Tackling treatment uncertainty: why experiments need to show results

Nick Medhurst

Manager, Good Clinical Trials Collaborative

During the global pandemic, randomised controlled trials (RCTs) have been essential in identifying effective treatments and vaccines for COVID-19.

Early in 2020, as the first COVID-19 patients arrived in hospitals, the medical world collectively faced a new unknown. With so little information available, we relied on the experience, judgement and bravery of countless healthcare professionals to do the best they could. They had to not only care for individuals but also learn and share as much as possible with severely limited knowledge, tools and resources.

With so much at stake, telling the difference between a promising idea and an effective treatment became a global priority. Vaccine studies demonstrated the importance of well-designed, sufficiently large RCTs that deliver clear, informative and publicly available results. Vaccines would simply not be in routine use without them.

Hope and evidence

The story was initially different for medicines to treat (rather than prevent) the disease. The urgency of caring for those already sick with COVID-19 created pressure to use drugs and other therapies where there was some plausible hope of benefit but no reliable evidence.

‘Something is better than nothing’ was a compelling rationale in a desperate situation and some health services looked to record, compare and share their findings. However, too little was done to design, coordinate and implement robust trials that could reliably compare and learn from the treatment of millions of patients. Time and lives were lost unnecessarily.

We must learn that when we face uncertainty and the unknown – now and in the future – we need our health systems to be ready and prepared to turn first to clinical trials.

Finding answers

Thanks to several large RCTs that were able to establish a system of learning, even while health services struggled to cope with and contain outbreaks, today we are better positioned globally to fight the pandemic with evidence-based strategies.

We must learn that when we face uncertainty and the unknown – now and in the future – we need our health systems to be ready and prepared to turn first to clinical trials, end the barriers between clinical care and research and rapidly turn hope into evidence.