A Framework for Estimating the Eligible Patient Population for New Migraine Acute Therapies in the United States

DOI: https://doi.org/10.1007/s12325-021-01781-z

Abstract-Summary New acute therapies may offer relief for this population of patients; however, popu- lation size and associated potential costs of new therapies are unclear.

A conceptual framework was developed to estimate anticipated use of new acute

therapies.

Targeted literature review (TLR) was conducted to identify factors affecting access to migraine-specific acute therapies, and characteristics of individuals who would be eligible for new acute therapies.

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Application of the framework suggested that 15–25% of individuals with

migraine would be eligible for new acute therapies.

A limited number of patients currently use migraine-specific acute therapies. A minority of individuals with migraine may be expected to use new acute

therapies.

The framework developed in this study is intended to facilitate estimating the

eligible patient population in assessments of costs of new acute therapies.

Such assessments should also consider recommendations that patients have access to multiple types of acute therapies, which may yield savings from reduced medication-overuse headache (MOH), progression to chronic migraine, and urgent- care costs.

Extended: We developed a conceptual framework for estimating anticipated use of new acute therapies, based on a targeted literature review (TLR) and insights from clinical experience.

We conducted a TLR which was used to inform development of a conceptual framework for estimating the eligible patient population for new acute therapies for migraine in the US.

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Introduction The American Headache Society (AHS) indicated that new acute treatments should be considered in patients who have contraindications to triptans or who have failed to respond to or tolerate at least two oral triptans [52].

The framework identifies factors affecting access to migraine-specific (i.e., pre- scription) acute therapies and the characteristics of patients who would use new acute therapies.

We then illustrated use of the framework by estimating the size of the patient population eligible for new migraine-specific acute therapies in the US, applying parameter values (1) identified in the TLR and (2) modeled in a recent budget- impact analysis (BIA) of new acute therapies conducted by the Institute for Clinical and Economic Review (ICER).

Considerations for modeling the costs of new acute treatments for migraine, beyond those for estimating the eligible patient population, are discussed on the basis of results of the TLR.

Methods Types of results excluded in the screening step included descriptive publications (e.g., reporting on symptoms and complications, pathophysiology, patient-reported outcome tools, and biological mechanisms of side effects of treatments), prospec- tive studies of efficacy of migraine therapies (including acute and preventive), cost- effectiveness analyses, and other studies of non-acute therapies (including non-migraine specific and preventive).

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Eligibility of remaining publications was confirmed on the basis of review of abstracts; publications were excluded if their abstract suggested they were review articles of efficacy and safety results or studies of non-US populations.

Following confirmation of eligibility and review of the publications, their find- ings were summarized by the reviewers and discussed among all authors of this study.

To estimate the patient population eligible for new acute therapies, factors affect-

ing access to migraine-specific acute therapies were first specified.

Results Further, a significant proportion of patients who might benefit from migraine-spe- cific acute therapy may not be candidates for use of triptans.

Beyond current users of triptans, a significant proportion of patients may have discontinued use; in the CaMEO study, compared to the 23% currently using migraine-specific acute therapy, 13% were former users, among whom only one in five reported being able to work or function normally with a headache [88].

In step 4, the percentage of migraine-specific acute therapy users inadequately managed with triptans was modeled on the basis of the OVERCOME (ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE) study [89], due to its recency and distinction of causes of inadequate management with triptans (i.e., lack of efficacy, intolerance, or contraindication).

The conceptual framework was also applied using parameter values reported in the ICER BIA, which yielded an estimate that 17.1% of prevalent migraine patients might be eligible for new acute therapies.

Discussion Considering that different acute therapies may be needed for effective management in different attacks or within an attack, patients may require access to an array of acute therapies in order to follow a successful management strategy [90–93].

If access to new acute therapies allows patients to follow their recommended management strategy more effectively, assessments of total costs should account for the potential cost offsets associated with improved effectiveness of manage- ment [94].

In assessing the costs of acute treatments for migraine, healthcare decision-mak- ers may benefit from considering patient and physician education initiatives around effective acute treatment strategies.

Many patients with migraine have never used migraine-specific acute therapies [95, 96] and among those who have, a significant proportion may delay use of acute treatments to a point where they are of lesser effectiveness [97, 98].

Guidelines for acute treatment therefore stress the importance of ensuring that patients understand the condition and their treatment strategy, and that acute therapy be taken early in attacks [52, 99].

Conclusions We conducted a TLR which was used to inform development of a conceptual frame- work for estimating the eligible patient population for new acute therapies for migraine in the US.

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Using the framework, we illustrated that applying population mean parameter values (1) identified in the TLR and (2) used in ICER’s BIA of new acute therapies suggested that 15–25% of prevalent migraine patients might be eligible for new acute therapies.

Beyond estimation of the eligible patient population, other important factors to consider in assessment of total costs of new acute therapies include that effective management strategies call for access to multiple acute therapies, and that ineffec- tive acute treatment is associated both with considerable burden for patients (includ- ing risk of MOH and progression to chronic migraine) as well as significantly higher healthcare resource-use costs.

Acknowledgement A machine generated summary based on the work of Harris, Linda; L’Italien, Gilbert; O’Connell, Thomas; Hasan, Zacharia; Hutchinson, Susan; Lucas, Sylvia. 2021 in Advances in Therapy.

Unmet Needs in Japanese Patients Who Report Insufficient Efficacy with Triptans for Acute Treatment of Migraine: Retrospective Analysis of Real-World Data