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

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