with the PBAC secretariat before presenting a submission. Further information on major and minor submissions is given in Section 3 .
Submissions should be presented on time and should be complete. No guarantee can be given that material supplied late will be incorporated into the submission or included in the agenda papers. Advice of committee decisions is provided to sponsors in writing within 15 working days of a meeting, and PBAC and PBPA meetings are coordinated to minimise processing time.
Box 1.3 shows a timeline for major actions and events in PBAC procedures, relative to PBAC meetings. PBAC public output in relation to a submission includes a brief summary of the outcome2 followed by a more extensive public summary document.3
|Action or event||Time relative to PBAC meeting|
|TGA delegate's overview/advice to ADEC and/or ADEC resolution and/or TGA registration granted|
|Cut-off date for major submissions to department||17 weeks before|
|Cut-off date for minor submissions to department||11 weeks before|
|Departmental papers to sponsors||6 weeks before|
|Sponsor's pre-subcommittee response to department||5 weeks before|
|Meeting of subcommittees||4 weeks before|
|Subcommittee papers to sponsors||2 weeks before|
|Sponsor's pre-PBAC response to department||1 week before|
|Verbal advice to sponsor||half a week after|
|Written advice to sponsor||3 weeks after|
|Publication of PBAC outcomes on departmental website||6 weeks after|
|PBAC ratified minutes to sponsor||10 weeks after|
|Publication of public summary document on departmental website||16 weeks after|
|Publication of public summary document (first time rejections)||18 weeks after|