Patient-reported experiences during and following treatment with belantamab mafodotin for relapsed/refractory multiple myeloma in the DREAMM-2 study.

Introduction: Patients with relapsed or refractory multiple myeloma (RRMM) are likely to be living with persistent symptoms, especially bone pain and fatigue, and experiencing restrictions in their physical and social functioning, which reduce health-related quality of life.

Methods: This qualitative interview study evaluated patients' perspectives about living with RRMM and their treatment with belantamab mafodotin, using interviews embedded in the Phase II DREAMM-2 trial (NCT03525678) with belantamab mafodotin. Patients consented to participate in up to 2 recorded telephone interviews (at treatment cycle 4 [C4] and at end of treatment [EOT]) comprising open-ended questions.

A toolbox of different approaches to analyze and present PRO-CTCAE data in oncology studies.

Background: The patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is used to assess symptomatic adverse events in oncology trials. Currently, no standard for PRO-CTCAE analysis exists.

Methods: Key methods of descriptive analysis and longitudinal modeling using PRO-CTCAE data from an oncology clinical trial, DRiving Excellence in Approaches to Multiple Myeloma-2 (DREAMM-2), a phase II trial of belantamab mafodotin in multiple myeloma (NCT03525678), were explored.

Assessing the treatment pattern, health care resource utilisation, and economic burden of multiple myeloma in France using the Système National des Données de Santé (SNDS) database: a retrospective cohort study.

Background: Real-world data on health care resource utilisation (HCRU) and costs for French patients with multiple myeloma (MM) are limited due to the quickly evolving MM treatment landscape. This retrospective, national-level study quantified the MM economic burden in France.

Methods: The study included patients with newly diagnosed MM from the Système National des Données de Santé coverage claims database between 2013 and 2018 who received active treatment within 30 days of diagnosis.

Belantamab mafodotin for the treatment of relapsed/refractory multiple myeloma in heavily pretreated patients: a US cost-effectiveness analysis.

Background: Patients with relapsed/refractory multiple myeloma (RRMM) require several lines of therapy, with typically shorter remission duration with each additional line.

Research Design And Methods: The cost-effectiveness of belantamab mafodotin (belamaf; DREAMM-2; NCT03525678) was compared with selinexor plus dexamethasone (SEL+DEX; STORM Part 2; NCT02336815) among patients with RRMM who have received at least four prior therapies. The base case used a US commercial payer's perspective over a 10-year time horizon.

Understanding the migraine treatment landscape prior to the introduction of calcitonin gene-related peptide inhibitors: Results from the Assessment of TolerabiliTy and Effectiveness in MigrAINe Patients using Preventive Treatment (ATTAIN) study.

Background: Calcitonin gene-related peptide (CGRP) inhibitors were introduced in the United States (US) in 2018. To understand the changing patterns of preventive treatment following the introduction of these new agents, we must first characterize the patterns which preceded their introduction.

Objective: To characterize the burden, unmet need, and treatment patterns in patients with migraine initiating preventive migraine medications before the introduction of CGRP inhibitors in the US.

Longitudinal assessment of utilities in patients with migraine: an analysis of erenumab randomized controlled trials.

Background: Cost-effectiveness analyses in patients with migraine require estimates of patients' utility values and how these relate to monthly migraine days (MMDs). This analysis examined four different modelling approaches to assess utility values as a function of MMDs.

Methods: Disease-specific patient-reported outcomes from three erenumab clinical studies (two in episodic migraine [NCT02456740 and NCT02483585] and one in chronic migraine [NCT02066415]) were mapped to the 5-dimension EuroQol questionnaire (EQ-5D) as a function of the Migraine-Specific Quality of Life Questionnaire (MSQ) and the Headache Impact Test (HIT-6™) using published algorithms.

Letter--Value-Driven Treatment Decisions: Need of the Hour for Relapsed/Refractory Multiple Myeloma.

No funding was involved in the writing of this letter. Medhekar, Sapra, Majer, Harrison, and Tekle are employees of and stockholders in Amgen.

Patient Preferences for Preventive Migraine Treatments: A Discrete-Choice Experiment.

Objective: To understand treatment preferences of people with migraine and the relative importance of improvements in efficacy and avoiding adverse events (AEs), such as cognition problems or weight gain.

Background: Current preventive migraine medicines are associated with poor adherence and tolerability. There is an unmet need for effective migraine-specific preventive treatments with fewer AEs.

Migraine day frequency in migraine prevention: longitudinal modelling approaches.

Background: Health economic models are critical tools to inform reimbursement agencies on health care interventions. Many clinical trials report outcomes using the frequency of an event over a set period of time, for example, the primary efficacy outcome in most clinical trials of migraine prevention is mean change in the frequency of migraine days (MDs) per 28 days (monthly MDs [MMD]) relative to baseline for active treatment versus placebo. Using these cohort-level endpoints in economic models, accounting for variation among patients is challenging.

Costs of Acute Headache Medication Use and Productivity Losses Among Patients with Migraine: Insights from Three Randomized Controlled Trials.

Background: Migraine is associated with a substantial physical and emotional burden for patients. There is also a large economic burden associated with migraine, in terms of lost productivity and healthcare resource use. By reducing the number of monthly migraine days (MMD) experienced by patients, effective preventive treatments can reduce acute medication use and costs of lost productivity.

Improving Medical Communication in Migraine Management: A Modified Delphi Study to Develop a Digital Migraine Tracker.

Objective: This study aimed to identify the essential content and amount of information to be collected from people with migraine via a patient-facing smartphone-based migraine tracker for them to share with clinicians during live discussions to assist in optimizing migraine management. The proposed tracker is intended for use in non-interventional research to evaluate disease burden in episodic migraine and chronic migraine patients as assessed by demographic and clinical characteristics and health resource utilization in an integrated delivery network setting. The proposed tracker is not intended for commercial purposes.

Migraine-related disability, impact, and health-related quality of life among patients with episodic migraine receiving preventive treatment with erenumab.

Background We evaluated the effect of erenumab, a fully human monoclonal antibody that inhibits the canonical calcitonin gene-related peptide receptor, on migraine-related disability, impact, and health-related quality of life among patients with episodic migraine. Methods Patients enrolled in a phase 3, 6-month, double-blind, placebo-controlled study of once-monthly erenumab 70 and 140 mg for migraine prevention (STRIVE) used an eDiary during the baseline and double-blind treatment phases to complete validated, specific questionnaires, including the modified (monthly) Migraine Disability Assessment Questionnaire; Headache Impact Test; and Migraine-Specific Quality of Life Questionnaire-role function-restrictive (MSQ-RFR), -role function-preventive (MSQ-RFP), and -emotional function (MSQ-EF). Results A total of 955 patients were randomized to receive erenumab 70 mg (n = 317), erenumab 140 mg (n = 319), or placebo (n = 319).

Estimating the clinical effectiveness and value-based price range of erenumab for the prevention of migraine in patients with prior treatment failures: a US societal perspective.

Background: Frequent migraine with four or more headache days per month is a common, disabling neurovascular disease. From a US societal perspective, this analysis models the clinical efficacy and estimates the value-based price (VBP) for erenumab, a fully human monoclonal antibody that inhibits the calcitonin gene-related peptide receptor.

Methods: A Markov health state transition model was developed to estimate the incremental costs, quality-adjusted life-years (QALYs), and value-based price range for erenumab in migraine prevention.

Direct and Indirect Healthcare Resource Utilization and Costs Among Migraine Patients in the United States.

Objective: The goal of this analysis was to provide a contemporary estimate of the burden of migraine, incorporating both direct and indirect costs, by comparing the costs of migraine patients to a matched group of patients without migraine in a large, nationally representative sample of commercially insured patients in the United States.

Background: Previous studies have shown that the economic burden of migraine in the United States is substantial for payers, patients, and employers. Despite the availability of multiple acute and preventive pharmacological treatment options and a relatively stable migraine prevalence in the United States, there has been a documented increase in migraine-related healthcare resource and pharmacy use.

A Controlled Trial of Erenumab for Episodic Migraine.

Background: We tested erenumab, a fully human monoclonal antibody that inhibits the calcitonin gene-related peptide receptor, for the prevention of episodic migraine.

Methods: We randomly assigned patients to receive a subcutaneous injection of either erenumab, at a dose of 70 mg or 140 mg, or placebo monthly for 6 months. The primary end point was the change from baseline to months 4 through 6 in the mean number of migraine days per month.

Development and exploration of the content validity of a patient-reported outcome measure to evaluate the impact of migraine- the migraine physical function impact diary (MPFID).

Background: Adults with migraine experience substantial reductions in quality of life during and in-between migraine attacks. Clinical and regulatory guidelines encourage the inclusion of patient reported outcomes for the evaluation of benefits of interventions for migraine.

Methods: The conceptual framework and items for a new patient-reported outcome (PRO) instrument, the Migraine Physical Function Impact Diary (MPFID), were developed using scientific methods recommended to ensure content validity of PRO instruments.

Factors Associated with Direct Health Care Costs Among Patients with Migraine.

Background: Migraine imposes substantial economic burden on patients and the health care system. Approximately 18% of women and 6% of men suffer from migraine in the United States. This is a heterogeneous group, and little data are available to evaluate factors associated with migraine costs.

Psychometric Evaluation of a Novel Instrument Assessing the Impact of Migraine on Physical Functioning: The Migraine Physical Function Impact Diary.

Objective: The objective of this study was to evaluate the measurement properties of the Migraine Physical Function Impact Diary (MPFID), a novel patient-reported outcome (PRO) measure for assessing the impact of migraine on physical functioning.

Methods: In a prospective, observational study, adults with episodic migraine (EM) or chronic migraine (CM) used an eDiary to complete the MPFID (assessing daily impacts of migraine on physical function) and a headache diary (capturing migraine days, migraine pain intensity, and migraine interference) each day, and other PRO instruments related to migraine. Item-level evaluation, item response theory (IRT), and exploratory factor analysis (EFA) methods were applied to identify domains, select final MPFID items, and develop scoring procedures.

Measuring the impact of migraine for evaluating outcomes of preventive treatments for migraine headaches.

Background: Migraine is characterized by headache with symptoms such as intense pain, nausea, vomiting, photophobia, and phonophobia that significantly impact individuals' lives. The objective of this study was to develop a strategy to measure outcomes from the patients' perspectives for use in evaluating preventive treatments for migraine.

Methods: This study used a multi-stage process.

Health state utilities associated with attributes of treatments for hepatitis C.

Background: Cost-utility analyses are frequently conducted to compare treatments for hepatitis C, which are often associated with complex regimens and serious adverse events. Thus, the purpose of this study was to estimate the utility associated with treatment administration and adverse events of hepatitis C treatments.

Design: Health states were drafted based on literature review and clinician interviews.

Incidence and cost of treatment-emergent comorbid events in insured patients with chronic hepatitis C virus infection: a retrospective cohort study.

Background: Treatment-emergent comorbid events (TECs) are common In patients initiating treatment with pegylated interferon alpha (PEG-IFN-alfa) and ribavirin for chronic hepatitis C virus (HCV) infection. The purpose of this study was to estimate the incidence and incremental cost of these events.

Methods: In a retrospective cohort analysis of healthcare claims, we studied patients with HCV who were newly treated with PEG-IFN-alfa/ribavirin between 2006 and 2008.

Measurement properties of the flu-like symptom index from the hepatitis physical symptom severity diary.

Purpose: Chronic Hepatitis C (CHC) Virus infection is a serious health issue in the US. Standard treatment involves peginterferon alpha and ribavirin, often associated with adverse side effects including flu-like symptoms. These adverse effects are common reasons for the discontinuation of treatment and therefore represent a major obstacle in the effective treatment of CHC.

Care of asthma patients in relation to guidelines.

Clinical asthma care may have to change to be brought in line with Expert Panel Report 3 (EPR3) guidelines, which recommend increased intensity of therapy (steps) to treat uncontrolled asthma. This study determined if asthma therapy steps can be identified using claims data and if patients have appropriate step-up in therapy if their disease is not controlled. A cohort study was performed using an administrative claims database and involving patients 12-64 years old with uncontrolled asthma events (either impairment or risk).