Explainer: what is comparative effectiveness research?

The aim of CER is to assess the effectiveness and worth of medical interventions in real-life scenarios. Image from shutterstock.com

The evidence gleaned from medical research directly affects the decisions made about health care in Australia, driving everything from clinical practice guidelines, to which health interventions will and won’t be reimbursed by the public health system.

For the most part, this evidence comes from highly specific studies called randomised clinical trials. These trials are conducted by commercial or academic sponsors to evaluate how effective a new drug, medical device or treatment is within a highly-selected population.

The problem is, it can be difficult for clinicians to work out how best to use or interpret this new evidence and compare it to the alternatives already available. This gives rise to a wide variation in treatment practices between clinicians treating the same illness, which may benefit some patients but disadvantage others.

Lately, however, clinicians and researchers from around the world have called for the introduction of a different type of research to evaluate the effectiveness of treatments, called comparative effectiveness research (CER).

The aim of CER is to assess the effectiveness and worth of those same medical interventions in real-life scenarios. These studies would be conducted by practicing clinicians to compare currently available treatment options to new agents or devices.

While randomised clinical trials involve carefully-selected participants and strict treatment regimens, in real life, patients typically present with multiple health problems and may be subjected to numerous variations in standard treatment.

As such, CER is designed to account for the plethora of treatment options by comparisons that are clinically meaningful in a broader representation of the affected population. CER may therefore reduce variability in clinical practice, helping to ensure high-quality care for all patients.

Cost effectiveness

The outcomes of recent studies demonstrate that CER makes an important contribution in confirming the effectiveness, or ineffectiveness of a treatment for a particular health issue. It could also reduce spending on less effective, or even unnecessary, procedures.

Randomised clinical trials don’t account for patient variability – that’s where CER can be of use. Anoto AB

Results of a study released in 2011, for example, looked at ways to prevent hospital patients dying of blood clots (thromboembolism). The researchers discovered that a well-accepted and frequently prescribed treatment involving a combination of compression stockings and an injected medication, was no more effective - but obviously more expensive - than the use of compression stockings on their own.

If more broadly applicable, the potential cost savings across the system of comparing newer treatments to other established alternatives is potentially enormous.

Next steps

While it might be assumed that existing research funding avenues provided by the National Health and Medical Research Council (NHMRC) are sufficient for conducting CER, this type of research may not be seen as novel or as innovative as other types of research that seek to redefine the boundaries of new knowledge. Therefore, CER may continue to miss out on necessary funds.

In light of this, a group of local researchers and clinicians has proposed that a new research funding program be established from the same financial streams that fund the nation’s health-care services. This would foster and encourage CER and ensure its initiatives don’t have to compete for funding with projects designed to develop new knowledge.

The advantage of using a research methodology to fund comparative effectiveness initiatives is that the very conduct of research engages clinicians in the change process by facilitating a cultural shift. It requires those involved to sift through currently available evidence, against which comparisons of alternative approaches will be made.

Importantly, CER allows clinicians to develop novel approaches to prevent, diagnose and treat illness for the benefit of the entire community. Hopefully this model will soon be adopted by health policymakers and become a reality.