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OBJECTIVES: Decision-analytic models assessing the value of emerging Alzheimer's disease (AD) treatments are challenged by limited evidence on short-term trial outcomes and uncertainty in extrapolating long-term patient-relevant outcomes. To improve understanding and foster transparency and credibility in modeling methods, we cross-compared AD decision models in a hypothetical context of disease-modifying treatment for mild cognitive impairment (MCI) due to AD. METHODS: A benchmark scenario (US setting) was used with target population MCI due to AD and a set of synthetically generated hypothetical trial efficacy estimates. Treatment costs were excluded. Model predictions (10-year horizon) were assessed and discussed during a 2-day workshop. RESULTS: Nine modeling groups provided model predictions. Implementation of treatment effectiveness varied across models based on trial efficacy outcome selection (clinical dementia rating - sum of boxes, clinical dementia rating - global, mini-mental state examination, functional activities questionnaire) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6 to 5.2 years for control strategy and from 0.1 to 1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0 to 0.6 and incremental costs (excluding treatment costs) from -US$66 897 to US$11 896. CONCLUSIONS: Trial data can be implemented in different ways across health-economic models leading to large variation in model predictions. We recommend (1) addressing the choice of outcome measure and treatment effectiveness assumptions in sensitivity analysis, (2) a standardized reporting table for model predictions, and (3) exploring the use of registries for future AD treatments measuring long-term disease progression to reduce uncertainty of extrapolating short-term trial results by health-economic models.
- MeSH
- Alzheimerova nemoc * ekonomika farmakoterapie MeSH
- analýza nákladů a výnosů * MeSH
- ekonomické modely MeSH
- kognitivní dysfunkce * ekonomika MeSH
- kvalitativně upravené roky života MeSH
- lidé MeSH
- metody pro podporu rozhodování MeSH
- progrese nemoci MeSH
- výsledek terapie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- srovnávací studie MeSH
This conceptual study introduces the "virtual waiting room," an innovative, interactive, web-based platform designed to enhance the waiting experience in oncology by providing personalized, educational, and supportive content. Central to our study is the implementation of the circular entry model, which allows for non-linear navigation of health information, empowering patients to access content based on their immediate needs and interests. This approach respects the individual journeys of patients, acknowledging the diverse pathways through which they seek understanding and manage their health. The virtual waiting room is designed not only to support patients but also to facilitate stronger communication and shared understanding between patients, caregivers, and families. By providing a shared digital space, the platform enables caregivers and family members to access the same information and resources, thereby promoting transparency and collective knowledge. This shared access is crucial in managing the emotional complexities of oncology care, where effective communication can significantly impact treatment outcomes and patient well-being. Furthermore, the study explores how the circular entry model within the virtual waiting room can enhance patient autonomy and engagement by offering customized interactions based on user feedback and preferences. This personalized approach aims to reduce anxiety, improve health literacy, and prepare patients more effectively for clinical interactions. By transforming passive waiting into active engagement, the virtual waiting room turns waiting time into a meaningful, informative period that supports both the psychological and informational needs of patients and their support networks.
- MeSH
- komunikace MeSH
- lékařská onkologie * MeSH
- lidé MeSH
- nádory * psychologie terapie MeSH
- vyprávění MeSH
- vzdělávání pacientů jako téma MeSH
- zdravotní gramotnost MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
Health Technology Assessment (HTA) je klíčovým nástrojem pro objektivní hodnocení dopadu inovativních technologií na zdraví pacientů a na ekonomiku veřejného zdravotnického systému. V ČR je HTA běžně používáno pro hodnocení léčivých přípravků používaných v ambulantní péči v rozhodovacím procesu o jejich zařazení do úhrady z veřejného zdravotního pojištění, přičemž celý proces zařazení do úhrady má předem jasně definovaná pravidla zakotvená v legislativě. V současné době neexistuje podobný systematický proces pro hodnocení vakcín, jejichž zařazení do úhrady je aktuálně naopak spojeno s jen velmi málo transparentním a s politicky ovlivnitelným schvalovacím procesem. Vzhledem k významu vakcín jako klíčové preventivní intervence v oblasti veřejného zdraví se jedná o významný nedostatek v českém systému, který má však potenciál na brzké zlepšení. Po vzoru zahraničních systémů by mohlo být HTA v blízké budoucnosti zařazeno jako povinná součást procesu přiznání úhrady novým vakcínám i v ČR. Záměr na zlepšení českého sytému v této oblasti odstartoval pilotním projektem Ministerstva zdravotnictví ČR zahájeným v roce 2023 a skončit by mohl legislativním upevněním HTA vakcín v zákoně č. 48/1997 Sb., o veřejném zdravotním pojištění a o změně a doplnění některých souvisejících zákonů. Pro úspěšnou implementaci HTA vakcín bude nezbytný kontinuální rozvoj lokální metodiky farmakoekonomických analýz a rozhodovacího procesu, který umožní co nejpřesněji zachytit všechny potenciální přínosy vakcíny pro společnost. V neposlední řadě je potřeba klást důraz na systematické sbírání lokálních dat o epidemiologii infekčních onemocnění, která jsou základním kamenem pro relevantní výsledek HTA vakcín. Špatná metodika a nereprezentativní data mohou vést k podhodnocení skutečné hodnoty vakcín pro společnost. Cílem článku je nastínit aktuální a budoucí roli HTA v rozhodování o úhradě vakcín z veřejného zdravotního pojištění v ČR.
Health Technology Assessment (HTA) is a key tool for objective assessment of the impact of innovative technologies on patient health and the economics of the public health system. In the Czech Republic, HTA is commonly used for the evaluation of medicinal products in the decision-making process on their reimbursement, with the whole process having clearly defined rules set out in advance in the legislation. Currently, there is no similar systematic process for the evaluation of vaccines, which, on the contrary, currently have very little transparency and a politically influenced approval process. Given the importance of vaccines as a key preventive intervention in public health, this is a significant gap in the Czech system, but one that has the potential for early improvement. Following the example of foreign systems, HTA could be included as a mandatory part of the reimbursement approval process for new vaccines in the Czech Republic soon. The intention to improve the Czech system in this area started with a pilot project of the Ministry of Health launched in 2023 and could end with the legislative consolidation of HTA of vaccines in Act No. 48/1997 Coll. on Public Health Insurance and on Amendments and Additions to Certain Related Acts. For successful implementation of HTA vaccines, the continuous development of a local pharmacoeconomic analysis methodology and decision-making process will be essential to capture all potential benefits of the vaccine to society as accurately as possible. Finally, emphasis should be placed on the systematic collection of local data on infectious diseases epidemiology, which is the cornerstone for a relevant outcome of HTA of vaccines. Poor methodology and unrepresentative data can lead to underestimation of the true value of vaccines to society. The aim of this article is to outline the current and future role of HTA in the decision-making process regarding the reimbursement of vaccines from public health insurance in the Czech Republic
Cohort studies are a robust analytical observational study design that explore the difference in outcomes between two cohorts, differentiated by their exposure status. Despite being observational in nature, they are often included in systematic reviews of effectiveness, particularly when randomized controlled trials are limited or not feasible. Like all studies included in a systematic review, cohort studies must undergo a critical appraisal process to assess the extent to which a study has considered potential bias in its design, conduct, or analysis. Critical appraisal tools facilitate this evaluation. This paper introduces the revised critical appraisal tool for cohort studies, completed by the JBI Effectiveness Methodology Group, who are currently revising the suite of JBI critical appraisal tools for quantitative study designs. The revised tool responds to updates in methodological guidance from the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) Working Group and reporting guidance from PRISMA 2020, providing a robust framework for evaluating risk of bias in a cohort study. Transparent and rigorous assessment using this tool will assist reviewers in understanding the validity and relevance of the results and conclusions drawn from a systematic review that includes cohort studies. This may contribute to better evidence-based decision-making in health care. This paper discusses the key changes made to the tool, outlines justifications for these changes, and provides practical guidance on how this tool should be interpreted and applied by systematic reviewers.
- MeSH
- kohortové studie MeSH
- lidé MeSH
- výzkumný projekt * normy MeSH
- zkreslení výsledků (epidemiologie) * MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
BACKGROUND: Randomized clinical trials (RCTs) are essential for advancing medical knowledge, especially in pediatrics, where they provide critical evidence for safe and effective treatments. Regulatory frameworks like the Pediatric Regulation in the EU and the Best Pharmaceuticals for Children Act in the U.S. have spurred growth in pediatric RCTs. However, these clinical trials face unique challenges, including ethical complexities, recruitment difficulties, and funding limitations, which can hinder their completion. The publication of RCTs' results is equally crucial, yet not all completed RCTs share their findings, contributing to publication bias. This bias, especially in pediatric clinical trials, can distort medical evidence, impact clinical decisions, and potentially compromise patient safety. METHODS: We conducted a cross-sectional analysis of pediatric RCTs registered on ClinicalTrials.gov between 2011 and 2013, with completion status by 2017. Inclusion criteria included clinical trials with drug interventions in participants aged 0-17 and randomization. Data on RCTs characteristics, including phase, funding source, participant age, and enrollment, were extracted. RCTs completion status was assessed, and reasons for incompletion were categorized. Publication status was evaluated through registry and manual searches on PubMed and Google Scholar. Statistical analyses, including logistic regression, were performed to identify factors associated with trial incompletion and non-publication. RESULTS: Out of 2875 pediatric clinical trials reviewed, 1088 met the inclusion criteria. Among these, 16.54% were uncompleted, primarily due to patient accrual issues (32.22%). Academic sponsors funded 48.53% of trials, and industrial sponsors funded 45.13%. Of 908 completed RCTs, 58.48% posted results in registries, while 70% had results published in peer-reviewed journals. Industrially funded RCTs were more likely to post results, but academic RCTs had a higher scientific publication rate. The median time to first result publication in registries was 21 months, with significant delays linked to the trial phase, funding, and participant enrolment size. CONCLUSION: Our study highlights significant challenges in pediatric RCTs, including high incompletion rates and delays in result reporting. Ethical, regulatory, and logistical barriers hinder progress, impacting evidence transparency. Strengthened regulatory oversight and enhanced compliance are essential to improve pediatric research outcomes and ensure timely dissemination of RCTs results.
- Publikační typ
- časopisecké články MeSH
BACKGROUND: Type 2 diabetes and prediabetes represent significant global health challenges, with physical activity (PA) being essential for disease management and prevention. Despite the well-documented benefits, many individuals with (pre)diabetes remain insufficiently active. General practitioners (GP) provide an accessible platform for delivering interventions; however, integrating PA interventions into routine care is hindered by resource constraints. OBJECTIVES: The ENERGISED trial aims to address these barriers through an innovative GP-initiated mHealth intervention combining wearable technology and just-in-time adaptive interventions. METHODS: The ENERGISED trial is a pragmatic, 12-month, multicentre, randomised controlled trial, assessing a GP-initiated mHealth intervention to increase PA and reduce sedentary behaviour in patients with type 2 diabetes and prediabetes. The primary outcome is daily step count, assessed via wrist-worn accelerometry. The primary analysis follows the intention-to-treat principle, using mixed models for repeated measures. Missing data will be handled under the missing-at-random assumption, with sensitivity analyses exploring robustness through reference-based multiple imputation. The trial incorporates the estimand framework to provide transparent and structured treatment effect estimation. DISCUSSION: This statistical analysis plan outlines a robust approach to addressing participant non-adherence, protocol violations, and missing data. By adopting the estimand framework and pre-specified sensitivity analyses, the plan ensures methodological rigour while enhancing the interpretability and applicability of results. CONCLUSIONS: The ENERGISED trial leverages innovative mHealth strategies within primary care to promote PA in individuals with (pre)diabetes. The pre-specified statistical framework provides a comprehensive guide for analysing trial data and contributes to advancing best practices in behavioural intervention trials for public health. TRIAL REGISTRATION: ClinicalTrials.gov NCT05351359 . Registered on April 28, 2022.
- MeSH
- akcelerometrie MeSH
- cvičení * MeSH
- diabetes mellitus 2. typu * terapie psychologie diagnóza MeSH
- fitness náramky MeSH
- lidé MeSH
- multicentrické studie jako téma MeSH
- nositelná elektronika MeSH
- pragmatické klinické studie jako téma MeSH
- praktické lékařství * metody MeSH
- prediabetes * terapie psychologie diagnóza MeSH
- sedavý životní styl * MeSH
- telemedicína * statistika a číselné údaje MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- protokol klinické studie MeSH
Tento přehledový článek se zaměřuje na základní principy technologií umělé inteligence (AI), možnosti jejich využití v medicíně a na příklady aplikací, které již byly začleněny do klinické praxe. Diskutuje také klíčové výzvy včetně etických otázek, jako je ochrana soukromí pacientů, algoritmická bias a problém transparentnosti modelů AI. Článek zdůrazňuje nutnost integrace AI do medicíny způsobem, který zajistí bezpečnost a důvěryhodnost, a současně vyzdvihuje význam vzdělávání zdravotnických profesionálů v oblasti AI. Umělá inteligence nabízí potenciál ke zlepšení přesnosti diagnostiky, efektivity péče a podpory při klinickém rozhodování, přičemž optimálních výsledků lze dosáhnout spoluprací mezi lékaři a systémy AI.
This review article focuses on the fundamental principles of artificial intelligence (AI) technologies, their utilisation in medicine, and examples of applications that have already been incorporated into clinical practice. It also discusses key challenges, including ethical issues such as patient data privacy, algorithmic bias, and the transparency problem of AI models. The article emphasizes the necessity of integrating AI into medicine in a manner that ensures safety and trustworthiness, while underscoring the importance of educating healthcare professionals about AI. Artificial intelligence offers the potential to enhance diagnostic accuracy, the efficiency of care, and support for clinical decision-making, with optimal outcomes being achieved through collaboration between physicians and AI systems.
- MeSH
- algoritmy MeSH
- lékařství * MeSH
- lidé MeSH
- nefrologie MeSH
- umělá inteligence * etika MeSH
- velké jazykové modely MeSH
- zabezpečení počítačových systémů MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- přehledy MeSH
Corneal dystrophies are a group of predominantly rare inherited disorders. They are by definition bilateral, relatively symmetrical, and without systemic involvement, affecting corneal transparency and/or refraction. Traditional classification of corneal dystrophies is based on slit-lamp appearance, affected corneal layer and histological features. Molecular genetics has provided ultimate proof for the existence of distinct corneal dystrophies and discarded duplicates in their terminology. Currently, there are at least 16 genes with identified pathogenic variants implicated in corneal dystrophies. Herein, we summarise contemporary knowledge on genotype-phenotype correlations of corneal dystrophies, including a critical review of some reported variants, along with the understanding of the underlying pathogenic dystrophic process; essential knowledge for the development of targeted therapies.
Eccrine hidrocystomas are rare, benign cystic lesions that usually affect the scalp, cheeks, and eyelids. They are thought to originate from the sweat glands. These lesions can be single or many in nature and frequently worsen in the summer from increased perspiration. They are caused by dilated ducts of eccrine sweat glands. Clinically, they seem like small, transparent cystic lesions that are painless, and they usually affect middle-aged or older people. However, a histological study is necessary to make a final diagnosis. We present a case of a 35-year-old woman who had an epidermal inclusion cyst first identified as a single, painless cystic growth on her right lower eyelid.
- MeSH
- diferenciální diagnóza MeSH
- dospělí MeSH
- ekrinní žlázy patologie MeSH
- epidermální cysta * diagnóza patologie MeSH
- hidrocystom * diagnóza patologie MeSH
- lidé MeSH
- nádory očního víčka diagnóza patologie MeSH
- nádory potních žláz * diagnóza patologie MeSH
- Check Tag
- dospělí MeSH
- lidé MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- kazuistiky MeSH
- přehledy MeSH
Cíl: Retrospektivně zhodnotit anatomickou a funkční úspěšnost chirurgického řešení rhegmatogenního odchlípení sítnice (ROS) na jediném, posledním vidoucím oku. Materiál a metody: Soubor tvoří 28 očí 28 pacientů, z toho 19 (68 %) mužů, průměrného věku 46 let operovaných jedním chirurgem pro ROS na Oční klinice FN a LF MU Brno od 1. 7. 2019 do 30. 4. 2023 kryochirurgickou technikou anebo 25G+ PPV. U 11 pacientů byla provedena 25G+ PPV s našitím preekvatoriální cerkláže. V anamnéze pacientů bylo přípustné tupé poranění oka a nekomplikovaná operace šedého zákalu s implantací zadněkomorové nitrooční čočky. Příčinou ROS byla/y retinální trhlina/y bez ohledu na jejich počet a lokalizaci. Transparentnost předního segmentu oka umožňovala spolehlivou vizualizaci zadního segmentu. Přípustná byla předoperační proliferativní vitreoretinopatie (PVR) stupně A-D2. Vyloučeni byli pacienti, s pronikajícím očním poraněním v anamnéze. Pacienti byli hodnoceni 1–3 měsíce po PPV. Operace byla anatomicky úspěšná, pokud byla sítnice přiložená v plném rozsahu. U každého pacienta byla hodnocena výsledná zraková ostrost (ZO) na Snellenově optotypu. Pro číselné vyjádření výsledků byl použit aritmetický průměr a numerické hodnoty byly rovněž vyjádřeny v procentech. Poněvadž nebyly vzájemně porovnávány různé skupiny, nebylo potřeba použít žádný statistický test. Výsledky: U 27 pacientů (97 %) jsme dosáhli přiložení sítnice v celém rozsahu, u 1 (3 %) pacienta zůstala sítnice odchlípená a konstatovali jsme anatomický neúspěch léčby. 9 (32%) pacientů dosáhlo ZO ≥ 4/8. Závěr: Kryochirurgickou techniku s využitím episklerálně fixovaných cerklážních pásků a plomb, 25G+ PPV a případně i jejich vzájemnou kombinaci považujeme za vhodné způsoby řešení ROS na jediném, posledním vidoucím oku.
Aim: To retrospectively evaluate the anatomical and functional success of surgical treatment of rhegmatogenous retinal detachment (RRD) in the only remaining seeing eye. Material and methods: The study included 28 eyes of 28 patients, 19 (68%) of whom were men, with an average age of 46 years. They were operated on by a single surgeon for RRD at the Eye Clinic of the University Hospital and Faculty of Medicine, Masaryk University in Brno, from July 1, 2019, to April 30, 2023, using cryosurgical techniques and/or 25G+ pars plana vitrectomy (PPV). In 11 patients, 25G+ PPV was performed with the application of a pre-equatorial cerclage. The Blunt ocular trauma and uncomplicated cataract surgery with implantation of a posterior chamber intraocular lens were admissible within the patient histories. The cause of RRD was retinal tear(s) regardless of their number and location. The transparency of the anterior segment of the eye enabled reliable visualization of the posterior segment. Preoperative proliferative vitreoretinopathy (PVR) grades A-D2 were acceptable. Patients with a history of penetrating eye injury were excluded. Patients were evaluated 1-3 months after the performance of PPV. The surgery was considered anatomically successful if the retina was completely reattached. Each patient's final visual acuity (VA) was assessed using a Snellen chart. Numerical results were expressed as arithmetic means and percentages. Since the different groups were not compared, no statistical tests were needed. Results: Retinal reattachment was achieved in 27 patients (97%), while 1 patient (3%) experienced retinal detachment, resulting in anatomical failure of the treatment. 9 patients (32%) achieved VA ≥ 4/8. Conclusion: We consider cryosurgical techniques using episclerally fixed cerclage bands and buckles, 25G+ PPV, and possibly a combination thereof, to be suitable methods for treating RRD in the only remaining seeing eye.
