Introduction The Royal College of Pathologists of Australasia by k9902mn



The Royal College of Pathologists of Australasia (RCPA) welcomes the opportunity to
contribute to this important Australian Government Review of Health Technology
Assessment (HTA) in Australia.

The RCPA is the leading organisation representing pathologists in Australasia. Its mission is
to train and support pathologists and to improve the use of pathology testing to achieve
better healthcare for patients. It achieves this mission through its continual focus on the
professional and quality issues pertaining to pathology testing. For these reasons the RCPA
is ideally placed to comment on the conduct of HTA as it relates to pathology testing.

The RCPA supports robust evaluation of health technologies with a view to safety,
effectiveness and cost effectiveness, as this is clearly in the best interests of the community.
However the RCPA has long been concerned about the difficulties experienced with HTA in
Australia, and specifically with the Medical Services Advisory Committee (MSAC) process as
it applies to pathology tests for the diagnosis, monitoring and prevention of disease.

Extensive delays occur with introducing new pathology tests to the Medicare Benefits
Schedule, and this is impacting adversely on the health and wellbeing of the Australian
community. This is particularly an issue for genetic testing, and consequently Australia has
been falling behind the rest of the developed world in terms of access to gene tests. The
RCPA has called for the establishment of a national framework for genetic testing to address
issues of access and qualityi and the HTA Review can contribute meaningfully to this.

MSAC uses a comparative assessment approach whereby the new technology “is compared
against the most commonly used currently funded treatment option/s”.ii This poses a
significant challenge for new pathology tests, which often have no ideal comparator and in
some cases no comparator at alliii. Whilst safety and clinical effectiveness may still be
demonstrable, it is difficult to prove that a new test is cost effective relative to the alternative
of doing nothing unless the broader consequences of doing nothing (in terms of overall
morbidity and mortality associated with the disease in question) are taken into account.
However such measures may not fall within the ambit of an MSAC model of cost
effectiveness and consequently the test would not be recommended for public funding,
leaving patients with the options of paying for the test or going without.

This difficulty is accentuated in the context of genetic tests where it may be the
asymptomatic relatives of a patient who would benefit from a test, but again the benefits that
would accrue will not be captured in a simple comparative cost effectiveness model even
though there would be clear savings to society from early intervention or potentially
prevention of the disease.

The MSAC model has been designed for technologies and devices used as treatment
modalities, and with some modification it may be well suited to that purpose. It is not
suitable for the analysis of diagnostic modalities however, and consequently the RCPA
recommends that technologies pertaining to the diagnostic medical specialties (pathology
and radiology) be subjected to an alternative method of HTA, possibly by extracting them
from the MSAC process. In particular, low volume gene tests should be managed outside
the MSAC process because there will never be enough literature to meet the requirements
of the existing MSAC model and this discriminates against those families unfortunate
enough to experience rare genetic conditions.

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
In addition to a separate assessment model for diagnostic medical specialties, there should
be greater reliance on HTA conducted by other (including overseas) organisations, more
liberal use of interim MSAC approvals, better coordination between HTA agencies and a
more flexible and responsive approach adopted.

Response to Terms of Reference

The RCPA would like to make the following comments and responses to the Review’s Terms
of Reference and key questions:

Term of Reference No 1

Simplification and better co-ordination between all Commonwealth health technology
assessments, including:

a) consideration of a single entry point and tracking system for applications for market
registration and funding and coverage purposes;

b) making time to affordable access as short as possible for new technologies while
maintaining rigour of evaluation process; and

c) examination of the feasibility of conducting concurrent assessments for market registration
and funding and coverage purposes, noting current work in this area.

Key Questions

1. How can the interaction between the different HTA agencies (ie TGA, MSAC and PDC)
and their processes for the registration and approval for market entry and public and private
health funding of new medical services and devices be improved?

•    There should be formalised communication channels to ensure that each agency,
     including the PBAC, is aware of the applications currently being considered by others.
     For example, if PBAC is considering a drug that requires genetic testing to determine the
     appropriate patients to receive it, MSAC should be aware so that the required genetic
     test can be evaluated in a comparable timeframe.

              Example: Gefitinib was listed on the PBS in late 2004, restricted to patients with
              lung cancer cells that have a particular class of genetic abnormality. There is,
              however, still no Medicare rebate towards the $1000 test that is necessary to
              determine whether that genetic abnormality is present.iv

•    Better coordination could be facilitated through one point of entry and a single tracking
     system for all applications, but improved communication between the committees and
     their administrative support staff is the most essential element for improving the
     interaction between the HTA agencies.

•    Consideration could be given to having a single agency conduct all HTA, and provide
     these assessments (with or without recommendations) to different decision making
     bodies such as the TGA.

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
2. How could the administrative processes of each individual HTA agency (ie TGA, MSAC
and PDC) be simplified without compromising the scientific rigour underpinning the HTA

•    There should be standard arrangements for groups of tests to be submitted and
     combined assessments done. This would enable a more efficient use of resources, save
     time for the experts involved in advisory panels, and reduce delays in making
     appropriate tests available for the community. Safety would be demonstrated once for
     each test as this does not change depending on clinical context. The tests could then be
     assessed for the range of clinical conditions to which they pertain from the perspectives
     of effectiveness and cost effectiveness.

•    This is particularly relevant for genetic tests. The Human Genome Project mapped
     25,000 genes and it is increasingly apparent that each gene can cause many diseases,
     and each disease can be caused by many genes; it would make sense in some
     instances to make an application for several gene tests that are undertaken
     simultaneously for a range of clinical conditions.

                Example: people with early onset phaeochromocytoma have a 20% chance of a
                genetic mutation (in one of several genes) that will increase their risk of
                developing other types of cancer. Identifying this mutation clarifies further
                management of the patient, and guides testing to identify at-risk relatives and
                plan any necessary surveillance for them. Current best practice undertaken
                through state laboratories is to test five genes, as each is associated with a
                different cancer spectrum. It would be an enormous waste of time and resources
                to develop applications for every one of these genes and for each possible clinical

•    MSAC encourages potential applicants to arrange a 15-20 minute pre-lodgement
     meeting to discuss the MSAC process, eligibility criteria, definition of a medical service
     and data requirements. It appears that this information is largely generic and uni-
     directional. The ambit of these meetings should be expanded to include a more detailed
     discussion of the proposed application, with foreseeable issues such as lack of Class 1
     evidence identified, and consideration of how these issues can be ameliorated. This
     would be of great assistance to individual applicants such as pathologists who struggle
     to provide the type and level of information required for an application to be accepted.

•    Establishment of expert advisory panels is too slow and can be a significant factor in
     overall delays. For pathology tests this could be addressed by using members of the
     Pathology Services Table Committee (PSTC) Subcommittees as a core group to form
     the panel, and supplementing this with other experts as required. Each of the PSTC
     Subcommittees has expertise in the relevant discipline as well as a sound working
     knowledge of the pathology tests in the schedule and the processes involved in making
     changes to the schedule, and consequently Subcommittee members are ideally placed
     to contribute to MSAC reviews.

•    Broadening the range of research that can be considered would not compromise
     scientific rigour. Studies reviewed could still be Class 1 evidence yet encompass
     additional attributes of measurement, such as evaluating the impact on relatives of
     testing breast/ovarian cancer patients for BRCA1 and BRCA2 gene mutations.

•    In the last decade it has been individual pathologists (rather than industry) who have
     prepared the most significant MSAC applications for new pathology tests. Each
     submission presents a sizable task, often necessitating the pathologist taking time off

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
     work to research and write it while their colleagues cover the requirements of the
     laboratory. Finding pathologists with the time and skills to do this is extremely difficult in
     a time of workforce crisis, and is a major reason for the small number of applications. To
     address this, a service could be funded to assist with preparing submissions by
     undertaking background research and partial drafting of the application. PSTC would be
     able to facilitate this process.

3. How can HTA undertaken by other countries be used in the Australian context? What are
the limitations, risks and opportunities that would need to be considered?

•    The need to collaborate and see HTA from a global perspective is recognised
     increasingly, through organisations such as Health Technology Assessment International
     (HTAi)v,vi. In keeping with this, HTA undertaken in other countries should be used more
     extensively to inform decision making by Australian agencies. This would replace the
     need for MSAC to initiate a full assessment for every application, which would result in
     considerable cost savings for the taxpayer (given the estimated average cost per MSAC
     assessment is $250,000) and reduced delays (both for the expedited application and for
     applications further down the queue).

•    MSAC relies on overseas and international trials as a primary source of information
     because it recognises that the Australian population is too small to support adequate
     scientific studies in a reasonable timeframe. It is only an extension of the same logic to
     use HTA undertaken by other countries.

•    The ‘Global Harmonisation Task Force’ has developed principles by which medical
     device regulations in a number of countries are aligned; a similar framework of principles
     could be established to align HTA done in different countries. This would engender a
     consistent HTA model to which local parameters could be applied.

•    The risks associated with using HTA from other countries – that they relate to another
     population, environment and context – do not outweigh the benefits in terms of saving
     time and resources. The safety and effectiveness of new technologies varies little
     between populations and clinical needs are similar between first world countries. It is a
     waste of time and resources to repeat assessments recently undertaken in other nations
     on the pretext that the Australian population or environment is unique.

•    Whilst funding mechanisms may differ from country to country, all nations are seeking
     value for money in the health care they provide to their constituents. The assessment of
     cost effectiveness may differ depending on the funding model used, but many are
     grappling with evidence requirements for gene testsvii.

•    It is recognised that there is no perfect model for evaluating testing modalities but
     Australia would do well to draw from experience in other countriesviii, and build on an
     existing model used elsewhere rather than creating one anew, particularly in relation to
     assessment of genetic tests. The UK, USA and Canada have well developed systems
     for HTA, and examples are included at Appendix A.

4. How can assessment of cost effectiveness be improved to ensure HTA can inform
government decisions in a timely manner?

•    Cost effectiveness should be considered in broad terms to engender a comprehensive
     understanding of the effects of a new technology on health care costs for the Australian

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
     community. Important health outcomes may not fit with traditional measures, and as a
     result standard cost effectiveness analyses will not capture the range of relevant
     information. Using a narrow definition thus leads to false economies and decisions
     based on incomplete information. Where Cost Effectiveness Analysis is difficult to apply,
     consideration should be given to the use of Cost Utility Analyses or Cost Benefit

•    A new test may change who is classified as having a disease, often resulting in more
     cases diagnosed relative to a pre-existing testx. This may be interpreted as having an
     adverse effect on cost effectiveness of the test, in that treating additional cases will incur
     additional healthcare costs. Yet treating additional cases earlier may prevent
     downstream health costs. For example, identifying more cases of Vitamin D deficiency
     can lead to more people classified as having osteoporosis and being treated with drugs
     such as biphosphonates, which in turn prevent fractures that are costly to treat and have
     an enormous impact on morbidity and mortality in the community. Ideally, from holistic
     health care perspective, there should be a mechanism whereby money saved
     downstream could be allocated to the cost of the test.

•    Genetic tests have many applications, being used to diagnose, predict risk, and direct
     therapy. Although considered as new technologies, to evaluate a genetic test properly it
     should be seen as a total clinical processxi. One aspect unique to gene tests is that a
     patient’s results can have implications for their relatives. This is an area where
     considerable savings can be made in terms of expenditure on health care and other
     costs associated with morbidity and mortality of a given condition.

           Example: A man has colon cancer associated with an error in the MSH2 gene; his
           siblings could be tested to see if they also carry this genetic error, and this would
           guide the need for regular colonoscopies. Relying on family history alone the siblings
           could be given annual colonoscopies even though they may not be at increased risk.
           In other words it would be more cost effective in terms of saving overall health dollars
           (not to mention less invasive) to determine the risk for each sibling before submitting
           for annual colonoscopies, yet this evidence would not necessarily be captured in an
           MSAC evaluation of a test for the MSH2 gene of the index case. xii

5. Are there regulatory impediments to enhancing the evidence base for items approved for
interim funding, either through collaboration or individually?

•    Impediments to enhancing the evidence base for items approved for interim funding
     relate primarily to the level of evidence required before an application will be considered
     for interim funding. If, for example, there is no clear comparator for a pathology test then
     the requirements of the initial assessment to determine eligibility for interim funding may
     not be met.

•    As the MSAC process is not well suited to pathology test evaluation, the criteria for
     interim funding should be invoked more readily to enable laboratories to assist with the
     collection of data when conclusive evidence of effectiveness and cost-effectiveness of a
     test is initially lacking.

•    This is particularly the case for

           o     Genetic tests that are already performed throughout Australian states yet not
                 funded through Medicare, so that patient access relates not to clinical need but to
                 place of residence, and

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
           o     Co-dependent technologies whereby access to lifesaving treatments can be
                 compromised by a patient’s ability to pay for a test that demonstrates eligibility.

•    Interim funding is generally approved for a small number of services to use the new
     technology; the mechanism for determining which laboratories should be able to perform
     the test for a rebate needs to be transparent and based on factors such as expertise of
     those who will perform the test, and accessibility by an appropriate group of patients.

•    Funding should be allocated to commission research where it is determined that
     additional evidence is required. Further more, it should be possible to fund research
     regardless of whether or not interim funding has been sought or approved for the test in
     question. MSAC is not currently resourced to do this and consequently it was the DoHA
     Quality Use of Pathology Program that was called upon to fund a trial to examine Point
     of Care Testing.

Term of Reference No 2

Improving role clarity and addressing duplication between processes, where it exists
including consideration of consolidating functions with the Australian HTA system.

Key Questions

1. What HTA roles and functions require clarification?

•    Australia is unusual in that HTA (in the context of MSAC and PBAC) is enmeshed in
     funding decisions at a federal level, whereas in other countries HTA is undertaken
     separately and lack of linkage to funding can compromise the uptake of HTA
     recommendations xiii. This HTA Review is a good opportunity to build on a system
     where HTA and funding are linked, but taking elements from the way HTA is done in
     other countries.

•    The Department of Health and Ageing (DoHA) is responsible for approximately one third
     of referrals to MSACxiv, yet the basis for such referral is reactive and does not appear to
     reflect any particular strategy. DoHA has a strategic role in management of health
     services for the Australian community, however, and this should be reflected in referral to
     MSAC based on clinical need and anticipated benefits. In turn MSAC should be able to
     prioritise and fast track applications based on strategic guidance as to clinical need
     rather than all applications being assessed in chronological order of receipt.

•    The role that state organisations are playing in HTA needs to be clarified in order to
     identify where overlap is occurring. The reasons for states establishing their own
     assessment processes should be examined, and opportunities for collaboration and/or
     removal of duplication are likely to be identified.

•    Responsibility for horizon scanning should be clarified as the importance of this role is
     increasing with technological advances and it is currently undertaken in a minor and
     overlapping way by several national and state bodies.

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
2. Does duplication and/or overlap of HTA processes occur? If so, where? How could this be

•    For each eligible application MSAC undertakes an “evidence-based assessment from
     the ground up”.xv This leads to overlap, because frequently the safety and effectiveness
     of the new technology is already well established, and the only issue to be determined is
     cost effectiveness, yet a full analysis of all three elements is still done. For example
     there is recognised overlap between TGA and MSAC regarding assessment of safety; it
     is essential to define how these analyses are complementary and eliminate the
     duplication. TGA is competent at analytic validity, whereas MSAC or another agency
     should be responsible for clinical validity and economic analysis.

•    Duplication occurs in the context of requiring a complete assessment even if

           o     one has recently been done by an HTA organisation in another country, or
           o     an MSAC assessment has already been done for the same test used in the
                 context of a different clinical conditions

•    In regard to genetic tests, considerable duplication can occur when an application is put
     forward to MSAC for a test that is already being done by state run laboratories. This is a
     significant issue, with a survey in 2006 finding that there were only five genetic tests on
     the Medicare Benefits Schedule, and a further 437 types of genetic test were being
     undertaken throughout the Australian states.xvi In other words, the laboratories
     performing these tests are satisfied that they are safe, effective and cost effective, yet
     none of this is taken into account in an MSAC evaluation that assesses the test “from the
     ground up”.

•    Duplication and overlap could be resolved with clearer role delineation, a unified
     approach with better communication between the agencies and a mandate not to repeat
     assessment of aspects that have already been evaluated by another agency or through
     the states.

•    Standardisation of information requirements is important in that there should be no
     obligation to provide the same information in different formats to separate government
     agencies merely because the links between them are ineffective.

•    However attempts to standardise the information required must be managed carefully.
     For example, PBAC assessments are based on the evidence provided by the applicant,
     and drug companies are well placed to provide adequate information, whereas
     diagnostic technology companies and individuals are less well resourced and to require
     the same level of information would prove onerous and act as a deterrent to making an
     application for HTA.

Term of Reference No 3

Enhancing post marketing surveillance mechanisms to ensure the ongoing safety and
efficacy of medical devices.

Key Questions

1. What changes, if any, are needed to current HTA arrangements for post market
surveillance of health technologies?

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
•    In regard to pathology tests approved through MSAC for Medicare funding, post market
     surveillance is managed effectively through the NATA/RCPA medical testing
     accreditation system, which accredits Australian laboratories against the standard ISO
     15189, involving a thorough assessment of all operations within the laboratory.
     NATA/RCPA accreditation is mandatory for any laboratory wanting to provide tests that
     will attract a Medicare rebate.

•    As part of their accreditation requirements, laboratories must have external quality
     assurance programs, which enable a robust scientific analysis of how their tests are
     operating relative to tests being conducted by other laboratories and can pick up
     problems evident in one testing modality relative to another. The RCPA QAP Pty Ltd
     has been providing external quality assurance programs to laboratories for over twenty

•    In other words, despite the difficulties with having pathology tests approved through the
     MSAC process, the post market surveillance of pathology tests once approved is
     arguably better than surveillance for many other health technologies.

•    There does, however, need to be better surveillance for “quality use” of new tests.
     Whilst the accreditation system works well to demonstrate safety, many new tests are
     slow to enter clinical use, and some patients referred may not be the most appropriate.
     Introduction of new tests should be linked to an education program. An education
     program for new tests could be undertaken by the proposed national referral service
     announced with the budget (to be managed by the National Prescribing Service), with
     targeting to different categories of specialist as required.

•    One significant concern relates to surveillance for genetic tests that are being marketed
     directly to consumers. Such surveillance is fraught with difficulty and can only be
     realistically achieved through collaboration with international agencies. It should be
     recognised, however, that a more effective system for delivery of genetic testing within
     Australia has the potential to reduce the number of patients seeking off shore testing and
     the consequent risks this involves.

2. How could the arrangements for post market surveillance of medical devices for ongoing
safety and clinical effectiveness be improved?

•    Post market surveillance could be further improved through networking between
     laboratories, both private and public, with an agreed dataset collected for new tests for
     an agreed period.

•    For low volume tests, surveillance should be conducted through clinical reference
     laboratories (one or two nationally), again with an agreed dataset.

•    Any new test listed on the schedule should have an external quality assurance program
     put in place to ensure the actual quality of the test being conducted reflects the quality
     assessed during the HTA

3. What additional arrangements for post market surveillance could be considered or

•    Additional arrangements that could be considered include

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
           o     Monitoring the rate of use of new items through Medicare data.

           o     Assessment of whether the rate of referrals is appropriate through surveys of

           o     Improvements in health outcomes and patient perceptions could be evaluated
                 over a longer timeframe.

           o     A higher rebate per test could be paid to laboratories who contribute agreed data
                 demonstrating cost utility to an audit process, and these data could be used for
                 decision making regarding ongoing funding for that test.

4. How should post market surveillance be managed?

•    NATA/RCPA should continue to manage surveillance of pathology tests through the
     existing accreditation system, supported by external quality assurance programs such as
     those provided by the RCPA QAP Pty Ltd.

•    PSTC Subcommittees could play a greater role in the review of data collected about new
     pathology tests following their introduction. In particular auditing of use patterns for new
     tests (in terms of rate of referral, distribution geographically, whether referred by
     specialist or GP etc) could be overseen by the PSTC Statistics Subcommittee.

•    RCPA QAP Pty Ltd currently has an informal arrangement whereby TGA will be advised
     if there appear to be problems with a new technology based on external quality
     assurance report findings. This arrangement could be formalised.

Term of Reference No 4

Strengthening transparency and procedural fairness in the assessment, decision making and
fee negotiation processes through improved communication with stakeholders about
processes, methodologies, outcomes and performance against key indicators.

Key Questions

1. What aspects of Australia’s HTA system are working well in relation to transparency and
procedural fairness? Provide specific examples.

•    The rights of individuals such as pathologists to make applications supports procedural
     fairness and this must be preserved in any HTA model going forward. Unlike subsidised
     drugs, where manufacturers negotiate a price directly with Government, diagnostic
     technology companies must negotiate prices with individual laboratories; if they predict
     that a test will be used irrespective of a Medicare rebate, there is little incentive to make
     an application to MSAC, which is time consuming and can be resource intensive even to
     demonstrate eligibility. This is particularly true for genetic tests; as indicated above, 437
     types of genetic tests were undertaken in Australian laboratories in 2006, yet only five
     were eligible for a Medicare rebate.xvii

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
•    The provision of published guidelines to assist potential applicants is supported, and the
     absence of a fee for making MSAC applications makes the process more accessible to
     individuals and consumer organisations than it would otherwise be.

•    Some may argue that using the same highly structured model for all MSAC applications
     underpins transparency and procedural fairness, however this model is far more
     conducive to evaluating new treatment modalities than to new tests for the prevention,
     diagnosis or monitoring of disease. In fact, of the 27 applications made to MSAC for
     pathology tests since 1999, only 11 have been approved for funding.xviii

•    This issue is accentuated for genetic tests. Following the MSAC process has resulted in
     an average of only one gene test being added to the MBS every two years in the last
     decade. Clearly it is not feasible to continue at this pace given the rapid expansion of
     genetic testing and inexorable move towards personalised medicine. Moreover,
     submitting a separate application for each genetic test could require thousands of
     submissions (in view of the potential number of genes for which tests may be developed)
     and this would bring the MSAC program to a complete standstill.

2. What could be improved to assist transparency and procedural fairness? Provide specific

•    There is a perception that MSAC processes are not transparent and can be steered
     towards predetermined agendas, with the approval of the subcommittees overseeing the
     analyses being described as a mere formality.

•    To counter this perception, it is important that

           -     potential conflicts of interest are declared and managed

           -     there are clear and structured pathways for addressing concerns raised by
                 applicants or committee members during the evaluation process and

           -     that details of completed assessments are distributed to relevant stakeholders
                 automatically (rather than on request) and in a timely manner.

•    MSAC should have an appeals process established that is consistent with the appeals
     processes already in place for other agencies.

•    The HTA system should be flexible and responsive; not only should it be possible to add
     new items (tests, drugs, treatment modalities etc) quickly, there should also be robust
     mechanisms for removing items that have become obsolete or have been superceded.
     It should be noted, however, that new tests often do not replace existing tests, as these
     may be required for adjunct purposes or other conditions.

3. What key performance indicators could be developed and reported on to improve
transparency for HTA processes?

•    The MSAC website states that “The time taken for an application to be assessed depends
     on a number of factors, including the completeness of the application form, the quality of
     available evidence and the complexity of the medical service, treatment or procedure. As a
     general guide, the more complete the application and the more robust the evidence
     available, the easier it will be to progress.” This statement seems to absolve MSAC of all

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
     responsibility for delays. MSAC should be required to publish annually all milestones for
     applications such as:
        o date application received
        o date literature review completed
        o date advisory panel established and when meetings held
        o date of decision
        o date of recommendation being sent to Minister
        o date of stakeholders being advised of outcome.

•    Key performance indicators (KPI) could be arranged along similar lines e.g. median
     turnaround time, as well as tracking average cost per HTA.

•    It would be valuable for both transparency and learning purposes if MSAC were to report
     on why applications are rejected. For example
         o If “no good comparator against which to evaluate cost effectiveness” is a frequent
             reason, this would demonstrate a failure in the assessment process and a
             compromise of procedural fairness.
         o If “inadequate evidence” is a frequent reason it would signal a need for more
             funding to be allocated to accruing evidence.

Term of Reference No 5

Enhanced arrangements for assessment of co-dependent and hybrid technologies.

Key Questions

1. What are the key issues for government, regulators, medical and health professionals,
industry and consumers in relation to the assessment of co-dependent and hybrid

•    There is a lack of coordination, between government bodies and also with regulators, QA
     providers, and the proponents of the proposed interventions.

•    The process for getting new tests onto the Medicare Benefits Schedule is too slow,
     denying patients timely access to investigations that they need,

           Example: Brain Natriuretic Peptides (BNP) assay for the diagnosis and monitoring of
           heart failure, was accepted as eligible for assessment by MSAC in July 2004, and
           was finally endorsed by the Minister for funding in a hospital setting in February 2007
           and in primary care in August 2007.

•    When approval is denied patients do not understand why there is no rebate, especially if
     the test has been in use for a long time. Liquid Based Cytology, for example, has been
     used widely for more than a decade, yet it has never been approved for government

•    Tests attracting a Medicare rebate must be undertaken in a NATA/RCPA accredited
     laboratory but the same is not the case for tests where there is no rebate. This makes it
     difficult to be assured that non rebatable tests are being conducted in a high quality

K/Operations/DCEO/DCEO 2009 HTA Review - Submission
           Example: Her2 testing is not Medicare rebatable and therefore sits external to
           standard accreditation processes; this has necessitated the establishment of a
           specific Quality Assurance Program for Her2 testing.

2. What enhancements to current arrangements for assessment of co-dependent and hybrid
technologies could be introduced?

•    Better links should be fostered between HTA agencies as outlined above.

•    Rather than undertaking each assessment sequentially and from the ground up,
     assessments should build on those completed by other agencies and/or be done in
     unison where possible. For example, if a gene test has been approved by the TGA (i.e.
     safety established), and it has been deemed a mandatory companion test for a drug
     recommended by the PBAC (i.e. evidence of clinical need and effectiveness), the MSAC
     assessment of the test should take these findings into account (not start from scratch)
     and this assessment should be commenced as soon as the PBAC advises it is likely to
     be a mandatory companion test.

•    Referrals for assessment of co-dependent tests should automatically be made through
     the DoHA; they should not await the development of an application from an individual or
     from someone in the industry.

•    When the PBAC recommends a drug for inclusion in the PBS for a subset of patients to
     be identified by a test, that test could then be analysed by a subcommittee of the PBAC
     rather leaving it to someone else to develop an application for MSAC.

•    Consideration should be given to undertaking hybrid (or co-dependent) HTA. This could
     be done by having a single HTA agency to advise decision makers as recommended
     above. For co-dependent technologies a joint meeting of representatives from decision
     making bodies could then be held to facilitate consistent and timely decisions.

3. What are the implications for assessment of clinical effectiveness and cost-effectiveness
for hybrid and co-dependent technologies in relation to decision making about funding?

•    As indicated above, there is considerable scope for improvement in the coordination of
     HTA for co-dependent technologies, and resolution of these issues has the potential to
     result in more appropriate allocation of health resources, enabling access where it is
     needed and avoiding waste.

           Example: The drug Imatinib targets a specific genetic abnormality in chronic myeloid
           leukaemia. It was listed on the PBS in 2004, with benefits restricted to patients with
           this genetic abnormality. A specific DNA-based assay is more precise and less
           expensive than the surrogate marker alternative yet it took four years for this assay
           to be evaluated by MSAC.

K/Operations/DCEO/DCEO 2009 HTA Review - Submission

• Canada has long been a leader in regard to HTA having invested in this area before
   other countries. Agencies involved include
          o Canadian Coordinating Office for Health Technology Assessment
          o Alberta Heritage Foundation for Medical Research
          o Institute of Health Economics Canada

• The Centers for Disease Control and Prevention (CDC) developed the ACCE framework,
  which considers Analytic validity, Clinical validity, Clinical usefulness and any Ethical,
  social, or legal implications for assessment of gene tests.
      - A “rapid-ACCE” model has been developed to enable expedient evaluation of
          emerging gene tests, using the same questions as the full ACCE model to guide
          a systematic review of literature and unpublished data, leading to a summary of
          available evidence and identification of gaps in knowledgexix.
• Subsequent to the ACCE Project, the CDC has established the Evaluation of Genomic
  Applications in Practice and Prevention (EGAPP) initiative to implement a robust
  evidence-based process for evaluating genetic tests under the guidance of an
  independent panel of multidisciplinary experts. The primary objectives of EGAPP include
  development of a transparent, publicly accountable process; building on existing review
  methods; and clearly linking evidence to recommendationsxx.

• National Institute for Health and Clinical Excellence – is able to set its own agenda in the
   form of a “needs-led” prioritisation of topics for HTAxxi
• The UK Genetic Testing Network (GTN) has expanded on the ACCE model to more
   clearly define test purpose and components of clinical utility, and to incorporate health
   quality measuresxxii. The GTN evaluates genetic tests proposed for the National Health
   Service using the following criteria:
       - the seriousness of the condition;
       - the prevalence of the condition;
       - the purpose of the test in relation to diagnosis, treatment, prognosis and
           management, pre-symptomatic testing, and risk assessment;
       - the complexity of the test;
       - the population in which the test is to be used;
       - the performance of the test, including clinical sensitivity, specificity and predictive
       - the clinical utility of the test;
       - ethical, legal and social considerations, and
       - the cost of the test.

K/Operations/DCEO/DCEO 2009 HTA Review - Submission

   RCPA Paper “Genetic Testing in the 21st Century - Are we ready?” RCPA May 2008 (attach.)
    Australian Government Department of Health and Ageing “Review of Health Technology
Assessment in Australia – A Discussion Paper” 2009
    Glasziou, Paul et al “When should a New Test Become the Current Reference Standard?” Ann
Intern Med. 2008; 149:816-821
    RCPA Paper “Genetic Testing in the 21st Century - Are we ready?” RCPA May 2008
v ;
    International Network of Agencies for Health Technology Assessment
      Lijmer J, et al “Evaluation of diagnostic tests: a problem based approach.” Annu Meet Int Soc
Technol Assess Health Care Int Soc Technol Assess Health Care Meet. 2000; 16: 357
    Grosse, SD et al “Economic methods for valuing the outcomes of genetic testing:beyond cost
effectiveness analysis” Genet Med 2008: 10(9): 648-654
    Glasziou, Paul et al “When should a New Test Become the Current Reference Standard?” Ann
Intern Med. 2008; 149:816-821
    Burke, W et al “Defining purpose: a key step in genetic test evaluation” Genetics in Medicine
October 2007; 9:10 pp 675-681
     RCPA Paper “Genetic Testing in the 21st Century - Are we ready?” RCPA May 2008
     Jackson, TJ “Health technology assessment in Australia: challenges ahead” MJA September 2007;
187:5 pp 262-264
     Jackson, TJ “Health technology assessment in Australia: challenges ahead” MJA September 2007;
187:5 pp 262-264
     Australian Government Department of Health and Ageing “Review of Health Technology
Assessment in Australia – A Discussion Paper” 2009
     Suthers, G “Report of the Australian Genetic Testing Survey 2006” RCPA March 2009
      Suthers, G “Report of the Australian Genetic Testing Survey 2006” RCPA March 2009
      Data sourced from
     Gudgeon, JM et al “Rapid ACCE: Experience with a rapid and structured approach for evaluating
gene-based testing” Genet Med 2007:9(7):473-478
     Teutsch, SM et al “The Evaluation of Genomic Applications in Practice and Prevention (EGAPP)
initiative: methods of the EGAPP Working Group Genet Med 2008:10(10):1-13
     Jackson, TJ “Health technology assessment in Australia: challenges ahead” MJA September 2007;
187:5 pp 262-264
      Burke, W and Zimmern, R “Moving Beyond ACCE: An expanded framework for genetic test
evaluation” PHG Foundation report for the UK Genetic Testing Network September 2007

K/Operations/DCEO/DCEO 2009 HTA Review - Submission

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