A recent article in Cardiovascular Business summarized the lack of quality research on surgeries for children with congenital heart disease.
Reviewers uncovered a total of 333 randomized clinical trials encompassing 23,902 children from 34 countries since 2000. Ninety-four percent of the studies were early phase and had small sample sizes, while only 4.5 percent of the trials made it to the late phases.
“Randomized controlled trials only represent the gold standard when appropriately designed, conducted and reported,” wrote Nigel E. Drury, PhD, and colleagues in their review published in the European Journal of Cardio-Thoracic Surgery. “Although phase II efficacy and safety trials have a role in determining whether potential treatments are worth pursuing, it is phase III clinical effectiveness trials that influence guidelines and change practice.”
It turns out enrolling patients for these trials isn’t the problem. Target sample size is achieved in 87 percent of the studies, the researchers reported.
With enough willing participants, the responsibility lies with the research community to create more studies and improve the design and conduct of the trials, so they reach the late stages. If this is achieved and clinically relevant endpoints are evaluated, the findings could be substantial enough to improve practice.
The reviewers suggested borrowing a page from pediatric oncology’s playbook in making “the opportunity to participate in late-phase clinical trials part of the routine care pathway.”
From my personal experience and anecdotal evidence from news reports and documentaries, families that experience a tragedy or have ailing children are incredibly sympathetic to others in a similar situation. If presented with the opportunity to help their own child with a novel heart procedure while also informing future best practice for other kids in the same predicament, most families would likely be willing to participate in a trial.
It’s up to researchers to ensure that participation doesn’t go to waste.
“As a congenital heart disease community, we have the responsibility to provide scientific leadership and work together to conduct well-designed, rigorously conducted, multicenter trials with clearly defined, clinically relevant endpoints that answer important questions to improve the outcomes of surgery for our patients and their families,” Drury and co-authors wrote.