Monday, 21 December 2015

ANZIC-RC wins $4.4 million for ground-breaking pneumonia trial

In a ground-breaking new platform trial, researchers at SPHPM from the Australian and New Zealand Intensive Care Research Centre (ANZIC-RC) in collaboration with the Platform for European Preparedness Against (Re-)emerging Epidemics (PREPARE) have secured $4.4 million from the National Health Medical Research Council (NHMRC).

The OPTIMISE CAP trial (Optimisation by Platform Trial Involving Multiple Interventions with Simultaneous Evaluation in Community Acquired Pneumonia), is planned as a perpetual trial to commence in 2016.

Leading the Australian and New Zealand component of the trial is Adjunct Professor Steve Webb, who is very enthusiastic to be at the cutting edge of trial design and believes that a platform trial is the best strategy to rapidly improve the strength of available evidence.

"This trial represents a paradigm shift in the way evidence is generated to help clinicians provide optimal treatment to their patients. The trial design is around five times more efficient that conventional trials and it is highly likely that this type of design will become used increasingly across all disciplines of medicine,” Professor Webb said.

The ground-breaking trial will use a highly adaptive and innovative trial design to evaluate the impact of interventions to reduce mortality and morbidity for patients with Severe Community Acquired Pneumonia (Severe CAP) in Intensive Care Units (ICUs) across Australia.

Currently, the mortality burden of severe community acquired pneumonia is similar to the annual road toll and it is responsible for more than 7,000 ICU admissions and 1,400 deaths each year.

Researchers said currently there is a stark contrast between the substantial public health impact of Severe CAP and the low quality of evidence that guides therapies.

“As a consequence of the limited evidence-base there are major differences, some contradictory, among international guidelines. For example, each major guideline recommends different, albeit overlapping, antibiotic choices,” Professor Webb said.

Clinical registry data estimates that the annual direct costs of ICU care of these patients is in excess of $200 million.

The trial will compare different types of ventilation, antibiotic and immunomodulation strategies which are currently used as standard care and will preferentially randomise patients to treatments that the trial is finding to be more effective, leading to better outcomes for patients within the trial.

Professor Webb said there is a strong case that this trial is one of the most advanced trial designs ever developed.

“This preferential randomisation, termed response adaptive randomisation, means that mortality for patients within the trial is lower than would have been the case than if the trial had been conducted with 50:50 randomisation. This is an impressive fusion of continuous quality improvement with generation of new knowledge,” Professor Webb said.

The trial will also provide a platform that can be adapted to test alternative interventions in the event of a pandemic that causes life-threatening Severe Acute Respiratory Illness (SARI), thus contributing a major component of Australia’s pandemic research response.

The trial will run in Europe coordinated by The Julius Center for Health Sciences and Primary Care at the University Medical Centre Utrecht in the Netherlands.