Результаты поиска

Global welfare burden of hematologic malignancies from 2015 to 2023.

Hematologic malignancies cause premature mortality and disability across the life course, but their welfare burden is difficult to compare across countries. We monetized global burden of disease (GBD) 2023 disability-adjusted life years (DALYs) for major hematologic malignancies in 188 countries from 2015 to 2023 using a value of lost welfare framework incorporating purchasing power parity adjusted gross domestic product (GDP), income transfer, age-specific valuation, and discounting. Global leukemia-related value of lost welfare (VLW) increased from US$1.40 trillion to US$1.70 trillion, while

Estimating the shares of the value of branded pharmaceuticals accruing to manufacturers and to patients served by health systems.

Previous studies have estimated that patients served by health systems accrue 59-98% of the value generated by new pharmaceuticals. This has led to questions about whether sufficient returns accrue to manufacturers to incentivize socially optimal levels of R&D. These studies have not, however, fully reflected the health opportunity costs imposed by payments for branded pharmaceuticals. We present a framework for estimating how the value generated by new branded pharmaceuticals is shared. We quantify value in net health effects and account for benefits and health opportunity costs in the patent

Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to ca

The Role of Real-World Evidence in UK Reimbursement: Case Study of Lenalidomide in Myelodysplastic Syndrome Deletion 5q.

BackgroundUncertainty within cost-effectiveness analysis, often driven by lack of mature data from large clinical trials, plays a key role in decisions made by the National Institute for Health and Care Excellence (NICE), particularly for early access medicines and orphan drugs.ObjectivesIn this case study, we used real-world evidence to address the uncertainty in the cost-effectiveness case for lenalidomide in transfusion-dependent low- and intermediate-1-risk myelodysplastic syndrome (MDS) deletion 5q [del(5q)], affecting a small but unique subpopulation with an orphan disease.MethodsAs part

Экономичность atumumumab для лечения повторной формы множественных склерозов в Объединенных Арабских Эмиратах.

Множественный склероз (MS) — аутоиммунное заболевание центральной нервной системы, связанное с обезвоживанием и воспалением. Повторные формы MS (RMS) охватывают всех пациентов, у которых MS испытывает рецидивы. Несмотря на то, что в настоящее время не существует лекарств для лечения MS, внедрение нескольких методов лечения, позволяющих регулировать заболевания (DMT), значительно уменьшило риск рецидива и инвалидности у пациентов с MS. Существует острая потребность в легком для использования DMT, который можно использовать на раннем этапе лечения.ЦЕЛИ: Данное исследование было направлено на оце

Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers.

BackgroundThere has been an increase in 'risk sharing' schemes for pharmaceuticals between healthcare institutions and pharmaceutical companies in Europe in recent years as an additional approach to provide continued comprehensive and equitable healthcare. There is though confusion surrounding the terminology as well as concerns with existing schemes.MethodsA literature review was undertaken to identify existing schemes supplemented with additional internal documents or web-based references known to the authors. This was combined with the extensive knowledge of health authority personnel from

Anticancer drug prices and clinical outcomes: a cross-sectional study in Italy.

ObjectiveTo investigate whether the prices of new anticancer drugs correlated with their relative benefit despite negotiation.DesignRetrospective cross-sectional study correlating new anticancer drugs prices with clinical outcomes.SettingWe did a retrospective cross-sectional study including all new anticancer drugs approved by the European Medicines Agency (EMA) (2010-2016) and reimbursed in Italy.Main outcomes and measuresInformation on clinical outcomes-in terms of median overall survival (OS), median progression-free survival (PFS) and objective response rate (ORR)-was extracted from pivot

Cost-Effectiveness of Post-Autotransplant Lenalidomide in Persons with Multiple Myeloma.

Considerable data indicate post-transplant lenalidomide prolongs progression-free survival and probably survival after an autotransplant for multiple myeloma (MM). However, optimal therapy duration is unknown, controversial and differs in the EU and US. We compared outcomes and cost-effectiveness of 3 post-transplant lenalidomide strategies in EU and US settings: (1) none; (2) until failure; and (3) 2-year fixed duration. We used a Markov decision model, which included six health states and informed by published data. The model estimated the lenalidomide strategy given to failure achieved 1.06

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,

Нейропсихологические аспекты расстройств контроля импульсов в болезни Паркинсона.

Синдромы импульсного контроля (ИДД), такие, как чрезмерные азартные игры, навязчивое сексуальное поведение, поедание бингов, навязчивые покупки, а также синдром регулирования дофамина, могут возникать как изнурительное нервно-психическое осложнение болезни Паркинсона (PD). Хотя патофизиология не в полной мере понятна, она, вероятно, включает мезолимбичную допаминергическую чрезмерную оптимизацию в сочетании с факторами уязвимости, связанными с заболеваниями, в плане поощрения, мотивации и ингибиционных сетей контроля. В настоящем описательном обзоре резюмируются данные о нейропсихологических,

Совпадение в полном тексте источника
… In contrast, another study found no differences between PD patients with and without ICDs on the Delay Discounting Task, which measures impulsive decision-making and preference for immediate over delayed rewards ( Hlavatá et al., 2020 ). … Temporal discounting Temporal discounting, also known as delay discounting, refers to the preference for smaller immediate rewards over larger delayed rewards. Tasks typically give hypothetical options (e.g., “Would you prefer 5€ today or 20€ in 6 months?”) …

Comparison of health technology assessment for new medicines in France and England: an example based on ixazomib for patients with relapsed or refractory multiple myeloma.

Introduction: The appraisal of medicines is often a complex and iterative process. We compared the health technology assessment (HTA) process in England and France taking as a case study the example of ixazomib for multiple myeloma. Methods: We undertook an analysis of eight relevant published documents identifed from the websites of the French and English HTA bodies (HAS and NICE, respectively). We analyse patients' availability of ixazomib resulting in the different stages of the appraisal process. Results: We identified differences in the assessment, one of these being the use of an apprais

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures.

Rare cancers are defined by an annual incidence of fewer than 6 per 100,000. Bearing similarities to rare diseases, they are associated with substantial health inequalities due to diagnostic complexity and delayed access to innovative therapies. This situation is further aggravated in Southeastern European countries like Bulgaria, where limited public resources and expertise underscore the need for additional policy and translational research on rare cancers. This study aimed to explore the availability and access to orphan drugs for rare cancers in Bulgaria for the period of 2020-2023. We cro

Developing shortcut algorithms for value-based pricing of pharmaceuticals.

Value-based pricing (VBP) involves regulating the reimbursement or pricing of pharmaceuticals based on their therapeutic value. We adopt VBP as the policy context for this study. Unlike alternative approaches, VBP depends on an explicit relationship between price and incremental health outcomes and costs. This dependence makes VBP decisions especially sensitive to uncertainty about downstream costs and outcomes. A central question is whether—and how far—these components must be modeled beyond the trial period. A key gap is that the literature provides limited, generalizable conditions for when

Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval.

ImportanceNumerous cancer drugs have received accelerated approval from the US Food and Drug Administration (FDA) based on clinical trial outcomes that are otherwise not acceptable for traditional FDA approval; the accelerated approval process allows outcomes based on surrogate measures that are only reasonably likely to estimate clinical benefits. In England, the National Institute for Health and Care Excellence (NICE) evaluates the clinical benefits and cost-effectiveness of drugs after they have received regulatory approval and issues recommendations regarding their coverage in the National

Valorising and creating access to innovative medicines in the European union.

This Perspective describes (a) the current situation, (b) challenges and initiatives, (c) and formulates recommendations to valorize and create access to innovative medicines in the EU. We are currently still far away from optimal assessment of value for money in the EU. On the one hand, valorizing innovative medicines involves a local appraisal by health technology assessment (HTA) bodies and competent authorities about the value for money, the budget impact, and the local medical need that can be filled with new medicines. Therefore, local priorities and national health care policy environme

Глобальное, региональное и национальное экономическое бремя гематологовых магнестов1990-2021) с прогнозами 2050.

Гематологические колебания (ГМ) обусловливают значительные затраты на здравоохранение и производительность труда во всем мире. Однако различия в экономическом воздействии между регионами и странами по-прежнему недостаточно изучены. Цель этого исследования заключалась в оценке глобального, регионального и национального экономического бремени ТМ и его подтипов (лейкемии, лимфомы без ходжкина, множественной миэломы и Ходжкинской лимфомы) 1990 в адрес 2021, с прогнозами 2050. Методические данные о глобальном бремени болезней 2021 Исследование использовалось для оценки экономического бремени ТМ с и

Patient perceptions of copay card utilization and policies.

BackgroundCopay cards are intended to mitigate patient out-of-pocket (OOP) expenses. This qualitative, exploratory focus group study aimed to capture patient perceptions of copay cards and copay adjustment programs (CAPs; insurers' accumulator and maximizer policies), which redirect the copay card utilization benefits intended for patients' OOP expenses.MethodsPatients with chronic conditions were recruited through Janssen's Patient Engagement Research Council program. They completed a survey and attended a live virtual session to provide feedback on copay cards.ResultsAmong 33 participants (m

Health economic evidence for the use of molecular biomarker tests in hematological malignancies: A systematic review.

ObjectivesMolecular biomarker tests can inform the clinical management of genomic heterogeneous hematological malignancies, yet their availability in routine care largely depends on the supporting health economic evidence. This study aims to systematically review the economic evidence for recent molecular biomarker tests in hematological malignancies.MethodsWe conducted a systematic search in five electronic databases for studies published between January 2010 and October 2020. Publications were independently screened by two reviewers. Clinical study characteristics, economic methodology, and

INvestigational Vertebroplasty Efficacy and Safety Trial (INVEST): a randomized controlled trial of percutaneous vertebroplasty.

BACKGROUND: The treatment of painful osteoporotic vertebral compression fractures has historically been limited to several weeks of bed rest, anti-inflammatory and analgesic medications, calcitonin injections, or external bracing. Percutaneous vertebroplasty (the injection of bone cement into the fractured vertebral body) is a relatively new procedure used to treat these fractures. There is increasing interest to examine the efficacy and safety of percutaneous vertebroplasty and to study the possibility of a placebo effect or whether the pain relief is from local anesthetics placed directly on

The Challenges of Outcomes-Based Contract Implementation for Medicines in Europe.

ObjectivesThe aim was to outline the challenges of implementing outcomes-based contracts (OBCs) in Europe.MethodsA scoping review was conducted, building on the searches of a previous systematic review and updating them for December 2017 until May 2021. The combined results were screened, based on inclusion and exclusion criteria. All identified studies published in the English language that described specific OBC schemes for medicines in European countries were included. Insights into the challenges of OBCs were extracted and analysed to develop a conceptual framework.ResultsTen articles from