Number Needed to Treat in Multiple Sclerosis Clinical Trials.

Area of Science:

• Neurology
• Clinical Trials
• Biostatistics

Background:

• Clinicians evaluate therapy benefits against risks.
• Relapsing-remitting multiple sclerosis (RRMS) management relies on evidence interpretation.
• Standard measures include risk reduction and MRI lesion reduction.

Purpose of the Study:

• To review the utility of the number needed to treat (NNT) in RRMS clinical trials.
• To discuss NNT's methodological characteristics and application in RRMS.
• To provide guidance on appropriate use of NNT in this context.

Main Methods:

• Literature review focusing on NNT methodology.
• Analysis of NNT application in RRMS controlled clinical trials.
• Examination of time-to-event and recurrent event endpoints.

Main Results:

• NNT interpretation can be unreliable in RRMS trials.
• NNT's utility is questionable for heterogeneous diseases like MS.
• Specific scenarios where NNT use is cautioned against are identified.

Conclusions:

• Caution is advised when using NNT for RRMS treatment effect interpretation.
• NNT should be used judiciously, especially in comparative trials and with specific endpoints.
• Alternative or complementary measures may be more reliable for RRMS evidence appraisal.