Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ).

OBJECTIVES: Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia. This study aimed to identify disease-related symptoms and impacts important to patients with CAD, and to develop a novel CAD-specific patient-reported outcome measure. METHODS: Adults with CAD were randomly selected from a United States patient panel to participate in concept elicitation (CE) interviews to identify important symptoms and impacts or cognitive debriefing (CD) interviews to assess the comprehension and relevance of the draft item set. RESULTS: Overall, 37 adults were included (mean [range] age 67.2 [35-87] years). In CE interviews (n = 16), the most frequently reported CAD-related symptoms were reactions to cold environments and fatigue (both 93.8%). CAD had negative impacts on enjoyable activities (81.3%) and daily activities (75.0%). Following CE, standard survey methodological principles were used to develop a draft item pool of 14 concepts. Items were refined through three iterative rounds of CD interviews (n = 21), yielding 11 final items: fatigue; cold sensitivity; dyspnea; wearing extra clothing; limited physical, social, and enjoyable activities; difficulty with usual activities; mood; frustration; and anxiety/stress. CONCLUSIONS: The novel 11-item CAD-Symptoms and Impact Questionnaire provides a measure of the symptoms and impacts of CAD that are important to patients.

Assessing What Matters to People Affected by Alzheimer's Disease: A Quantitative Analysis.

INTRODUCTION: In this phase of the ongoing What Matters Most study series, designed to evaluate concepts that are meaningful to people affected by Alzheimer's disease (AD), we quantified the importance of symptoms, impacts, and outcomes of AD to people at risk for or with AD and care partners of people with AD. METHODS: We administered a web-based survey to individuals at risk for or with AD (Group 1: unimpaired cognition with evidence of AD pathology; Group 2: AD risk factors and subjective cognitive complaints/mild cognitive impairment; Group 3: mild AD) and to care partners of individuals with moderate AD (Group 4) or severe AD (Group 5). Respondents rated the importance of 42 symptoms, impacts, and outcomes on a scale ranging from 1 ("not at all important") to 5 ("extremely important"). RESULTS: Among the 274 respondents (70.4% female; 63.1% white), over half of patient respondents rated all 42 items as "very important" or "extremely important," while care partners rated fewer items as "very important" or "extremely important." Among the three patient groups, the minimum (maximum) mean importance rating for any item was 3.4 (4.6), indicating that all items were at least moderately to very important. Among care partners of people with moderate or severe AD, the minimum (maximum) mean importance rating was 2.1 (4.4), indicating that most items were rated as at least moderately important. Overall, taking medications correctly, not feeling down or depressed, and staying safe had the highest importance ratings among both patients and care partners, regardless of AD phase. CONCLUSION: Concepts of importance to individuals affected by AD go beyond the common understanding of "cognition" or "function" alone, reflecting a desire to maintain independence, overall physical and mental health, emotional well-being, and safety. Preservation of these attributes may be key to understanding whether interventions deliver clinically meaningful outcomes.

Technical Review of Clinical Outcomes Assessments Across the Continuum of Alzheimer's Disease.

INTRODUCTION: Insight into the relationship between concepts that matter to the people affected by Alzheimer's disease (AD) and the clinical outcome assessments (COAs) commonly used in AD clinical studies is limited. Phases 1 and 2 of the What Matters Most (WMM) study series identified and quantitatively confirmed 42 treatment-related outcomes that are important to people affected by AD. METHODS: We compared WMM concepts rated as "very important" or higher to items included in COAs used commonly in AD studies. RESULTS: Twenty COAs designed to assess signs, symptoms, and impacts across the spectrum of AD were selected for review. Among these 20 COAs, only 5 reflected 12 or more WMM concepts [Integrated Alzheimer's Disease Rating Scale (iADRS), Alzheimer's Disease Cooperative Study-Activities of Daily Living Inventory (ADCS-ADL), Alzheimer's Disease Cooperative Study-Activities of Daily Living Inventory-Mild Cognitive Impairment (ADCS-ADL-MCI), Alzheimer's Disease Composite Scores (ADCOMS), and Clinical Dementia Rating; Clinical Dementia Rating-Sum of Boxes (CDR/CDR-SB)]. Multiple symptoms and impacts of AD identified as important and meaningful in the WMM studies map only indirectly at best to 7 of the 20 most widely used COAs. CONCLUSION: While many frequently used COAs in AD capture some concepts identified as important to AD populations and their care partners, overlap between any single measure and the concepts that matter to people affected by AD is limited. The highest singly matched COA reflects fewer than half (45%) of WMM concepts. Use of multiple COAs expands coverage of meaningful concepts. Future research should explore the content validity of AD COAs planned for AD trials based on further confirmation of the ecological validity of the WMM items. This research should inform development and use of core outcome sets that capture WMM items and selection or development of new companion tools to fully demonstrate clinically meaningful outcomes spanning WMM.

Practice patterns and perspectives regarding treatment for symptoms of menopause: qualitative interviews with US health care providers.

OBJECTIVE: To document health care providers' views regarding treatments for symptoms associated with menopause and discussions with patients about symptoms and treatment decisions. Results informed development of a data collection form for a retrospective medical record review (reported separately). METHODS: Registered US gynecologists or primary care providers from all US regions were identified from local association directories and an in-house database and were invited to participate in a qualitative interview if they consulted with three or more patients per week presenting with menopausal symptoms. Participants provided demographic data, information about patients' symptoms, and health care provider and patient views on prescription and nonprescription therapies. Key concepts/themes from interviews were identified. RESULTS: Participating health care providers (10 gynecologists, 10 primary care providers) agreed there are effective treatment options for menopausal symptoms, particularly vasomotor symptoms and vaginal dryness and/or atrophy. Health care providers reported that treatment was generally dictated by symptoms that interfered with quality of life and/or daily activities, although patients often had symptoms for months before presentation. All health care providers said they prescribe hormone and/or nonhormone therapies for treatment of menopausal symptoms; half stated that they typically inquire about patients' nonprescription therapy use, and 45% recommend specific nonprescription therapies. The most commonly cited barriers to initiation of any therapy for menopausal symptoms were patient concerns about risks and financial considerations (ie, insurance or cost). CONCLUSIONS: US health care providers reported prescribing therapies for menopausal symptoms and noted that these therapies were perceived as generally effective; however, barriers to initiation of prescription therapy exist, and new treatment options are needed.

Assessing what matters most to patients with or at risk for Alzheimer's and care partners: a qualitative study evaluating symptoms, impacts, and outcomes.

BACKGROUND: The What Matters Most (WMM) study was initiated to evaluate symptoms, AD-related impacts, treatment-related needs, preferences, and priorities among individuals with or at risk for Alzheimer's disease (AD) and their care partners. The objective of this qualitative study phase was to identify a comprehensive set of concepts of interest that are meaningful to individuals across the AD continuum. METHODS: Interviews were conducted with 60 clinically referred individuals and care partners across 5 AD stages (n = 12 each): group 1 (non-clinically impaired individuals with AD pathology), group 2 (individuals with mild cognitive impairment and AD pathology), group 3 (individuals with mild AD), group 4 (individuals with moderate AD and their care partners), and group 5 (care partners of individuals with severe AD). Interviews were conducted by experienced interviewers, audio-recorded, and transcribed. Dominant trends were identified in each interview and compared across subsequent interviews to generate themes or patterns in descriptions of AD symptoms, impacts, and desired treatment outcomes. RESULTS: All participants endorsed current issues related to memory; nearly all participants (n = 55; 92%) across the five groups endorsed symptoms related to communication and language. Groups 1-3 reported an impact on mood/emotions (n = 23; 64%) and a decrease in social activities or outgoingness (n = 17; 47%). Current and future concerns reported by the overall sample included memory (n = 48; 80%), dependence (n = 40; 67%), and "other" concerns (n = 33; 55.0%) (e.g., uncertainty about the future, burdening others). The most desired AD treatment outcomes were improvement or restoration of memory (n = 40; 67%) and stopping AD progression (n = 35; 58.3%). Group-level differences were observed in the symptoms, impacts, and desired treatment outcomes among patients and care partners across the AD continuum. CONCLUSIONS: Cognitive functioning issues-particularly in memory and communication-are present even in preclinical and early-stage AD, including among those without a formal AD diagnosis. While the impacts of AD vary across the disease-severity spectrum, improved memory and disease modification were treatment outcomes considered most important to participants across all 5 AD stages. Neuropsychological assessments traditionally used in AD clinical trials may not evaluate the often-subtle concepts that are important to patients and care partners. Results from this study will inform the second phase of the WMM project-a quantitative study to elicit the relative importance of these concepts of interest to people at risk for and living with AD and their care partners.

Development of the Painful Periods Screening Tool for endometriosis.

OBJECTIVES: Diagnostic delay is common in endometriosis. There is an unmet need for a symptom-based, patient-completed screening tool to facilitate discussions between patients and physicians about potential endometriosis symptoms. The objective of this study was to develop and assess the patient-completed Painful Periods Screening Tool (PPST) to assess the presence of potential endometriosis symptoms. METHODS: To develop and refine the PPST, a cross-sectional qualitative study was conducted with women with endometriosis and healthy controls. Following identification of potentially relevant concepts in the literature and input from clinical experts, a draft version of the PPST was tested during in-depth individual interviews with 16 women: 11 with endometriosis and 5 healthy controls. RESULTS: The six draft items of the PPST were refined iteratively in two rounds of interviews, and one item was deleted following the second set of interviews. All concepts included in the final five-item PPST were found to be relevant to women with endometriosis, and all 11 participants with endometriosis endorsed at least one of the items. No core symptoms of endometriosis were noted as missing from the PPST. CONCLUSION: The PPST assesses the most important endometriosis-related symptoms and may help facilitate discussions between patients and physicians, promoting earlier diagnosis and treatment of endometriosis.

Experiences, attitudes, and perceptions of caregivers of individuals with treatment-resistant schizophrenia: a qualitative study.

BACKGROUND: Treatment-resistant schizophrenia (TRS) affects about one-third of individuals with schizophrenia. People with TRS do not experience sustained symptom relief and at the same time have the most severe disease-related disability and associated costs among individuals with severe mental disorders. Like caregivers of people with treatment-responsive schizophrenia, caregivers of individuals with TRS experience the disease burden along with their care recipients; however, for those providing care for individuals with TRS, the stress of the burden is unrelenting due to uncontrolled symptoms and a lack of effective treatment options. The objective of this study is to better understand the burden of TRS from the caregiver perspective and to explore their perception of available treatments. METHODS: Eight focus groups with non-professional, informal caregivers of individuals with TRS were conducted in 5 US locations. TRS was defined as failure of ≥2 antipsychotics and persistent moderate-to-severe positive symptoms of schizophrenia, per caregiver report. RESULTS: The 27 caregivers reported an average of 37 h/week providing direct care, and 21 reported being on call "24/7." Caregivers commonly reported that their care recipients exhibited symptoms of auditory hallucinations (89%), agitation/irritability/hostility (81%), suspiciousness (78%), tangentiality (74%), and cognitive impairment (74%); 70% of caregivers ranked suspiciousness/persecution as the most challenging symptom category. Caring for an individual with TRS impacted many caregivers' finances, career prospects, social relationships, and sense of freedom. Additionally, multiple medication failures led to a sense of hopelessness for many caregivers. CONCLUSIONS: Persistent positive symptoms caused significant perceived burden, feelings of being overwhelmed and having no relief, and substantial negative impacts on caregivers' emotional and physical health. To address these substantial unmet needs, policy makers should be aware of the need for practical, social, and emotional support for these caregivers and their families. Additionally, new treatment options for TRS should be developed.

Screening and treatment for short cervical length in pregnancy: a physician survey in the United States.

PURPOSE: To evaluate how physicians in the United States (US) screen for, define, and treat a short cervix to prevent preterm birth. METHODS: This was a cross-sectional, web-based survey of 500 physicians treating pregnant patients with a short cervix in the US. Respondents' geographic region was monitored to ensure balance across the nine US Census divisions. RESULTS: Respondents were predominantly obstetrician/gynecologists (86%, 429/500; mean age 49 years). Physicians reported that a median of 90% of their pregnant patients undergo cervical length screening; 81% (407/500) use transvaginal ultrasound. Physicians consult multiple evidence sources to inform their patient care, most commonly clinical guidelines (83%; 413/500) and published research (70%; 349/500). Most physicians (98%; 490/500) reported treating pregnant patients with a short cervix; 95% (474/500) use synthetic and/or natural progestogen, alone or in combination with other treatment modalities. If reimbursement was not a concern, 47% of physicians (230/500) would choose vaginal progesterone as their preferred treatment to prevent preterm birth in all patients with a short cervix, and 45% (218/500) would choose a synthetic progestogen. CONCLUSION: US guidelines recommend transvaginal ultrasound for cervical length screening; 81% of physicians in this study reported using this method. Most physicians surveyed use progestogens to treat a short cervix, with approximately half choosing a synthetic progestin (45%) and half choosing natural progesterone (47%) as their preferred treatment, despite national guidelines recommending only vaginal natural progesterone for this indication. Additional physician education is required to implement current and best practices.

Understanding the Patient Experience with Carcinoid Syndrome: Exit Interviews from a Randomized, Placebo-controlled Study of Telotristat Ethyl.

PURPOSE: Telotristat ethyl, an oral tryptophan hydroxylase inhibitor, is intended to treat carcinoid syndrome by reducing serotonin production. Telotristat ethyl was evaluated in TELESTAR, a Phase III study for patients who had carcinoid syndrome with at least 4 bowel movements (BMs) per day and who were receiving somatostatin analogue therapy. This interview substudy was conducted to provide insight into the patient experience in TELESTAR and to help understand whether reductions in BM frequency (the primary end point) and other symptoms were clinically meaningful. METHODS: Participating sites were asked to invite (before randomization) all eligible patients to telephone interviews scheduled at the end of the double-blind treatment period. Patients and interviewers were blinded to treatment. FINDINGS: All 35 interviewed participants reported diarrhea and/or excessive BMs at baseline. Patients reported that these symptoms negatively affected emotional, social, physical, and occupational well-being. Prespecified criteria for treatment response (achieving ≥30% reduction in BM frequency for at least 50% of the days) were met by 8 of 26 patients taking telotristat ethyl and 1 of 9 patients taking placebo. All 8 patients taking telotristat ethyl described clinically meaningful reductions in BM frequency and were very satisfied with the ability of the study drug to control their carcinoid syndrome symptoms. Overall, reports of being very satisfied were observed in 12 patients taking telotristat ethyl and 0 taking placebo. IMPLICATIONS: Patient interviews revealed that TELESTAR patients, at baseline, were significantly affected by their high BM frequency. Patient reports of their clinical trial experience supported the significance of the primary end point and clinical responder analysis in TELESTAR, helping identify and understand clinically meaningful change produced by telotristat ethyl.

Use and Value of the 7-Item Binge Eating Disorder Screener in Clinical Practice.

OBJECTIVE: To evaluate physician knowledge of and attitudes about binge-eating disorder (BED) and the value and ease-of-use of the 7-item Binge Eating Disorder Screener (BEDS-7) in clinical practice. METHODS: Two internet surveys (wave 1: April 15-May 6, 2015; wave 2: August 19-25, 2015) were administered to primary care physicians serving adults (PCPs-adults) and psychiatrists. Wave 1 invitees were US-based physicians spending ≥ 50% of their time in direct patient care and reporting "no" to "some to average" experience with eating-disorder patients. Respondents completing wave 1 qualified for wave 2. RESULTS: Among the 1,047 physicians who responded, 313 did not meet at least 1 of the screening criteria, including 3.15% of respondents who spent < 50% of their time in direct patient care. Overall, 122 PCPs-adults and 123 psychiatrists completed both waves. Physician groups spent similar mean ± SD amounts of time providing direct patient care (PCPs-adults: 94.66% ± 8.4%, psychiatrists: 91.15% ± 12.2%). Based on composite scores, BED knowledge increased from wave 1 to wave 2 in PCPs-adults (P < .001) and psychiatrists (P < .05). Composite scores pertaining to knowledge of and comfort with diagnosing and treating BED were lower for PCPs-adults than psychiatrists in both waves (all P < .001). Based on wave 2 responses, the BEDS-7 was used by 32.0% of PCPs-adults and 26.8% of psychiatrists. All BEDS-7 users (100%) indicated the screener was "very" or "somewhat" valuable, and nearly all users (psychiatrists: 100%, PCPs-adults: 97.4%) reported it was "very" or "reasonably" easy to use. BEDS-7 users reported that important uses of the screener included assisting clinicians in identifying BED patients and encouraging/initiating doctor-patient discussions about BED. CONCLUSIONS: These results support the utility of the BEDS-7 in clinical practice, with BEDS-7 users reporting that it is a highly valued and easy-to-use screener. Furthermore, both PCPs-adults and psychiatrists acknowledged the importance of being knowledgeable about BED.

Patient-completed or symptom-based screening tools for endometriosis: a scoping review.

PURPOSE: The objective of this review was to evaluate existing patient-completed screening questionnaires and/or symptom-based predictive models with respect to their potential for use as screening tools for endometriosis in adult women. Validated instruments were of particular interest. METHODS: We conducted structured searches of PubMed and targeted searches of the gray literature to identify studies reporting on screening instruments used in endometriosis. Studies were screened according to inclusion and exclusion criteria that followed the PICOS (population, intervention, comparison, outcomes, study design) framework. RESULTS: A total of 16 studies were identified, of which 10 described measures for endometriosis in general, 2 described measures for endometriosis at specific sites, and 4 described measures for deep-infiltrating endometriosis. Only 1 study evaluated a questionnaire that was solely patient-completed. Most measures required physician, imaging, or laboratory assessments in addition to patient-completed questionnaires, and several measures relied on complex scoring. Validation for use as a screening tool in adult women with potential endometriosis was lacking in all studies, as most studies focused on diagnosis versus screening. CONCLUSIONS: This literature review did not identify any fully validated, symptom-based, patient-reported questionnaires for endometriosis screening in adult women.

Assessing the burden of treatment-emergent adverse events associated with atypical antipsychotic medications.

BACKGROUND: Treatment of schizophrenia and major depressive disorder (MDD) with atypical antipsychotics (AAPs) show improved efficacy and reduced side effect burden compared with older antipsychotic medications. However, a risk of treatment-emergent adverse events (TEAEs) remains. TEAEs are hard to quantify and perspectives on the importance of TEAEs differ across patients and between patients and physicians. The current study is a qualitative assessment that investigates TEAEs of AAPs from both patient and physician perspectives to provide better understanding of the occurrence and burden of TEAEs associated with these medications. METHODS: Focus groups comprised of patients with MDD and interviews with patients with schizophrenia were conducted at two qualitative research facilities, along with a physician focus group at one of the facilities. Information collected from patients included an exhaustive list of TEAEs experienced, and the frequency and level of bother of each TEAE; from psychiatrists, information included an exhaustive list of TEAEs based on personal observations and patient report, frequency of TEAEs, clinically important TEAEs, and levels of patient-perceived bother. Standard qualitative analysis methods were used to identify, quantify, characterize, and summarize patterns found in the data collected. RESULTS: A total of 42 patients (25 with MDD and 17 with schizophrenia) and 4 psychiatrists participated in the study. TEAEs reported as bothersome across both patients groups included cognitive issues, weight gain and/or increased appetite, low energy, extrapyramidal symptoms (EPS), and need to sleep/excessive sleep/excessive sleepiness. TEAEs considered more bothersome by patients with schizophrenia were weight gain, low energy, EPS, mental anxiety, and increased positive symptoms; those considered more bothersome by patients with MDD were cognitive issues, somnolence/sedation, and flat/restricted affect. TEAEs considered most clinically important by psychiatrists included metabolic syndrome, weight gain, neutropenia, hyperglycemia, and QT prolongation; those TEAEs considered most bothersome to patients from physicians' perspectives included weight gain, reduced sexual desire or performance, EPS, akathisia, and hormonal issues. CONCLUSIONS: The wide range of TEAEs that are both frequent and bothersome and the variation in perceived burden according to diagnosis highlight the need for a tailored TEAE-awareness approach when choosing an AAP.

Development of the 7-Item Binge-Eating Disorder Screener (BEDS-7).

OBJECTIVE: Develop a brief, patient-reported screening tool designed to identify individuals with probable binge-eating disorder (BED) for further evaluation or referral to specialists. METHODS: Items were developed on the basis of the DSM-5 diagnostic criteria, existing tools, and input from 3 clinical experts (January 2014). Items were then refined in cognitive debriefing interviews with participants self-reporting BED characteristics (March 2014) and piloted in a multisite, cross-sectional, prospective, noninterventional study consisting of a semistructured diagnostic interview (to diagnose BED) and administration of the pilot Binge-Eating Disorder Screener (BEDS), Binge Eating Scale (BES), and RAND 36-Item Short-Form Health Survey (RAND-36) (June 2014-July 2014). The sensitivity and specificity of classification algorithms (formed from the pilot BEDS item-level responses) in predicting BED diagnosis were evaluated. The final algorithm was selected to minimize false negatives and false positives, while utilizing the fewest number of BEDS items. RESULTS: Starting with the initial BEDS item pool (20 items), the 13-item pilot BEDS resulted from the cognitive debriefing interviews (n = 13). Of the 97 participants in the noninterventional study, 16 were diagnosed with BED (10/62 female, 16%; 6/35 male, 17%). Seven BEDS items (BEDS-7) yielded 100% sensitivity and 38.7% specificity. Participants correctly identified (true positives) had poorer BES scores and RAND-36 scores than participants identified as true negatives. CONCLUSIONS: Implementation of the brief, patient-reported BEDS-7 in real-world clinical practice is expected to promote better understanding of BED characteristics and help physicians identify patients who may have BED.

Patient-centered assessment of cognitive symptoms of depression.

OBJECTIVE: To identify and explore concepts important to patients with cognitive symptoms of major depressive disorder (MDD) and adapt an existing patient-reported outcome (PRO) measure to assess these symptoms. METHODS: Four focus groups were conducted with MDD patients (n = 33) to elicit relevant concepts and determine whether one of several PRO scales could be used to assess cognitive symptoms of depression. Following selection and minor modification of the Perceived Deficits Questionnaire (PDQ), cognitive debriefing interviews were conducted with additional patients (n = 17) to further refine and adapt this measure for use in MDD. Minor revisions based on patient input yielded the PDQ for Depression (PDQ-D). RESULTS: Focus group participants reported a variety of cognitive symptoms that were classified into 7 general categories: lack of focus and clear thought, memory problems, difficulty with lexical access, difficulty with divided attention, difficulty with decision making, difficulty thinking quickly, and difficulty learning new things. Limitations in work productivity were the most commonly reported impacts of cognitive symptoms. While suggesting a few modifications, focus group participants reacted positively to the PDQ based on the breadth, specificity, and relevance of the items. Cognitive debriefing participants indicated that the modified PDQ items were generally easy to understand and relevant to their experiences with MDD. CONCLUSION: Because cognitive symptoms are burdensome to patients with MDD, their assessment is important to optimize treatment outcomes. The PDQ-D has the potential to supplement existing assessment methods, providing unique information important for both comprehensive evaluation of individuals with MDD and evaluation of new treatments.

Development and validation of the ADHD Benefits of Coaching Scale (ABCS).

OBJECTIVE: Develop and validate the ADHD Benefits of Coaching Scale (ABCS), a self-report instrument designed to measure benefits of ADHD coaching in college students. METHOD: Concepts were elicited from interviews with ADHD coaches and college students receiving ADHD coaching. Draft items were tested in cognitive interviews among ADHD students. Item and scale reliability and validity of ABCS were evaluated using web-survey data from students reporting ADHD. Mean ABCS scores for coached and noncoached groups were compared. RESULTS: Five coaches and 14 students participated in concept elicitation interviews; 10 students participated in the cognitive interviews; 209 students completed the web-survey. The 10-item ABCS showed satisfactory internal consistency reliability and validity and differentiated between coached (n = 17) and noncoached groups (n = 192; p < .01). CONCLUSION: To assess benefits of ADHD coaching in college students, the ABCS was developed and psychometrically validated in accordance with Food and Drug Administration's (FDA) patient-reported outcome guidance.

Assessing Methotrexate Adherence in Rheumatoid Arthritis: A Cross-Sectional Survey.

INTRODUCTION: Limited data are available to explain nonadherence to methotrexate (MTX) therapy in patients with rheumatoid arthritis (RA). Better understanding of patterns of MTX use and reasons for nonadherence may help identify patients who would benefit from alternative RA treatments and potentially aid in developing strategies to increase overall adherence. The purpose of this study was to assess patients' self-reported adherence to MTX and to identify reasons for nonadherence. METHODS: Patient panel members in the US self-reporting a diagnosis of RA of ≥3 months' and current MTX use of ≥4 weeks' duration, with or without concomitant use of another RA prescription medication, participated in this cross-sectional, web-based survey. RESULTS: The sample population (251 MTX monotherapy, 250 MTX combination therapy) was predominantly female, white, non-Hispanic, and educated; 48% were 18-44 years-old, 47% had medical comorbidities, 66% were first diagnosed with RA ≤5 years earlier, 51% reported MTX use of <1 year, and 83% reported oral MTX use. Forty-two percent reported not taking MTX exactly as prescribed. Reasons for nonadherence included forgetting to take it (33%), not needing it when feeling well (24%), and concern about long-term safety (24%). Among nonadherent patients, 53% took smaller doses, 52% skipped doses, and 6% reported other nonprescribed ways of taking MTX. Younger age, male sex, and shorter duration of MTX use were associated with poorer self-reported adherence. Compared with monotherapy patients, combination therapy patients, particularly those taking ≥2 other RA prescriptions, were less likely to report high adherence. CONCLUSION: Nearly half the sample reported poor MTX adherence because they forgot to take it, thought it was not needed when they felt well, or had long-term safety concerns. Patients taking ≥2 other RA prescription medications were less likely to report good adherence. Reducing treatment burden without sacrificing efficacy may be a strategy worth evaluating.

Cognitive interviewing in risk minimization survey development: patient and healthcare professional surveys.

Risk minimization programs are often required for selected drugs and other products to ensure that the benefits of these prescription products outweigh their risks. Regulators in the USA and Europe have recently called for more rigorous standards in developing measures for risk minimization program assessment. Cognitive pretesting interviews are a critical step in the development of survey instruments used to evaluate patients' and healthcare professionals' knowledge and behaviors associated with the safe use of products requiring a risk minimization program. This article is intended as a guide for the researcher who is charged with the development of survey instruments used in these programs and focuses on the role of cognitive pretesting interviews in successful survey instrument design, data analysis and interpretation.

Successful implementation of cognitive interviews in special populations.

The cognitive interview, with a focus on debriefing methods, was developed in the 1980s to identify sources of potential response error in surveys or questionnaires. With the release of the final US FDA guidance, titled 'Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims', cognitive interviews have gained importance and relevance both for concept elicitation and debriefing purposes in the context of instrument development. This article is intended as a guide for the researcher working with special populations in methods to foster successful cognitive interviews that meet FDA standards. While many of these techniques are broadly applicable, specific recommendations are provided for working with pediatric and cognitively challenged populations, as well as with individuals with communication difficulties.

Qualitative Development of the Rhinitis Control Assessment Test (RCAT), an Instrument for Evaluating Rhinitis Symptom Control.

BACKGROUND: : Effective management of allergic rhinitis requires ongoing monitoring of its control. This article describes the qualitative phase of development of a patient-completed instrument, the Rhinitis Control Assessment Test (RCAT), designed to assist patients and providers in the detection of problems with rhinitis symptom control. OBJECTIVE: : To identify concepts to be measured and to develop initial questionnaire items to be tested further in the next phase of development. METHODS: : A literature review and input from patient focus groups were utilized to generate concepts to be measured in the draft questionnaire. The draft items were subjected to cognitive testing to ensure that the items were understood by patients and to eliminate ambiguity as well as to select an optimal recall period and meaningful response scales. Patients aged ≥18 years self-reporting a rhinitis diagnosis who had experienced rhinitis symptoms in the past 12 months were eligible for participation in the focus groups (n = 39) and the cognitive interviews (n = 23). RESULTS: : This qualitative process yielded a draft instrument with 26 items assessing five constructs of rhinitis symptom control: frequency and bothersomeness of nasal and non-nasal symptoms, symptom impact, activity interference, symptom control, and medication use. A five-point Likert response scale and a 1-week recall period for each item were adopted based on patient input. CONCLUSION: : The qualitative phase of development of the RCAT produced constructs and items for an instrument that is anticipated to facilitate accurate assessment of rhinitis control and improve the quality of care for patients with allergic rhinitis.