Use of pragmatic randomized trials in multiple sclerosis: A systematic overview

Area of Science:

• Clinical Research Methodology
• Real-World Evidence Generation
• Neurology and Multiple Sclerosis Studies

Background:

• Pragmatic trials are crucial for generating real-world evidence on treatment effectiveness.
• Their application in multiple sclerosis (MS) research is growing but requires further investigation.
• Understanding the characteristics of existing pragmatic trials in MS is essential for future study design.

Purpose of the Study:

• To investigate the utilization and design characteristics of pragmatic trials within multiple sclerosis (MS) research.
• To analyze the scope of interventions and participant numbers in MS pragmatic trials.
• To assess the degree of pragmatism in MS trials using the PRECIS-2 tool.

Main Methods:

• A systematic literature search was conducted for pragmatic trials related to any intervention up to 2022.
• The PRagmatic Explanatory Continuum Indicator Summary-2 (PRECIS-2) tool was employed to assess the pragmatism of identified trials.
• Data on trial characteristics, interventions, participant numbers, geographical distribution, and outcomes were analyzed.

Main Results:

• 48 pragmatic trials in MS were identified, published between 1967 and 2022, with a median of 82 participants.
• The majority of trials (85%) focused on supportive care, with only 15% assessing drug interventions.
• Most trials originated from the UK, Italy, and the US; only 2% utilized routinely collected data, and 29% were considered widely pragmatic.

Conclusions:

• The field of pragmatic trials in multiple sclerosis (MS) is characterized by a limited number of mostly small studies, with a low focus on drug interventions.
• Despite the availability of routine data infrastructure, its use in MS pragmatic trials remains minimal.
• There is a significant need to enhance the design and execution of pragmatic trials in MS to effectively generate valuable real-world evidence.