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Global, regional, and national disability-adjusted life-years (DALYs) for 359 diseases and injuries and healthy life expectancy (HALE) for 195 countries and territories, 1990-2017: a systematic analysis for the Global Burden of Disease Study 2017.

BACKGROUND: How long one lives, how many years of life are spent in good and poor health, and how the population's state of health and leading causes of disability change over time all have implications for policy, planning, and provision of services. We comparatively assessed the patterns and trends of healthy life expectancy (HALE), which quantifies the number of years of life expected to be lived in good health, and the complementary measure of disability-adjusted life-years (DALYs), a composite measure of disease burden capturing both premature mortality and prevalence and severity of ill

Use of real-world evidence in the Medicare Drug Price Negotiation Program: A checklist for the Centers for Medicare and Medicaid Services and manufacturers.

Under the Inflation Reduction Act's (IRA's) - Medicare Drug Price Negotiation Program, the Centers for Medicare & Medicaid Services' (CMS's) "maximum fair price" must be informed by evidence on factors such as therapeutic advance of the selected drug compared with its alternative, comparative effectiveness across clinical and patient-reported outcomes, the impact on specific populations, and the ability to address unmet medical needs. This paper describes how real-world evidence could improve CMS decision-making and creates a best practices checklist to help CMS evaluate the quality of any man

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… Introduction With the passage of the Inflation Reduction Act (IRA), the federal government can now “negotiate” prices directly with drug manufacturers for certain high-cost, sole-source drugs. 1 , 2 The law states that the Centers for Medicare and Medicaid Services (CMS) will negotiate a “maximum fair …

Искусственные разведывательные методологии для ранней диагностики точности и индивидуализированных терапевтических стратегий в нейро-офтальмических и нейродегенеративных патологиях.

Совершенствование нейроимации, в частности методов диффузной магнитной резонансной съемки (MRI) и молекулярной съемки с помощью позитронной томографии (ПТЭ), значительно повысило раннее обнаружение биомаркеров в нейродегенеративных и нейро-офтальмических расстройствах. К ним относятся болезнь Альцгеймера, болезнь Паркинсона, множественный склероз, оптика нейромиелита и болезнь антител миэлина олигодендроцита гликопротеина. В настоящем обзоре подчеркивается преобразующая роль современных методов диффузии MRI - Неевритная ориентация - диспергация и интерпретация плотности и дифлюстрация Куртоза

Surrogate Biomarkers in Gene Therapy for Orphan Diseases: Validation, Application, and Regulatory Aspects.

The development of gene therapies for rare hereditary disorders is hindered by small patient cohorts, incomplete characterization of natural disease history, and the impracticality of conducting long-term clinical trials. Surrogate biomarkers-quantifiable indicators predictive of clinical outcomes-represent a promising strategy to accelerate the evaluation of therapeutic efficacy. This review examines the role of surrogate endpoints in gene therapy, outlining essential validation criteria, including biological plausibility, analytical reproducibility, and clinical predictive value. Regulatory

Pharmacovigilance in Cell and Gene Therapy: Evolving Challenges in Risk Management and Long-Term Follow-Up.

Cell and gene therapies, including CAR T-cells, CRISPR-based genome editing, and next-generation viral and non-viral delivery platforms, are transforming treatment paradigms across cancer, rare genetic disorders, immune dysregulation, and neurodegenerative disease. These therapies offer curative potential but also present safety challenges owing to prolonged biological activity, systemic immune engagement, and lasting genomic alterations. This review examines the range of related toxicities, including immune complications, genotoxicity, and organ-specific effects, with attention to atypical pr

The Danish health care system and epidemiological research: from health care contacts to database records.

Denmark has a large network of population-based medical databases, which routinely collect high-quality data as a by-product of health care provision. The Danish medical databases include administrative, health, and clinical quality databases. Understanding the full research potential of these data sources requires insight into the underlying health care system. This review describes key elements of the Danish health care system from planning and delivery to record generation. First, it presents the history of the health care system, its overall organization and financing. Second, it details d

Известная эволюция полимер-связывающего пептида LCI для улучшения связывания полипропилена.

Функционализация поверхностей полимеров с помощью полимерных связывающих пептидов предоставляет значительные возможности для направленногоImmobilization ферментов, биоактивных пептидов и антигенов. Применение полимерных связывающих пептидов в качестве адгезивов требует надежного и стабильного связывания при условиях процесса. Молекулярные режимы взаимодействия между материалами поверхностей, пептидами и растворителем часто не понимаются на уровне, который позволяет (semi-) рационально проектировать полимерные связывающие пептиды, что мешает полному использованию их потенциала. Концепция направ

SARS-CoV-2 Persistent Viral Shedding in the Context of Hydroxychloroquine-Azithromycin Treatment.

SARS-CoV-2 nasopharyngeal shedding contributes to the spread of the COVID-19 epidemic. Among 3271 COVID-19 patients treated at the Hospital University Institute Méditerranée Infection, Marseille, France from 3 March to 27 April 2020, tested at least twice by qRT-PCR, the median SARS-CoV-2 nasopharyngeal shedding duration was 6 days (range 2-54 days). Compared with short shedders (qRT-PCR positivity < 10 days), 34 (1.04%) persistent shedders (qRT-PCR positivity ≥ 17 days; mean ± SD: 23.3 ± 3.8 days) were significantly older, with associated comorbidities, exhibiting lymphopenia, eosinopenia, in

Recent advances in precision oncology research.

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The value-for-money assessment and funding arrangements for high-priced drugs in an era of uncertainty: a comparative analysis of national health technology assessment agencies in South Korea, England, Australia, and Canada.

BackgroundInnovative health technologies have increasingly emerged as a promising solution for patients with untreatable or challenging conditions. However, these technologies often come with expensive costs and limited evidence at the time of launch. This study assessed how these high-priced drugs with limited evidence were appraised and introduced in South Korea, England, Australia, and Canada, where cost-effectiveness analysis (CEA) generally plays a central role in pricing and reimbursement decisions.MethodsThe study analysed 22 high-priced drugs (24 indications) introduced in South Korea,

Последствия применения обеспеченных предписаний для амбулаторного использования прегабалина во Франции: региональная оценка во Французском Нувеле-Аквитане.

AIM: Французские власти разрешили использовать в мае безопасные рецепты на догабалин. 2021. Цель этого исследования заключалась в оценке воздействия этой меры на использование и неправильное использование догабалинов в Нувеле-Акитане, юго-западном французском регионе, расположенном вокруг six million Жители. МЕТОДЫ: Это исследование было проведено на основе региональных баз данных французской системы медицинского страхования &quot; Нувел-Аквитейн &quot; . В нее входят все лица, которые получают по крайней мере one Возмещение расходов на догабалин между 24 Май 2020 и 24 Май 2022. Мы проанализир

HealthTree Cure Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles and Accelerate Research in Multiple Myeloma.

PurposeThe study of rare diseases, such as multiple myeloma (MM), often experiences unique research hurdles that can delay or prevent lifesaving discoveries. HealthTree Cure Hub is a first-in-class software program designed to overcome these potential research hurdles.MethodsWe assessed whether HealthTree Cure Hub improved four commonly experienced research hurdles such as (1) small numbers of patient accrual to clinical trials and research studies, (2) shallow and isolated data sets, (3) high costs to answer research questions, and (4) lack of long-term follow-up patient data.ResultsAs of Jun

The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health.

Since late 2020, the Canadian Agency of Drugs and Technologies in Health (CADTH) has been using a threshold of $50,000 (CAD) per quality-adjusted life-year (QALY) for both oncology and non-oncology drugs. When used for oncology products, this threshold is hypothesized to have a higher impact on the time to access these drugs in Canada. We studied the impact of price reductions on time to engagement and negotiation with the pan-Canadian Pharmaceutical Alliance for oncology drugs reviewed by CADTH between January 2020 and December 2022. Overall, 103 assessments reported data on price reductions

The Persistence of Stigma and Other Challenges Experienced by Medical Cannabis Users: A Thematic Synthesis of Qualitative Studies.

BackgroundThe legislation, practices, and guidance shaping patient and professional access to medical cannabis (MC) for managing chronic pain and other conditions vary widely across jurisdictions. Despite promising evidence of effectiveness, challenges persist for those trying to access or prescribe MC. Little is known about how patients navigate these complex environments. We aimed to understand the evidence related to personal lived experiences associated with using MC for chronic pain and other conditions in negotiating these challenges.SummaryWe used a framework by Thomas and Harden for co

Primed for global coronavirus pandemic: Emerging research and clinical outcome.

The global effort to combat and contain the coronavirus disease 2019 (COVID-19) caused by the recently discovered severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is now proceeding on a war footing. The world was slow to react to the developing crisis, but once the contours of the impending calamity became evident, the different state and non-state actors have raced to put their act together. The COVID-19 pandemic has blatantly exposed the shortcomings of our healthcare system and the limitations of medical science, despite considerable advances in recent years. To effectively tack

Repurposing drugs for highly prevalent diseases: pentoxifylline, an old drug and a new opportunity for diabetic kidney disease.

Diabetic kidney disease is one of the most frequent complications in patients with diabetes and constitutes a major cause of end-stage kidney disease. The prevalence of diabetic kidney disease continues to increase as a result of the growing epidemic of diabetes and obesity. Therefore, there is mounting urgency to design and optimize novel strategies and drugs that delay the progression of this pathology and contain this trend. The new approaches should go beyond the current therapy focussed on the control of traditional risk factors such as hyperglycaemia and hypertension. In this scenario, d

10th Trends in Medical Mycology Held on 8 to 11 October 2021, Aberdeen, Scotland, Organized by the European Confederation of Medical Mycology (ECMM).

Plenary Sessions: [...].

Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand.

BackgroundPublic payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec). CDR has been criticized for negative recommendations for orphan drugs and contributing to delays in patient access to these drugs. However, it is unclear how CDR makes recommendations for orphan drugs and the role clinical and economic factors play in decision making. The objective of the present study was to analyze the basis for CDR orphan drug recommendations and to compare recommendations to those in other

Искусственный интеллект и наука о вкладе пациента: точка зрения людей с множественным склерозом.

Искусственный интеллект (АИ) может сыграть жизненно важную роль в переходе к прогнозной, профилактической и индивидуализированной медицине при условии, что мы руководствуемся наукой с помощью и с помощью пациента. Меры по достижению результатов, о которых сообщают пациенты (ПРОМ), представляют собой уникальную возможность для получения практических знаний от людей, живущих в условиях здоровья, и обеспечения их научной актуальности для всех других заинтересованных сторон. Несмотря на это, использование стандартизированных результатов, включая ПРОМ, в рамках научно-исследовательской системы и си

Patient-reported outcomes following autologous stem cell transplant for patients with multiple myeloma.

We evaluated changes in patient-reported outcomes and cognitive function from pre- to 3-6 months post-treatment among 42 newly diagnosed patients with multiple myeloma undergoing transplant with complete data using PROMIS-29. There were statistically significant improvements in physical (p < .001) and mental health (p < .001) but not cognition from pre-treatment to 3-6 month follow-up. Similar results were seen within age or comorbidity strata. Patients with myeloma undergoing transplant experienced generally improved short-term health outcomes with no significant declines in cognition.

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… CONFLICTS OF INTEREST The article was prepared as part of one of the author's (REJ) official duties as employees of the US Federal Government. …