Researchers call for more pragmatic clinical trials in MS

Few multiple sclerosis (MS) clinical trials have used a so-called pragmatic design, which evaluates the effectiveness of interventions in real-life routine practice, a study has found.

Because such trials are better able to emulate conditions in real-world clinical practice and provide solid and more actionable evidence to inform treatment decisions, scientists are calling for their increased use in the MS field.

The researchers made their argument in a paper, “Use of pragmatic randomized trials in multiple sclerosis: A systematic overview,” published in Multiple Sclerosis Journal.

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Participating in traditional trial different from experience in clinical practice

Clinical trials are carefully designed studies using human participants. Most clinical trials (such as studies investigating experimental MS therapies) have intensive monitoring, strict inclusion criteria, and other structures that are designed to ensure results from the study are as clear and detailed as possible.

These structures make traditional trials helpful for answering scientific questions, but at the same time, it means the experience of participating in a trial is usually very different from what it’s like to receive care in a clinic.

Pragmatic clinical trials offer an alternative to this setup. The basic idea behind a pragmatic trial is to design the study so participants have an experience that’s as close as possible to day-to-day clinical care. These trials are also generally more focused on answering the question of what the best treatment choice is, rather than investigating a novel intervention, as traditional clinical trials do.

While the inclusion of real-world populations means data are messier, comparisons of existing treatment approaches provide results that are more readily translated into clinical practice.

Here, researchers conducted a review of the medical literature, looking for pragmatic clinical trials that involved people with MS.

The team identified 48 studies published from 1967 to 2022, with about half published after 2015. In total, 12 studies were described by their authors as “pragmatic,” and another 36 weren’t described with those exact terms but generally met criteria for a pragmatic trial. The researchers noted, however, that many studies also included non-pragmatic elements, such as excluding pregnant or older patients.

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Most studies assessed lifestyle or behavioral interventions

The vast majority of the studies assessed non-pharmacological interventions, such as lifestyle or behavioral changes. Seven of the trials compared the effects of different medications.

The outcomes assessed by the different studies varied. Some trials tested the effects of interventions on quality of life, others on physical function, and others on disease activity.

The studies generally were small: most had fewer than 100 participants, and a handful had more than 500 participants. Most of the trials were conducted in the U.S. or Europe, and most were funded by public sources.

Overall, the researchers concluded “there are only very few and mostly small pragmatic trials in MS and rarely assessing drug treatments.”

The scientists called for more studies using this design, highlighting “there is an urgent need to leverage the potential of this pioneering study design to provide useful randomized real-world evidence.”