Comparative Effectiveness of Novel Pharmacologic and Evidence-Based Nonpharmacologic Treatments for Migraine Prevention -- Cycle 2 2023

The Patient-Centered Outcomes Research Institute (PCORI) seeks to fund rigorous, large-scale pragmatic trials that compare newly available pharmacologic and/or evidence-based nonpharmacologic treatments for the prevention of migraine. For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures, and care services) for potential funding by PCORI.

Research Initiative Highlights

Preventive treatments include both pharmacological and nonpharmacological options, with the goal of reducing the frequency, severity, and duration of attacks. Although there are many pharmacological options used for migraine prevention, most options were originally developed for the treatment of other conditions. Newly approved treatments such as calcitonin gene-related peptide (CGRP) antagonists have been developed specifically for migraine prevention and have been shown to be efficacious. However, there are uncertainties regarding the long-term comparative effectiveness of CGRP inhibitors compared with commonly prescribed preventives. Further, there is growing interest in the use of nonpharmacological therapies and additional research is needed to understand the role of such options in the prevention of migraine. 

This Targeted PCORI Funding Announcement will solicit applications that respond to the following question:

What is the comparative effectiveness of novel pharmacologic and/or evidence-based nonpharmacologic treatments for the prevention of migraine?

PCORI is particularly interested in studies that compare emerging pharmacological options such as CGRP antagonists to standard prophylactic therapy or to each other. PCORI is also interested in studies that examine the comparative effectiveness of evidence-based nonpharmacological options for migraine prevention. As appropriate, studies may include nonpharmacological interventions as standalone therapy or as an adjunct to pharmacological options.

Proposed studies should examine large and diverse populations, as appropriate, with an overall sample size that allows precision in the estimation of hypothesized effect sizes. The application must also have planned subgroup analyses aimed at generating meaningful information to inform clinical practice. Applicants should also clearly define criteria for the initiation of preventive therapy based on guideline recommendations.

Applicants should consider, as appropriate, the full range of clinical and patient-centered outcomes data relevant to patients and other stakeholders. PCORI’s Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research inform the expectations for applicants and the corresponding evaluation of applications submitted in response to this PFA. Pharmacologic studies should be adequately powered to assess changes in the frequency of headache or migraine days consistent with and/or exceeding prior efficacy trials, while considering evidence gaps regarding long-term effectiveness (follow-up of at least 24 weeks). Other important outcomes of interest include quality of life, headache-related disability, functional impact, tolerability, adverse events, and use of abortive treatment.

PCORI encourages all investigators interested in applying to request a conversation with a PCORI Program Officer via [email protected] prior to submitting a Letter of Intent. Investigators considering a request for patient care cost coverage are especially encouraged to reach out.