Making Purposeful Treatment Decisions for Meaningful Change

NeurologyLiveAugust 2019
Volume 2
Issue 4

NeurologyLive Editor in Chief Stephen D. Silberstein, MD discusses the importance of reaching a treatment effect that makes a significant difference in patients' lives.

Stephen D. Silberstein, MD

Stephen D. Silberstein, MD

In July, I attended the American Headache Society’s Annual Scientific Meeting in Philadelphia, Pennsylvania, where hundreds of my colleagues in headache medicine convened to discuss the latest trial data and clinical applications. However, amid the lively debates and poster tours, I noticed one topic of special interest: clinically meaningful change.

Indeed, clinically meaningful change has long been a part of the conversation surrounding headache and migraine, but now, as our treatment armamentarium continues to grow, it is more important than ever to consider the impact that these treatments have on symptoms that our patients consider most bothersome.

After all, if a preventive therapy reduces monthly migraine days by 5, but the patient still experiences debilitating pain and photophobia during the remaining 10 migraine days, have we really helped them? We must repeatedly ask ourselves, “Is what we consider significant in a trial really significant to a patient?”

“Statistical tests inform on the probability of a result being a chance finding; however, they convey no information on whether a given effect will be experienced as important by patients,” wrote the authors of a 2017 systematic review on the topic.1 “In acute pain, it seems reasonable to anchor clinical relevance to the patients’ experience. This approach is in accordance with the increasing awareness of the relevance of patient-reported outcomes in clinical research.”

The topic was the focus of a symposium at the meeting, where it was reported that 40% of patients on migraine therapy still experience at least 1 migraine-related issue. This, combined with the fact that it can take weeks to months for patients to see therapeutic effects, ultimately contributes to the high treatment discontinuation rate we see in this population.

Although anti—calcitonin gene-related peptide therapies have brought us into a new era of migraine treatment—an era with significant reductions in migraine frequency and severity—we can always do better.

And “better” doesn’t have to be just more effective therapies; setting appropriate efficacy expectations “should be a lengthy convo with your patient,” advised Jelena Pavlovic, MD, PhD, an assistant professor of neurology at Albert Einstein College of Medicine in Bronx, New York, who spoke on the topic. “It’s easy for the FDA’s first-line measures to spill over to what we think falls within clinical expectations, but that does not mean that that’s all we should care about or what patients care about."

“Knowing your patients, and…what their biggest problem is, is vital.”

Join Stephen D. Silberstein, MD and other top neurologists in New York City this September 27-28 for the 1st Annual International Congress on the Future of Neurology. The 2-day Congress will feature a rigorous agenda of presentations, question and answer sessions, and lightning rounds highlighting topics across the breadth of neurology, including the latest in headache, multiple sclerosis, dementia, movement disorders, epilepsy, and more. Click here to register and receive 25% off with code Neuro19SI.


1. Olsen MF, Bjerre E, Hansen MD, et al. Pain relief that matters to patients: systematic review of empirical studies assessing the minimum clinically important difference in acute pain. BMC Med. 2017;15(1):35. doi: 10.1186/s12916-016-0775-3.

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