The Perceived Usability of Virtual Visits Among Black Adults' Receiving Oncology Care: A Qualitative Analysis.

BACKGROUND: With the COVID-19 pandemic came rapid uptake in virtual oncology care. During this, sociodemographic inequities in access to virtual visits (VVs) have become apparent. To better understand these issues, we conducted a qualitative study to describe the perceived usability and acceptability of VVs among Black adults diagnosed with cancer. METHODS: Adults who self-identified as Black and had a diagnosis of prostate, multiple myeloma, or head and neck cancer were recruited from 2 academic medical centers, and their community affiliates to participate in a semi-structured interview, regardless of prior VV experience. A patient and family advisory board was formed to inform all components of the study. Interviews were conducted between September 2, 2021 and February 23, 2022. Transcripts were organized topically, and themes and subthemes were determined through iterative and interpretive immersion/crystallization cycles. RESULTS: Of the 49 adults interviewed, 29 (59%) had participated in at least one VV. Three overarching themes were derived: (1) VVs felt comfortable and convenient in the right contexts; (2) the technology required for VVs with video presented new challenges, which were often resolved by an audio-only telephone call; and (3) participants reported preferring in-person visits, citing concerns regarding gaps in nonverbal communication, trusting providers, and distractions during VV. CONCLUSION: While VVs were reported to be acceptable in specific circumstances, Black adults reported preferring in-person care, in part due to a perceived lack of interpersonal connectedness. Nonetheless, retaining reimbursement for audio-only options for VVs is essential to ensure equitable access for those with less technology savvy and/or limited device/internet capabilities.

Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study.

BACKGROUND: Trofinetide was approved for the treatment of Rett syndrome (RTT) in patients aged ≥2 years based on the results of the 12-week, randomized, phase 3 LAVENDER study. In LILAC, a 40-week, open-label extension study of LAVENDER, trofinetide continued to improve the symptoms of RTT, with a similar safety profile as LAVENDER. Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study. METHODS: Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2. Safety and tolerability were assessed with the incidence of adverse events (AEs). Efficacy was assessed with Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scores. Caregiver interviews explored the patient's experience with RTT and the efficacy of trofinetide during study participation. FINDINGS: In total, 77 participants were enrolled in LILAC-2. The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%). The mean (standard error [SE]) change in RSBQ score from LAVENDER baseline to week 104 of LILAC-2 was -11.8 (2.45). The mean (SE) CGI-I score from LILAC baseline to week 12 of LILAC-2 was 3.1 (0.10). Most caregivers (96%; n = 24/25) were satisfied or very satisfied with the benefits of trofinetide. CONCLUSIONS: Long-term treatment with trofinetide continued to improve RTT symptoms, without new safety concerns. Caregivers reported satisfaction with trofinetide related to improvements that were meaningful for their child and themselves. FUNDING: The study was supported by Acadia Pharmaceuticals (San Diego, CA, USA). This study was registered at ClinicalTrials.gov: NCT04776746.

Residential crowding and severe respiratory syncytial virus disease among infants and young children: a systematic literature review.

BACKGROUND: The objective of this literature review was to determine whether crowding in the home is associated with an increased risk of severe respiratory syncytial virus (RSV) disease in children younger than 5 years. METHODS: A computerized literature search of PubMed and EMBASE was conducted on residential crowding as a risk factor for laboratory-confirmed RSV illness in children younger than 5 years. Study populations were stratified by high-risk populations, defined by prematurity, chronic lung disease of prematurity, hemodynamically significant congenital heart disease, or specific at-risk ethnicity (i.e. Alaska Native, Inuit), and mixed-risk populations, including general populations of mostly healthy children. The search was conducted for articles published from January 1, 1985, to October 8, 2009, and was limited to studies reported in English. To avoid indexing bias in the computerized databases, the search included terms for multivariate analysis and risk factors to identify studies in which residential crowding was evaluated but was not significant. Methodological quality of included studies was assessed using a Cochrane risk of bias tool. RESULTS: The search identified 20 relevant studies that were conducted in geographically diverse locations. Among studies of patients in high-risk populations, 7 of 9 found a statistically significant association with a crowding variable; in studies in mixed-risk populations, 9 of 11 found a significant association with a crowding variable. In studies of high-risk children, residential crowding significantly increased the odds of laboratory-confirmed RSV hospitalization (i.e. odds ratio ranged from 1.45 to 2.85). In studies of mixed-risk populations, the adjusted odds ratios ranged from 1.23 to 9.1. The findings on the effect of residential crowding on outpatient RSV lower respiratory tract infection were inconsistent. CONCLUSIONS: Residential crowding was associated with an increased risk of laboratory-confirmed RSV hospitalization among high-risk infants and young children. This association was consistent despite differences in definitions of residential crowding, populations, or geographic locations.

Systematic literature review assessing tobacco smoke exposure as a risk factor for serious respiratory syncytial virus disease among infants and young children.

BACKGROUND: The role of environmental tobacco smoke (ETS) exposure as a risk factor for serious respiratory syncytial virus (RSV) disease among infants and young children has not been clearly established. This systematic review was conducted to explore the association between ETS exposure and serious RSV disease in children younger than 5 years, including infants and young children with elevated risk for serious RSV disease. METHODS: A systematic review of English-language studies using the PubMed and EMBASE databases (1990-2009) was performed to retrieve studies that evaluated ETS as a potential risk factor for serious RSV illness. Studies assessing risk factors associated with hospitalization, emergency department visit, or physician visit due to RSV (based on laboratory confirmation of RSV or clinical diagnosis of RSV) in children under the age of 5 years were included. RESULTS: The literature search identified 30 relevant articles, categorized by laboratory confirmation of RSV infection (n = 14), clinical diagnosis of RSV disease (n = 8), and assessment of RSV disease severity (n = 8). Across these three categories of studies, at least 1 type of ETS exposure was associated with statistically significant increases in risk in multivariate or bivariate analysis, as follows: 12 of 14 studies on risk of hospitalization or ED visit for laboratory-confirmed RSV infection; 6 of 8 studies of RSV disease based on clinical diagnosis; and 5 of the 8 studies assessing severity of RSV as shown by hospitalization rates or degree of hypoxia. Also, 7 of the 30 studies focused on populations of premature infants, and the majority (5 studies) found a significant association between ETS exposure and RSV risk in the multivariate or bivariate analyses. CONCLUSION: We found ample evidence that ETS exposure places infants and young children at increased risk of hospitalization for RSV-attributable lower respiratory tract infection and increases the severity of illness among hospitalized children. Additional evidence is needed regarding the association of ETS exposure and outpatient RSV lower respiratory tract illness. Challenges and potential pitfalls of assessing ETS exposure in children are discussed.

Review and evaluation of patient-centered psychosocial assessments for children with central precocious puberty or early puberty.

The objective of this study was to assess the current use of patient-centered psychosocial assessments for the evaluation of children with central precocious puberty (CPP). Studies evaluating the psychosocial impact of CPP were identified through searches of the PubMed and Cochrane Library databases, ClinicalTrials.gov, a drug prescribing information database, and regulatory websites. Studies were screened using prespecified inclusion and exclusion criteria. Potentially relevant patient-centered outcome assessments (including patient-, parent- or observer-reported measures) used in the identified studies were evaluated in detail for their relevance in CPP. Of the 467 studies identified, 15 met the inclusion criteria. Frequently assessed concepts included depression and anxiety, behavior and behavioral problems, body image and self-esteem and personality type/characteristics. Among the assessments used in the identified studies, the Child Behavior Checklist, Pediatric Quality of Life Inventory (PedsQL), SF-10 for Children and Child Health Questionnaire were comprehensively evaluated. The PedsQL showed promise as a patient-centered outcome measure in CPP. Although there is a lack of validated tools measuring psychosocial health and health-related quality of life in patients with CPP, the PedsQL captures issues seen in this patient population and is relatively easy to administer. Further studies using this and other tools in children with CPP are needed.

Payer Perspectives on Patient-Reported Outcomes in Health Care Decision Making: Oncology Examples.

BACKGROUND: Health authorities and payers increasingly recognize the importance of patient perspectives and patient-reported outcomes (PROs) in health care decision making. However, given the broad variety of PRO endpoints included in clinical programs and variations in the timing of PRO data collection and country-specific needs, the role of PRO data in reimbursement decisions requires characterization. OBJECTIVES: To (a) determine the effect of PRO data on market access and reimbursement decisions for oncology products in multiple markets and (b) assess the effect of PRO data collected after clinical progression on payer decision making. METHODS: A 3-part assessment (targeted literature review, qualitative one-on-one interviews, and online survey) was undertaken. Published literature was identified through searches in PubMed/MEDLINE and Embase. In addition, a targeted search was conducted of health technology assessment (HTA) agency websites in the United States, the United Kingdom, France, and Germany. Qualitative one-on-one interviews were conducted with 16 payers from the RTI Health Solutions global advisory panel in 14 markets (Australia, Brazil, France, Germany, Italy, South Korea, Netherlands, Poland, Spain, Sweden, Taiwan, Turkey, the United Kingdom, and the United States [n = 3]). Of the 200 payers and payer advisors from the global advisory panel invited to participate in the online survey, 20 respondents (China, France, Germany, Spain [n = 2], Taiwan, the United Kingdom, and the United States [n = 13]) completed the survey, and 6 respondents (Australia, South Korea, and the United States [n = 4]) partially completed the survey. RESULTS: Reviews of the literature and publicly available HTAs and reimbursement decisions suggested that HTA bodies and payers have varying experience with and confidence in PRO data. Payers participating in the survey indicated that PRO data may be especially influential in oncology compared with other therapeutic areas. Payers surveyed offered little differentiation by cancer type in the importance of PRO data but felt that it was most important to collect PRO data in phase 3 and postmarketing studies. Payers surveyed also anticipated an increasing significance for PRO data over the next 5-10 years. Characteristics of PRO data that maximize influence on payer decision making were reported to be (a) quality, well-controlled, and transparent PRO evidence; (b) psychometric validation of the PRO measure in targeted populations; and (c) publication in peer-reviewed journals. In markets with decentralized health care decision making, PRO data currently have more influence at the local level. Inclusion of PRO data in cancer treatment guidelines is key for centralized markets. Payers surveyed generally considered collecting PRO data postprogression to be useful. Of the 16 interviewees, 11 indicated that it is worthwhile to collect PRO data postprogression and that positive PRO data may support continued therapy at the physician's discretion upon regulatory approval, even in progressive disease. CONCLUSIONS: PRO data may help to differentiate treatments, particularly after clinical progression in oncology. Payers worldwide recognize high-quality PRO data as a key component of their decision-making process and anticipate the growing importance of PRO data in the future. DISCLOSURES: This study and preparation of this article were funded by Novartis Pharmaceuticals. This research was performed under a research contract between RTI Health Solutions and Novartis Pharmaceuticals. Brogan, Hogue, Demuro, and Barrett are employees of RTI Health Solutions. D'Alessio and Bal are employees of Novartis Pharmaceuticals. Study concept and design were contributed by DeMuro, Barrett, Bal, and Hogue. Brogan and Hogue took the lead in data collection, assisted by DeMuro and Bal. Data interpretation was performed by Brogan and Hogue, assisted by the other authors. The manuscript was written by D'Alessio and Brogan, along with the other authors, and revised primarily by Brogan, along with Hogue and assisted by the other authors. The abstract for this article was presented as a research poster at the following meetings: Hogue SL, Brogan A P, De Muro C, D'Alessio D, Bal V. Patient-reported outcomes (PRO) in post-progression oncology: implications in health technology assessments and payer decision making. Poster presented at the ISPOR 18th Annual European Meeting; November 7-11, 2015. Milan, Italy. Hogue SL, Brogan AP, De Muro C, D'Alessio D, Bal V. Influence of patient-reported outcomes (PRO) on market access decisions in markets with centralized healthcare systems. Poster presented at the ISPOR 18th Annual European Meeting; November 7-11, 2015. Milan, Italy. Hogue SL, Brogan AP, De Muro C, Barrett A, D'Alessio D, Bal V. Influence of patient-reported outcomes on market access decisions in decentralized markets (Brazil, Italy, Spain and the United States). Poster presented at the ISPOR 20th Annual Meeting; May 16-20, 2015. Philadelphia PA. Hogue SL, Brogan A P, De Muro C, Barrett A, McLeod L, D'Alessio D, et al. Payer Perspectives of Patient-Reported Outcomes Data: An Online Assessment. Poster presented at the ISOQOL 22nd Annual Meeting; October 21-24, 2015. Vancouver, British Columbia, Canada.

A comprehensive review of nongenetic prognostic and predictive factors influencing the heterogeneity of outcomes in advanced non-small-cell lung cancer.

While there have been advances in treatment options for those with advanced non-small-cell lung cancer, unmet medical needs remain, partly due to the heterogeneity of treatment effect observed among patients. The goals of this literature review were to provide updated information to complement past reviews and to identify a comprehensive set of nongenetic prognostic and predictive baseline factors that may account for heterogeneity of outcomes in advanced non-small-cell lung cancer. A review of the literature between 2000 and 2010 was performed using PubMed, Embase, and Cochrane Library. All relevant studies that met the inclusion criteria were selected and data elements were abstracted. A classification system was developed to evaluate the level of evidence for each study. A total of 54 studies were selected for inclusion. Patient-related factors (eg, performance status, sex, and age) were the most extensively researched nongenetic prognostic factors, followed by disease stage and histology. Moderately researched prognostic factors were weight-related variables and number or site of metastases, and the least studied were comorbidities, previous therapy, smoking status, hemoglobin level, and health-related quality of life/symptom severity. The prognostic factors with the most consistently demonstrated associations with outcomes were performance status, number or site of metastases, previous therapy, smoking status, and health-related quality of life. Of the small number of studies that assessed predictive factors, those that were found to be significantly predictive of outcomes were performance status, age, disease stage, previous therapy, race, smoking status, sex, and histology. These results provide a comprehensive overview of nongenetic prognostic and predictive factors assessed in advanced non-small-cell lung cancer over the last decade. This information can be used to inform the design of future clinical trials by suggesting additional subgroups based on nongenetic factors that may be analyzed to further investigate potential prognostic and predictive factors.

Epidemiology, public health burden, and treatment of diabetic peripheral neuropathic pain: a review.

OBJECTIVE: The literature examining the epidemiology, quality of life burden, cost, and treatment of diabetic peripheral neuropathy pain (DPNP) in U.S. adults was reviewed. DESIGN: A comprehensive computerized literature review of DPNP was conducted using MEDLINE and other databases, which were searched from 1995 through August 2004 using the Medical Subject Headings diabetic neuropathies and pain combined with relevant terms. A supplementary MEDLINE search of clinical trials of pharmacological treatments for DPNP was conducted through July 2005. RESULTS: The search resulted in 321 articles. Several epidemiological studies assessed diabetic peripheral neuropathy among patients with diabetes and reported prevalence rates of 26-47%. No estimates of DPNP prevalence were reported, although one study (N = 2,405) reported that 26.8% of participants with diabetes experienced either pain or tingling. Randomized clinical trials have been conducted of several medications and classes of medication in patients with DPNP, and the U.S. Food and Drug Administration has approved two drugs for DPNP. Several published studies reported that DPNP impairs quality of life. Estimates of the costs of DPNP in the United States were limited. One study estimated average annual pain medication costs of $1,004 per DPNP patient. CONCLUSIONS: This review of DPNP identifies gaps in the literature and highlights the need for further study. The establishment of a consistent definition and diagnostic code for DPNP would improve ability to collect data and understand the impact of DPNP on patients and the health care system. Well-designed, prospective studies are needed to better define the epidemiology and public health burden of DPNP.