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Discussion paper 5 – Enhanced Post Market Surveillance

This paper puts forward a number of proposals to address different elements of post market surveillance.

Discussion paper 5 – Enhanced Post Market Surveillance (PDF 58 KB)
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Disclaimer

The proposals for reform to Commonwealth Health Technology Assessment (HTA) processes as outlined in this Discussion Paper represent a range of responses to the issues raised in submissions to the HTA Review and during stakeholder consultation. The Discussion Papers are intended to stimulate discussion at the forthcoming consultations and any proposal presented or omitted should not be taken to represent the policy position of the Government. The HTA Review is required to put forward proposals that can be sustained within existing funding levels and that are consistent with Government policy objectives.

Introduction

Because of the uncertainties in the evidence base around particular health technologies at the time of regulatory and reimbursement decisions, it is necessary to ensure the ongoing monitoring of health technology safety and performance. Post market (and post reimbursement) surveillance of health technology safety and performance is important because:

• it can provide valuable new evidence on the performance of a health technology on an ongoing basis especially where pre market and pre reimbursement evidence is limited; and
• the information collected helps to inform potential decisions such as:
  • should a technology be removed from the market?
  • should a technology continue to be publicly funded?
  • should the eligible population be reviewed?
  • should price (benefit) paid for technologies increase, decrease or stay the same?

This paper puts forward a number of proposals to address different elements of post market surveillance. They require further considered consultation with stakeholders including on how they may be appropriately resourced for effective implementation. Because the Review of Health Technology Assessment in Australia (the HTA Review) is constrained to put forward recommendations that can be implemented within existing funding levels, some of the proposals may be considered to be medium to longer term strategies to improve post market surveillance. They may also potentially impose additional regulatory requirements that would need to be carefully considered prior to recommendation or implementation.

Concerns Raised in HTA Review Consultations

The following concerns were raised about Australia’s post market surveillance system:

• it is too passive;
• it is heavily weighted in favour of safety concerns, with minimal comparative effectiveness focus. It needs to be more wide reaching and systematic across the health system by being proactive in seeking information about the comparative safety and effectiveness of particular technologies;
• it is over reliant on notification of concerns from suppliers and manufacturers which gives rise to potential conflict of interest;
• there is no nationally consistent process for alerting different jurisdictions in the event of a serious fault in a health technology;
• it needs to move beyond signal generation and into the realm of quantifying risks and benefits¹ ; and
• an expanded but targeted use of registers is needed to generate data that are more representative of health technology performance.

There were concerns also expressed around the post reimbursement evidence gathering arrangements (‘interim funding’) of the Medical Services Advisory Committee (MSAC):

• sponsors (and practitioners rendering the service) are not compelled to collect evidence to support a review (data collection is an expectation rather than requirement);
• it is not always clear who should pay for the collection of evidence, and the design and execution of the data collection is often methodologically weak;
• where data are collected, the quality of the data is such that definitive conclusions cannot be made; and
• there is currently no clear mechanism for the Government to have specific trials performed for new technologies and procedures for Medicare funding whether or not Medicare Benefits Schedule (MBS) listing is approved on an ‘interim funding’ basis² .

Proposal 12 – Widening the Scope of Post Market Surveillance

Instead of primarily evaluating and acting on intrinsic safety, efficacy and short-term effectiveness data, Australia’s post market surveillance system could expand its scope and consider comparative safety, clinical effectiveness and cost effectiveness. This could include an analysis of information about longer term risks, benefits, and costs in real life settings (in the hands of typical clinicians as applied to the broad range of patients encountered outside the usual investigational context).

Discussion
Traditionally, post market surveillance systems collect data of interest primarily to regulators. This proposal is about to what extent post market surveillance systems should collect comparative data which is of interest primarily to funders, but which may also be of interest to regulators.

There are many definitions of surveillance, but a widely used definition describes surveillance as the ‘ongoing, systematic collection, analysis, interpretation, and dissemination of data about a health related event for use in public health action to reduce morbidity and mortality and to improve health”³ . This definition could be interpreted as inclusive of any data, including comparative, that inform a decision to reduce harm and maximise benefit (regardless of whether it is a regulatory or reimbursement decision). However it may be more feasible to have separate but closely related evidence generating systems that support the distinct needs of each these decision makers rather than mushrooming the size of the current post market surveillance system. A situation would need to be avoided where loosely connected ‘silos’ of evidence gathering are created.

¹Submission 85. Medical Services Advisory Committee (MSAC). 1st July 2009
² It is important to note that MSAC is not the principal funder of clinical trials. Rather, it may recommend temporary MBS listing “to support data collection to address specific areas of uncertainty with respect to MBS listing decisions."
³ Centre for Disease Control and Prevention. Morbidity and Mortality Weekly Report; 50:1-35. 2001

Proposal 13 – Establish a More Rigorous Coverage with Evidence Development Framework

Coverage with Evidence Development (CED) is defined as any health policy initiative that links use of health care technology to a requirement for data collection, with the intent of informing future decision making⁴. CED is one of several policy options that have been postulated to overcome the problems of remaining uncertainty associated with making decisions to cover (i.e. reimburse the use of) a health technology. Conditionality allows a promising technology to be made available under specific conditions, usually for a defined period, after which the benefits of the technology are reviewed. Within this proposal, there are at least two possible options.

Option 13A – More stringent data collection requirements following MSAC recommendation of ‘interim funding’
This option would simply tighten the rules around existing interim funding arrangements including the enforcement of data collection. The disadvantage of this option is that it remains confined to services that have been considered by MSAC.

Option 13B – An independent CED research program
This could be coordinated by a separate research agency that conducts post HTA assessments on behalf of the HTA advisory committees such as MSAC, the Pharmaceutical Benefits Advisory Committee (PBAC) and the Prostheses and Devices Committee (PDC) with its own guidelines, protocols and earmarked funding.

Discussion
There are several methods of evidence development that could be adopted under a CED type program depending on the type of uncertainty in the evidence base that needs addressing. If the uncertainty is over the cost-effectiveness of a technology, then CED offers a way of generating further evidence on these variables without delaying access to treatments with clinical benefits adequately demonstrated in prior evidence⁵ . It may be possible to identify the range within which the values of the uncertain variables must fall to give reasonable confidence that an appropriate level of clinical benefit and/or cost-effectiveness will be achieved⁵. The new data collection could be designed to show more robustly whether the variables fall within the required ranges.

When the uncertainty is around the clinical effectiveness of a technology, there are multiple choices in study design depending on the research question for example:

• randomised pragmatic clinical trials (where feasible); or
• observational studies (for example through the use of registers).

Whatever study design is chosen, care must be taken to ensure that, in the pursuit of seeking evidence of more practical value to clinicians and patients in the real life local health care environment, this is not at the expense of robust study design or else this may inadvertently increase the uncertainty in the evidence base.

Given potential ethical issues ⁶ associated with CED, a robust consent framework would be required that makes it clear to all participants (patients and medical professionals) that further evidence about the new procedure or technology is being collected about identifiable patients and that informed consent and data collection are required for participation and funding. CED has several consequences for stakeholders5:

⁴ HTAi Special Interest Group on Conditional Coverage and Evidence Development for Promising Technologies. Found at: http://www.cmtpnet.org/cmtp-research/applied-policy-and-methods/coverage-with-evidence-development/htai-special-interest-group.
⁵ Hutton J, Trueman P, Henshall C. Coverage with Evidence Development: an examination of conceptual and policy issues. International Journal of Technology Assessment in Health Care 23: 4; 425-435. 2007
⁶ Miller FG, Pearson SD. Coverage with Evidence Development. Ethical issues and Policy implications. Medical Care; 46 (7): 746-751. 2008

For manufacturers and sponsors:

• it might allow promising technologies to be made available sooner that might otherwise be rejected, but might also introduce additional regulatory burden through more stringent data collection requirements;
• there might be little interest in conducting research if it carries the risk of proving that a product is not what it was hoped to be; and
• it might allow sponsors to reconsider the pricing of the technology, although this might not always be feasible.

For decision makers:

• it might allow a health technology to be made available in a controlled manner while also allowing the decision maker to define what evidence is required to support further reimbursed use of the technology;
• it would potentially place extra demands of approving the study design and monitoring and reviewing the data collected; and
• it might introduce the challenge of withdrawing coverage if this is the conclusion reached.

For health care providers:

• it might allow for earlier access to promising health technologies, thus increasing treatment options available for their patients but health care providers might also remain reluctant to use a technology that remains under evaluation.

For patients:

• it might allow access to health technologies that have apparent benefits, but patients might also fail to recognize or appreciate that there are distinctive risks, burdens, or greater uncertainties relating to benefit associated with CED participation. They might also incorrectly presume that coverage might imply that the risks of the intervention are well-known and considered reasonably low for all patients; and
• Alternatively, patients (and, in some instances, providers) might develop an inflated perception of the intervention’s benefit because it already attracts reimbursement and thus infers that sound evidence has already justified the use of the intervention.

If a CED type program were formally adopted, care would need to be taken to safeguard against creating unrealistic expectations about CED assessment outcomes for the various stakeholders. It is critical that the potential implications of embarking on a CED process (in terms of future decisions) are well defined at the outset. Timeframes set for the completion of CED research would need to take into account the time it takes to put a research team together, recruit sufficient number of participants and allow for collection and analysis of data.

Proposal 14 – Expanded use of Registers for Post Market and Post Reimbursement DataCollection

Many of the submissions advocate the setting up of additional registers as a data collection tool to reduce the uncertainty in the technology evidence base after both regulatory and reimbursement approval. This tool for post market surveillance could be used in conjunction with CED, or independent of it.

The primary criterion for implementing any register is that there is widespread confidence that it would be capable of generating convincing data on safety and relative effectiveness to support any subsequent decision about ARTG listing and reimbursement. By definition, registers are databases that systematically collect health-related information on individuals who are⁷ :

• treated with a particular surgical procedure, device or drug (e.g. joint replacement);
• diagnosed with a particular illness (e.g. stroke); or
• managed via a specific healthcare resource (e.g. treated in an intensive care unit).

The system or organisation governing the register is known as the registry. The purpose of the register would dictate what type of data collection is needed. Within this proposal there are at least three possible options in terms of type of registers that could be adopted ⁸
Option 14A – Device or Procedure Specific Registers
These types of registers are established to assess the specifics of a single device. They are often required by regulators to address questions about the real life characteristics of a procedure or device. The downside of this option is that if a register was set up for every single device or procedure, then the number of registers may become unmanageable. This option is likely to be reserved for certain circumstances (for example if considerable concerns have been raised about the safety and efficacy of a particular device or procedure, then this type of register could be set up to specifically investigate these concerns).

Option 14B – Class Registers
Class Registers collect data on all devices and procedures used in a specific class of intervention. The National Joint Replacement Register (NJRR) is an example of a class register. Several submissions supported the wider use of registers based on the NJRR model and this type of register may be well suited to support the PDC which is currently sorting prostheses on the Prostheses List into groups for the purpose of benefit negotiation.

⁷ The NHMRC Centre of Research Excellence in Patient Safety. Guidelines for the establishment and management of clinical registries. A report prepared for The Australian Commission on Safety and Quality in Health Care
⁸Submission 39. Medical Technology Association of Australia. 22 May 2009

Option 14C – Comparative Registers
Comparative Registers look at a range of different types of treatment options for a specific type of disease. They collect information on the different treatment pathways that might apply, including the use of surgical procedures, pharmaceuticals, and devices. This type of register is more suited to answering questions of interest primarily to reimbursement decision makers but may also be of interest to regulators. They can be very complex and expensive to operate and hence careful consideration would need to be given before their adoption.

Discussion
Because of the potential expense involved in setting up and maintaining a register, careful consideration would need to be given to defining criteria of high risk for selection and inclusion of a health technology into any future registers. For example, in relation to devices (or class of devices) these criteria could be informed by the risk classification of medical devices set down by the TGA⁹ , and also by the risk classification model proposed for HTA processes, which might also capture the longevity of each device.

If clinical registers were set up as a conduit to collect data as part of any enhanced active post market surveillance (or post reimbursement evidence collection system), there are several important requirements of a clinical registers that need careful consideration8. There must be good clinical buy in (clinicians are more likely to want to participate if the method of contributing information is relatively straight forward in terms of time and effort). Care would need to be taken to avoid duplication of data collection across registers, so that clinicians do not have to repeatedly supply the same information to a number of registers for the same procedure. The potential users of the register data must feel that the information is not tainted by bias and that interpretation is not made out of context. Financial incentives to encourage register participation would also need to be carefully constructed.

If poorly designed and resourced, registers have several limitations including:

• a lack of timely reporting, with some registers taking significant periods of time to provide reports;
• lack of clarity around ownership and privacy of data and compliance by users of the health technology being assessed;
• lack of comparability with other data sources in the absence of standardised definitions and practice;
• variable approaches to data audit, especially with regard to the capacity to recruit an eligible population and the completeness of the assessments;
• validity and comprehensiveness of data reporting; and
• potential for conflicts of interest in registry management, regardless of the manager.

⁹Therapeutic Goods Administration. Australian Medical Devices Guidance Document Number 25. Classification of Medical Devices. January 2005

It is therefore essential that the purpose and design of a register be carefully considered before embarking on establishing it. Draft operating principles and technical standards for registers developed by the Australian Commission for Safety and Quality in Health Care could be drawn upon in the first instance.

Proposal 15– Enhanced Data Linkage to Support Post Market Surveillance

There are at least two possible options in terms of improving linking of data for post market surveillance.

Option 15A – ‘Piggy back’ on existing national data linkage and e-health initiatives that would then feed information into post market surveillance system
The issue of data linkage is one the broader Australian health care system continues to grapple with and is being addressed elsewhere by various national data linkage and e-health initiatives coordinated by the Australian Health Ministers’ Advisory Council (AHMAC). Given that there are already some highly sophisticated infrastructure projects being put in place nationally aiming for cross-jurisdictional data linkage on a de-identified unit record level, any recommendation for improved post market surveillance data collection could be incorporated into these existing data linkage initiatives.

Option 15B – ‘Pilot’ a specific data linkage project for post market surveillance purposes
This project could initially link appropriate datasets at a Commonwealth level followed by datasets at a State and Territory level to specifically extract information relevant for post market surveillance purposes. Care would need to be taken that this option does not duplicate existing efforts to improve data linkage nationally. A clearer idea about what information is being sought for post market surveillance would need to be resolved before considering what data linkage systems are needed to best extract this information.

Discussion
The current inability to link datasets within and across jurisdictional boundaries was stated amongst the submissions to the HTA Review as an impediment to post market surveillance. Building a capability for post market surveillance using data linkage will require investment in structures and frameworks for governance, privacy, ethics and community involvement; information technology and information management; methods and tools for data linkage and analysis of linked datasets; and human capacity¹⁰ . The potential benefits of data linkage include¹¹ :

• increased cost-efficiency of research compared with performing de novo longitudinal studies and other more traditional approaches to epidemiologic and health services research;
• adding value to existing information assets and generating a research return on the substantial existing investment in routine administrative and clinical data sets within health;
• fostering collaborative research involving population health researchers, clinical researchers and biomedical scientists, with direct benefits for clinical outcomes; and
• community development through enhancing interactions among researchers, clinicians, administrators, consumer groups and the mass media.

¹⁰Australian Government. Department of Innovation, Industry, Science and Research. National Collaborative Research infrastructure Strategy (2005-2011) Scoping Paper - Population Health and Data Linkage. 2005.
¹¹Data Linkage Australia. Scoping paper: a model for a data linkage facility in New South Wales. Sydney: the Sax Institute, 2005.

Proposal 16 – A Review Process with Capacity to Recommend Disinvestment

The discipline of HTA could play a larger role in making recommendations around the disinvestment of health technologies including the:

• identification of ineffective technologies;
• provision of advice recommending reducing or refining the use of technologies; and
• provision of advice recommending the removal of technologies from government and insurance funding schedules altogether¹² .

This would allow reallocation (or reinvestment) of funding to interventions and programs that offer overall health gains more efficiently and could encourage more robust and efficient processes around all health care decision making, not just disinvestment.
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Discussion
In the discipline of HTA, criteria have already been developed for determining priorities for assessing new and emerging health interventions. Preliminary work has begun at an academic level to build upon these criteria to develop a framework to facilitate systematic and transparent identification of existing, potentially cost-ineffective health interventions for formal review12. The preliminary criteria are as follows:

• new evidence on safety, clinical effectiveness and/or cost effectiveness may come to light that changes previously held conclusions;
• provider and geographical variations in care where choice of intervention significantly varies for a particular condition;
• when an intervention has evolved to the point that it differs markedly from the initial or prototype intervention that was originally assessed or funded;
• temporal variation in volume of use i.e. a trend in MBS item volume between time points (eg 2, 3, 5 years) of a substantial percentage (say 30%, 50% or 80%). This may be a decrease or increase;
• public interest or controversy;
• nominated by expert clinical groups for formal review: This could be through a consultation process via an overseeing ‘disinvestment committee’ with broad stakeholder representation;
• when a new intervention is presented to the relevant HTA advisory committee and is considered a potential replacement for (an) established comparator(s) for that indication, then that comparator could also be considered for formal review;
• technology use (with reimbursement) is outside the evidence based indications or conflicts with clinical practice which is accepted as cost-effective. This has also been ‘leakage’ or ‘indication creep’; and
• ‘legacy’ items – long established interventions (or class of interventions) that have never had their cost-effectiveness assessed.

¹²Elshaug AG, Moss JR, Littlejohns P, Karnon J, Merlin TL, Hiller JE. Identifying existing health care services that do not provide value for money. Medical Journal of Australia; 190: 269-273. 2009

Criteria would also need to be developed to inform prioritization of candidates for formal review after identification such as12:

• high cost per item of intervention or high overall due to high volume;
• the potential health, cost and equity impacts of disinvestment versus maintaining the status quo;
• a cost-effective alternative;
• a high level of disease burden; and/or
• a pre-designated time for follow up review has arrived.

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Given the potential negative connotations around the term disinvestment (i.e. stakeholders may perceive this term as implied criticism of clinical practice or an intrusion on clinical autonomy), it is important that any implementation also includes a transparent and consultative process that actively engages all affected parties. In the short to medium term, a disinvestment review framework that traverses across all Commonwealth HTA processes (the Medicare Benefit Schedule, the Pharmaceutical Benefit Scheme and the Prostheses List) could be established. This could be in the form of a specialist subcommittee, with broad stakeholder representation, supporting each of the HTA advisory committees (MSAC, PDC and PBAC).

In the 2009-10 Budget, the Government provided $9.3 million over two years under the Medicare Benefits Schedule — a quality framework for reviewing services measure to put in place a new evidence-based framework for reviewing services listed on the MBS. The new framework will take effect from 1 January 2010. Under the new arrangements, services will be evaluated and aligned with contemporary evidence to ensure clinical relevance and appropriate pricing. New services will be evaluated three years after being listed. The aim of this budget measure is to improve health outcomes for patients and help maintain the financial sustainability of the MBS. The Department of Health and Ageing has commenced work on implementing this budget measure.


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