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Elke Hunsche,1 Martha Gauthier,2 Brooke Witherspoon,2 Viatcheslav Rakov,1 Sanjay K Agarwal3 1Myovant Sciences GmbH, Basel, Switzerland; 2Endpoint Outcomes, A Lumanity Company, Boston, MA, USA; 3Center for Endometriosis Research and Treatment, University of California San Diego, La Jolla, CA, USACorrespondence: Elke Hunsche, Global Market Access and Health Economics / Outcomes Research, Myovant Sciences GmbH, Aeschengraben 27, Basel, 4051, Switzerland, Email elke.hunsche@myovant.comObjective: This interview study sought to capture patients’ experiences and perceptions of endometriosis symptoms and their impacts on daily life, as described by women in their own words. Using open-ended questions and a concept-elicitation approach, this study assessed the signs and symptoms of endometriosis and their impacts on different aspects of quality of life, including daily activities, functioning, and well-being.Materials and Methods: This interview study included US women with moderate-to-severe endometriosis-associated pain who completed one of two Phase 3, randomized, double-blind, placebo-controlled trials (SPIRIT 1 or SPIRIT 2; ClinicalTrials.gov identifiers: NCT03204318, NCT03204331). Interviews were conducted via a web/Internet-based video platform or telephone by trained interviewers, using open-ended questions in a concept-elicitation approach, and probes as needed to obtain additional feedback on the burden of endometriosis. Qualitative data from the interviews were analyzed, and emerging concepts were coded by independent coders. Concept saturation was evaluated to determine if all endometriosis-related symptoms and impacts had been described by the sample of women interviewed.Results: Forty women participated in this study. In total, 18 unique symptoms of endometriosis emerged from the interviews; pelvic pain (92.5%), dyspareunia (80.0%), and heavy bleeding (75.0%) were the most commonly reported endometriosis symptoms. A total of 33 unique impacts of endometriosis symptoms were identified across 11 concepts: physical impacts, impacts on activities of daily living, social impacts, sleep impacts, emotional impacts, appearance impacts, financial impacts, sex-related impacts, work/school-related impacts, fertility impacts, and cognitive impacts. Concept saturation was achieved for both symptoms and impacts of endometriosis.Conclusion: This interview study provides substantive qualitative data on the burden of endometriosis, from the perspective of affected women in the US. The findings demonstrate the debilitating effect of endometriosis symptoms, which limit and adversely impact women’s daily lives.Keywords: endometriosis, interview, concept-elicitation, gynecology, patient perspective, pain

PURPOSE: To describe three commercial weight loss programs (Jenny Craig, LA Weight Loss, and Weight Watchers), including efficacy data when available, and to provide clinicians with information for patient counseling. DATA SOURCES: Personal interviews, journal articles, and Web site information. CONCLUSIONS: Commercial weight loss programs provide weight loss services. Benefits are experience, planned menus, and psychological support. Limitations are cost, sales promotions that encourage on-the-spot commitment to prepaid contracts, and the cost of program food and additional vitamins. IMPLICATIONS FOR PRACTICE: Weight loss is a necessary goal for many patients. Clinicians are mandated to provide counseling regarding weight loss. [ABSTRACT FROM AUTHOR]

What Is This Summary About?: This summary describes what researchers learned during interviews of women with uterine fibroids and heavy menstrual bleeding (or period bleeding). At this time, little is known about how women perceive the impact of uterine fibroids on their lives and more information is needed. The goal of this study was to provide new information about the symptoms women have and how these symptoms affect their everyday lives. These interviews were done to better understand how uterine fibroid symptoms affect the lives of women in their own words., What Were the Results?: Thirty women from the United States, who had completed a clinical trial for a new treatment for heavy menstrual bleeding and uterine fibroids, agreed to be interviewed. The women described what their experiences with uterine fibroids were and the impact these experiences with uterine fibroids had on their lives before participating in the clinical trial. The most common symptoms of uterine fibroids the women described were heavy bleeding with their menstrual periods, pain in the pelvis or groin area, the passing of blood clots, and anemia (or low hemoglobin in red blood cells). Women said their symptoms affected them physically, emotionally, socially, and financially. They also said their symptoms made it hard to do daily activities, sleep, have a sex life, and go to work or school., What Do the Results Mean?: Women who have heavy menstrual bleeding and uterine fibroids experience various uterine fibroid symptoms, and these symptoms affect most parts of the their lives.

This paper delves into the critical materials supply chain of the battery market with an emphasis on long-term energy security. The study recognizes electric vehicle battery packs as reservoirs of "locked reserves" for extended periods, typically 10 years or more. A comprehensive understanding of material flows and end-of-life battery management is essential to establish a sustainable, durable, and secure domestic supply chain for lithium-ion batteries. In addressing these concerns, the paper introduces a metric designed to assess the "per mile" consumption of critical reserves called "Materials Per Gallon-Electric (MPGe)". The study emphasizes the immediate need for critical materials to meet the accelerated demand for large-scale electric vehicle adoption in the short term. Furthermore, the paper also emphasizes the urgent need to advance recycling technologies to recover the critical mineral reserves "locked" in end-of-life battery packs., Competing Interests: The authors declare no competing financial interest., (© 2024 The Authors. Published by American Chemical Society.)

Background: Tardive dyskinesia (TD), a movement disorder in which patients experience abnormal involuntary movements, can have profound negative impacts on physical, cognitive, and psychosocial functioning. The Abnormal Involuntary Movement Scale (AIMS), a clinician-rated outcome, is considered the gold standard for evaluating treatment efficacy in TD clinical trials. However, it provides little information about the impacts of uncontrolled movements from a patient perspective and can be cumbersome to administer in clinical settings. The Tardive Dyskinesia Impact Scale (TDIS) was developed as a patient-reported outcome measure to fulfill the need for a disease-specific impact assessment in TD. The objective of the present study was to develop and evaluate the psychometric properties of the TDIS to determine whether it is fit-for-purpose to measure TD impact., Methods: Data from qualitative studies and phase 3 trials of a VMAT2 inhibitor for the treatment of TD (KINECT3 and KINECT4) were used to determine the psychometric properties of the TDIS. Qualitative research included concept elicitation and cognitive debriefing interviews with TD patients and their caregivers in order to assess how well the TDIS captured key domains of TD impact. Quantitative analyses to examine the psychometric properties of the TDIS included assessing construct validity (factor structure, known groups, and predictive validity) and responsiveness to change., Results: Qualitative results showed that the TDIS captures the key TD impacts reported by patients and caregivers and that the TDIS was interpreted as intended and relevant to patients' experiences. Quantitative results found evidence of 2 underlying domains of the TDIS: physical and socioemotional (Comparative Fit Index > 0.9). Known groups and predictive validity indicated that, compared with the AIMS, the TDIS captures unique content (correlation between AIMS and TDIS = 0.2-0.28). The TDIS showed responsiveness to change in treatment, with TDIS scores improving over 48 weeks in the 2 phase 3 trials., Conclusions: The TDIS captures relevant information about the impact of TD and is easily administered in a clinician's office or patient's home. It may be used longitudinally to show changes in TD burden over time. The TDIS complements the AIMS; using these assessments together provides a more holistic assessment of TD., (© 2024. The Author(s).)

Background: Research on women's perspective of uterine fibroids (UF) experiences using their own words is limited. This study aimed to provide new insights on the symptoms experienced and their impacts on daily life., Methods: Interview substudy in 30 US women with heavy menstrual bleeding (HMB) associated with UF who completed one of two phase 3, randomized, double-blind, placebo-controlled trials (LIBERTY 1 and 2; ClinicalTrials.gov identifiers: NCT03049735, NCT03103087). Women who consented to participate in this substudy were interviewed after their last clinical trial study visit. Concepts ( i.e. , symptoms and impacts) of importance to women were determined via open-ended questions, and the frequency of symptoms and their impacts, including the relationship between pain and menstruation, were assessed. Data were analyzed using established qualitative research methods, including grounded theory and constant comparative methods, and concept saturation was assessed., Results: Fifteen unique symptoms of UF emerged: the most commonly reported were HMB ( n  = 30, 100.0%), pelvic pain ( n  = 28, 93.3%), and passing of blood clots ( n  = 24, 80.0%). In total, 25 unique impacts were identified across eight concepts: physical impacts, activities of daily living, sleep, emotional impacts, sex life, social impacts, work and school, and financial impacts. Concept saturation was achieved for both symptoms and impacts., Conclusion: This study provides data on the symptoms experienced by women with HMB associated with UF, as well as the negative impacts of these symptoms as reported using their own words. The study findings confirm the significant burden associated with symptomatic UF., Competing Interests: E.H. and V.R. are employed by Myovant Sciences and are shareholders of the company. L.M. was an employee and a shareholder of Myovant Sciences at the time of writing. B.W. is employed by Endpoint Outcomes, who was compensated by Myovant Sciences to conduct this research. K.S. was an employee of Endpoint Outcomes at the time this research was conducted., (© Elke Hunsche et al., 2022; Published by Mary Ann Liebert, Inc.)

Progressive multifocal leukoencephalopathy (PML) is a John-Cunningham virus-related central nervous system that is rarely observed in persons treated with the anti-CD20 monoclonal antibody, rituximab., (© 2022. The Author(s), under exclusive license to Springer Nature Switzerland AG.)

More than half of all serious adverse drug reactions are identified seven years after FDA approval. One recent and unusual example involves a syndrome initially termed nephrogenic dermatopathic fibrosis, and then called nephrogenic systemic fibrosis (NSF)., (© 2022. The Author(s), under exclusive license to Springer Nature Switzerland AG.)

This study describes cases of individuals who report adverse events following consumption of the most commonly prescribed fluoroquinolone (FQ) antibiotics: ciprofloxacin, levofloxacin, or moxifloxacin. Fluoroquinolone (FQ) antibiotics are some of the most widely prescribed antibiotics in the world. Although these antibiotics have been on the market for more than 20 years, a wide range of serious FQ-associated adverse events first became apparent in 2006 and continued to be recognized for the next 15 years., (© 2022. The Author(s), under exclusive license to Springer Nature Switzerland AG.)

Biosimilar drugs, close copies of patented biologicals, are intended to provide access to less expensive, highly similar versions of reference (previously approved) biological agents (Kozlowski et al. in N Engl J Med 365:385-388, 2011). The biological epoetin accounts for $1.8 billion in drug spending annually worldwide (primarily for the treatment of anemia due to chronic kidney disease or anemia due to cancer chemotherapy.)., (© 2022. The Author(s), under exclusive license to Springer Nature Switzerland AG.)

Biosimilars are biologic drug products that are highly similar to reference products in analytic features, pharmacokinetics and pharmacodynamics, immunogenicity, safety, and efficacy. Biosimilar epoetin received Food and Drug Administration (FDA) approval in 2018. The manufacturer received an FDA nonapproval letter in 2017, despite receiving a favorable review by FDA's Oncologic Drugs Advisory Committee (ODAC) and an FDA nonapproval letter in 2015 for an earlier formulation. We discuss the 2018 FDA approval, the 2017 FDA ODAC Committee review, and the FDA complete response letters in 2015 and 2017; review concepts of litigation, naming, labeling, substitution, interchangeability, and pharmacovigilance; review European and U.S. oncology experiences with biosimilar epoetin; and review the safety of erythropoiesis-stimulating agents. In 2020, policy statements from AETNA, United Health Care, and Humana indicated that new epoetin oncology starts must be for biosimilar epoetin unless medical need for other epoetins is documented. Empirical studies report that as of 2012, reference epoetin use decreased from 40%-60% of all patients with cancer with chemotherapy-induced anemia to <5% of such patients because of safety concerns. Between 2018 and 2020, biosimilar epoetin use varied, increasing to 81% among one private insurer's patients covered by Medicare whose cancer care is administered with Oncology Analytics and to 41% with the same private insurer's patients with cancer covered by commercial health insurance and administered by the private insurer, to 0% in several Veterans Administration Hospitals, increasing to 100% in one large county hospital in California, and with yet-to-be-reported data from most oncology settings. We conclude that biosimilar epoetin appears to have overcome some barriers since 2015, although current uptake in the U.S. is variable. Pricing and safety considerations for all erythropoiesis-stimulating agents are primary determinants of biosimilar epoetin oncology uptake. IMPLICATIONS FOR PRACTICE: Few oncologists understand substitution and interchangeability of biosimilars with reference drugs. Epoetin biosimilar is new to the market, and physician and patient understanding is limited. The development of epoetin biosimilar is not familiar to oncologists., (© 2021 AlphaMed Press.)

Objectives: This study aimed to examine the validity of the modified Reflux Symptom Questionnaire-electronic Diary (mRESQ-eD) through patient input and psychometric testing of the questionnaire to support use in clinical trials in patients with persistent gastroesophageal reflux disease (GERD) and in accordance with Food and Drug Administration guidance on patient-reported outcome instruments., Methods: Cognitive interviews were conducted with patients (n = 30) to evaluate the interpretability and content validity of draft mRESQ-eD items. Patient data from a phase 2b clinical study (ClinicalTrials.gov identifier: NCT02637557) on persistent GERD served to aid in the construction of weekly scores for heartburn severity, regurgitation severity, and total GERD severity. These scores' psychometric properties were also evaluated., Results: Minor modifications were made to the draft mRESQ-eD based on patient feedback to improve interpretability and clarity of the instrument. Psychometric analysis suggested that an 8-item version of the mRESQ-eD was best suited to the clinical data. The internal consistency was found to be high (Coefficient ω = 0.95). Retest reliability and convergent validity were strong for a heartburn weekly severity score, regurgitation weekly severity score, and total GERD severity score., Discussion: The final 8-item mRESQ-eD is a reliable and valid instrument with good psychometric properties for use in clinical trials in patients with persistent GERD. The mRESQ-eD may be considered for inclusion in clinical trials for persistent GERD and potentially positioned, in consultation with Food and Drug Administration, as endpoints to characterize treatment benefit.

Objectives: Collection of patient-reported outcome (PRO) measures is critical to fully understand chronic obstructive pulmonary disease (COPD) management and progression, as the impact on health-related quality of life is not well understood by objective measures alone. Electronic PROs (ePROs) are increasingly used because of their advantages over paper data collection, including elimination of transcription errors, increased accuracy and data quality, real-time data reporting, and increased compliance. The objective of this study was to characterize how patients with COPD prefer to use various types of technology to report disease symptoms, and their preferences for ePRO design and display., Methods: The sample consisted of subjects with COPD (N = 103) who completed in-person surveys on their ePRO preferences., Results: The majority of subjects prefer to use a form of electronic media over paper to report their disease symptoms. Of these electronic methods, subjects most often prefer to use a smartphone provided by their physician. Subjects were also interested in ePRO features, such as knowing estimated PRO completion time at the outset, tracking their progress in real time as they complete a questionnaire, seeing the data that they report in order to track their health status, being encouraged to complete their diary if they fall behind by positive messaging, and being thanked for their completion of a daily diary., Conclusions: Investigators should consider including these preferences when designing ePRO assessments. Incorporating patient preferences for ePRO design can ultimately help reduce patient burden and increase engagement, compliance, and improve data quality.

Biosimilar filgrastims are primarily indicated for chemotherapy-induced neutropenia prevention. They are less expensive formulations of branded filgrastim, and biosimilar filgrastim was the first biosimilar oncology drug administered in European Union (EU) countries, Japan, and the U.S. Fourteen biosimilar filgrastims have been marketed in EU countries, Japan, the U.S., and Canada since 2008, 2012, 2015, and 2016, respectively. We reviewed experiences and policies for biosimilar filgrastim markets in EU countries and Japan, where uptake has been rapid, and in the U.S. and Canada, where experience is rapidly emerging. U.S. regulations for designating biosimilar interchangeability are under development, and such regulations have not been developed in most other countries. Pharmaceutical substitution is allowed for new filgrastim starts in some EU countries and in Canada, but not Japan and the U.S. In EU countries, biosimilar adoption is facilitated with favorable hospital tender offers. U.S. adoption is reportedly 24%, while the second filgrastim biosimilar is priced 30% lower than branded filgrastim and 20% lower than the first biosimilar filgrastim approved by the U.S. Food and Drug Administration. Utilization is about 60% in EU countries, where biosimilar filgrastim is marketed at a 30%-40% discount. In Japan, biosimilar filgrastim utilization is 45%, primarily because of 35% discounts negotiated by Central Insurance and hospital-only markets. Overall, biosimilar filgrastim adoption barriers are small in many EU countries and Japan and are diminishing in Canada in the U.S. Policies facilitating improved U.S. adoption of biosimilar filgrastim, based on positive experiences in EU countries and Japan, including favorable insurance coverage; larger price discount relative to reference filgrastim pricing; closing of the "rebate trap" with transparent pricing information; formal educational efforts of patients, physicians, caregivers, and providers; and allowance of pharmaceutical substitution of biosimilar versus reference filgrastim, should be considered. IMPLICATIONS FOR PRACTICE: We reviewed experiences and policies for biosimilar filgrastims in Europe, Japan, Canada, and the U.S. Postmarketing harmonization of regulatory policies for biosimilar filgrastims has not occurred. Acceptance of biosimilar filgrastims for branded filgrastim, increasing in the U.S. and in Canada, is commonplace in Japan and Europe. In the U.S., some factors, accepted in Europe or Japan, could improve uptake, including acceptance of biosimilars as safe and effective; larger cost savings, decreasing "rebate traps" where pharmaceutical benefit managers support branded filgrastim, decreased use of patent litigation/challenges, and allowing pharmacists to routinely substitute biosimilar for branded filgrastim., Competing Interests: Disclosures of potential conflicts of interest may be found at the end of this article., (© AlphaMed Press 2019.)

Objectives: This study explored how signs and symptoms of pyruvate kinase (PK) deficiency, a rare hemolytic anemia caused by mutations in the PKLR gene, impacts patients' health-related quality of life (HRQoL)., Methods: Interviews with 21 adults with PK deficiency in the United States, Netherlands, and Germany were conducted. Participants were asked to describe signs, symptoms, and impacts of the disease on their daily lives. Interviews were transcribed and analyzed using qualitative analysis methods., Results: The most common signs and symptoms reported were yellow eyes (n = 19), tiredness (n = 18), yellow skin (n = 17), fatigue (n = 15), low energy (n = 13), and shortness of breath (n = 13). Furthermore, signs and symptoms of PK deficiency negatively impact the ability to perform physical activities, appearance, social activities, emotional states, activities of daily living, leisure activities, work and/or school, sleep, and cognitive states of those living with PK deficiency. A conceptual model is presented that further demonstrates the profound impact that signs and symptoms of PK deficiency have on dimensions of patients' HRQoL., Conclusions: This is the first study that provides patient perspective on the burden of living with PK deficiency and lays the foundation for future studies to examine the effect of pharmacologic interventions on overall HRQoL for patients living with PK deficiency., (© 2018 The Authors. European Journal of Haematology Published by John Wiley & Sons Ltd.)

Patients with increased intracranial pressure generally require pharmacologic therapies and often more definitive treatments, such as surgical intervention. The overall goal of these interventions is to maintain or re-establish adequate cerebral blood flow and prevent herniation. Regardless of the cause of increased intracranial pressure, osmotherapy is considered the mainstay of medical therapy, and should be administered as soon as possible. This article reviews the history of hyperosmolar and hypertonic therapies, the Monro-Kellie hypothesis, and types of cerebral edema. Pharmacologic properties, clinical applications, complications, recommended monitoring during therapy, and risks versus benefits are also discussed., (Copyright © 2017 Elsevier Inc. All rights reserved.)

Ultrasonography is a first-line diagnostic tool when evaluating volume status in the critical care patient population. Ultrasonography leads to a prompt diagnosis and more appropriate management plan, while decreasing health care costs, time to diagnosis, hospital length of stay, time to definitive operation, and mortality. It is recommended that critical care providers treating critically ill patients be skilled and competent in critical care ultrasonography. As the critical care population and the shortage of critical care physicians increases, advanced practice providers are becoming more prevalent in critical care areas and should be competent in this skill as well., (Copyright © 2017 Elsevier Inc. All rights reserved.)

Background: Systems to meet the on-demand learning needs of nurses in intensive care units are not well studied beyond the traditional classroom models., Objective: To study the feasibility and effect of implementing an online discussion forum for nurses in a busy neuroscience intensive care unit., Methods: A baseline survey was done to highlight the areas of educational need in the unit. Freeform-a password-protected, online discussion forum supported by the university-was used for the pilot project. Freeform has functions similar to Facebook, with "likes," "follow," discussion/comment spaces, and the capacity for uploading images and files. A page called "All things NeuroCritical Care" was created. All nurses working in the intensive care unit were automatically enrolled. Clinical vignettes relevant to neurocritical care were posted once a month with 1 to 2 lead questions. All participation was voluntary, and topics were chosen on the basis of the needs survey. At the end of each case, a recent review article on the topic was posted for secure download., Results: Eight sentinel diagnoses have been presented as clinical vignettes, and 34 of 76 members formally follow the page. The mean number of discussion strings per case is 8.3 posts. The number of unique visitors to the page during active case discussions exceeds 100., Conclusion: A secure, online, problem-based learning discussion format is a feasible point-of-care learning opportunity that can help overcome some of the traditional barriers to ongoing nursing education needs in a busy intensive care unit., (©2016 American Association of Critical-Care Nurses.)

Purpose: To describe three commercial weight loss programs (Jenny Craig, LA Weight Loss, and Weight Watchers), including efficacy data when available, and to provide clinicians with information for patient counseling., Data Sources: Personal interviews, journal articles, and Web site information., Conclusions: Commercial weight loss programs provide weight loss services. Benefits are experience, planned menus, and psychological support. Limitations are cost, sales promotions that encourage on-the-spot commitment to prepaid contracts, and the cost of program food and additional vitamins., Implications for Practice: Weight loss is a necessary goal for many patients. Clinicians are mandated to provide counseling regarding weight loss.

This study compared scores on future time perspective of 50 female prison inmates who were participating in a vocational training program within the Julia Tutwiler Prison. There was no control group. Zimbardo's measure of future time perspective, administered a year earlier, was associated with success or failure in the program. Associations of future time perspectives with education, age, and ethnicity were investigated. An analysis of covariance between the 21 graduates and 16 dropouts while controlling for education was significant. Despite the incomplete design, some implications are of interest.

Increased attention has been given to cytologic and histopathologic technic in the detection of endometrial lesions because of an increasing incidence of endometrial malignancy. The cervical-vaginal smear as performed by most clinicians is inadequate for the detection of endometrial neoplasia. Existing technics for the detection of endometrial lesions are costly and time consuming. An effective, reliable and economical technic has been developed for rapid smearing and fixation of endometrial cells. The routine endometrial screening of 739 patients (1143 specimens) using the Milan-Markley technic detected 4 cases of endometrial carcinoma, 1 case of carcinoma of the tube, 22 cases of endometrial hyperplasia, and 9 of endometrial polypi. It is hoped that this cytologic method will complement existing histologic detection procedures.

Fine-needle aspiration of solid tumors other than cervical neoplasms has, until recently, been held in low esteem by US physicians. Its disfavor is undeserved, contend the authors of this article, who base their opinion on the findings from 545 aspirations done over a two-year period. They assert that in experienced hands, in the appropriate setting, and with discriminate use, this diagnostic technique yields accurate results safely and rapidly.