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Preference: Administration and Health Services

2

Watchdog wants to dob on doctors

Health Minister Sussan Ley has asked AHPRA to suggest changes to National Law

The medical workforce watchdog will have the power to inform employers and places of practice of any change in the registration status of doctors under legislative changes being sought by the Australian Health Practitioner Regulation Agency.

Responding to a report critical of its handling of a serious case involving the preventable deaths of several babies, the Agency will gather and share more information regarding doctors under investigation and has asked for changes to its legislation to enable it to disclose more details to employers.

The changes are part of a detailed set of measures intended to address serious shortcomings identified by an investigation into its handling of the Djerriwarrh Health Service scandal, in which 10 babies died in two years – including seven deaths considered to have been avoidable.

AHPRA commissioned consultancy KPMG to examine how its Victorian office handled the matter after it was revealed it took 28 months to investigate a complaint about one of the doctors working at the health service in Bacchus Marsh, Victoria.

The Victorian Health Minister said the case involved a “catastrophic failure” of clinical governance at the health service, and ordered sweeping changes to its management.

For its part, AHPRA admitted that it had “taken longer than it should have” to investigate the complaint made to it about on the health service’s doctors.

In its report, KPMG said the watchdog needed to exercise better risk assessment, to throw more resources at investigating high-risk complaints, to operate with greater transparency, to address perceptions of being “pro-practitioner” and to critically evaluate its performance.

AHPRA accepted all the recommendations, and said that although the review concentrated on the agency’s Victorian operations, many of the changes it was making would apply nationally.

Aside from improving its process to assess high risk cases and speed investigations, the watchdog has detailed measures that will mean greater disclosure of information about matters and people under investigation.

“We are using the National Law more flexibly to improve information sharing with key stakeholders, such as notifiers, employers, [and the] Department of Health and Human Services (DHHS),” AHPRA said.

It said it had adopted a broader definition of employer to improve the gathering and sharing of practitioner employment details where action is to be taken, and was working with DHHS on an agreement setting out what sensitive information can be shared between agencies, and under what circumstances.

In addition, Health Minister Sussan Ley has asked AHPRA to suggest any changes to its legislation it might consider necessary, and it has suggested requiring registered practitioners to inform AHPRA of their employers and places of practice, and to give the regulator the power to “advise employers and places of practice of any changes to their registration status”.

The latest development come after a meeting between AHPRA, the Medical Board of Australia and AMA officials including Vice President Dr Stephen Parnis which discussed more timely investigations, and the need for greater consultation with the medical profession on ways to improve the complaints process both for those making complaints and those subject of notifications.

Adrian Rollins

 

Primary Health Networks and Aboriginal and Torres Strait Islander health

One of six priorities set by the Australian Government is for Primary Health Networks (PHNs) to focus on the health of Aboriginal and Torres Strait Islander peoples.1 Announced in the 2014–15 federal Budget, PHNs aim to coordinate primary health care provision especially for those at risk of poor health outcomes. There are 31 PHNs across Australia with several formed from consortia of Local Hospital Networks (LHNs). Operational and flexible funding of up to $842 million was committed for PHNs over 3 years from 2015–16.2 It is timely for PHNs to consider how they will improve health care coordination in partnership with Aboriginal and Torres Strait Islander communities in their respective regions.

Efforts to reduce the high hospitalisation rates of Aboriginal and Torres Strait Islander (hereafter referred to as Aboriginal) people will require PHNs to build formal participatory structures to support best practice service models. Comprehensive primary health care can then be shaped by the needs of the community rather than by ad hoc factors or reactions to financial incentives and health care funding arrangements.3,4 Collaborations with Aboriginal community controlled health services (ACCHSs) within PHN regions have been recommended.5,6

This article outlines how PHNs might support health services to systematically and strategically improve their responsiveness to Aboriginal people within their boundaries according to ten proposals. These best practice models and examples can assist PHNs to adapt their strategic plans to optimally respond to this priority.

1. Collaborate with ACCHSs

ACCHSs are authorities on comprehensive primary health care matters at the local level4,7 and do much more than just cure illness.8 As authentic representational advocates, they can guide PHN responsiveness to Aboriginal health issues and, with more than 150 services across Australia, there are ACCHSs within the regional boundaries of every PHN.

The predecessors of PHNs — the Medicare Locals — were expected to engage with ACCHSs for many Closing the Gap initiatives, such as the Indigenous Chronic Disease Package (ICDP), from 2008.9 Where meaningful partnerships between ACCHSs and Medicare Locals were established in the delivery of these programs, health outcomes for Aboriginal people substantially improved (Box 1).10

2. Establish an Aboriginal and Torres Strait Islander steering committee

PHNs can foster meaningful Aboriginal community engagement by establishing an Aboriginal and Torres Strait Islander steering committee (and Aboriginal representation on the PHN board) with membership led by ACCHSs representatives inclusive of other Aboriginal health service organisations. Similar partnership forums established between the ACCHS, general practice sectors, and state and territory governments have set Aboriginal health priorities at the jurisdictional and regional level for decades.11 The steering committee aims might be modelled on current partnerships between the LHN and ACCHSs (Appendix 1) to develop a strategic plan across the life course.

3. Establish formal agreements to support the strategic plan

PHNs should aim for partnerships to reorient health services from reactionary care to comprehensive primary health care. For example, in remote Western Australia, a partnership agreement between an ACCHS and state government health services was associated with a reversal of the increasing trend in hospital emergency department attendances among other substantial health improvements in only 6 years (Box 2).12

Partnership agreements between PHNs, ACCHSs and other agencies should support Aboriginal leadership, quality care, accountability and patient-centredness, and should be formalised from non-binding memoranda of understanding to binding contracts (Box 1 and Box 2) to support a long term vision for core activity that is flexible to local priorities.

4. Support health services to assess their systems of care

There are now health system assessment tools specifically adapted to optimise the primary health care of Indigenous Australians based on the Chronic Care Model.13 Over 200 Aboriginal primary health care services have used such tools (Appendix 2). Many ACCHSs self-audit their performance using clinical audit tools for chronic disease, maternal and child health and other health priorities, and undertake generic health systems assessment as part of continuous quality improvement (CQI).14

Health system assessment and audits of actual practice against best practice standards should be used to guide PHN (and LHN where there is conjoint responsibility) priorities to systematically enhance quality care within all primary care services in PHN boundaries. Barriers to and enablers for systems improvement, and gaps in health service responsiveness to Aboriginal health needs, will be clearer. These include improving systems for follow-up of patients, use of electronic registers and recalls, Aboriginal community engagement and leadership, the commitment of workforce and management, service infrastructure, and staff training and support.14,15

5. Embed quality assurance activity within primary health care services

A commitment to CQI is a key strategy for disease prevention (Appendix 3) and the prevention of avoidable hospitalisations (Appendix 4), and should be a universal feature of primary health care services providing care to Aboriginal and Torres Strait Islander peoples.15

A national Aboriginal and Torres Strait Islander CQI framework supported by the Australian Government will shortly be released to guide jurisdictions to assess and deliver better quality primary health care.16 PHNs should endorse and adapt this framework to coordinate efforts and develop CQI implementation plans. For example, most state and territory affiliates of the National Aboriginal Community Controlled Health Organisation provide support to ACCHSs for CQI activities; and in some jurisdictions (Queensland and the Northern Territory), CQI support programs are well developed.14

PHNs will need to engage with existing programs to identify strategies for and barriers to CQI. Supporting CQI within the network boundary will require regional facilitators, trained staff, the coordinated use of shared electronic medical records and use of local information management systems by all providers (including locums and visiting services), regular monitoring of CQI indicators, performance reporting, and agreements on data use, ownership and reporting.14

6. Expand primary health care performance reporting

All primary health care services within each PHN delivering care to Aboriginal people (and especially in receipt of financial grants or incentives specific to Indigenous Australians) should be required to undertake CQI, and to participate in regional or centralised performance reporting which can be disaggregated by Aboriginality. Primary health care performance should be a core responsibility of quality, safety and risk subcommittees of both PHNs and LHNs.3 Aggregated CQI data at PHN levels can identify health service gaps and areas that need to be improved.16

The Australian Government reporting framework for PHNs will include national, local and organisational performance indicators.17 National indicators for PHNs will include primary and community health indicators such as potentially preventable hospitalisations (these will be sourced from existing datasets such as the National Hospital Morbidity Database) not unlike what is currently reported for LHNs. Potentially preventable hospitalisations are an indirect measure of whether people are receiving adequate primary health care. The disproportionately high rate of illness affecting Aboriginal people and their poorer access to primary health care explains higher potentially preventable hospitalisation rates independent of age, sex and remoteness (Appendix 4). Age-standardised potentially preventable hospitalisation rates within PHN boundaries should be disaggregated by Aboriginality and incorporated as a performance indicator within PHN strategic plans.

The selection of local and organisational performance indicators by PHNs should be guided by the Aboriginal steering committee. Benchmarking PHN progress using Aboriginal and Torres Strait Islander national key performance indicators18 should be considered. National key performance indicators serve as both a CQI tool and performance measure in the provision of primary health care to Indigenous Australians. For example, ACCHSs are required to report on 19 key performance indicators through a standardised portal supported by the Australian Government.7 Organisational performance reporting of PHN activity should quantify the allocation of funds towards Aboriginal programs and contractors and identify whether these are ACCHSs or other services.

7. Align and endorse PHN and LHN strategic plans

Commitment to region-specific Aboriginal primary health care strategic plans should be the goal for both PHN and LHN boards so that actions are informed by both and integrated to avoid cross purposes.3 These linkages might be streamlined in regions where PHNs have been established by LHNs. However, it is unclear how many LHNs have established Aboriginal health subcommittees or effective and formalised Aboriginal community engagement mechanisms to facilitate endorsement of strategic plans.

All PHNs are expected to complete baseline needs assessment and strategies to respond to service gaps.19 If these submissions pertain to the Aboriginal and Torres Strait Islander population, they should be accompanied by evidence of endorsement by the Aboriginal representative bodies in their region.

8. Strengthen the primary health care service model

Many visiting health providers can overburden Aboriginal people in remote communities with overlapping and poorly explained services.20 A core priority for PHNs is to review the coordination of care and improve clinical pathways in all geographic regions. PHNs will need to review the efficiency of current services including generalist and specialist outreach if they are to avoid duplication, foster local or residential health services,21 and sustain local CQI systems.

Specialist outreach should complement local health services through a bottom-up approach integrated with primary health care. Specialist outreach services operating independently of existing primary health care services will need review. Service reforms might mean building hub-and-spoke models involving ACCHSs, supporting regional Aboriginal health networks (Box 1), using telehealth adapted for Aboriginal and Torres Strait Islander settings, renegotiating clinical pathways, empowering local outreach coordinators of hospitals to support primary health care models, substituting workforce tasks through nurse and Aboriginal and Torres Strait Islander health practitioners, rural generalists and physician assistants,22 and reorienting health services towards primary health care (Box 2, Appendix 5).

9. Enhance cultural competence of primary health care services

Strategies to merely increase the awareness of non-Indigenous health staff to Aboriginal cultural protocols are often recommended to reduce Aboriginal health disparities, but may not lead to cultural competence.23 Some staff still struggle with how to make services culturally responsive beyond the posting of Aboriginal artwork.20 The ICDP invested in cultural awareness training of over 6000 general practice staff but change in practice was not universally embraced.8,15

Enhancing the cultural competence of health services within PHN boundaries will require strategies best managed by the Aboriginal steering committee and may include subcontracting ACCHSs and expanding their outreach role, person-centred and family-oriented care, fostering a culturally identified workforce that reflects the patient population and health needs, staff training in cultural safety, performance measures for cultural competence, and future planning.

10. Transition primary health care services to Aboriginal community control

PHNs are to be the health “providers of last resort and their decision to directly provide services should require the approval of the Department of Health”.3 Agreement from local ACCHSs in the region should be required if a PHN opts to directly provide health services to Aboriginal communities rather than make purchasing arrangements with existing Aboriginal services (Box 1). ACCHSs should also be supported to choose their involvement in programs within the PHN boundary.

Coalitions of Aboriginal organisations have advised that future funding on Indigenous health programs be prioritised to ACCHSs being better placed to meet Aboriginal health needs with better returns on investment.24 For example, according to the ICDP evaluation, it was unclear whether Closing the Gap measures (such as financial incentives to general practices) increased the provision of services to Aboriginal people who are “hard to reach” or increased their access to primary health care.9,15 For PHNs, it makes sense to direct Aboriginal health strategies to health services with the desire and potential to provide quality care to Aboriginal patients, which is also more cost-effective.15

The Queensland and Northern Territory governments have commenced processes to transition certain health services in remote areas to Aboriginal community control.25,26 The aim is to reform remote area services from doctor-focused, illness-centred, acute hospital-based primary care services to community-engaged, comprehensive, preventive and responsive systems. Policy frameworks propose staged approaches and capacity building of existing ACCHSs. Strategic plans developed by PHNs should consider transitioning health services and responding to existing transition plans26 to ensure alignment with them.

Conclusion

These proposals offer policy makers and PHNs a framework for health service planning within newly established boundaries, and may inform PHN organisational performance reporting on efforts to close the gap in Aboriginal health disparity.

Australians report that our health system is not sufficiently patient focused. Primary care is reactive and episodic, funding structures support providers and not patients, and there is little accountability for health outcomes.4 Through existing and better targeted additional investments, PHNs can offer Aboriginal and Torres Strait Islander people some hope towards reforming access to and quality of primary health care in their localities, but only if programs and systems can better fit in with community needs. To close the gap, PHNs need to support Aboriginal communities towards greater participation in primary health care, ultimately through the expression of community control.

Box 1 –
Example: collaboration between Medicare Locals and Aboriginal community controlled health services

In 2008, to close the gap in Aboriginal health disparity, the Indigenous Chronic Disease Package funded the Care Coordination and Supplementary Services (CCSS) program. The program supports Aboriginal and Torres Strait Islander patients with complex care needs, by coordinating clinical care and providing supplementary funding for allied health, specialists, transport services and medical aids. Implementation required collaborations between Aboriginal and mainstream health services. In South-East Queensland, the Metro North Brisbane Medicare Local (MNBML) was funded to deliver the CCSS program on behalf of four other Medicare Locals in South-East Queensland. A consortium of ACCHSs — the Institute for Urban Indigenous Health (IUIH) — was subcontracted by the MNBML to implement the program in 2013–14. IUIH employed a manager to oversee the program delivered by 20.5 full-time equivalent care coordinators.

The IUIH reported that subcontracting delivered significantly more services to significantly more Indigenous Australians with complex chronic conditions than any other part of the country: “In 2013–14 IUIH and members [ACCHSs] delivered over 57 000 episodes of care via the CCSS Program. The delivery of intensive case management and access to a comprehensive range of specialist and allied health services and medical aides for this population has avoided costly hospital admissions for Government and significantly improved the health and wellbeing of some of our most vulnerable and unwell patients.”10

Box 2 –
Example: a partnership to reorient acute care to comprehensive primary health care

Clinic services in the very remote Fitzroy Valley in Western Australia are delivered by state government health services (Fitzroy Crossing Hospital and the Kimberley Population Health Unit for community health services). Non-clinical health services are delivered by the Aboriginal community controlled health service (Nindilingarri Cultural Health Services) to a population of 3500 (80% Aboriginal). These services comprise healthy lifestyle programs designed around Aboriginal culture. A formal agreement between the agencies was negotiated in 2006 to form a single governance structure to allocate funding, share an e-health record, and coordinate health promotion, cultural safety, acute inpatient care, primary care and specialist care, and population-based screening. Commonwealth funding supported the development of a shared e-health record for quality improvement and additional staff (through the Healthy for Life and Indigenous Chronic Disease programs), and provided Medicare rebates to patients for primary care services delivered at the hospital clinic (an exemption from section 19(2) of the National Health Insurance Act 1973). Medicare billings were reinvested to support this reorientation under the guidance of the partners.

This reorientation enhanced health promotion programs and was associated with a reversal of the increasing trend in emergency department attendances. In the primary care clinic, there was a substantial increase in the number of patients seen, the number of health checks, the detection of risk factors, the proportion of patients with diabetes having care plans, transport provision, cultural security and follow-up attendances in only 6 years.12

Serious adverse event reporting in investigator-initiated clinical trials

New drugs and medical devices offer improvements in health care. Clinical research is undertaken to elucidate such benefits, but also to identify potential harms. Adverse event (AE) and serious AE (SAE) data are crucial information in drug and device development studies (Box 1). Definitions and requirements for safety reporting in Australia are outlined by the international guidelines,1 and the National Health and Medical Research Council (NHMRC) National statement on ethical conduct in human research.2 Further, the NHMRC has provided clarification on how AEs should be reported and who has responsibility for reviewing and acting on them.3 Such guidelines are practical steps to ensure safety for participants in all research involving interventions, including post-marketing surveillance and Phase IV trials (Box 2) of approved medicines, treatments and devices.

However, there is quite marked variation in how, to whom and indeed if such safety reporting is required in investigator-initiated clinical trials. Any such requirement needs to take into account the context of the study, including its phase, the existing health conditions of the participants, and the risks of the proposed intervention over and above standard care. Moreover, the burden and cost of reporting should be proportionate with the public health benefit of this information and be closely linked to a clear need to establish a safety profile, as opposed to simply fulfilling a perceived reporting requirement.

Unfortunately, in Australia there are ever-increasing complexities and burdensome requirements from sponsors and human research ethics committees (HRECs) to report everything without necessarily paying heed to the principles outlined in the guidance documents. The increased burden on investigators and research coordinators is increasing the workload and costs of clinical trials,4 and decreasing opportunities for trial recruitment and participation of centres at the local level. This has led to a decrease in the uptake of research, particularly less well funded investigator-initiated trials.5

We, and others,6,7 believe that individual AE reports to HRECs serve no useful purpose because in most cases the study group identity (drug exposure) is not known, and in any case their value is limited (being a single case report). Further, such reporting is not explicitly mandated by existing guidelines; it is only an option that an HREC may decide to require. In a recent public consultation of safety reporting in Australia, the NHMRC identified several areas for potential reform but did not make specific reference to the issues arising in investigator-initiated studies employing comparisons of approved interventions.8 The purpose of this commentary is to draw attention to these issues and to present some solutions.

Post-marketing surveillance and Phase IV pragmatic drug trials

Although safety reporting is essential for investigational drugs and devices when they are being used for the first time or in a novel indication (Phase I and II, and some Phase III studies), the manner in which they are collected, reported and reviewed should always be proportionate to the size, complexity and risk of the proposed use.9 In situations in which they are being used within their approved indication but may be delivered in the setting of a pragmatic Phase IV drug trial, it can be argued that the threshold for reporting should be commensurate with that expected of their use in routine clinical practice. That is, as all health care professionals are expected to report adverse reactions to medicines through national reporting systems,10–13 we contend that this system should also guide appropriate safety reporting for Phase IV trials.

Known events, as listed in the product information document, need not be reported as AE reporting in post-marketing Phase IV trials. Known AEs should be compiled in an appendix to the study protocol so that investigators and research staff will know of these facts.

By defining safety endpoints in a study protocol, appropriate data can be collated and reviewed for safety and efficacy by a properly constituted trial data safety and monitoring committee (DSMC). Therefore data review can occur in a meaningful way, by independent experts, with blinded separation of treatment groups enabling useful comparisons for the whole dataset. This then will enable the generation of a concise and relevant safety report for dissemination and review by all participating HRECs. This will relieve overburdened HRECs from having to deal with large numbers of reports which contain minimal information relevant to the true safety of the product under investigation.

Reporting requirements in the United States and the European Union

In Australia, it has been our experience that some HRECs insist on blanket reporting of any event (expected or unexpected, severe or otherwise, treatment-related or otherwise) during a predefined period, using a stringent interpretation of NHMRC guidelines.14 The US Food and Drug Administration (FDA) issued updated guidelines in 2009,15 stating that AEs listed in the investigator’s brochure would, by definition, not be considered unexpected and thus do not require reporting to HRECs. The European Union has adopted similar guidelines.12 That is, an AE should only be reported if the site investigator would be prepared to report the event as part of routine clinical care.

An Australian solution: defining what should be reported

The role of the DSMC is to assess, at predefined intervals, the progress of a clinical trial, safety data and critical efficacy variables, and to recommend to the sponsor whether to continue, modify or terminate a trial.16,17 It is the responsibility of the DSMC to safeguard the individual trial subjects while enhancing the integrity of the trial.18 A DSMC should be made up of individuals who are independent from the trial, but with an understanding of the trial and its components.19 The steering committee of the trial should produce a charter or operating procedures for the DSMC before commencement of the trial,22 and this document should outline the defined role and reporting pathways of the DSMC.

An important role of an HREC is to ensure, with few exceptions, that a clinical trial protocol requires safety (and other) data to be reviewed by a DSMC. In the US, the Office for Human Research Protections and the FDA have agreed that such duplicate reporting (to a DSMC and an institutional review board [IRB; equivalent to an Australian HREC]) is not required,20 and that it has the effect of “inhibiting, rather than enhancing, the ability of IRBs to adequately protect human subjects”.15

The DSMC is in the unique position of being able to review safety information according to the blinded, and sometimes unblinded, treatment arms of the study; that is, it can view the safety data by group and also by site.21 The DSMC can then provide a summary report to the trial steering committee, principal investigators, and in turn the relevant HRECs in a meaningful way (Box 3).

What should be reported?

For Phase IV trials, universal safety endpoints should be routinely collected,16,21 reducing the administrative and AE reporting burden as well as enhance vigilance. The methods used to document these safety endpoints should be described in the protocol, and the data analysed at appropriate time points as determined by the risk profile of the intervention. The International Council for Harmonisation (ICH) guidelines for good clinical practice have identified such universal safety points.12 Universal safety endpoints should include death, readmission or admission to hospital, and return to surgery (if relevant) — these being the major concerns for increased risk to patients, clinicians, HRECs and regulatory bodies. Data collection and AE reporting need to be more meticulous if a study is primarily designed to specifically evaluate safety.

In line with the ICH guidelines12 adopted by the regulatory bodies of the EU, Japan and the US, reportable safety data should be related to drug reactions deemed serious and unexpected. This is consistent with the recommendations of the Council for International Organizations of Medical Sciences,22 which provides essential risk management components. Therefore, reportable events should be defined by:

  • A single occurrence of a serious, unexpected event that is uncommon and strongly associated with drug exposure (eg, angioedema, agranulocytosis, Stevens-Johnson syndrome).

  • A single occurrence, or more often a small number of occurrences, of a serious, unexpected event that is not commonly associated with drug exposure, but uncommon in the study population (eg, tendon rupture, progressive multifocal leukoencephalopathy).

  • AEs should be collected and classified enabling identification of relevant signals in the study, according to treatment group.

  • An AE that is described or addressed in the investigator’s brochure, protocol, or informed consent documents, but occurs at a specificity or severity that is inconsistent with prior observations (eg, if transaminase elevation is listed in the investigator’s brochure and hepatic necrosis is observed in trial subjects, hepatic necrosis would be considered a reportable AE).15

Conclusions and recommendations

Pragmatic, investigator-initiated Phase IV clinical trials of post-marketed drugs or devices play a critical part in understanding their role in the “real world” of everyday clinical practice.13 In this setting, the workload and costs of systematic, complete reporting of all AEs and SAEs (independent of whether these are treatment-related) is wasteful and mostly unnecessary. There needs to be consistency and relevance applied to SAE reporting in clinical trials.

Definitions of safety events, inclusion of expected trial events for the population under study, interim analyses, DSMC review and a statistical analysis plan should be detailed in the protocol for Phase IV clinical trials.

Defined trial endpoints do not need to be reported as safety events because they are being properly monitored and analysed. Commonly reported SAEs such as events leading to significantly increased hospital length of stay, readmission to hospital or death should be mandated as endpoints in all Phase IV clinical trials.

To reassure HRECs, study participants and the general public, good clinical practice training and certification should be mandated when accepting the role of principal investigator in any clinical trial.

Box 1 –
Types of adverse advents

Adverse event (AE): An AE is any untoward medical occurrence in a patient administered a medicinal product which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavourable and unintended sign (eg, an abnormal laboratory finding), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product.

Serious adverse event (SAE): An AE is serious and should be reported if it results in death, is life-threatening, requires inpatient hospitalisation or results in prolongation of existing hospitalisation, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is a medically important event or reaction that may require medical or surgical intervention to prevent one of the other outcomes.

Suspected unexpected serious adverse reactions (SUSARs) and unanticipated serious adverse device effects (USADEs): A serious adverse event with a possible relationship to the study drug, device or procedure should be considered unexpected or unanticipated if the nature, severity or frequency of that event is not consistent with the information on the investigator’s brochure, device information or in the current Australian product information.

Box 2 –
Types of clinical trials

Phase I study: A new drug or treatment is evaluated in a small number of participants (usually young, healthy males) for the first time to assess safety, dosage range, and identify any adverse effects. The aim is to quantify pharmacokinetic or other performance characteristics, as well as gain an initial safety profile in humans.

Phase II study: The drug or treatment is given to a larger group of participants (often including those with a specific condition) to assess whether it is effective and to further evaluate safety. The main aim is to establish an appropriate dosage schedule to test in later phase studies.

Phase III study: The drug or treatment is given to large groups of participants with a specific condition, most often in the setting of a double-blind randomised trial, to assess its efficacy and adverse effects in comparison to placebo or other established treatments. Results will inform the drug/device product information document. The aim is to use these data to gain marketing approval for use in clinical practice.

Phase IV study: Further studies are carried out after the drug or treatment has been marketed to gather information on the drug or device’s effectiveness in broader populations, and any side effects associated with long term use.

Box 3 –
Trial reporting pathways for investigator-initiated research

Australia’s health: being accountable for prevention

Stronger policy and a comprehensive approach to prevention is a good investment

The global action plan of the World Health Organization (WHO) for preventing and controlling non-communicable diseases1 aims to substantially reduce the burden of premature mortality caused by cancer, diabetes, cardiovascular disease and chronic lung disease by 2025, through action on nine targets measured by 25 indicators of performance.1 As a member state of the WHO, Australia is committed to the global action plan. However, we argue that the Australian government devotes insufficient attention to health policy, funding and program implementation for effective prevention. In this article, we review Australia’s progress against national non-communicable disease (NCD) targets set in 2009, and suggest that a more comprehensive approach is required.

NCDs are responsible for nine out of ten deaths in Australia, and often reduce people’s quality of life and ability to function. Tackling the growing personal, social and national economic impact of NCDs (also referred to as chronic diseases) is imperative, particularly in a country with an ageing population. Some population groups, such as Indigenous Australians and socio-economically disadvantaged people, are affected more than others.

Many NCDs share common, preventable risk factors such as smoking, harmful alcohol use, poor nutrition and physical inactivity. They contribute to biomedical risk factors such as obesity, hypertension and high cholesterol levels. Modifiable risk factors account for a substantial proportion of the burden of disease in Australia, with, for example, high body mass and physical inactivity explaining 60% of the health loss associated with type 2 diabetes.2 Seventy per cent of mortality from cardiovascular disease in Australia has been attributed to high blood pressure, high cholesterol levels and physical inactivity.3

Preventive health efforts

Effective prevention can improve health and reduce pressure on clinical services. Australia has had some outstanding successes in areas such as smoking, road trauma and heart disease. However, we lack a sustained, comprehensive and strategic approach to prevention, together with adequate funding, coordination and monitoring. In the 2013–14 financial year, spending on public health (which includes prevention activities) was only 1.53% of total recurrent health spending, and this proportion is declining.4 This places Australia out of step with similar countries. The Organisation for Economic Co-operation and Development reported in 2011 that Australia’s spending on prevention and public health as a share of total recurrent health spending was 2.0%, much less than in New Zealand (6.4%), Finland (6.1%) and Canada (5.9%).5

Health goals and targets can provide important foci for action. The National Preventative Health Taskforce was established in 2008 to develop a National Preventative Health Strategy (NPHS). The NPHS focused on obesity, tobacco and alcohol, and set a number of targets for prevention. Also in 2009, the Council of Australian Governments (COAG) National Partnership Agreement on Preventive Health (NPAPH) set a number of complementary targets.

In addition to targets, the NPHS outlined actions, including 32 alcohol-specific actions and 27 obesity-specific actions. A review of progress against the alcohol-specific actions in 2013 by the Foundation for Alcohol Research and Education found that four actions had been completed, 18 were progressing, and no progress had been made against ten actions.6 With respect to the obesity actions, a similar recent analysis by the Obesity Policy Coalition found that of these three had been completed, 17 were progressing and no progress had been made against seven (Jane Martin, Obesity Policy Coalition, personal communication).

The recommendations of the NPHS included establishing the Australian National Preventive Health Agency, which was set up in 2011, but abolished in June 2014. The NPAPH was also abolished in 2014, which resulted in the removal of $374 million of funding to the states. In the context of substantial policy change, and disinvestment in prevention, it is timely to review the progress made against the NPHS and NPAPH goals. This review is shown in the Box.

What needs to change?

With the exception of tobacco control, the data suggest there is little or no progress being made in preventing and controlling risk factors for chronic diseases in Australia.

Failure to make progress in relation to poor nutrition, physical inactivity and harmful alcohol use is linked to a failure of implementation. Expert advice about these risk factors has been sought and obtained by many governments, but implementation of expert recommendations has often been lacking.

Despite government concern about the sustainability of the health system, Australia is not currently investing significantly in disease prevention. Assessment of progress against national goals and targets from 2009 suggests that Australia is failing to take adequate steps to reduce these modifiable risk factors, although some progress is being made with Indigenous child health.

Our country is a global leader in tobacco control, and this has generated considerable benefits for the population, health system, and economy. A comprehensive approach, including taxation to make tobacco products more expensive, media campaigns, regulation of tobacco products and targeted interventions for vulnerable groups has contributed to the reduction in smoking. Sustained bi-partisan commitment has been important to our success in tobacco control.

The reduction in the prevalence of smoking in Australia has occurred in the face of considerable opposition from tobacco companies. Progressive undermining of evidence-based policy by unhealthy commodity industries,13 and weakening of public health approaches to chronic disease prevention is common in Australia and other countries.14

Comprehensive and courageous approaches to other risk factors, such as poor nutrition, physical inactivity and alcohol would significantly benefit the health of Australians. Evidence-based interventions, including taxation, media campaigns, regulation of unhealthy products and targeted interventions could reduce the chronic disease burden in this country,15 and hence the pressure on our health system. Often, these are also the most cost-effective interventions, and a number are cost-saving. Improving diet and nutrition presents a different set of challenges than do single-risk-factor problems such as smoking or excessive salt intake. However, it is important that diet and nutrition are tackled, given that most Australian adults are overweight or obese, and (unlike England and the United States) rates have continued to rise in Australia.16 All interventions, and their impact on NCDs and risk factors, should be rigorously evaluated.

Continuity and progressive change towards improved health are important. Taking on the problems of tobacco and the road toll has involved many governments implementing a range of actions over decades. Many of the states and territories are active in the area of preventive health, and the federal government is currently developing a National Strategic Framework for Chronic Conditions. The COAG Community Care and Population Health Principal Committee may provide a forum where a preventive health agenda can again be taken forward.

Conclusion

Australia invests less in prevention than do other comparable countries, and our investment is declining. The burden of NCDs is high; more than seven million Australians are living with a chronic condition, and we are failing to meet most of the national targets set by COAG and the NPHS in 2009.

NCDs have a high personal, social and national economic impact. If we seek to achieve significant reductions in the burden of chronic disease in Australia, sustained, comprehensive and courageous approaches are required.

Box –
Preventive health targets and progress in Australia since 2009

Target and source

Target year

Progress against target

Results

Halt and reverse the rise in overweight and obesity (NPHT)

2020

Not on track

71% of Australian men and 56% of women are overweight.7 One in four adults are obese, and 27% of children are overweight or obese. Australia is not on track to meet either COAG or NPHT targets for overweight and obesity7

Increase the proportion of children and adults meeting national guidelines for healthy body weight by 3% within 10 years (COAG)

2019

Not on track

Increase the proportion of children and adults meeting national guidelines for healthy eating and physical activity by 15% within 6 years (COAG)

2015

Healthy eating not on track; physical activity on track

In 2014–15, only 5.1% of adults and children ate enough fruit and vegetables7 — the COAG target for adults is 21.2%55% of adults met the physical activity recommendations in 2014–15.7 The COAG physical activity target for adults for 2015 is on track

Reduce the prevalence of daily smoking among adult Australians aged 18 + years from 17.4% in 2007 to 10% or lower (NPHT)

2020

On track

Progress with regard to smoking has been very encouraging. Between 1993 and 2013, the proportion of daily smokers aged 18 years or older halved from 26.1% to 13.3%8

Reduce the proportion of Australians who drink at short-term risky or high-risk levels to 14%, and the proportion of Australians who drink at long-term risky or high-risk levels to 7% (NPHT)

2020

Unlikely to be met

19.1% of Australians drank at long-term risky levels in 2013.9 A recent study showed increases in medium- and high-risk drinking among adults from 2001 to 2011–12,10 consistent with very high levels of alcohol-related hospital emergency department presentations.11 Of concern are high levels of risky drinking among people aged 15–18 years and increasing risky drinking levels among young women

Close the gap in life expectancy within a generation (COAG)

2031

Not on track

Limited progress. The current rate of progress will have to gather considerable pace if the target is to be met12

Halve the gap in mortality rates for Indigenous children under five within a decade (COAG)

2018

On track

Long-term progress. Between 1998 and 2013, the Indigenous child death rate declined by 31%, and in the same period, there has been a 35% narrowing of the gap in child death rates between Indigenous and non-Indigenous children12

COAG = Council of Australian Governments. NPHT = National Preventative Health Taskforce.

Time to raise the minimum purchasing age for tobacco in Australia

Increasing the minimum purchasing age may be the most straightforward way towards a tobacco-free generation

Australia is often described as a global leader in tobacco control. While this is true, it has taken two generations to reduce current smoking rates to around 15% of the population, and there is still a long way to go, especially in disadvantaged communities.

Tasmania’s tobacco-free generation

One innovative idea on the road towards minimising smoking rates is currently before the Parliament of Tasmania. Put simply, passage of the Public Health Amendment (Tobacco-free Generation) Bill 20141 would ensure that people born after 1999 could not lawfully purchase tobacco at retail outlets within Tasmania in their lifetimes.2

The Tobacco-free Generation Bill was introduced into the Legislative Council by independent member Ivan Dean, whose father was a chain smoker and who suffered terribly before dying from lung cancer.2 The Bill is currently being considered by a Parliamentary Committee, and public submissions can be viewed online.3

The Tobacco-free Generation Bill defines a member of the tobacco-free generation as “a person born on or after 1 January 2000”.1 Although the Bill does not prohibit smoking by a member of the tobacco-free generation, it does create a new offence for retailing tobacco within Tasmania to such a person, with a first-time penalty of up to $7700.1 By contrast, there would be no offence for retailing tobacco to adults born before 2000. As time passes, the legislation would create two distinct classes: adults who, by virtue of being born before the year 2000, would be entitled to continue to purchase tobacco during their lives; and adults of the tobacco-free generation, born after 31 December 1999, who would be forever unable to purchase tobacco lawfully in Tasmania.

The Bill would create an offence for a member of the tobacco-free generation to use a false proof of age to purchase tobacco, with a fine of up to $540. However, it would not penalise friends or family members from purchasing tobacco that was subsequently consumed by a member of the tobacco-free generation, provided that the tobacco was given, rather than sold, to that person.1,2

In its submission to the Committee, the Tasmanian Government raised concerns that the Bill would encourage interstate and internet purchases of tobacco by smokers who were members of the tobacco-free generation, thereby undermining the goals of the Bill. It pointed to the Tasmanian Population Health Survey as evidence that current policies were reducing smoking rates effectively.4

Predictably, the Bill has been opposed by tobacco manufacturers. One submission by a retailer on Flinders Island suggests that Imperial Tobacco systematically urged retailers to lobby the Legislative Council against the Bill.5

Seizing the opportunity for leadership in tobacco control in Tasmania

Tobacco is more than just the “risky product” that tobacco companies claim it to be. If today’s smokers do not quit, a majority will die prematurely from tobacco-related diseases. A study by Banks and colleagues published in 2015 suggests that about two-thirds of deaths among current smokers are caused by smoking.6 The authors followed more than 200 000 smokers aged over 45 years, for 4 years, showing that even ten cigarettes daily doubles a person’s risk of dying prematurely. As Tasmania’s popular former Premier, Jim Bacon, said in 2004, when he quit politics following a diagnosis of lung cancer: “The message from me to everyone is please don’t be a fool like me, don’t keep smoking, try and give it up and if you are young and you haven’t started, don’t start”.7

The idea of legislating for a tobacco-free generation is one of a growing number of tobacco endgame scenarios promoted by tobacco control advocates.8 While it is plausible that the Bill could discourage smoking initiation by members of the tobacco-free generation, it is possible that the Bill might also inadvertently create a permanent “outlaw class” of adults addicted to tobacco but unable to purchase it lawfully within Tasmania. Achieving acceptance about why two adults of different ages should be treated differently in terms of their right to access tobacco is one of the challenges the Bill faces.

If the Tobacco-free Generation Bill does not succeed, the opportunity remains for Tasmania to show national leadership by raising the minimum purchase age for tobacco to at least 21 years. A higher minimum purchase age would make it more difficult for youth to access tobacco at the ages when they are most vulnerable to smoking initiation.

The case for raising the minimum purchasing age for tobacco

The case for a higher minimum purchasing age is supported by four key factors. According to the National Drug Strategy Household Survey (NDSHS), in 2013 Tasmania’s daily smoking rate for people aged 14 years and above was 16.1%. This is second only to the Northern Territory (21.3%) and well above the national average of 12.8%.9 The same survey found that 31.6% of Tasmanians aged 18–24 years were daily smokers — more than twice the Australian average of 13.4% for this age category.9 In its submission to the Parliamentary Committee, the Tasmanian Government quoted the Tasmanian Population Health Survey 2013, which estimated a 19.7% smoking rate for 18–24-year-olds, and a 15% smoking rate overall.4 The Tasmanian Department of Health and Human Services has acknowledged that due to its methodology, the Tasmanian survey significantly under-represented males and younger age groups.10 NDSHS estimates suggest that smoking rates remain alarmingly high in young adults and that innovative strategies are needed to ensure that Tasmania does not fall further behind the mainland states.

Second, a higher minimum purchasing age for tobacco could help to reduce high smoking rates among young pregnant women. In 2013, the average rate of smoking while pregnant was 12% nationally,11 but was 15% in Tasmania and in excess of 33% among pregnant Tasmanian women aged under 20 years.12

Third, a higher minimum purchasing age for tobacco could go a long way towards encouraging the creation of a tobacco-free generation, since few people begin smoking for the first time after their mid-20s. A 2013 study of smoking initiation rates in New Zealand confirmed that while initiation after age 24 is rare, the highest initiation rates occur among those aged 15–21 years. Over a 4-year period, the rate of smoking initiation for those aged 15–17, 18 and 19, and 20–24 years was 14.2%, 7.0% and 3.1%, respectively.13

Finally, in the United States, at least 30 cities and counties, including New York City, have set 21 years as the minimum purchasing age for tobacco products.14 Four states have raised the minimum purchase age to 19 years,14 and in June 2015, Hawaii raised it to 21 years for both tobacco products and e-cigarettes.15 If linked to a proof-of-age swipe card or so-called smoker’s licence, the right to purchase tobacco could be verified instantaneously.16

In its recent report, an expert committee of the US Institute of Medicine concluded that raising the minimum purchasing age for tobacco would delay initiation by adolescents, with the largest proportion of reductions being among teenagers aged 15–17 years.14 Based on modelling, the Committee concluded that while raising the minimum purchase age to 19 years would likely yield only a modest reduction in smoking initiation by 18-year-olds, raising it to 21 years would substantially reduce smoking prevalence and smoking-related mortality, given the numerous life transitions that young adults experience between 18 and 20 years. The Committee concluded that increasing the minimum purchasing age would also improve fetal, maternal and infant health outcomes by reducing the likelihood of maternal or paternal smoking.14

A survey of Australian secondary school students’ use of tobacco found that in 2014, nearly 18% of 16–17-year-old smokers purchased their last cigarette from a commercial source.17 This finding underscores the need for rigorous enforcement of existing laws to further reduce the retail availability of tobacco to underage youth. However, the fact that current underage smokers mostly obtain their tobacco from non-commercial sources does not demonstrate that current age restrictions have no impact on youth smoking rates, nor that a higher minimum age would be similarly ineffective. Rigorously enforced age restrictions reduce tobacco purchase attempts and youth smoking rates,14,18,19 although their impact is likely to be strengthened when integrated within a comprehensive set of tobacco control policies that maintain high retail prices and de-normalise tobacco use.14,20 These features would likely boost the impact of youth access laws by reducing or interrupting sources of social supply, inhibiting initiation and leading to decreased tobacco use rather than mere substitution of purchased tobacco with tobacco from social sources.20

Summary

The time has come for Australian state governments to seriously consider raising the minimum purchasing age for tobacco. If the Tasmanian Parliament fails to support the Tobacco-free Generation Bill, as seems likely, a higher minimum purchasing age could reinforce efforts to reduce smoking initiation and help to address the significant health inequalities that Tasmanians experience relative to the mainland states. Simple amendments to the provisions in the Public Health Act 1997 (Tas) that govern underage smoking could raise the minimum purchasing age and encourage the creation of a tobacco-free generation. In late December 2015, the Tasmanian Government released a 5-year strategic plan for health that includes raising the minimum legal smoking age to 21 or 25 as an option for consideration.21 This is welcome news. Rigorous monitoring and enforcement of legislative amendments to raise the minimum purchase age for tobacco would strengthen social norms against youth smoking, and generate data that could inform tobacco control policies in other states.

Closing the million patient gap of uncontrolled asthma

Australia’s burden of asthma requires structural reform in health care delivery

Asthma control is the principal aim of asthma management. Uncontrolled asthma impairs quality of life, increases exacerbation frequency, heightens risk of death, and is four times more costly to treat than controlled asthma. Therefore, results from a web-based Australian asthma survey are disappointing and disquieting.1

One-quarter of respondents did not regularly use asthma preventers, despite having uncontrolled asthma. Another 20% of respondents had uncontrolled symptoms even while regularly using preventers. If these figures are truly representative of the nation’s 2.3 million people with asthma, they suggest that about one million Australians have uncontrolled asthma. This is despite the fact that asthma guidelines have been available for 26 years.2,3 Fundamental reforms to providing asthma care are therefore needed. A new National Asthma Strategy is on its way, and may provide a platform for structural changes.4

The first therapeutic gap highlighted by the web-based survey was the lack of regular preventer use by many patients, despite having uncontrolled symptoms.1 These patients seemed to favour immediate symptom relief over long term disease control.1 Ironically, the present dispensing system reinforces such behaviour. Relievers are readily available over the counter, but preventers require prescriptions, necessitating additional effort, time and expense.

The logical solution to this problem is to re-design access to asthma medications. Preventers must be made more accessible. It is encouraging that the possibility of dispensing low-dose inhaled corticosteroids without prescription is now under discussion.4

A less palatable but arguably more important measure would be to detect and attempt to reduce the high volume dispensing of relievers without adequate concomitant preventers, because this pattern of medication use is implicated in asthma deaths.5 Such a move would require electronic coordination across pharmacies, with the ability to trigger referral for asthma review.6

These proposals would increase the rate of preventer dispensing, but they cannot guarantee adherence. One reliable way to improve preventer use would be to launch and promote a combined short-acting reliever and steroid preventer in a single device. This would ensure that every dose of reliever was accompanied by a corresponding dose of preventer. There is now evidence that as-needed use of an inhaled corticosteroid combined with a short-acting β-agonist improves symptoms in mild persistent asthma.7 There is less support for this approach in moderate to severe asthma, but an inhaled corticosteroid combined with a long-acting β-agonist may be used instead in such patients, for prevention and relief.8

The second therapeutic gap identified by the survey relates to patients who claim to take regular preventers, but whose asthma remains uncontrolled. The drivers for this situation are complex. Important contributing factors to this problem probably include limited understanding of the disease, incorrect inhaler technique, ongoing smoking and insufficient attention to other asthma triggers, such as aero-allergens, occupational exposures and non-specific irritants.3 These problems are challenging to solve within general practice consultations, and rebates may need to be adjusted so they are based on realistic consultation times. An alternative approach also under consideration is to fund asthma educators and organise the appropriate credentials for them.4 Finally, more patients could be encouraged to schedule regular reviews; for example, by discounting medication costs for those who do.

Even with optimal asthma management in primary care, a small proportion of patients will continue to have uncontrolled asthma, some of whom may be insensitive to corticosteroid-based therapies.3,9 These patients need to be reviewed by respiratory specialists, and automated prompts to activate referrals should be built into asthma review programs.

For the most challenging patients, evaluation at a dedicated “difficult asthma” centre provides additional benefits for outpatient respiratory consultations.10 Many of these patients will have truly severe asthma, but there are also high rates of misdiagnosis, comorbidities and psychosocial factors. According to results from a recent uncontrolled study, dealing with these issues through comprehensive multidisciplinary assessment can improve quality of life and use of health care services, and can also define the patient subgroups most likely to respond to the expensive biological agents now entering clinical practice.11

In the United Kingdom, there are at least 11 specialised centres for treating difficult asthma that operate along similar lines.11 In Australia, this concept is less well developed, and services with interest in difficult asthma vary widely in the scope of their protocols and the extent of multidisciplinary support. Agreement is needed on which patients warrant extensive assessment, and how such patients should be evaluated. Resources could then be channelled to match demand.

We suggest radical steps to curb excessive reliance on relievers, enhance preventer adherence, encourage asthma review, and provide specialised evaluation for the most complex patients. The ultimate challenge is to fully integrate all these measures for maximal impact. Technological solutions are necessary for unhindered data sharing and seamless clinical transition across all levels of asthma care.6

Asthma management in Australia has come a long way, but innovative strategies are needed to bridge the remaining gaps.

Affordable access to innovative cancer medicines — don’t forget the prices

Efforts to improve access to cancer medicines should not overlook exorbitant prices

On 17 September 2015, the much anticipated Senate report on the Availability of new, innovative and specialist cancer drugs in Australia was released.1 The inquiry preceding the report, which was triggered by concerns about inadequate and inequitable access to cancer medicines, had attracted over 200 submissions from doctors, patients, patient advocacy groups and government decision makers.

The report addressed the health burden of cancer on our society; the impact on patients of delayed access to cancer medicines; and the challenges of assessing cost-effectiveness, particularly for rare cancers. It also focused on ways of improving Australia’s processes of health technology assessment (HTA), by which we determine whether medicines are safe, effective and cost-effective.

Australia’s health technology assessment processes

In Australia, HTAs for medicines are carried out in two phases. First, a pharmaceutical company makes a submission to the Therapeutic Goods Administration, which assesses a medicine’s efficacy and safety. If the medicine is approved, an application can be made to the Pharmaceutical Benefits Advisory Committee (PBAC) to have the medicine subsidised by the Pharmaceutical Benefits Scheme (PBS). The PBAC assesses whether the medicine is cost-effective in comparison with existing therapies. For targeted therapies, approval may also be sought from the Medical Services Advisory Committee for “companion diagnostics” that determine whether patients are likely to respond to the treatment. If medicines are not subsidised by the PBS, patients and their doctors have to find other means to gain access to them, which may include enrolling in clinical trials, seeking treatment through public hospitals or appealing to pharmaceutical companies for free or subsidised access. If unsuccessful, patients are left with the pressure of raising the money themselves or having to forgo treatment.

Those advocating in the Senate report for reform argued that patients are forced into these situations far too often because Australia’s HTA processes are antiquated, inflexible, unpredictable and inequitable — particularly for those with rare cancers, young people with cancer, and cancer patients located in rural and remote regions.

Proposed solutions to these problems included:

  • providing multiple HTA pathways;

  • prioritising the resources of regulators and payers so that the most important and complex medicine applications are given the most attention;

  • enabling better coordination between decision-making bodies to speed up decisions;

  • enabling better communication with pharmaceutical companies to set expectations early and thereby reduce failures;

  • leveraging off decisions made by overseas regulators with comparable evidence standards;

  • taking greater account of indirect economic benefits and outcomes, such as improvements in productivity; and

  • having greater focus on outcomes important to patients and doctors.

It was also suggested that because companies may not be commercially motivated to seek approval for non-commercially attractive uses of their products, it should be made easier for physicians, patient advocates and other stakeholders to make applications. To help regulators and payers make timely decisions, often in the midst of great uncertainty about real benefits, harms and costs, it was also proposed that there should be broader use of “managed entry” schemes in Australia — that is, schemes in which further evidence is generated after approval by the regulator or payer.

Cost of new cancer drugs

While it is important for Australia to refine its HTA principles and processes, what was notably absent from the Senate report was an in-depth consideration of why new cancer medicines cost so much, and what can be done about it. Many new cancer drugs cost more than $100 000 per treatment,2,3 and it has been shown that in the United States the launch price of cancer medicines has increased by 10% per annum over almost 20 years.4 These prices mean that unsubsidised medicines are well out of the reach of all but the wealthiest individuals, and they place intense political pressure on governments to subsidise medicines that would otherwise have been considered too expensive or supported by insufficient evidence.

The report’s overlooking of drug prices is significant because adjusting HTA processes to provide earlier access to more drugs without reforming the way we price cancer drugs will mean an increasingly large proportion of our health budget will be directed to medicines in general, and cancer medicines in particular. This has the potential to create enormous opportunity costs and inequities elsewhere in the system.

In this regard, there are lessons to be learned from other jurisdictions. In its submission, the Society of Hospital Pharmacists of Australia poignantly notes that the United Kingdom’s Cancer Drugs Fund, which was set up to provide access to cancer drugs not approved by the National Institute for Health and Care Excellence, has inadvertently resulted in the UK paying more for cancer drugs than most other European countries, and ultimately resulted in 25 of the 84 previously listed cancer medicines not being funded in 2015–16.

The pressure on governments is likely to get worse. According to the Pharmaceutical Research and Manufacturers of America, there are almost 800 drugs in development for cancer, of which 98 are for lung cancer, 87 for leukaemia, 78 for lymphoma, 73 for breast cancer, 56 for skin cancer and 48 for ovarian cancer.5 A recent report by the IMS Institute predicts that 225 new medicines will enter the market over the next 5 years, and that cancer treatments represent the highest proportion of these drugs.6 Of the cancer medicines being developed, 91% will be targeted therapies, which is likely to make these medicines more expensive. Pressure on budgets will therefore only increase if something is not done now about cancer drug prices.

Perhaps one reason the Senate report focused so much on HTA, and not on drug prices, is that price and profit expectations for pharmaceutical markets are set internationally, and Australia is a small player in this market. Part of the pharmaceutical industry’s global strategy includes setting high pricing precedents, typically in the US market. Although companies do negotiate different prices elsewhere in the world, there is a limit to their willingness to do so.

It is interesting, however, to observe that the US — traditionally the bastion of medicine price deregulation — now recognises that high drug prices are the biggest barrier to patient access, and questions are beginning to emerge about the legitimacy of the prices being charged. A new Bill has recently been submitted to the US Congress seeking to empower the nation’s Medicare system (which provides public health care primarily to people aged 65 years and older) to drive down prices, and to demand reports about expenditure and profits for each drug listed with the US Food and Drug Administration, including overseas sales.7 No doubt, recent scandals relating to unjustifiable price hikes — most notably the more than 5000% increase for 60-year-old drug pyrimethamine (Daraprim), used to treat infections such as malaria8 — has contributed to the recent spike in unease about medicine pricing.

A few submissions to the Senate report did make mention of the need for new approaches to purchasing medicines. Rare Cancers Australia, for example, recommended treating medicines as a service, wherein licences to use medicines, rather than the medicines themselves, are bought and sold. The advantage of this approach is that regardless of how much of a medicine is used, the licensing fee remains fixed, removing any incentive to overprescribe or aggressively promote use of a medicine. If such licences are not linked to specific indications, this model may also provide subsidised access to off-label drugs to treat patients with rare cancers.

Social impact bonds are another possible approach that was recommended by the Cancer Drugs Alliance. A social impact bond is a means to attract non-government investment into projects that resolve social problems that have traditionally relied on relatively small-scale support from trusts and foundations. The premise is that dealing with acute social problems early (eg, severe suffering from cancer) will lead to less expensive interventions and therefore savings for governments, of which a proportion is provided to investors as reward.9

Where to from here?

Such dramatic changes to how we procure medicines will need to be considered carefully and adopted gradually, and perhaps all Australia can do for now is wait for global drug pricing trends to adjust. Meanwhile, we need to be cautious about demands to radically overhaul HTA processes that might actually be working quite well. For example, when it comes to managed entry programs, it has to be recognised that current evidence standards have evolved for a reason, and it is extremely difficult to disinvest if a medicine subsequently proves to be ineffective, unsafe, poor value for money or simply unaffordable. It is therefore crucial for decision makers to separate the real value of cancer medicines from the hype that often surrounds them — using, for example, a tool developed by the European Society for Medical Oncology that ranks the “clinically meaningful benefit” that can be expected from new cancer treatments.10

One change that we can safely make now is to advocate for greater transparency surrounding both HTA and price negotiations. At present, decisions about access to cancer medicines are made behind closed doors, largely because of the perceived need to maintain commercial confidentiality. It is understandable that companies would not want to completely reveal their commercial interests, but without greater openness about how funding decisions are made, and how medicine prices are linked to underlying research and development, manufacturing and operational costs, we will remain unable to optimise the utilisation of our health resources in a way that works for both society and the pharmaceutical industry.

Resistance sans frontières: containing antimicrobial resistance nationally and globally

Coordinated action on several fronts is required

Antimicrobial resistance is everywhere, and everywhere invisible. Bacteria, which comprise the bulk of microscopic life, have lived on planet Earth for 3.4 billion years, giving them a huge amount of time to diversify, to establish themselves in almost all terrestrial and aquatic niches, and to develop advanced survival skills. Antimicrobial resistance is one of these skills. The agility with which bacteria acquire resistance to antimicrobial drugs is a perfect demonstration of those skills and of Darwin’s “survival of the fittest”.

Antimicrobials developed for therapeutic use are a very recent addition to the range of toxins in the bacterial environment. The introduction of sulfonamides in the 1930s was followed in the early 1940s by the development of penicillin, the first “miracle drug”, capable of killing bacteria causing infection in host tissues while causing no harm to the host.1 Resistance to both drug types emerged quite rapidly after their introduction into medical practice.2 Resistance has since developed, sooner or later, to all other classes of antimicrobials that have made their way into human and veterinary medicine, and into other fields of human activity.

Wherever antimicrobials are used, bacteria will be exposed and ultimately acquire resistance, by mutation or, more commonly, by acquiring resistance genes from other bacteria or the environment. The same is true for antiseptic agents, which are, in reality, antimicrobials that can only be safely administered topically. Although we are familiar with their use in humans, antimicrobials are currently used in a variety of other settings for the treatment and control of bacterial infections: in food-producing animals, companion animals, performance animals; in aquaculture, apiculture, and agriculture. We are extending the reach of antimicrobials by including antiseptics in home cleaning and personal hygiene products.

The alarm bells about resistance have been ringing for some time, but it has taken more than 20 years and several false starts before minds have responded collectively and focused on controlling resistance nationally and internationally. Antimicrobial resistance is now a major item on the agendas of the World Health Assembly3 and the World Organisation for Animal Health (OIE),4 and has been revived as a major work focus for both the World Health Organization and the OIE. Many developed and some developing countries have generated strategies and action plans in recent years; Australia did so in 2015, when it released Australia’s First National Antimicrobial Resistance Strategy.5 Although not the first attempt in this country to address the problem of resistance,6,7 it was the first to fully embrace the idea that resistance has no borders, ensuring that all aspects of antimicrobial use and resistance were considered. Both the Department of Health, and the Department of Agriculture and Water Resources drove the development of the Strategy.

What does the Strategy hope to achieve? It incorporates seven objectives, each with a strong motivation to cut through and achieve the changes needed to make a difference.

Increase awareness and understanding: There is ample evidence that most in our community have a poor understanding of what antibiotics can and cannot do, and what resistance is. At least half believe that antibiotics will help with the common cold, and many also believe that antimicrobial resistance means that they personally become resistant to antibiotics. NPS MedicineWise has been running advertising and other programs in response to this problem,8 but the impact has yet to be fully felt. Awareness and understanding are also sometimes lacking among prescribers. Although almost all doctors and veterinarians prescribe antimicrobials as part of their daily practice, few are aware of rational prescribing principles and their benefits. Doctors and vets need to improve their own awareness and understanding, as well as that of their clients, of the negative effects of using antimicrobials inappropriately.

Implement effective antimicrobial stewardship: Antimicrobial stewardship is the coordination of activities to ensure and promote rational prescribing in a defined context; for example, in hospitals, in the community, or in veterinary practice. Having a stewardship program is now part of hospital accreditation requirements, thanks to the efforts over many years of the Australian Commission on Safety and Quality in Health Care (ACSQHC).9 There is an obvious need to extend stewardship into residential aged care, general practice, small animal and equine practice, and food animal practice. The establishment last year of a Centre for Research Excellence, the National Centre for Antimicrobial Stewardship, will lay the groundwork for the development of stewardship programs in all these areas.

Develop national surveillance: Without surveillance data it is impossible to know which control strategies are effective, or how effective they are. Although Australia has for some decades had several antimicrobial use and resistance surveillance programs in human medicine, their work has lacked coordination and correlation. The ACSQHC has received funding for the development of a national coordinated use and resistance surveillance system for human health, due for completion by June 2016. This project will coordinate all existing programs, enhance them as needed, and fill important gaps, including through regular and timely reporting and trend analysis of antimicrobial dispensing data from the Pharmaceutical Benefits Scheme, and linking data on antimicrobial resistance from laboratory information systems around the country. On the veterinary and agriculture side, there have been a number of small pilot programs. Funding was recently found for a project in pig production, and there is interest in extending this initiative to the poultry sector. However, more needs to be done to establish a national surveillance program in the non-human sector that is integrated with surveillance in the human community.

Improve infection prevention and control: Control of antimicrobial use is essential, but by itself is insufficient to control the spread of antimicrobial resistance. Controlling the spread of bacterial diseases (eg, with vaccines) is a very effective way of reducing the need for antimicrobials. Infection prevention and control systems are essential components of resistance containment. Australia has national infection control guidelines for human health,10 and infection control systems are a mandatory requirement of hospital accreditation. In the non-human sectors, infection control is a key part of animal husbandry in intensive food animal industries. In veterinary practice, guidelines on infection prevention and control are available, including information on personal protection for vets and staff;11 however, more could be done to prevent the spread of infection between animals.

Agree on a national research agenda: Containing antimicrobial resistance is not currently an explicit research priority in Australia. The National Health and Medical Research Council, the Australian Research Council and other funding bodies support research in antimicrobial resistance, but only on a competitive funding basis (ie, in competition with all other types of research). While some excellent studies have been supported by these organisations, there is no strategic or targeted approach which ensures that the most important research questions are prioritised, such as new drug discovery and development, rapid diagnostics, and the identification of optimal education, community and professional strategies.

Strengthen international collaboration: Australia may be an island, but we are certainly not protected from exposure to new resistances. We have a long history of effective control of the introduction of exotic infectious diseases, but have not yet recognised that the same objective should apply to exotic resistances. The recent introduction in Victoria of an exotic resistance to last-line antibiotics (carbapenem resistance), with subsequent spread in the human population, highlights the fact that this aspect of resistance crossing borders cannot be neglected.12 Developing partnerships with countries across the world will assist Australia to learn from international best practice, avoid duplication of effort, contribute to public health outcomes in our region, and provide early warning of emerging threats.

Establish and support clear governance: None of these objectives can work without a clear, forward-looking and stable governance structure. As a federation, our national strategy requires the cooperation and coordination of the activities of nine governments and, more importantly, of numerous ministries and agencies. This is where the national Antimicrobial Resistance Prevention and Containment (AMRPC) Steering Group, reporting to the federal ministers for Health and Agriculture, supported by the Australian Strategic and Technical Advisory Group, and working in collaboration with the Australian Health Protection Principal Committee, is forging the way forward. A coordinated approach is essential. The efforts of these groups will align with international efforts and contribute to the global control of antimicrobial resistance.

All prescribers and users of antimicrobials have a responsibility to preserve their long term effectiveness and to protect the health of their nation’s citizens, animals and ecosystems. With the ever increasing global movements of people, animals and goods, all nations must work together to protect each other. Resistant bugs don’t respect borders.

Hospital handout ‘dismally short’ of need

The injection of an extra $2.9 billion of Commonwealth funding for public hospitals will provide some relief for cash-strapped health systems, but still leaves institutions desperately short of the resources they need to meet growing demand for their services, state premiers have warned.

Echoing AMA concerns that the extra money fails to bridge the huge $57 billion hospital funding shortfall created by the 2014 Budget, the leaders of New South Wales, Victoria, Queensland and South Australia said the additional funds would be helpful, but details of how hospitals were to be adequately funded in the longer term remained unresolved.

AMA President Professor Brian Owler said the states would be grateful for any new funding, but the outcome of the COAG meeting was disappointing.

“Today’s agreement goes nowhere near meeting the long-term needs of the nation’s public hospitals, and falls dismally short of replacing the funding taken away from the states in the 2014 Federal Budget,” Professor Owler said.

He said the extra funds would relieve some of the pressure on hospitals in the short-term, but did not provide the funding certainty that was vital for the decade ahead.

Prime Minister Malcolm Turnbull, who hopes to neutralise public hospital funding as an issue in the forthcoming Federal election, used the 1 April Council of Australian Governments meeting to thrash out a deal under which the states will receive an additional $2.9 billion between July 2017 and June 2020, capped at an annual growth rate of 6.5 per cent, in exchange for greater efforts to reduce hospitalisation rates through improved chronic disease care and fewer hospital-acquired infections and other complications.

The deal means that activity based funding and national efficient price mechanism, two reforms that were driving hospital efficiency and which were facing the axe next year, will be sustained until at least 2020.

In announcing the deal, Mr Turnbull acknowledged that the Commonwealth shared responsibility with the states and territories to provide universal health care, but warned that it had less revenue to pay for it.

“We are recognising that we have a serious structural budget problem,” the Prime Minister said. “We have to be clear eyed about our choices”.

Mr Turnbull had hoped the COAG meeting would back his radical plan to give the states the power to raise their own income taxes as a way to increase hospital funding, giving his Government scope to go into the election promising Federal income tax cuts.

The Prime Minister said the change would give states access to a broad revenue base that grew in line with the economy, would reduce the number of tied Commonwealth grants they receive, giving them greater autonomy and flexibility in tailoring their spending.

But the proposal failed to win consensus support among the premiers, and has been held over for further discussion and development.

Instead, while welcoming the short-term funding injection, state leaders warned the Commonwealth needed to provide much more significant funding if a looming crisis in public hospitals was to be averted.

Victorian Premier Daniel Andrews said that while the agreement signed at the COAG meeting would provide his state’s hospitals with “hundreds of millions in extra funding…it doesn’t replace the billions taken away”.

“I would ask to remain focused on the context here. The fact is that many billions of dollars which will not be flowing to our hospitals because of decisions made in 2014,” Mr Andrews said.

New South Wales Premier Mike Baird struck a more positive note, expressing the hope that there would be a “coming together” between the states and the Commonwealth on long-term hospital funding.

Queensland Premier Annastacia Palaszczuk said Mr Turnbull had recognised that health was the “most fundamental issue” facing the nation, and welcomed the short-term funding deal, which would inject an extra $445 million into Queensland’s public hospitals.

“But there is still a huge gap [in funding] that is going to place a huge strain on our hospitals,” Ms Palaszczuk said.

Adrian Rollins

PM’s tax plan no hospital fix

The Prime Minister’s suggestion that states be given the power to levy their own income taxes will not fix the funding crisis hitting the nation’s public hospitals, according to AMA President Professor Brian Owler.

Professor Owler said public hospitals needed a significant ongoing increase in funding if they were to meet growing demand for their services, and it was “very unclear” how Malcolm Turnbull’s proposal to allow the states to impose income taxes would provide that.

Mr Turnbull has floated a radical change in the tax system under which the Commonwealth would cut its income tax take and allow the states to set their own income taxes while keeping the overall tax burden the same.

The idea, which has been rejected by the Opposition and received only lukewarm responses from State leaders, is due to be discussed at a meeting of Federal, State and Territory leaders and treasurers tonight before formal discussions at tomorrow’s Council of Australian Governments meeting.

The Prime Minister is under pressure to unwind $57 billion of cuts to public hospital funding unveiled by his predecessor Tony Abbott in 2014, and is expected to propose a short-term injection of up to $7 billion to help ensure the issue does not figure prominently in the forthcoming federal election.

But Professor Owler said that although extra money would be welcome, an extra $7 billion would not fix the long-term problem of inadequate Commonwealth funding.

And he said simply reallocating income tax responsibilities between levels of government was not an answer.

“Unless there’s going to be extra funding that’s put into health by the Commonwealth, I fail to see how this new policy is going to fix the problem of funding public hospitals,” the AMA President told ABC News 24.

Professor Owler said the policy was likely to exacerbate existing differences in access to care between the states, because New South Wales, Victoria and Queensland had a much bigger potential revenue base to draw on than smaller states like Tasmania and South Australia.

“While the larger states and territories might be okay, we know that the smaller states already struggle,” he said. “If you’re relying on income tax, we know that their economies and revenue would be much less, in a proportional sense, than the revenue raised from some of the bigger states. So I think we just create more inequality across the country.”

Instead, he has called for COAG use any breathing space created by a short-term injection of funds to engage in “a much broader, in-depth discussion about how we are going to fix the problem around Federal reform, about long-term funding of public hospitals.”

And he dismissed suggestions by Mr Turnbull that there needed to be a greater focus on improving hospital efficiency.

Professor Owler said that doctors and nurses had been working to improve hospital efficiency for decades, and had achieved huge improvements, such as in reducing patient length of stay.

But instead of supporting this process, the Abbott Government had axed the activity-based funding mechanism which was specifically designed to drive greater efficiencies, and the AMA President said the Turnbull Government should “get back” to some of those structural mechanisms.

Adrian Rollins