Project Summary

Background and Significance 

Migraine affects 14 percent of the general population each year and is a leading cause of pain and disability. Migraine treatment consists of symptomatic therapies (treatments taken during a migraine attack to relieve symptoms) and preventive therapies (regularly scheduled treatments that reduce migraine attack frequency). Symptomatic medication is indicated for and utilized by nearly all individuals with migraine. Preventive medication is indicated in approximately 40 percent of those with episodic migraine and nearly all individuals with chronic migraine, meaning that over 21 million people in the United States should be offered preventive treatment.   

Several medications from different medication classes are commonly used for the prevention of migraine. Any one of these preventive medications has a 30 percent to 55 percent likelihood of providing adequate benefit, typically defined as at least a 50 percent reduction in frequency of days with headache or migraine. However, due to a lack of studies that have directly compared these migraine medications, there is insufficient knowledge about how the benefits and side effects from these different medications compare to one another.  

Current guidelines and payer coverage rules typically require “step therapy” in which older and less expensive medications must be tried prior to newer and more expensive medications. This approach is appropriate if the newer medications are not superior to the older ones. However, if the newer medications are superior, step therapy is inappropriately resulting in many patients going through a process of trial and error during which they change from one preventive medication to the next over many months or years in search of a treatment that is best suited for them.   

Study Aims 

The study objective is to compare the benefits and tolerability of a newly approved migraine medication (atogepant) to two older medications (propranolol, topiramate) that are commonly used in clinical practice and have strong evidence for their efficacy.   

Study Description 

In this study, 1,200 adults with migraine who should be offered preventive therapy according to established guidelines will be randomly assigned to one of three oral medications used for migraine prevention and will complete the study. Patients will be enrolled from primary care, general neurology and headache specialty clinics.   

The primary outcome will be the proportion of patients who are “treatment responders,” defined as: 

  • Completing 12 weeks on the assigned medication without stopping the medication for any reason 
  • Having a 50 percent or greater reduction in headache frequency at weeks 9-12 after starting the medication compared to the four weeks prior to treatment 

Additional study outcomes will provide more information to clinicians and patients about choosing between migraine preventive medications, including patient quality of life, disability caused by migraine, side effects, benefits from the medications, patient satisfaction with the medications, and work productivity and impairment. Longer-term outcomes will be measured at weeks 21-24 and 45-48.   

Numerous stakeholders will contribute to this research, including individuals with migraine, scientists, clinicians, clinical trial experts, statisticians, experts in information dissemination, pharmacists, leaders from headache and neurological societies that educate and advocate for people with migraine and their clinicians, and representatives from payor organizations and healthcare systems. These stakeholders have participated in the process of defining the research question, the primary and secondary outcomes, and the study design. They will continue to participate in every aspect of the research so that it is patient centered, the outcomes are meaningful to patients, and the results will truly inform future treatment decisions and impact medication coverage.   

Results from this study will provide valuable information to patients, clinicians, guideline developers and payors about the relative benefits, side effects and persistence with migraine preventive medications. Since each of the study medications is already available in clinical practice, the results should directly and immediately impact the treatment of migraine.

Project Information

Todd Schwedt, B.S., M.D., M.S.
Mayo Clinic Arizona
$12,561,288 *

Key Dates

60 months *
April 2024
2024

*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.

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Last updated: April 23, 2024