“Once a medication is approved by the regulatory agencies and enters into clinical practice, we start using it. That’s where effectiveness [versus efficacy] comes into play. That’s not how something works in a clinical trial compared to something else, it’s how well it works in a real-world scenario.”

When ocrelizumab (Ocrevus, Genentech) was granted FDA approval in May 2017, multiple sclerosis (MS) got its first treatment for the primary-progressive form of the disease. And while it showed impressive efficacy in the clinical trials, there was some hesitation as to whether or not this would be replicable in the real world.

To determine this, a group of physicians at the Cleveland Clinic’s Mellen Center for MS, including Daniel Ontaneda, MD, undertook a study to observe the real-world effectiveness of ocrelizumab. The center similarly conducted these observational studies with other recently approved treatments such as fingolimod (Gilenya, Novartis).

At the Americas Committee for Treatment and Research in MS (ACTRIMS) Forum in Dallas, Texas, the staff neurologist at the Mellen Center shared his real-world experience with the treatment in primary-progressive MS patients. He detailed the importance of these observations in determining the effectiveness of a treatment in patients in clinical practice, as opposed to knowing the efficacy of a treatment—which is entirely measured in clinical trials in comparison to placebo or another treatment.

To find out more about the group’s work, NeurologyLive spoke with the staff neurologist at Cleveland Clinic's Mellen Center in an interview.