Opportunities and Challenges for Decentralized Clinical Trial Approaches: European Health Technology Assessment Perspective.

OBJECTIVES: Decentralized clinical trial (DCT) approaches are clinical trials in which some or all trial activities take place closer to participants' proximities instead of a traditional investigative site. Data from DCTs may be used for clinical and economic evaluations by health technology assessment (HTA) bodies to support reimbursement decision making. This study aimed to explore the opportunities and challenges for DCT approaches from an HTA perspective by interviewing representatives from European HTA bodies. METHODS: We conducted semistructured interviews with 25 European HTA representatives between September 2022 and February 2023, and transcripts were analyzed after thematic analysis. RESULTS: Two main themes were identified from the data relating to (1) DCT approaches in HTA and (2) trial-level acceptance and relevance. Experience with assessing DCTs was limited and a variety of knowledge about DCTs was observed. The respondents recognized the opportunity of DCTs to reduce recall bias when participant-reported outcome data can be collected more frequently and conveniently from home. Concerns were expressed about the data quality when participants become responsible for data collection. Despite this challenge, the respondents recognized the potential of DCTs to increase the generalizability of results because data can be collected in a setting reflective of the everyday situation potentially from a more diverse participant group. CONCLUSIONS: DCTs could generate relevant results for HTA decision making when data are collected in a real-world setting from a diverse participant group. Increased awareness of the opportunities and challenges could help HTA assessors in their appraisal of DCT approaches.

Left ventricular ejection fraction: time to revise the metric?

In this paper, we highlight the prevalent misconception among patients regarding the normal value of left ventricular ejection fraction in cardiac function assessment. This misconception arises from the proportion dominance effect, wherein individuals tend to judge based on proportions rather than absolute values. We explain how this misunderstanding impacts patient demoralization and medical adherence, leading to worse clinical outcomes. To address this, the concept of "Left Ventricular Ejection Fraction - Proportion of Normal" is introduced, which adjusts left ventricular ejection fraction to a patient-specific normal range. This patient-centric approach aims to enhance comprehension, engagement, and adherence by conveying accurate information through the lens of proportions, thereby potentially improving clinical outcomes.

Defining human-centricity in Industry 5.0 and assessing the readiness of ergonomics/human factors communities in UK.

There is a lack of a clear and consistent definition of human-centricity in Industry 5.0. This study identified the definition of human-centricity in Industry 5.0 through a systematic literature review and used it to assess the readiness of Ergonomics/Human Factors communities in the UK. The assessment of the communities readiness was conducted by reviewing UK accredited courses and events of three professional bodies; and interviewing practitioners (n = 8). Eleven themes were identified as elements of human-centricity from the thematic analysis of 30 publications. Gaps that had to be addressed to better equip UK practitioners to support the realisation of human-centricity in Industry 5.0 were also identified.

The meaning of human-centricity in Industry 5.0 and its bearing on Ergonomics/Human Factors communities are not fully understood. Eleven themes that define human-centricity in Industry 5.0 are extracted. Gaps that have to be addressed by Ergonomics/Human Factors communities in UK are also identified.

Contribution of the Dynamic Intestinal Absorption Model (Diamod) to the Development of a Patient-Centric Drug Formulation.

Compound X is a weak basic drug targeting the early stages of Parkinson's disease, for which a theoretical risk assessment has indicated that elevated gastric pH conditions could potentially result in reduced plasma concentrations. Different in vitro dissolution methodologies varying in level of complexity and a physiologically based pharmacokinetic (PBPK) absorption model demonstrated that the dissolution, solubility, and intestinal absorption of compound X was indeed reduced under elevated gastric pH conditions. These observations were confirmed in a crossover pharmacokinetic study in Beagle dogs. As a result, the development of a formulation resulting in robust performance that is not sensitive to the exposed gastric pH levels is of crucial importance. The dynamic intestinal absorption MODel (Diamod), an advanced in vitro gastrointestinal transfer tool that allows to study the gastrointestinal dissolution and interconnected permeation of drugs, was selected as an in vitro tool for the formulation optimization activities given its promising predictive capacity and its capability to generate insights into the mechanisms driving formulation performance. Different pH-modifiers were screened for their potential to mitigate the pH-effect by decreasing the microenvironmental pH at the dissolution surface. Finally, an optimized formulation containing a clinically relevant dose of the drug and a functional amount of the selected pH-modifier was evaluated for its performance in the Diamod. This monolayer tablet formulation resulted in rapid gastric dissolution and supersaturation, inducing adequate intestinal supersaturation and permeation of compound X, irrespective of the gastric acidity level in the stomach. In conclusion, this study describes the holistic biopharmaceutics approach driving the development of a patient-centric formulation of compound X.

A Human Digital-Twin-Based Framework Driving Human Centricity towards Industry 5.0.

This work presents a digital-twin-based framework focused on orchestrating human-centered processes toward Industry 5.0. By including workers and their digital replicas in the loop of the digital twin, the proposed framework extends the traditional model of the factory's digital twin, which instead does not adequately consider the human component. The overall goal of the authors is to provide a reference architecture to manufacturing companies for a digital-twin-based platform that promotes harmonization and orchestration between humans and (physical and virtual) machines through the monitoring, simulation, and optimization of their interactions. In addition, the platform enhances the interactions of the stakeholders with the digital twin, considering that the latter cannot always be fully autonomous, and it can require human intervention. The paper also presents an implemented scenario adhering to the proposed framework's specifications, which is also validated with a real case study set in a factory plant that produces wooden furniture, thus demonstrating the validity of the overall proposed approach.

T Cell Receptor Chain Centricity: The Phenomenon and Potential Applications in Cancer Immunotherapy.

T cells are crucial players in adaptive anti-cancer immunity. The gene modification of T cells with tumor antigen-specific T cell receptors (TCRs) was a milestone in personalized cancer immunotherapy. TCR is a heterodimer (either α/ß or γ/δ) able to recognize a peptide antigen in a complex with self-MHC molecules. Although traditional concepts assume that an α- and ß-chain contribute equally to antigen recognition, mounting data reveal that certain receptors possess chain centricity, i.e., one hemi-chain TCR dominates antigen recognition and dictates its specificity. Chain-centric TCRs are currently poorly understood in terms of their origin and the functional T cell subsets that express them. In addition, the ratio of α- and ß-chain-centric TCRs, as well as the exact proportion of chain-centric TCRs in the native repertoire, is generally still unknown today. In this review, we provide a retrospective analysis of studies that evidence chain-centric TCRs, propose patterns of their generation, and discuss the potential applications of such receptors in T cell gene modification for adoptive cancer immunotherapy.

[The loyalty of patients during coronavirus period: investigation experience in the metropolitan children polyclinic].

Two years after COVID-19 pandemic starting, the studies of patient satisfaction with medical services quality become topical again. The article presents the results of three-year project (2019-2022) of studying level of loyalty (Net Promoter Score) of legal representatives of patients of one of municipal children polyclinics. It is established that in Moscow, with implementation of first restrictive measures level of loyalty in the children polyclinic increased from 45% to 70%. In following years, level of loyalty was fixed at the level of 60%. The four groups of factors determining such changes as high level of panic moods due to pandemic, changes in organization of polyclinic functioning, energetic activity of mass media and users of social networks in glorifying image of medical workers and cultural psychological characteristics of Russians. The "optimistic", "realistic" and "pessimistic" prognoses concerning further dynamics of level of loyalty are proposed. The conclusion was made that COVID-19 pandemic served as key factor of positive changes in assessments of patients (and their legal representatives) of functioning both of particular polyclinic and the Russian health care in general. As far as each following time coronavirus infection will cause less and less concern among Russians it can be expected the requirements presented to medical services will soon increase, that means workload of medical personnel will only intensify. The following prospective directions of organizing functioning of medical institutions are proposed: monitoring of psycho-social indicators of medical personnel, implementation of telemedicine technologies, transfer of part of functionality of physicians and nurses to specialists without medical education.

Sensitivity to Change and Patient-Centricity of the Unified Multiple System Atrophy Rating Scale Items: A Data-Driven Analysis.

BACKGROUND: The Unified Multiple System Atrophy Rating Scale (UMSARS) is a commonly used semiquantitative rating scale to assess symptoms and measure disease progression in multiple system atrophy (MSA). However, it is currently incompletely understood which UMSARS items are the most sensitive to change and most relevant to the patient. OBJECTIVE: The objective of this study was to assess sensitivity to change and patient-centricity of single UMSARS items. METHODS: Data were taken from the European Multiple System Atrophy Study Group Natural History Study and the Rasagiline for Multiple System Atrophy trial. Sensitivity of change of an item of the UMSARS was assessed by calculation of a sensitivity-to-change ratio using its mean slope of progression divided by the standard deviation of the slope when modeling its progression over time. Patient-centricity was assessed through correlation of UMSARS items with quality-of-life measures. RESULTS: Progression rates above the mean in at least one of the two studies examined here were seen for seven items of UMSARS I and 11 items of UMSARS II. These items related to key motor functions such as swallowing, speech, handwriting, cutting food, hygiene, and dressing or walking, whereas items related to autonomic dysfunction were generally less sensitive to change in either data set. More UMSARS I items were identified as patient-centric compared with UMSARS II items, and items most strongly impacting patients' quality of life were those affecting verbal communication skills, personal hygiene, and walking. CONCLUSION: The present results illustrate the potential to optimize the UMSARS to enhance sensitivity to change and patient centricity. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

The Current Landscape of Research Advocacy and Education for Patients with Colorectal Cancer.

OPINION STATEMENT: Research advocacy is an evolving concept and should be tailored for the colorectal cancer research community. Research advocacy training and evaluation must be designed for the patient community with their insight included at each step of engagement, training, and implementation. Patient advocates bring a great deal of expertise to the research review process, but it is important to ensure that their insight is appropriately placed, and they bring an appropriate orientation to the research process as the most informed patient. This can be accomplished in part by providing advocates with the proper training, employing universal core competencies, and applying principles of adult learning. Additionally, the research community, advocacy organizations, and industry partners must understand the need to diversify the voices that are being leveraged to guide research, recognizing the importance of adequate mental health tools and compensation commensurate with their experience. As a community, it is necessary that we create and implement training programs, as well as evaluate and measure their impact to continually improve and tailor the delivery of this specific education. Research advocacy has become a necessity to the field, and when implemented effectively, research advocates can have a significant impact on the delivery of health care research, improving health outcomes for all those affected by colorectal cancer.

The future of personalized care for vascular patients: an industry perspective.

Healthcare has entered a brave new world in the early part of the 21st century: the landscape has changed and continues to change rapidly, evolving at a rate as never seen before. Fuelled by technological advancement, big data analytics, and the explosion of apps and sensors, as well as by telemedicine and remote monitoring needs driven by the COVID-19 pandemic, the healthcare ecosystem is metamorphosing literally before our eyes. So, what is the role for the Medtech industry as healthcare systems reshape themselves to address emerging patients' needs and desires, and how can the use of data and novel technologies be leveraged to bring about the kind of change needed to deliver truly holistic patient care?

[FROM "CAPRICE MEDICINE" TO "EQUAL RESPONSIBILITY MEDICINE": TASKS, TECHNOLOGIES AND SPECIFICS OF CONFLICT MANAGEMENT IN A MULTIDISCIPLINARY PRIVATE CLINIC].

The article substantiates the tasks of introducing a conflict management system into the work of a private sector medical organization, reducing the number of ethical and legal conflicts that arise during the provision of medical care. The analysis of the conceptual foundations, regulatory framework and specifics of the use of conflict management tools in the context of solving organizational and managerial tasks in a private multidisciplinary clinic is presented.

Leveraging the Similarities Between Cost-Effectiveness Analysis and Value-Based Healthcare.

OBJECTIVES: This article aims to explore overlaps and differences between the emerging concept of value-based healthcare (VBHC) and the established field of cost-effectiveness analysis (CEA), as well as the feasibility of integrating them together. Interest in VBHC has grown significantly in developed countries that seek to preserve the sustainability of their healthcare systems. Consequently, it is likely that VBHC will soon play a meaningful role in health economic policy and decision making. Because VBHC and CEA share many similarities, academics have pointed out that integration could lead to opportunities for improvements in both fields. METHODS: An exploration of overlapping topics in VBHC and CEA literature was performed to establish initial links between them. A new methodologic approach is described to consolidate key value frameworks from the respective fields. RESULTS: Several key themes emerged in which these 2 concepts can reinforce each other: interpretation of value, sensitivity to outcome changes, scientific credibility, methodology and measurement, and usability in decision making. Subsequently, an initial method is described of how the VBHC framework for value could be integrated into CEA through a so-called value-based healthcare quality-adjusted life year (VBHC-QALY). CONCLUSION: This article introduces the existing VBHC value framework to the cornerstone of modern CEA and substantiates the presumption of health economists that valuable synergies arise from consolidating the individual strengths of CEA and VBHC. Through integration CEA can help establish robust methods for VBHC implementation, while the latter can complement the former with a new viewpoint and conceptual toolbox for patient centricity and the definition of value.

Sensor Data Integration: A New Cross-Industry Collaboration to Articulate Value, Define Needs, and Advance a Framework for Best Practices.

Data integration, the processes by which data are aggregated, combined, and made available for use, has been key to the development and growth of many technological solutions. In health care, we are experiencing a revolution in the use of sensors to collect data on patient behaviors and experiences. Yet, the potential of this data to transform health outcomes is being held back. Deficits in standards, lexicons, data rights, permissioning, and security have been well documented, less so the cultural adoption of sensor data integration as a priority for large-scale deployment and impact on patient lives. The use and reuse of trustworthy data to make better and faster decisions across drug development and care delivery will require an understanding of all stakeholder needs and best practices to ensure these needs are met. The Digital Medicine Society is launching a new multistakeholder Sensor Data Integration Tour of Duty to address these challenges and more, providing a clear direction on how sensor data can fulfill its potential to enhance patient lives.

Research on Financial Systemic Risk in ASEAN Region.

The research of financial systemic risk is an important issue, however the research on the financial systemic risk in ASEAN region lacks. This paper uses the minimum density method to calculate the interbank network of ASEAN countries and uses the node centrality to judge the systemically important banks of various countries. Then the DebtRank algorithm is constructed to calculate the systemic risk value based on the interbank network. By comparing the systemic risk values obtained through the initial impact on the system important banks and non-important banks, we find that the systemic risk tends to reach the peak in the case of the initial impact on the system important banks. Furthermore, it is found that countries with high intermediary centrality and closeness centrality have higher systemic risk. It suggests that the regulatory authorities should implement legal supervision, strict supervision, and comprehensive supervision for key risk areas and weak links.

Challenges and opportunities to include patient-centric product design in industrial medicines development to improve therapeutic goals.

In the past, drug developers in industry chose approaches mainly focusing on the drug product's efficacy, safety and quality according to the level required by regulatory expectations stipulated in guidelines, pharmacopoeia and other regulatory provisions. By putting more focus on the patient perspective, regulatory authorities are currently raising their requirements regarding successful product submissions. The increasing involvement of patients in the product development process (e.g. conduction of human factor use tests, integration of feedback from patient and patient advisory groups into clinical programmes) requires adaptations to the existing and established industrial drug development processes without compromising fast patient access to innovative therapies. This review provides an expert opinion on the emerging challenges and opportunities to implement a patient-centric approach into new drug development programmes. The aim is to better understand the challenge of finding the right balance between bringing innovative drugs fast to the patients and to develop these in parallel in a patient-centric product form as well as why this is an opportunity and how stakeholder parties (e.g. patients, clinicians, pharmacists, caregivers, regulators) can provide support to achieve desired outcomes.

Real-world data and the patient perspective: the PROmise of social media?

Understanding the patient perspective is fundamental to delivering patient-centred care. In most healthcare systems, however, patient-reported outcomes are not regularly collected or recorded as part of routine clinical care, despite evidence that doing so can have tangible clinical benefit. In the absence of the routine collection of these data, research is beginning to turn to social media as a novel means to capture the patient voice. Publicly available social media data can now be analysed with relative ease, bypassing many logistical hurdles associated with traditional approaches and allowing for accelerated and cost-effective data collection. Existing work has shown these data can offer credible insight into the patient experience, although more work is needed to understand limitations with respect to patient representativeness and nuances of captured experience. Nevertheless, linking social media to electronic medical records offers a significant opportunity for patient views to be systematically collected for health services research and ultimately to improve patient care.

The Impact of Focality and Centricity on Vulvar Intraepithelial Neoplasia on Disease Progression in HIV+ Patients: A 10-Year Retrospective Study.

BACKGROUND: The impact of lesion focality and centricity in relation to patient outcome and disease recurrence of vulvar intraepithelial neoplasia (VIN) is an understudied area of research, especially in immunocompromised women. The prevalence and incidence of VIN have increased steadily since the 1980s because of the co-existence of human papillomavirus (HPV) and human immunodeficiency virus (HIV). In this study, we retrospectively examined the records of VIN patients to determine the effect of lesion focality and centricity with respect to the interval to disease recurrence. MATERIALS AND METHODS: All women diagnosed with VIN and managed between January 2002 and December 2011 were included (n = 90) and followed up until December 2017. Symptoms at the time of presentation, including HIV positivity (n = 75), were collated, including the influences of multifocality and multicentricity on time to disease recurrence. RESULTS: Multicentricity caused a more rapid recurrence of disease than unicentricity (p = 0.006), whereas multifocality increased the risk of recurrence more than unifocality (p < 0.0001). Viral load in the HIV+ patients was not associated with time to disease recurrence, but the reduced number of CD4+ lymphocytes present in HIV+ patients was. Treatment modalities had no effect on disease recurrence. CONCLUSION: Both focality and centricity have effects on interval to recurrence and final patient outcome, with multifocal disease having a poorer prognosis. Centricity and focality should be recorded at the time of diagnosis and act as a warning for disease recurrence. HIV+ VIN patients with multifocal disease and/or known immunosuppression (low CD4+ lymphocyte counts) should be regarded as "high-risk" patients and treated accordingly.

The Patient Research Partner Network Matures: A Report from the GRAPPA 2017 Annual Meeting.

The Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) has reached the third of 5 stages of organizational maturity regarding incorporating patient research partners (PRP) into psoriatic arthritis (PsA) and psoriasis research and educational efforts. Herein, we report the involvement of PRP at the GRAPPA 2017 annual meeting and plans for future PRP engagement.

A cloud-based home health care information sharing system to connect patients with home healthcare staff -A case report of a study in a mountainous region.

We have developed a cloud system, the e-Renraku Notebook (e-RN) for sharing of home care information based on the concept of "patient-centricity". In order to assess the likelihood that our system will enhance the communication and sharing of information between home healthcare staff members and home-care patients, we selected patients who were residing in mountainous regions for inclusion in our study. We herein report the findings.Eighteen staff members from 7 medical facilities and 9 patients participated in the present study.The e-RN was developed for two reasons: to allow patients to independently report their health status and to have staff members view and respond to the information received. The patients and staff members were given iPads with the pre-installed applications and the information being exchanged was reviewed over a 54-day period.Information was mainly input by the patients (61.6%), followed by the nurses who performed home visits (19.9%). The amount of information input by patients requiring high-level nursing care and their corresponding staff member was significantly greater than that input by patients who required low-level of nursing care.This patient-centric system in which patients can independently report and share information with a member of the healthcare staff provides a sense of security. It also allows staff members to understand the patient's health status before making a home visit, thereby giving them a sense of security and confidence. It was also noteworthy that elderly patients requiring high-level nursing care and their staff counterpart input information in the system significantly more frequently than patients who required low-level care.

Familial history of non-medullary thyroid cancer is an independent prognostic factor for tumor recurrence in younger patients with conventional papillary thyroid carcinoma.

BACKGROUND: It is not clear whether familial non-medullary thyroid cancer (FNMTC) is more aggressive and has a poorer prognosis, than sporadic carcinoma. Therefore, the optimal clinical approach for FNMTC is yet to be established. In this study, we investigated the biological behavior and prognosis of FNMTC compared with its sporadic counterpart. METHODS: Between 1996 and 2004, 1,262 patients underwent a total thyroidectomy for conventional PTC at Asan Medical Center and 113 (9.0%) were diagnosed with FNMTC. We compared the clinico-pathologic characteristics, treatment modalities, and prognosis between familial and sporadic NMTC. RESULTS: FNMTC was significantly more multi-centric than sporadic. We also found that family history itself was an independent risk factor for recurrence. Moreover, disease-free survival in the familial group was significantly shorter than in the sporadic group in the subgroups in which age was <45 years, and in which the tumors were multi-centric, bilateral, and of N1b node status. CONCLUSION: FNMTC may be considered as a separate clinical entity with a higher rate of recurrence and worse DFS than its sporadic counterpart. Furthermore, familial history of NMTC is an independent risk factor for recurrence, especially in younger patients with conventional PTC.