Ethics in medicine: is it a futile exercise?

In this issue of the MJA, we are launching a new occasional series of articles discussing current controversies and challenges in medical ethics. This first trio of articles addresses the provision of apparently futile treatment at the end of life. Koczwara, a medical oncologist, describes a difficult, but unfortunately all-too-common, case involving conflict between health professionals and family members about the appropriateness of life-prolonging treatment. Complementary clinical, ethical and legal perspectives follow.

There are two main groups of ethical challenges facing Australian health professionals in the 21st century. Some of them, like that of “futile” medical treatment, are questions that have been asked for a long time but remain unresolved. These contentious questions often involve conflicts between different ethical principles; for example, between the need to respect the patient’s autonomy and the need to allocate limited medical resources to those most able to benefit from them. It can sometimes appear that ethical debate on these questions is futile, since the arguments are intractable, values incommensurable, and solutions elusive. However, even if there is no single right answer to many ethical questions, there are often some answers that are clearly wrong, and can be rejected. Recent Australian work suggests the possibility of progress in the specific area of medical futility.1 Advance care planning significantly increases the chance that patients’ own wishes are known and respected at the end of life.2 This is likely to reduce the provision of unwanted and inappropriate treatment and may reduce disputes. Legal attention has shifted from attempts at defining futility to delineating a clear and fair process for resolving disputes when they occur.3,4

Other ethical challenges are new; for example, those arising from novel medical technologies and procedures. In forthcoming issues, the Journal will look at questions related to direct-to-consumer genetic testing, and umbilical cord blood storage and use. Both of these exemplify the challenge of regulating medical technologies and diagnostic tests that are available in both the public and private sectors. The problem for regulators is partly the speed of technological development; however, it is also the real difficulty of restricting access to direct-to-consumer testing in an age in which such testing can be simply arranged online or through the post and performed offshore.

One issue that has been a major source of debate in the medical ethics literature in the past decade and is likely to face practitioners in the near future is medical enhancement — drugs and technologies that are used to improve normal function rather than to treat illness or infirmity.5,6 In recent surveys, a small proportion of Australian adults have admitted to use of prescription medicines in the absence of a medical diagnosis to improve concentration or alertness.7 Should such enhancements be proscribed? Should students be tested for evidence of enhancers before sitting exams? As with performance enhancement in sport, could such an approach be futile, and should we accept that enhancement will occur despite any attempts to prevent it? If so, it may be better to focus attention on harm minimisation and education.

Finally, value pluralism creates an extra challenge for medical ethics in Australia, with our diversity of Indigenous and immigrant cultures.8,9 Without a shared set of values it may seem fruitless to even embark on ethical debate. It may be possible, though, to draw on higher-order principles that transcend religious and philosophical differences. First, we could seek universalisable solutions to ethical problems — could we apply to ourselves what we are proposing to do to others? This involves applying a version of the Christian “golden rule” or the Kantian categorical imperative shared by most philosophies.10 Second, we should be consistent in our ethical judgements and decision making. If our approach differs between cases, are there ethically relevant factors to justify that difference? (The philosopher Hare argued that the very language of ethics demands such consistency.11) This is a particular challenge in countries like Australia, where the legal framework for end-of-life decisions can differ significantly between states. Third, where there is a range of viewpoints that could reasonably dictate our actions, we should be guided by the wishes and values of the individual most affected by a decision, and avoid imposing on them the values of others, unless this would harm others.12 This approach recognises the diversity of values and beliefs held in our society. We respect this diversity by accepting that we will not all agree about contentious questions.

If the principles seem straightforward, their application is not — particularly when it comes to developing policy or legal solutions. As the articles in this issue illustrate, we have some way to go before our approach to conflicts about end-of-life treatment is universalisable, consistent and respectful of different values.

Futile treatment: the ethicist’s perspective

We need to pay attention to the reasons why treatment is judged to be futile

When doctors are confronted with making treatment decisions in the context of a terminally ill patient, the way forward is often difficult and confused. Bringing an ethics perspective to bear on the problem can help to resolve what the essential issues are, in turn enabling a clearer path to appropriate decisions by the people who ought to be making those decisions. In many cases where further treatment of a patient may be thought to be futile, such as John’s case, described by Koczwara,1 the central ethical question is whether treatment is medically inappropriate or futile, and whether, as a consequence, it may be withdrawn or withheld against the wishes of his family.

Is treatment inappropriate or futile?

There are many different terms that are used to describe treatment that medical professionals feel should not be provided, even if the patient or family request it.2 Sometimes, as in John’s case, it is called “futile”;1 at other times it is referred to as “inappropriate”, “non-beneficial”3 or “not indicated”.4 These terms suggest that there is a simple yes or no answer — treatment is either futile or it is not — and that this answer can be readily determined. However, in practice there are often disagreements between different medical professionals and between clinicians and families about whether treatment is futile. These disagreements partly stem from differences in assessment of the facts, including the nature of a patient’s illness and the prognosis with treatment. But they are also crucially based on different assessments of the goals of treatment, and on what would best reflect the interests of the patient. A doctor might regard treatment as futile if it is unlikely to lead to the patient being discharged alive from hospital, while a patient may see the same treatment as not futile if it gives him a chance of living long enough to see a new grandchild. One important step, wherever there are disputes, is to identify the goals of treatment for each party.5,6

May treatment be withheld or withdrawn?

What happens in the case where the patient or their surrogate decisionmaker may well have identified a different goal from clinicians? There are ultimately only two ethical reasons for refusing to provide treatment that a patient or his or her surrogate is requesting (Box).2 The first reason for refusal is that providing treatment would harm the patient. Either the chance or duration of survival is so small, or the quality of life is so poor (or both) that the physician judges that the harms of treatment outweigh the benefits. The second reason is that providing treatment would harm other patients. Where there are limited health care resources, providing treatment would mean that other patients who stand a greater chance of benefit are prevented from accessing it.7 Sometimes, perhaps often, both of these reasons will apply.

Would treatment be harmful to the patient?

In John’s case, the clinicians involved may well have believed that treatment was harmful for him, but his son disagreed. The critical question then is what the patient’s values are or were. Would he have judged treatment to be worth continuing, or thought that the harms outweigh the benefits? If the evidence says yes, then we have a strong prima facie reason to provide it. This reflects the importance of patient autonomy in Western medical ethics. But it also reflects the genuine philosophical uncertainty about how we should assess the benefits and burdens of treatment and determine whether treatment is harmful.8,9 Given this uncertainty, it is not clear why we should privilege a medical view over the patient’s view. To do so appears unjustifiably paternalistic.

However, in John’s case, there does appear to be evidence from his recent behaviour and interactions that he would not have wanted further treatment. Other members of the family appeared to support this. (We should nevertheless be careful to distinguish between what families want and what they think the patient would have wanted, since these may not coincide). If this was the case, then it is questionable whether the son was genuinely representing John’s wishes. There are a range of reasons why surrogates might request treatment that is contrary to the patient’s own wishes and interests, including guilt, denial, personal fear of losing a loved family member, religious reasons or, even occasionally, financial advantage.10,11 In such situations, an alternative surrogate decisionmaker should be sought.

Would treatment be harmful to others?

Resource allocation is not often explicitly acknowledged as a justification for forgoing treatment.12 Yet it is frequently present below the surface, and arguably provides the strongest justification for not providing treatment. This is based on the widely held principle, articulated by philosopher John Stuart Mill, that individual freedom may only be limited to prevent harm to others.13,14

If it did transpire that a patient such as John would have wanted cardiopulmonary resuscitation (CPR) in the event of a cardiac arrest, should it be provided? The chance of “success” is low in a patient with malignancy and multiorgan failure.15 Some ethicists have argued that in some circumstances it would be appropriate to provide futile CPR.16 However, the strongest reason for declining to provide CPR in John’s particular case is not that it would not be successful — on the contrary, it is because it may be successful and lead to John being intubated and ventilated and transferred to the intensive care unit (ICU). John’s son apparently did not want him to be admitted to the ICU, so there may not be a strong resource-based reason for declining to provide CPR. But what if the son had insisted on John being admitted to the ICU? In public health systems with limited intensive care beds, there are often patients who are declined admission to intensive care who would potentially benefit.17 Refusing to admit John to intensive care would not be paternalistic (ie, assuming values that he does not share). Rather, it would reflect the common values of wider society and the imperative to use scarce public resources fairly.

The important practical question is how resource-based decisions to decline treatment should be made. Such decisions need to be transparent and accountable,18 and should ideally be based on consensus guidelines or hospital policy.19 In their absence, hospital ethics committees may provide a mechanism for fairly considering differing points of view, and arbitrating whether it is reasonable to withhold treatment.

Conclusions

Ultimately, many cases are resolved by consultation and discussion. However, it is ethical for clinicians to refuse to provide treatment either where this would be contrary to the best interests of the patient, or where resources are insufficient. Giving attention to the reason for judging treatment to be futile may help in identifying the best way to resolve a dispute.

Resolving disputes about treatment judged to be medically inappropriate or futile*

* Adapted from Curr Opin Anaesthesiol 2011; 24: 160-165.2 Used with permission.

Key issues

Can we improve our ability to determine what the patient would have wanted?
How do we deal with differences between families and medical staff about the appropriate goals of treatment?
Can we develop fair rules or guidelines for determining which costly treatments (of low efficacy or chance of success) should not be provided?
What is the best process for fairly resolving disputes about medically inappropriate treatment?

Legal perspectives on consent in disputes about futile care

Futility disputes highlight some important legal principles concerning the right to refuse treatment, advance directives and the role of substitute decisionmakers

The need for legal resolution of futility disputes is a rare occurrence in Australia and the jurisprudence is still emerging.1 The legal approach to futility disputes differs in each state jurisdiction, and a full analysis of the Australian law is beyond the scope of this article. Most recent discussions have focused on the law in Queensland and New South Wales,2,3 so for this hypothetical I will outline the relevant South Australian law, given the SA connections of the other authors of this set of articles.4,5 However, this discussion has clear relevance to other jurisdictions in Australia.

Five established legal principles in
South Australia

Adult patients are presumed to be mentally competent

Australian law presumes that all adults are mentally competent. But this presumption can be rebutted. In Re MB (Medical Treatment) [1997] 2 FLR 426 it was said that a person lacks capacity if he or she is unable to comprehend and retain treatment information or is unable to use information, weigh it and make a choice. In the case described by Koczwara,4 John’s decisions to refuse tube feeding and chemotherapy were made competently. His capacity had not been disproved.

Patients can refuse life-sustaining treatment

In H Ltd v J [2010] SASC 176, J was a 73-year-old woman who was suffering from post-polio syndrome and diabetes. She was partially paralysed and confined to a wheelchair. She resided in a nursing home owned by H Ltd. J decided to end her life by refusing to eat and drink. She also refused insulin. J made an advance directive in accordance with the SA legislation and refused artificial nutrition and hydration. The nursing home approached the court for directions as to the lawfulness of J’s decision. Kourakis J found that there was no common law duty imposed on J to eat and drink. Consequently, it was not suicide for her to die from self-starvation, and there was no duty on the part of the nursing home to force-feed her or give her insulin.

Patients can make advance directives

Patients can make advance decisions to refuse treatment. These decisions are commonly referred to as “advance directives”, but in SA they are officially known as “anticipatory refusals” or “anticipatory directions”. In SA, such decisions are regulated by the Consent to Medical Treatment and Palliative Care Act 1995. A direction becomes operative when the patient is incompetent and in the terminal phase of a terminal illness. The SA Parliament is currently considering a new Advance Directives Act that will recognise decisions to refuse treatment recorded in a standard form, without the requirement that the patient be in the terminal phase of a terminal illness.

John did not make an anticipatory direction. The best time to have completed this would have been after he decided to refuse tube feeding and agreed to stop chemotherapy. A discussion was already taking place about his care, and this would have been an ideal time to record his wishes concerning life-sustaining treatments. A direction may have prevented the later conflict over his care.

Patients can appoint substitute decisionmakers

In SA, a substitute decisionmaker can be arranged in two ways. “Medical agents” can be appointed with powers to make decisions about medical treatment that appear to include power to refuse treatment (Consent to Medical Treatment and Palliative Care Act, s. 8). Medical agents must act in accordance with any lawful conditions and directions contained in the medical power of attorney document and they must generally act in the best interests of the patient. They cannot refuse natural administration of food and water, palliative care or treatment which would return the grantor to capacity (s. 8). Alternatively, John could have appointed an “enduring guardian”. Enduring guardians have all the powers of a guardian in law and equity and can consent to or refuse medical treatment for the person.

The new Advance Directives Act, if passed, will replace the medical agent and enduring guardian processes with an advance directive that allows for the appointment of a substitute decisionmaker. The substitute decisionmaker must follow directions as written by the patient, and, in the absence of a direction, must endeavour to make a decision that the patient would have made in the circumstances had he or she been competent.

From the facts in John’s case, it would appear that neither form of decisionmaker was appointed. It is important to enquire whether a patient has any agents or guardians, preferably on admission. A conversation about whether John would have liked to appoint a medical agent or enduring guardian could have occurred at the time he refused enteral feeding. This again may have reduced the chance of conflict occurring later.

Intervention by tribunals may be necessary in cases of dispute

In the absence of a direction, a medical agent or an enduring guardian, we are left with the professional opinions of the health care team and the role of relatives under the Guardianship and Administration Act 1993 (SA). Under s. 59 of that Act, relatives can give consent to treatment, but there is no express power to refuse treatment. Nevertheless, s. 17(2) of the Consent to Medical Treatment and Palliative Care Act states:

A medical practitioner responsible for the treatment or care of a patient in the terminal phase of a terminal illness, or a person participating in the treatment or care of the patient under the medical practitioner’s supervision, is, in the absence of an express direction by the patient or the patient’s representative to the contrary, under no duty to use, or to continue to use, life sustaining measures in treating the patient if the effect of doing so would be merely to prolong life in a moribund state without any real prospect of recovery or in a persistent vegetative state. [my emphasis]

The section has clearly been written to enable health care professionals to omit treatment that is futile. The differing professional opinions about John’s prognosis made it initially difficult to apply this section to John’s situation. Later, when opinions coalesced and united, the section clearly applied.

Section 17(2) also indicates that, to have the protection of the section, one needs the consent of the patient or the patient’s representative. “Patient representative” is defined to include “relatives”. Under the Guardianship and Administration Act, both John’s wife and son would qualify as a “relative”, but nothing is said about what to do when disputes between relatives occur.

If the new Act is passed it will replace the current arrangements with a “person responsible”, who will ordinarily be a relative, carer or friend of the patient. The person responsible will be given the power to refuse treatments in situations where he or she reasonably believes that the patient would have refused treatment had he or she been competent.

Given the difference of opinion between John’s wife and son, resort to the Guardianship Board was appropriate. The Public Advocate, as John’s guardian, had the power to refuse treatment, which resolved the legal uncertainty. Perhaps more importantly, the Public Advocate’s appointment seems to have acted like a circuit breaker and enabled all the parties to reach a consensus about the best way forward for John. That decision clearly fell under s. 17 of the Consent to Medical Treatment and Palliative Care Act. That section also provides protection to health care professionals when it states that treatment discontinuation decisions, made in accordance with s. 17(2), do not constitute a legal cause of John’s death.

Conclusion

Perhaps what is surprising is that futility concepts may play a subsidiary role to issues of consent. Some have argued that consent to withhold or withdraw futile treatment should not be necessary, as futile treatment cannot be demanded and such treatment is never in a patient’s best interests.6 These are undoubtedly true positions. However, they belie the fact that futility assessment is difficult and requires consideration of medical and social factors in the assessment of the patient’s best interests. A procedural approach to futility assessment, involving the treatment team and substitute decisionmakers, is the best way forward for an assessment, and this necessitates a good-faith attempt at reaching consensus.3 Consensus may be difficult to achieve, in which case resort to guardianship bodies or the Supreme Court may prove necessary.

Advance care planning provides a way to avoid conflicts in end-of-life care. The creation of a direction or the appointment of an agent or enduring guardian may reduce the likelihood of conflict and provide greater certainty to the health care team.

In search of a good ending

Despite much development and increasing complexity in health care, humankind’s mortality rate remains at 100%. Given the ubiquity of this experience, it could be argued that an important, universal health outcome measure is a “good death”. In the late 1990s, a qualitative study identified five things that people want when they approach the end of life: avoiding suffering; avoiding the prolongation of dying; achieving a sense of control; and relieving burdens on, and strengthening relationships with, loved ones (JAMA 1999; 281: 163-168). More recently, Australian researchers involved with the Respecting Patient Choices Program in Victoria conducted a randomised controlled trial that objectively demonstrated that advance care planning — “whereby a patient, in consultation with health care providers, family members and important others, makes decisions about his or her future health care … should the patient become incapable of participating in treatment decisions” (CMAJ 1996; 155: 1689-1692) — improved end-of-life care and patient and family satisfaction and reduced stress, anxiety and depression in surviving relatives (BMJ 2010; 340: c1345). In this issue of the MJA, several contributions examine end-of-life care from different perspectives.

Since 1995, adults in Tasmania have been able to appoint and legally register a substitute decisionmaker (known as an enduring guardian) to make health care decisions when they lack capacity to do so themselves. Ashby and colleagues (doi: 10.5694/mja12.10498) report that uptake of this option from 1995 to 2010 in the eligible population was only 2.7%: a missed opportunity, considering the authors’ belief that the process of advance care planning is just as beneficial as the legal documents arising from it. Another Tasmanian project, the Healthy Dying Initiative, seeks to improve care and decision making in and out of hospitals at the end of life. It includes a “goals-of-care” framework for clinicians to state whether goals are curative, palliative or terminal, and advance care directives.

We are getting better at planning care at the end of life for cancer patients, but what about the many Australians who will die from chronic disease? Burgess and colleagues (doi: 10.5694/mja12.11058) say this scenario holds particular challenges. As patients with multiple morbidities slowly deteriorate, they require active disease management, but they also need help to control distressing symptoms, and emotional, spiritual and psychological support for themselves and their families. The authors call for a system that allows and supports both these needs simultaneously.

Some doctors believe we should go a step further in assisting patients at the end of life. Willoughby and colleagues (doi: 10.5694/mja12.11610) put forward the position of Doctors for Voluntary Euthanasia Choice (www.drs4vechoice.org). Under current health care provisions and professional ethics guidelines, they are concerned that some patients still have unrelievable forms of pain and others may be forced to endure a wretched but ongoing existence. They call for voluntary euthanasia to be legalised, following the precedent set in several other countries.

As Wilkinson points out in the introduction to our new occasional Ethics series (doi: 10.5694/mja13.10115), we have some way to go before our conflicts about the big issues in medicine — such as how far we should go to assist in a good death — are resolved. He offers three principles that might help guide the way: universalisability (could we apply to ourselves what we are proposing to do to others?), consistency, and a willingness to be guided by the wishes and values of the individual most affected by a decision.

When it comes to the third principle, some of the advance care directives analysed by Ashby and colleagues indicate that, for many, the wishes for end-of-life care are simple, practical and clearly articulated: “I do not want to be force fed” or “When I am unable to walk outside by myself, I would like to be taken out to the sun for a few hours every day whenever possible”.

These wishes, at least, we should be able to grant.

Advance care planning: lessons from a study of Tasmanian enduring guardianship forms

Uptake of available instruments is low, and death is the “elephant in the room”

There is an ongoing global conversation about death and the process of dying. Despite half a century of clinical, academic and public policy activity by specialist palliative care workers — and by many others, including health administrators, academics, artists and writers — it is common to hear the same issues recycled with the oft-repeated comment that we “do not do this well”. Clinicians struggle with treatment abatement decisions and issues relating to causes of and responsibility for death. Pathways to death (“death ways”) are changing, with increasing numbers of people dying in old age, slowly over 1–2 years, with multiple comorbidities (which often include dementia) and greater numbers of significant medical decision points.

The public often has expectations of curative capacity that exceed reality (fed by a technically optimistic health industry) on the one hand, and exhibits widespread concern about bad dying on the other. Although improvement of clinical care is essential, it needs to be supported by public health promotion activities aimed at improving preparation for the end of life.

Lack of capacity has been identified as a major barrier to timely and appropriate limitation of life-prolonging medical treatment, and deployment of palliative and terminal care. Despite considerable global enthusiasm for the potential role of advance care planning to solve the challenge of decision making at the end of life, uptake is low and utility in clinical situations is contested.1 However, there has been recent common law endorsement of the status of advance care directives in Australia,2 and an Australian randomised controlled trial has shown that advance care planning improves adherence to patient wishes and family satisfaction.3

Enduring guardianship is a specific legal instrument that can be part of the broader context of advance care planning,1 a process that enables individuals to consider and make decisions about their future health care in consultation with their doctor and other health care professionals, family members and other important people in their lives. Consultations with lawyers for will drafting and with superannuation or financial planners may also bring up these issues.

Under the provisions of the Guardianship and Administration Act 1995 in Tasmania, people aged 18 years or older can appoint a substitute decisionmaker known as an enduring guardian, and the document of appointment is registrable with the Guardianship and Administration Board. Tasmania is the only Australian jurisdiction, and one of few in the world, to operate a state-funded registry and to require registration of completed enduring guardianship forms with a guardianship board.

From 1995 to 2010, 10 040 enduring guardianship forms were lodged with the Guardianship and Administration Board. This represents uptake by about 2.7% of the state’s eligible adult population, despite information being widely disseminated across the state.4 It sets a benchmark for comparing uptake of substitute decisionmaker instruments in Australia, and possibly overseas.

To assess uptake and use of the Tasmanian legislative provisions, and to obtain data to inform future developments in advance care planning in Tasmania and elsewhere, a study of enduring guardianship forms was commissioned by the Office of the Public Guardian for Tasmania.5,6

A one-in-20 sequential sample of forms was analysed (n = 502) in this study.5,6 Forms tended to be completed by retired people aged over 60 years (63% by women and 37% by men, for forms on which sex was specified). Despite legal assistance to complete an enduring guardianship form not being mandated, 39% of the forms showed evidence of legal input. Textual analysis showed substantial evidence that people tend to copy examples used in advisory material provided.

Nearly half the forms (47%) contained a statement about end-of-life care and indicated a preference for the deployment of timely and appropriate palliative care. These directions were usually made in general terms, and copied verbatim from examples. Quality-of-life considerations appear to be underpinning these statements, as seen from these extracts from the forms that were analysed:5

When I am unable to walk outside by myself I would like to be taken out to the sun for a few hours every day whenever possible.

As a lover of fine music I would like to be able to listen to classical music in the evenings.

I wish to hear Christian music even if I seem to show no response. Care should be taken to ensure that others are not annoyed.

I do not want to be force fed.

If I am bed ridden and only able to verbally communicate by sounds, not speech, and if the sounds which I make annoy others around me I do not wish to be given medication to silence me unless the noise is extreme and all other measures have been tried.

Despite the fact that enduring guardianship powers lapse at the time of death, 14% of the forms analysed provided direction to appointed guardians on wishes regarding organ donation.5 So it does not seem to be understood that the powers of substitute decisionmakers lapse at the time of death.

These data show what a generic legal instrument such as a guardianship tool can achieve in terms of advance care planning without a dedicated public health campaign focused on medical care or death and dying.

While the enduring guardianship instrument is clearly a statutory provision, advance care directives do not have statutory force in Tasmania, but have been supported in common law in Australia and other common law jurisdictions. It seems that the strongest position in medicine, ethics and law is to appoint a substitute decisionmaker under the relevant jurisdictional provisions, and preferably provide that person with oral or written instructions about values and wishes. Regardless of the legal status of advance care directives, doctors surely have an ethical obligation to respect what they know of patient wishes.

In addition, it is widely recognised that it is the process of advance care planning that matters: that people reflect on their values, beliefs and wishes about end-of-life health outcomes, and then advise those around them and their health care attendants. This process is seen as having pre-eminence over the completion of formal legal forms.7

All adults should be encouraged to appoint an enduring guardian (or equivalent) and write an advance care directive, using a format that addresses end-of-life issues, preferences, beliefs, values and unacceptable treatment outcomes. Directions on enduring guardianship forms, including those that specify aspects of personal care, should be actively sought and incorporated into a care plan if the person is admitted to a hospital or an aged care facility.

While capacity is important, broader action is required. For example, a Healthy Dying Initiative has been developed in Tasmania to improve care and decision making at the end of life.8 This consists of three components: a goals-of-care framework, an advance care directive for the end of life, and a number of health-promoting interventions aimed at raising awareness about death and dying. A goals-of-care form has replaced the not-for-resuscitation form at the Royal Hobart Hospital. It is a medical order form that requires clinicians to state whether the goals are curative, palliative or terminal and then to clarify any appropriately negotiated limitations of treatment, and to revise the form as needed, according to changing clinical picture and/or patient wishes. This form can also be endorsed for continued out-of-hospital use.

The public have significant concerns about death and dying, and advance care planning needs to meet the challenge of limitation of medical treatment and the dying process head-on. The concept of medical futility only has traction when allied with public engagement about how we approach death. We have no choice about whether or not we ultimately die, but, as increasing age and chronic disease burden take their toll, we can have some influence on the way that we die, and this choice is real.9

Glossary

Advance care planning: “a process of planning for future health and personal care whereby the person’s values, beliefs and preferences are made known so they can guide decision-making at a future time when that person cannot make or communicate his or her decisions. Formal advance care planning programs usually operate within a health, institutional or aged care setting after a life-limiting condition has been diagnosed, frequently requiring the assistance of trained professionals. However, people can choose to discuss their advance care plans in an informal family setting.”1

Advance care directive: “a type of written advance care plan recognised by common law or authorised by legislation that is completed and signed by a competent adult. An ACD can record the person’s preferences for future care and appoint an SDM to make decisions about health care and personal life management.”1

Substitute decisionmaker: “[a person] appointed or identified by law to make substitute decisions on behalf of a person whose decision-making capacity is impaired … An SDM may be appointed by the person, appointed for (on behalf of) the person, or identified as the default decision-maker by Guardianship Acts around Australia. More than one SDM can be appointed under an ACD.”1

Enduring guardian: a person appointed as an SDM under the Guardianship and Administration Act 1995 (Tas).

SDM = substitute decisionmaker. ACD = advance care directive.

Clinical effectiveness research: a critical need for health sector research governance capacity

The barriers to conduct of clinical research will require solutions if we are to implement evidence-based health care reform

Reforms in the funding of health services, such
as “activity-based” funding initiatives, seek to facilitate changes in how health care is delivered, leading to greater efficiency while maintaining effectiveness. However, often these changes in treatment strategies and service provision evolve without evidence demonstrating effectiveness in terms of patient outcomes. The pressures on health care expenditure (currently around 9% of gross domestic product1) make such an approach untenable and unsustainable. The evidence necessary to support these initiatives can only be derived through carefully conducted clinical research. Most readers would immediately think of clinical trials in terms of pharmaceuticals or clinical devices, and this type of research is critically important, although continuing to decline, in Australia.2 Other questions relate to the effectiveness of changes in health practice or policy, usually (but not always) based on sensible ideas that seem self-evident. However, in order to function with an evidence base, these ideas need to be proven to be clinically effective and cost-effective. Such research can be costly, and many of the questions to be addressed are not ones that would be the subject of an industry-sponsored trial. Researchers, clinicians and health administrators are therefore faced with the problem of how best to measure the outcomes of changes to health care strategies, without the necessary resources to ask and answer the question.

The MJA Clinical Trials Research Summit held in Sydney on 18 May 2012 included a working group addressing issues of research governance and ethics. The key discussion outcomes of that group were:

confusion exists regarding the differences between ethics and governance;
variability continues in state and federal legislation and regulations, despite attempts at harmonisation;
processes for improvement at government and institutional levels are underway but are not yet complete or implemented;
hospital boards and chief executive officers need to have incentives to make the infrastructure work;
substantial challenges exist when working with international investigator-initiated trials;
trials involving the private health sector include specific difficulties such as insurance and contracts; and
national accreditation of researchers and training should be considered.

Costs are not the only barrier. Efforts to rationalise health care provision on the basis of evidence provided through the conduct of clinical research are also hampered by existing or perceived obstacles in the form of cumbersome institutional research governance and ethics approval processes. Substantial changes and streamlining of the processes of ethical review are underway across Australia, addressing inconsistencies and inefficiencies of human research ethics committee approval, financial processes, and contractual clinical research governance processes. Nevertheless, the system remains complex, slow and expensive. Unfortunately, the old adage of “good, quick or cheap: pick two” still applies.

Many researchers fail to distinguish between research governance and ethics. Clinical research in Australia is governed by the National Health and Medical Research Council (NHMRC) National statement on ethical conduct in human research3 and the Australian code for the responsible conduct of research.4 Research governance can “be understood as comprising distinct elements ranging
from the consideration of budgets and insurance, to the management and conduct of scientific and ethics review”.5 Research governance thus includes oversight of all processes of ethics review, but also includes responsibilities of both investigators and institutions for the quality and safety of their research.3

The Harmonisation of Multi-centre Ethical Review (HoMER) initiative by the NHMRC is a significant step forward, enabling a single ethics review process that has been adapted for several states. This process, if used effectively, should reduce the resources required to obtain ethics approval for multicentre research, but it has also created some challenges in ensuring that research governance obligations are maintained within various health service jurisdictions.6 Currently, no incentives or requirements exist for health services or hospital chief executive officers to ensure that appropriate infrastructure is in place and working. Similarly, a different set of challenges arises when considering performing research in the private sector, where insurance and contractual issues may differ substantially from those in the public sector.

Much of the non-industry-sponsored clinical research performed in Australia is investigator-initiated research, supported by funding organisations such as the NHMRC, state governments, and other non-government organisations such as Cancer Council Australia, the National Heart Foundation of Australia and cooperative clinical trial groups. At present, investigator-initiated trials require comparable levels of research governance and are certainly subject to the same requirements for good clinical practice as industry-sponsored trials. The research questions addressed by these studies are based on clinical imperatives, a broad understanding of the underlying science, and a necessary ability to work on a shoestring — the latter being the main point of distinction from industry-sponsored trials. Current models of competitive research grant funding do not recognise the complexities, duration, costs and distribution of costs across the length of a clinical trial, especially when considering late clinical outcomes that are often the most clinically relevant ones. As an example, an NHMRC project grant can be funded for at most 5 years and therefore necessitates a focus on end points occurring within end-point time frames. The clinical questions that we and the community recognise as important might not be able to be answered with such designs. The resources required to meet these requirements continue to escalate and we currently run the risk that these trials will soon be untenable in Australia. Anecdotally, many academic clinical research units are already questioning what level of involvement they should have in such relatively underresourced trials or if they should be involved at all, for the most part purely for financial reasons.

Within the current Australian health care environment, clinical research is being conducted in the face of significant headwinds. These inefficiencies arise from resource costs due to complex governance arrangements combined with those of research conduct (Box). Processes to be considered that will improve clinical research capacity might include:

continued adoption of electronic health records that span clinical, investigative (ie, pathology and radiology) and therapeutic information (eg, the Australian Orthopaedic Association National Joint Replacement Registry);
data-linkage techniques to obtain clinical outcomes
(eg, hospital readmission data, Medicare Benefits Schedule and Pharmaceutical Benefits Scheme use data, the National Death Index);
better integration of research into routine clinical practice;
national accreditation of investigators;
standardised good clinical practice training;
increased profile for research participation at the clinician–patient level, enabling the conduct of studies that are more representative of a wide spectrum of patients;
development of a clinically relevant strategic research agenda led by collaborations between clinicians, researchers and health policy decisionmakers;
a culture shift where lawyers and hospitals communicate and quantify the risk or research appropriately.

Research developed through partnerships between health policymakers and health service providers should lead to outcomes that are more immediately relevant and translatable to the care we provide, the outcomes we achieve and the costs incurred by the health system. Reinvestment of financial and efficiency gains realised from initial research outcomes back into the next relevant translational research question provides a model for a sustainable health system that evolves with the support of a robust clinical research-driven evidence base. These financial windfalls currently go back into government coffers and ideally should be seen as a potential funding stream to support future clinical research.

As the demands on our health system continue to mount, the need for clinical effectiveness research to build a robust evidence base upon which to reform care has become even more acute. It will be critical to align the clinical and policy research agenda while strengthening the governance structures that facilitate the conduct of research within the clinical space if we are to develop “an agile, responsive and self-improving health system for future generations”.7

Key points

Barriers to clinical research include:

regulatory complexity
inflexibility of ethical review and oversight
funding models that are not designed to support clinical trials
lack of incentive for engagement of health services in research support.

Solutions may include:

different funding models, including support for longer time frames
simplification of ethical and governance processes recognising the different goals of industry- versus investigator-initiated research
better involvement by health services in supporting research
return of savings from clinical research to support further research
clinical research key performance indicators for health service administrators.