Targeted Literature Review Research Articles

Impact of HIV-Related Stigma on Antiretroviral Therapy Adherence, Engagement and Retention in HIV Care, and Transition to Adult HIV Care in Pediatric and Young Adult Populations Living With HIV: A Literature Review.

HIV-related stigma is associated with negative effects on mental health and lower health-related quality of life in pediatric and young adult populations living with HIV. We reviewed literature on the impact of HIV-related stigma on suboptimal antiretroviral therapy (ART) adherence, engagement and retention in HIV care, and transition to adult HIV care in children, adolescents, and young adults living with HIV. PubMed and Embase® were searched for publications reporting relevant data published from January 1, 2012, to April 13, 2023. The output was not a systematic review; it was a targeted literature review. Overall, 67 studies were selected for analysis based on pre-specified criteria (eg, quality). Most quantitative studies supported negative associations between HIV-related stigma and ART adherence (n = 8/11), engagement and retention in HIV care (n = 3/4), and transition to adult HIV care (n = 2/3) in pediatric and young adult populations living with HIV. Qualitative studies reported that stigma was a barrier to ART adherence (n = 26), engagement and retention in HIV care (n = 18), and transitioning to adult HIV care (n = 11). Prominent interview themes across all topics included anticipated stigma, enacted stigma, and fear of HIV status disclosure. Results reaffirm that HIV-related stigma is a significant barrier to ART adherence, engagement and retention in HIV care, and transition to adult HIV care among pediatric and young adult populations living with HIV, potentially impacting virologic suppression, onward transmission, and longer-term health. Additional interventional studies are needed to evaluate and reduce the impact of stigma in these important populations.

Adverse Impacts of PEGylated Protein Therapeutics: A Targeted Literature Review.

The beneficial effects of polyethylene glycol (PEG)-conjugated therapeutics, such as increased half-life, solubility, stability, and decreased immunogenicity, have been well described. There have been concerns, however, about adverse outcomes with their use, but understanding of those adverse outcomes is still relatively limited. The present study aimed to characterize adverse outcomes associated with PEGylation of protein-based therapeutics on immunogenicity, pharmacologic properties, and safety. A targeted review of English language articles published from 1990 to September 29, 2023, was conducted. Of the 29 studies included in this review, 18 reported adverse safety outcomes such as hematologic complications, hepatic toxicity, injection site reactions, arthralgia, nausea, infections, grade 3 or 4 adverse events (AEs), and AE-related discontinuations and dose modifications. Fifteen studies reported immunogenicity-related outcomes, such as the prevalence of pre-existing antibodies to PEG, treatment-emergent antibody response, and hypersensitivity reactions to PEGylated drugs. Seven studies reported pharmacological outcomes such as increased clearance and reduced activity in response to PEGylated drugs. This review aims to contribute to a balanced view of PEGylated therapies by summarizing the adverse outcomes or lack of benefit associated with PEGylated therapeutics reported in the literature. We identified several studies characterizing adverse outcomes, pharmacological effects, and immunogenicity associated with the use of PEGylated therapeutics. Our findings suggest that using PEGylated therapeutics may require careful monitoring for adverse safety outcomes, including screening and monitoring for pre-existing antibodies and those induced in response to PEGylated therapy, as well as monitoring and adjusting the dosing of PEGylated therapeutics.

Organisational cyber resilience: a heuristic for bridging foundations and applications

PurposeThe drive for digitalisation has increased the scope of cyber threats which can exploit the growing footprint of information and communication technology infrastructure supporting modern societies. Despite substantial interest and efforts in researching and building organisational cyber resilience, the resulting body of work is heterogeneous and has yet to reach maturity. This paper aims to address the gap in the conceptualisation of cyber resilience in academic and practice-oriented grey literature.Design/methodology/approachIn this conceptual paper, we firstly seek to explore the available foundations of resilience as a construct and consider how these can be applied to organisational cybersecurity. To that aim, this study employs a targeted literature review approach, incorporating systematic elements to ensure rigour. Literature was identified through comprehensive searches in key academic databases, reference chaining and expert recommendations. Articles were selected based on relevance and contribution to the field, resulting in a thematic analysis to identify gaps and propose a heuristic model for cyber resilience. With this approach, we aim to position the emerging view of cyber resilience relative to risk analysis, while highlighting its domain of “conceptual comparative advantage” – the types of applications it is best suited to address. Finally, a high-level heuristic model for cyber resilience is proposed, which functions across the relevant policy, strategy and operational dimensions while also considering its relationship with cyber risk management.FindingsA conceptual model for organisational cyber resilience is proposed which helps position and frame research contributions in this domain relative to risk analysis, highlighting its domain of comparative advantage. The model integrates policy, strategy and operational dimensions, in a manner conducive to bridging foundations and applications of the concept of cyber risk management. The proposed model provides a critical point of reference to evaluate individual models, frameworks and tools.Originality/valueThis paper is a pioneering effort to overcome the current gaps between conceptual and practical views of cyber resilience. It proposes a new, risk-aligned view of the concept of cyber resilience and provides a structural foundation for further research and practice in the field.

Use of transportability methods for real-world evidence generation: a review of current applications.

Aim: To evaluate how transportability methods are currently used for real-world evidence (RWE) generation to inform good practices and support adoption and acceptance of these methods in the RWE context. Methods: We conducted a targeted literature review to identify studies that transported an effect estimate of the clinical effectiveness or safety of a biomedical exposure to a target real-world population. Records were identified from PubMed-indexed articles published any time before 25 July 2023 (inclusive). Two reviewers screened abstracts/titles and reviewed the full text of candidate studies to identify the final set of articles. Data on the therapeutic area, exposure(s), outcome(s), original and target populations and details of the transportability analysis (e.g., analytic method used, estimate transported, stated assumptions) were abstracted from each article. Results: Of 458 unique records identified, six were retained in the final review. Articles were published during 2021-2023, focused on the US/Canada context, and covered a range of therapeutic areas. Four studies transported an RCT effect estimate, while two transported effect estimates derived from real-world data. Almost all articles used weighting methods to transport estimates. Two studies discussed all transportability assumptions, and one evaluated the likelihood of meeting all assumptions and the impact of potential violations. Conclusion: The use of transportability methods for RWE generation is an emerging and promising area of research to address evidence gaps in settings with limited data and infrastructure. More transparent and rigorous reporting of methods, assumptions and limitations may increase the use and acceptability of transportability for producing robust evidence on treatment effectiveness and safety.

Patient, Care Partner, and Physician Voices in Treatment Decision-Making for Multiple Myeloma.

Treatment decision-making for multiple myeloma (MM) is complex. Individuals involved in decision-making may value treatment attributes differently based on their role as a patient, care partner, or physician. This study describes those attributes, and what is most important by role. We conducted a cross-sectional online survey with consenting adult patients with MM, MM care partners, and physicians treating MM. Respondents were recruited from US panels (Inspire and M3 Global Research) between September and December 2022. Survey items were informed by a targeted literature review, qualitative interviews, and a steering committee comprising clinical experts, a patient advocate, patient, and care partner. Descriptive statistics were generated and reported in aggregate. Email invitations were sent to 8071 Inspire members interested in or posting about MM. Of these, 4427 viewed the invitation, 941 responded, and 156 patients and care partners completed the survey (17% of respondents). For physicians, 5588 were invited via Email by M3 Global Research, with 761 viewing the invitation, 214 accessing the survey link, and 137 completing the survey (64% of respondents). Duration of response, side effects, and patients' quality-of-life (QoL) were the top three treatment attributes selected across the three cohorts; alignment of these attributes was consistent among patients regardless of disease severity. Separately, patients rated QoL and the amount of caregiving needed during/after treatment as the most important factors for future treatment decisions. If more effective MM treatments were offered, care partners were more willing to assume greater family burden (77%) compared to patients (49%), and patients were more accepting of potential serious side effects (50%) than were care partners (34%). Patients with MM, care partners, and physicians consider and value various treatment decision-making factors. Recognizing and addressing these differences is critical to meeting patients' preferences, needs, and optimizing patient outcomes.

Preferences of patients and physicians in the United States for relapsed/refractory follicular lymphoma treatments.

Patients with follicular lymphoma (FL) often relapse or become refractory to treatment (R/R). While the R/R FL treatment landscape evolves, little is known about the priorities of patients and physicians. This discrete-choice experiment (DCE) study assessed patients' and physicians' treatment preferences, and the trade-offs they would be willing to make between efficacy, tolerability, and administration. An online survey was conducted in US-based patients (≥18 years) with R/R FL and FL-treating physicians. The DCE was informed by a targeted literature review, clinical data, expert oncologist input, and pilot interviews. Participants completed eight experimental choice tasks where they chose between two hypothetical treatment profiles defined by six attributes: progression-free survival (PFS), administration/monitoring, risks of laboratory abnormalities requiring intervention, severe infections, diarrhea, and cytokine release syndrome (CRS). Relative attribute importance (RAI) and willingness to trade-off between PFS and other attributes were estimated. Two-hundred patients (mean age 63.5 years; median three prior lines of therapy) and 151 FL-treating physicians participated. Increasing PFS was most important for both groups, although it was relatively less important to patients than physicians (RAI 35.2% vs. 45.7%). Administration/monitoring was three times more important to patients than physicians (RAI 28.8% vs. 9.5%); patients preferred oral treatment and would be willing to tolerate a significant reduction in PFS for oral administration over weekly intravenous infusions. Avoiding CRS was less important to patients than to physicians (RAI 7.7% vs. 15.8%). Both groups would accept shorter PFS for reduced risks of side effects (especially of laboratory abnormalities for patients and of CRS for physicians). Although PFS was the most important attribute to patients and physicians, both would tolerate lower PFS for reduced side effects. Patients would also accept a substantial reduction in PFS for oral administration. Differences between the preferences/priorities of patients and physicians highlight the importance of shared decision-making.

Vessel wall MRI in giant cell arteritis: standardized protocol and scoring approach developed by an international working group.

There are an increasing number of centers performing research on high-resolution vessel wall magnetic resonance imaging (VW-MRI) in giant cell arteritis (GCA). However, harmonized approaches to VW-MRI in GCA are lacking and are essential to performing multicentre studies. Using a data-driven, consensus-based approach, an international expert group developed a standardized MRI protocol and scoring system to advance multi-centered research in cranial GCA. A targeted literature review of VW-MRI in cranial GCA was conducted. A working group comprised of radiologists, rheumatologists, and ophthalmologists with expertise in VW-MRI and GCA reviewed the results of the literature search, presented relevant data and images from their respective centers, and then reached consensus on recommendations related to key MRI structures, MRI sequences, scoring system, and other important considerations. A total of 21 relevant articles were identified and reviewed. Based on published literature, structures to be evaluated on MRI were categorized based on anatomic location (extradural cranial, intradural cranial, and orbits) and prioritization (core vs elective). Essential and elective sequences to comprehensively image cranial and orbital structures while minimizing scan time were determined along with scoring systems to grade contrast enhancement. This report describes a standardized approach to facilitate research of VW-MRI in cranial GCA that is the result of a multi-disciplinary, international collaboration of experts in VW-MRI and/or GCA.

The Epidemiology of Biliary Tract Cancer and Associated Prevalence of MDM2 Amplification: A Targeted Literature Review

The Epidemiology of Biliary Tract Cancer and Associated Prevalence of MDM2 Amplification: A Targeted Literature Review

Abstract P307: A Review Of The Burden, Management And Outcomes Of Patients With Aldosterone Dysregulation

Introduction: Aldosterone dysregulation (excess aldosterone production) is a complex physiological driver of uncontrolled hypertension as well as cardiovascular (CV) and kidney outcomes. Understanding the clinical and economic burden of excess aldosterone in routine practice is critical to optimizing treatment and improving long-term outcomes. This review examines the burden of excess aldosterone production as a driver of disease and its impact on clinical and economic outcomes. Hypothesis: Excess aldosterone production has a broad impact on long-term outcomes, and left untreated, contributes to worsened patient outcomes. Methods: A targeted literature review was conducted to identify articles on the burden, management and outcomes of patients with excess aldosterone production (2013 – 2024) with 27 manuscripts included. Results: More studies document the impact of excess aldosterone on organ function in the cardiovascular system and kidney (10, 8 manuscripts) compared to liver (1 manuscript). Excess aldosterone levels and aldosterone-induced oxidative stress and inflammation are associated with increased risk of CV events including myocardial infarction, heart failure, stroke, and increased morbidity and mortality. For example, Framingham Offspring Study showed increased CV disease risk with each rise in serum aldosterone levels (HR, 1.11; 95% CI, 1.02–1.21). Kidney, hepatic and metabolic manifestations are also well-documented, with implications for progression to chronic kidney disease, liver disease and metabolic syndrome. Another study reported an 11% increased risk of CKD progression associated with each doubling of serum aldosterone. Current pharmacological interventions for hypertension, including mineralocorticoid receptor antagonists, were not reported to restore normal aldosterone levels. No literature was found on the economic burden of excess aldosterone or the association between excess aldosterone production and clinical outcomes independent of blood pressure control. Conclusions: Currently available literature emphasizes the broad impact of excess aldosterone production on CV, kidney, hepatic, and metabolic outcomes. The impact on outcomes indicates that excess aldosterone production is underappreciated, with prognostic implications beyond primary aldosteronism. Current hypertension treatments do not address excess aldosterone production. These findings underscore the unmet need for novel management strategies that target excess aldosterone production.

Do we consent to change? An evaluation of oral and maxillofacial outpatient department dentoalveolar consent forms

Obtaining informed consent before performing any dentoalveolar procedure is a professional, ethical, and legal obligation. Prior to this work, in this oral and maxillofacial department, no standardised consent process existed for dentoalveolar procedures. A targeted literature review established localised gold standards for dentoalveolar procedures, and retrospective data from 56 dentoalveolar consent forms over a four-week period assessed baseline compliance with these standards. Data analysis identified that risks of dry socket and trismus were documented in only 7% of cases, and maxillary tuberosity fracture was never documented. Thirty-three per cent of lower third molar cases did not include consent for inferior alveolar nerve injury. Findings were discussed in a departmental audit meeting, and a root cause analysis identified memory failure, repetition fatigue, and varying clinician knowledge and experience, as key drivers. The evidence base and localised gold standards were discussed. A standardised, bilingual, triple-copy dentoalveolar consent form was developed, including predefined indications, additional procedures, and risks, in a tick-box format. This form was presented to the Department of Patient Safety and Quality and gained local approval. Cases completed with the new form will now be evaluated against the localised gold standards, with a mixed-methods feedback survey investigating staff and patient opinions. Local processes for obtaining and documenting valid consent for dentoalveolar treatment were found to be inconsistent and frequently inadequate. Fifteen localised gold standards were set, and a new standardised consent form developed, which will be evaluated for effectiveness and acceptability.

Development of stakeholder-informed recommendations for inclusion of family spillover effects in health technology assessment.

The impacts of disease and treatment on a patient's family members and informal caregivers are known as "family spillover effects." Although many formal value frameworks call for the consideration of these effects, they are often not included in health technology assessments (HTAs) and cost-effectiveness analyses (CEAs). A formal evaluation of stakeholder perspectives may help address the disconnect for inclusion of family spillover effects observed in practice. To develop stakeholder-driven recommendations for the measurement and use of family spillover effects in the United States and to identify research opportunities. We first conducted a targeted literature review of US-based CEAs and HTA reports from the past 10 years to assess the current use of family spillover effects. We then used a purposeful sampling technique to conduct 25 qualitative interviews with outcomes researchers, patient advocates, health economists, and health policy and payer experts to gather perspectives on when and how family spillover effects should be considered in HTA processes. We conducted a thematic analysis of the interview transcripts to identify key themes and develop preliminary recommendations. Finally, we conducted an online workshop with 8 stakeholders to discuss, rate, and refine preliminary recommendations to develop final recommendations. A key theme identified in the stakeholder interviews was the role that data availability, analyst preferences, and prior precedence play in limiting the inclusion of spillover effects in HTAs. Additional themes included support for the inclusion of both qualitative and quantitative spillover effects and the need to capture broad and diverse impacts across populations. We developed 15 recommendations from the consensus building workshop addressing measurement, CEA modeling, and HTA processes. Key recommendations included (1) a transparent process for deciding when family spillover effects should be included, (2) measurement of direct and indirect costs with priority based on the magnitude of impact, (3) the use of validated measures, (4) the use of proxy information and expert elicitation when quality data are unavailable, and (5) the use of a modified impact inventory table for transparency of included effects. Research opportunities included patient involvement in family spillover effect research and HTAs, mapping algorithms and non-preference-based caregiver measures to generate utilities, and consensus best practices for modeling. The inconsistent inclusion of family spillover effects in HTAs and CEAs remains a persistent challenge. The stakeholder-driven recommendations and research opportunities identified in this study may help improve the transparency, measurement, and use of family spillover effects in assessing the clinical and economic value of novel medical technologies.

Participation in Multidisciplinary Teams Among Healthcare Professionals: A Discrete Choice Experiment in Tertiary Public Hospitals in China.

Healthcare professionals' participation is crucial for the efficient implementation of multidisciplinary team (MDT) collaboration models. We identified the key factors influencing healthcare professionals' preference to participate in MDTs in tertiary hospitals. To clarify the attributes and levels of the discrete choice experiment (DCE), we conducted a targeted literature review and conducted in-depth interviews with MDT service providers. Following this, a DCE was designed to evaluate healthcare professionals' preferences for MDT participation, and the influence of factors such as salary subsidies, leadership attention, patient participation, quality assessment, working intensity, and case complexity. A conditional logit model estimated the utility of each attribute. Willingness-to-pay estimates were derived by taking the negative ratio of the coefficients of non-economic and economic attributes. A series of policy simulation analyses were conducted. Two hundred healthcare professionals completed the questionnaire, with 180 valid responses used for analysis. All attributes were statistically significant. Leadership attention and working intensity were the primary factors influencing staff willingness to participate in MDTs, followed by quality assessment and salary subsidies. Significant preference differences were observed between respondents; compared with mid-level staff, senior-level healthcare professionals believed patient engagement would be more helpful in boosting participation. The policy simulation showed that changing leadership attention from "neglect" to "emphasis" would increase the probability of staff choosing to participate in MDTs from 24.4% to 66.98%. Leadership attention was the primary concern for healthcare professionals in MDTs. To effectively motivate staff participation in MDTs, policymakers should adopt a holistic approach that considers work motivation and individual backgrounds, including competitive salary packages and a positive work environment. They should concurrently introduce MDT case complexity measurement tools to optimize resource allocation. Addressing staff members' unique needs and career aspirations by creating targeted training programs, pathways for advancement, and personalized career development plans are also crucial.

Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding.

Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments. An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0-100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mildtomoderate nausea, mildtomoderate skin reactions). A Dirichlet regression was used to estimate average preference weights. Marginal utility and the reduction in ORR that patients would accept in exchange for a 10-pointdecrease or a10% decrease in other attributes were calculated. A total of 100 patients were recruited and self-completed the survey and MDT. Mean patient age was 64.9 years (standard deviation, 7.6), 54% were female, and 38% identified as white. All included treatment attributes had a statistically significant impact on preferences. Changes in ORR had the largest impact, followed by cancer-related pain and treatment-related risks. Patients were willing to accept an 8.4% decrease in ORR to reduce their pain level by 10 points or a 7.8% decrease in ORR to reduce the risk of peripheral neuropathy by 10%. For a 10% decrease in severe side effects, mildtomoderate nausea, or skin reaction, patients would accept decreases in ORR of 5.5%, 3.7%, or 3.4%, respectively. Of the attributes tested, changes in ORR were most important to patients. Patients made tradeoffs between treatment attributes indicating that a lower ORR may be acceptable for an improvement in other attributes such as reduced cancer-related pain or the risk of treatment-related adverse events.

Treatment Effect Waning in Immuno-oncology Health Technology Assessments: A Review of Assumptions and Supporting Evidence with Proposals to Guide Modelling.

Treatment effect waning (TEW) refers to the attenuation of treatment effects over time. Assumptions of a sustained immuno-oncologic treatment effect have been a source of contention in health technology assessment (HTA). We review how TEW has been addressed in HTA and in the wider scientific literature. We analysed company submissions to English language HTA agencies and summarised methods and assumptions used. We subsequently reviewed TEW-related work in the ISPOR Scientific Presentations Database and conducted a targeted literature review (TLR) for evidence of the maintenance of immuno-oncology (IO) treatment effects post-treatment discontinuation. We found no standardised approach adopted by companies in submissions to HTA agencies, with immediate TEW most used in scenario analyses. Independently fitted survival models do however suggest TEW may often be implicitly modelled. Materials in the ISPOR scientific database suggest gradual TEW is more plausible than immediate TEW. The TLR uncovered evidence of durable survival in patients treated with IOs but no evidence that directly addresses the presence or absence of TEW. Our HTA review shows the need for a consistent and appropriate implementation of TEW in oncology appraisals. However, the TLR highlights the absence of direct evidence on TEW in literature, as TEW is defined in terms of relative treatment effects-not absolute survival. We propose a sequence of steps for analysts to use when assessing whether a TEW scenario is necessary and appropriate to present in appraisals of IOs.

Development of a new patient-reported outcome measure for complex cryptoglandular fistulas (20-Item complex cryptoglandular fistula questionnaire™): a qualitative study

BackgroundThere are limited tools to measure the burden of disease and effectiveness of medical/surgical interventions in patients with cryptoglandular fistulas. The aim of this study was to explore concepts that are relevant and important to patients with complex cryptoglandular fistulas (CCF) and to develop a patient-centred, disease-specific, patient-reported outcome measure (PROM) to assess symptom burden and impacts of CCF.MethodsA targeted literature review was conducted, followed by one-to-one telephone interviews with five colorectal surgeons (USA, n = 3; UK, n = 1; Spain, n = 1) and 20 US adult patients with CCF to inform the development of a conceptual model and a CCF-specific PROM. The targeted literature review informed the development of the preliminary conceptual model and identified a PROM in the literature that was used as a reference to generate the draft CCF-specific PROM. The colorectal surgeon interviews provided insights on the experience of patients with CCF to refine the conceptual model, formulate probing questions for use in patient interviews, and to develop the draft CCF-specific PROM. Patients’ descriptions of their experiences with symptoms and the impacts on their lives and evaluation of the draft CCF-specific PROM in concept elicitation and cognitive interviews were used to develop the final conceptual model and final CCF-specific PROM.ResultsTen symptoms (odour, pain during bowel movement, abscess, post-operative pain, discharge/drainage/leakage, anal/perianal pain, inflammation/swelling, skin irritation, bleeding and itchiness) and 11 impacts (discomfort, inability to exercise, embarrassment, difficulty sitting, worry about disease, adapted life to maintain hygiene, negatively impacted social life/isolation, inability to perform daily activities, reduced interest in sex, negatively impacted intimate relationships and negatively impacted mood) were reported as most salient by patients. The patient experience, clinician perspective, and literature review provided input to item generation. Evaluation of relevance and patient understanding through cognitive interviews with patients provided evidence for the content validity of the new patient-reported outcome measure: the 20-item Complex Cryptoglandular Fistula Questionnaire™ (CCFQ-20™).ConclusionThe CCFQ-20™ is a new clinician-guided, patient-validated, disease-specific patient-reported outcome measure that measures disease impact and quality of life in patients with CCF.

2352 Disease burden of respiratory syncytial virus (RSV) in adults 60+ in the United Kingdom: targeted literature review (TLR)

Abstract Introduction It is increasingly clear that RSV infections in older adults significantly impact the UKs already strained public health system. However, the true burden remains underestimated (Korsten K, Eur Respir J, 2021, 57, 2,002,688; Sharp A, Influenza Other Respir Viruses, 2022, 16, 125–131). We conducted a TLR to scrutinise the available data and reveal evidence gaps. Methods A TLR search was conducted in OVID Medline, Embase, and ECON Lit to identify existing literature from Jan 2011-Aug 2023 and grey literature. Eligibility criteria were defined based on population (intervention/comparator (no limit), outcomes (clinical, epidemiological, economic and QoL) and limited to UK-only results. Results Despite the broad criteria, only 14 out of 1013 studies were included. Nine studies reported on epidemiological outcomes, one on epidemiology and costs, and four on model outcomes. Due to the small number of studies and the heterogeneity in study design and outcomes, it was not possible to draw reliable conclusions on incidence, prevalence, and mortality. One model paper showed that a hypothetical RSV vaccine could be cost effective in the UK, depending on incidence, vaccine effectiveness and cost. All identified studies emphasise the challenges in estimating the true burden due to limitations in testing and a lack of standardised disease definition in older adults. There is a lack of evidence for risk groups, particularly clinical and economic consequences for RSV patients at a higher risk of infection and severe sequelae. Conclusion Only a limited number of studies meeting the criteria focused on RSV infection in older adults were found, with nearly no data on risk groups and related cost. It is imperative that surveillance and testing are improved to understand the true burden. This will serve as a basis for evaluating cost-effectiveness and decisions on the most effective measures to lower the burden, including vaccination programmes.

Patient Experience with Chronic Refractory Gout and Its Impact on Health-Related Quality of Life: Literature Review and Qualitative Analysis.

Patients with chronic refractory gout face a considerable burden of disease due to unexpected flares characterized by severe and debilitating pain, which can lead to chronic pain and joint damage. This study aimed to understand the symptoms and impacts of chronic refractory gout on health-related quality of life (HRQoL). A targeted literature review was conducted to identify and review key articles describing the symptoms and impacts of gout, and articles examining the psychometric performance of the Medical Outcomes Survey Short Form-36 (SF-36) and Health Assessment Questionnaire-Disability Index (HAQ-DI) in gout. Qualitative interviews were conducted with 20 participants with chronic refractory gout. The results were used to develop the conceptual model and determine the appropriateness of the SF-36 and HAQ-DI in evaluating HRQoL in this population. Most frequently reported symptoms included bodily pain (n = 18, 90.0%), joint swelling (n = 18, 90.0%), joint tenderness (n = 18, 90.0%), and joint pain (n = 16, 80.0%). Most frequently reported impacts were difficulties climbing a flight (n = 20, 100.0%) or several flights of stairs (n = 20, 100.0%), climbing five steps (n = 19, 95.0%), completing chores (n = 19, 95.0%), and running errands and shopping (n = 19, 95.0%). All assessed items from SF-36 and HAQ-DI were reported by ≥ 25% (n = 5) of participants and mapped sufficiently to concepts elicited by participants. Patients with chronic refractory gout report symptoms and impacts that are highly bothersome and burdensome to everyday life. Items included in the HAQ-DI and SF-36 mapped directly to these symptoms and impacts and are relevant to understand the burden of disease of chronic refractory gout.

Advancing the future of equitable access to health care: recommendations from international health care leaders.

Disparities in access to health care are persistent and contribute to poor health outcomes for many populations around the world. Barriers to access are often similar across countries, despite differences in how health systems are structured. Health care leaders can work to address these barriers through bold, evidence-based actions. The Future of Health (FOH), an international community of senior health leaders, collaborated with the Duke-Margolis Institute for Health Policy to identify priority organizational and policy actions needed to improve equitable access to health care through a consensus-building exercise, a targeted literature review, and an expert discussion group. This paper describes four key action areas for health care leaders that FOH members identified as critical to enabling the future of equitable access to health care: ensuring prioritization of and accountability for equitable access to care; establishing comprehensive, organization-wide strategies to address barriers to access; clearly defining and incentivizing improvement on key measures related to reducing disparities in access; and establishing cross-sector partnerships to improve equitable access.

Adult vaccination coverage in the United States: A database analysis and literature review of improvement strategies

ABSTRACT Despite vaccines being instrumental in reducing vaccine-preventable disease, adult vaccination rates in the United States (US) are below optimal levels. To better understand factors affecting vaccination rates, we analyzed trends in adult vaccination coverage using data from the Behavioral Risk Factor Surveillance System (BRFSS) and conducted a targeted literature review (TLR) on interventions to improve adult vaccination rates in the US. Both the BRFSS analysis and the TLR focused on influenza; pneumococcal disease; tetanus and diphtheria or tetanus, diphtheria, and acellular pertussis; herpes zoster; and human papillomavirus vaccination for US adults aged 18–64 years. The TLR additionally included hepatitis A and hepatitis B vaccination. Vaccination coverage rates (VCRs) and changes in VCRs were calculated using the 2011–2019 BRFSS survey data. For the TLR, the MEDLINE and MEDLINE In-Process databases were searched for articles on vaccination interventions published between January 2015 and June 2021. The BRFSS analysis showed that changes in VCRs were generally modest and positive for most states over the study period. The TLR included 32 articles that met the eligibility criteria; intervention strategies that improved adult vaccination outcomes incorporated an educational component, vaccination reminders or reinforcement at the point of care, or authorized non-clinician members of the healthcare team to vaccinate. Furthermore, interventions combining more than one approach appeared to enhance effectiveness. The strategies identified in this TLR will be valuable for policymakers and stakeholders to inform the development and implementation of evidence-based policies and practices to improve adult vaccination coverage.

Capturing patient-reported sleep disturbance in atopic dermatitis clinical trials

BackgroundPatient-focused approaches to capturing day-to-day variability in sleep disturbance are needed to properly evaluate the sleep benefits of new treatments. Such approaches rely on patient-reported outcome (PRO) measures validated in the target patient population.MethodsUsing atopic dermatitis (AD) as an example of a disease in which sleep is commonly disturbed, we developed a strategy for measuring sleep disturbance in AD trials. In developing this strategy, we conducted a targeted literature review and held concept elicitation interviews with adolescents and adults with AD. We subsequently identified potentially suitable PRO measures and cognitively debriefed them. Finally, we evaluated their psychometric properties using data from phase 2b (NCT03100344) and phase 3 (NCT03985943 and NCT03989349) clinical trials.ResultsThe literature review confirmed that sleep disturbance is a key impact of AD but failed to identify validated PRO measures for assessing fluctuations in sleep disturbance. Subsequent concept elicitation interviews confirmed the multidimensional nature of sleep disturbance in AD and supported use of a single-item measure to assess overall sleep disturbance severity, complemented by a diary to capture individual components of sleep disturbance. The single-item sleep disturbance numerical rating scale (SD NRS) and multi-item Subject Sleep Diary (SSD)—an AD-adapted version of the Consensus Sleep Diary—were identified as potentially suitable PRO measures. Cognitive debriefing of the SD NRS and SSD demonstrated their content validity and their understandability to patients. Psychometric analyses based on AD trial data showed that the SD NRS is a well-defined, reliable, and fit-for-purpose measure of sleep disturbance in adults with AD. Furthermore, the SD NRS correlated with many SSD sleep parameters, suggesting that most concepts from the SSD can be covered using the SD NRS.ConclusionsUsing these findings, we developed an approach for measuring sleep disturbance in AD trials. Subject to further research, the same approach could also be applied to future trials of other skin diseases where itch causes sleep disturbance.