Результаты поиска

Plain language summaries supporting patient involvement: lessons and guidance from HTAi Patient and Citizen Involvement Interest Group (PCIG).

Patient involvement is an increasingly recognized cornerstone of effective Health Technology Assessment (HTA). Clear, accessible information empowers patient organizations to contribute meaningfully to HTA. Therefore, an international Summary Information for Patient Groups template was developed to provide plain language summaries of new medicines being assessed. Pilots using the template were conducted in Australia in 2021 and England in 2022, providing a trial within the HTA process. In Australia, the Consumer Evidence and Engagement Unit (CEEU) used a workshop and survey, together with key

Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007-13) and prospective (2014-18) MIDAS sales data analysis.

BackgroundHealth Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians suffer from a rare disease. Drugs treating rare diseases (DRDs) are also known as orphan drugs. While Canada is currently developing an orphan drug framework, in the United States (US), the Orphan Drug Act (ODA) of 1983 established incentives for the development of orphan drugs. This study measured total annual expenditure of orphan drugs in Canada (2007-13) and estimated futur

Regulatory insights on gene therapies, adeno-associated virus-based gene therapies, cell/tissue-based products, and medical care/practice in cell and gene therapies: Report from the 7th Asia partnership conference - April 25, 2024.

The 7th Asia Partnership Conference of Regenerative Medicine (APACRM) was held both in person and online on April 25, 2024, to promote the regulatory harmonization of regenerative medicine products across Asia. Recognizing domestic regulatory guidelines and their underlying rationales within each country and region is an important initial step toward achieving harmonious regulation. The 7th APACRM featured open dialogues on the non-clinical evaluations of adeno-associated virus gene therapy products, regulation of gene therapies, considerations for conducting clinical trials, and provision of

The Value of Pharmaceutical Industry-Sponsored Patient Registries in Oncology Clinical Research.

In May 2019, the US Food and Drug Administration (FDA) released the Framework for FDA's Real-World Evidence (RWE) Program, a draft guidance to evaluate the potential use of real-world data in facilitating regulatory decisions. As a result, pharmaceutical companies and medical communities see patient registries, which are large, prospective, noninterventional cohort studies, as becoming increasingly important in providing evidence of treatment effectiveness and safety in clinical practice. Patient registries are designed to collect longitudinal clinical data on a broad population to address cri

The impact of government reimbursement negotiation on targeted anticancer medicines use and cost in China: A cohort study based on national health insurance data.

BackgroundHigh prices of targeted anticancer medicines (TAMs) result in financial toxicity for patients and the health insurance system. How national price negotiation and reimbursement policy affect the accessibility of TAMs for cancer patients remains unknown.MethodsIn this population-based cohort study, we used national health insurance claims data in 2017 and identified adult patients with cancer diagnoses for which price-negotiated TAMs were indicated. We estimated the half-month prevalence of price-negotiated TAMs use before and after the policy implementation in September 2017. We calcu

Linzagolix, with and without add-back therapy, in women with endometriosis-associated pain: results from EDELWEISS 6, a double-blind randomized extension and withdrawal study.

STUDY QUESTION: Was the efficacy of linzagolix, observed at 6 months in women with endometriosis-associated pain, maintained when administered for an additional 6 months? SUMMARY ANSWER: Efficacy from this extension study complements the results of the main trial, by showing that linzagolix is effective at reducing endometriosis-associated pain for a majority of women. WHAT IS KNOWN ALREADY: In the pivotal EDELWEISS 3 randomized controlled trial, the efficacy and safety of 24 weeks of once-daily oral linzagolix (a GnRH antagonist) were reported in women with endometriosis-related pain. Risks

Ten years of AMNOG from an oncological perspective: new horizons and continuing expansion.

Purpose2021 marks the tenth anniversary of the AMNOG process and brought with it a new German administration-two good reasons to take stock of where we stand today, what has been achieved so far, and how the path of early benefit assessments in Germany should continue.ResultsFrom the perspective of manufacturers of cancer drugs, the AMNOG process, as a constantly evolving system, has for the most part proved itself-which does not mean that there is no longer room for improvement. Significant improvements have been achieved in the area of early consultation of medical societies regarding the se

Results of a quality control on non-interventional studies.

Non-interventional studies (NIS) have for decades been an established part of post-authorisation medicinal research. As early as the mid-nineties, there were at least rudimentary demands for controllable data quality. Beginning with the recommendations of the Federal Institute for Drugs and Medical Devices (BfArM) on the execution of non interventional (observational) studies of 1998 and finally with the guidelines and recommendations for ensuring Good Epidemiological Practice (GEP), with the VFA (Verband der forschenden Arzneimittelhersteller [German Association of Research-Based Pharmaceutic

Real-world survival of US patients with intermediate- to high-risk myelofibrosis: impact of ruxolitinib approval.

The Janus kinase inhibitor ruxolitinib is approved for the treatment of myelofibrosis (MF) and improved overall survival (OS) versus control therapy in the phase 3 COMFORT trials. The aim of this retrospective analysis was to examine the real-world impact of ruxolitinib on OS in patients with MF. The US Medicare Fee-for-Service claims database (parts A/B/D) was used to identify patients with ≥ 1 inpatient or ≥ 2 outpatient claims with an MF diagnosis (January 2010-December 2017). Eligible patients with MF were ≥ 65 years old (intermediate-1 or higher risk based on age). Patients were divided i

Financial toxicity in female patients with breast cancer: a national cross-sectional study in China.

PurposeTo quantify financial toxicity of female patients with breast cancer in China and investigate its factors and patients' coping strategies.MethodsThe Comprehensive Score for Financial Toxicity (COST) is defined by using a structured questionnaire containing 12 items measuring perceived affordability of healthcare services, with the range of scoring of which being from 0 to 44 (higher score indicates lower financial toxicity). From January to March 2021, a total of 664 female patients diagnosed with stage 0-IV breast cancer were recruited from 33 public tertiary cancer hospitals located i

Cancer treatment-related financial toxicity in Japan: a scoping review.

Financial toxicity during cancer survival has been studied mainly in the United States; 47-49% of cancer survivors reported financial hardships and 12-63% reported debt owing to treatment costs. Financial toxicity is influenced by each country's economic status and healthcare system. We aimed to review the evidence on financial toxicity in Japan. A systematic search was performed using PubMed and Ichushi databases. We included English or Japanese peer-reviewed articles that (1) explored the experiences of cancer patients facing financial toxicity due to cancer diagnosis and treatment, (2) were

Влияние корпоративного управления и вознаграждения ГАС на стратегии капитализации банков и решения о выплате компенсации в период шоковых колебаний доходов.

UNLABELELED: Цель этого исследования состоит в том, чтобы изучить вопросы корпоративного управления и вознаграждения исполнительного директора с учетом стратегий капитализации банков и политики выплат в пакистанском банковском контексте. Данные были получены из финансовых ведомостей банков, зарегистрированных на пакистанской фондовой бирже 2005 в адрес 2020. Результаты исследования показали, что механизмы корпоративного управления, поощряющие интересы акционеров банка, связаны со стратегиями низкой капитализации. Размер совета директоров оказывает значительное влияние на капитализацию банков.

Socioeconomic and physical health status changes after visual impairment in Korea using difference-in-difference estimations.

This study analyzed the impact of visual impairment on socioeconomic and physical health status and its heterogeneity by severity of visual impairment. We used nationally representative cohort data based on Korean national health insurance claims (2002-2013), which were extracted for 11,030 persons (2206 visually impaired, 8824 control). This was restructured as monthly data for each person (person-month). Multivariate and ordered logistic regressions were conducted, and the pre-impairment status between the visually impaired and non-visually impaired people was adjusted by difference-in-diffe

Transplant Programs in APBMT: Blood and Marrow Transplant Activity Report from Myanmar APBMT Meeting 2018, Taiwan.

Hematopoietic stem cell transplantation (HSCT) was introduced in Myanmar in 2014 and was first performed in a patient with multiple myeloma at the North Okkalapa General and Teaching Hospital. From 2014 to 2016,transplantation activities were in the preliminary stage of establishing the infrastructure and gradually developing capacity-building. In 2016, the Yangon General Hospital also commenced autologous transplantation for myeloma. Five autologous transplants were performed in Myanmar during 2016 in patients with myeloma, using high-dose melphalan and non-cryopreserved peripheral blood stem

Реальные модели лечения и результаты лечения пациентов с множественной миеломой, инициирующих эгранатамаб: результаты ALTITUDE-1 и ALTITUDE-2 Ретроспективные когортные исследования.

AIM: Elranatamab в настоящее время является антителом с биенной спецификой, утвержденным в Соединенных Штатах (США) для лечения повторной/охлажденной множественной миеломы (ММ). С учетом его недавнего одобрения реальные данные являются ограниченными. МЕТОДЫ: РЕЗУЛЬТАТЫ РАБОТЫ two исследования реального мира (РВ) с использованием административных требований (см.ALTITUDE-1) и данные о плате за услуги (ALTITUDE-2) сообща сообщаются здесь. Американские пациенты с MM, которые запустили Elranatamab после августа 2023 в сентябре/октябре 2025. Сообщалось о характеристиках пациентов, клинической истори

A cost-utility and budget impact analysis of allogeneic hematopoietic stem cell transplantation for severe thalassemic patients in Thailand.

BackgroundHematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients. The treatment, however, is very costly, particularly in the context of low and middle income countries, and no studies have been carried out to explore its economic justifiability. This study aimed to estimate the cost-utility of HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for severe thalassemia in Thailand, and to investigate the affordability of HSCT using a budget impact analysis.MethodsA Markov model was used to estimate t

Совпадение в полном тексте источника
… HSCT is provided with full coverage to thalassemic patients who are government employees and their dependents enrolled under the Civil Servant Medical Benefit Scheme (9% of the Thai population) as well as employees enrolled under the Social Security Scheme (11% of the population). …

Opioid-Induced Constipation among a Convenience Sample of Patients with Cancer Pain.

BACKGROUND: Little is known regarding the burden of opioid-induced constipation (OIC) among patients who suffer from cancer-related pain. METHODS: A prospective longitudinal study was conducted among cancer patients in the United Kingdom (UK), Canada, and Germany, which included medical record data abstraction, Internet-based patient surveys, and physician surveys. Patients on daily opioid therapy (≥30 mg for ≥4 weeks) for treatment of cancer pain with self-reported OIC were recruited. Response to laxatives was defined by classifying participants into categories of laxative use and evaluating

Research in haematological cancers: What do patients in the Netherlands prioritise?

IntroductionThe experiential knowledge of patients can provide research communities with complementary perspectives on disease. The aim of this study was to identify and prioritise everyday problems and research needs of haematological cancer patients and people who have undergone a stem cell transplantation.MethodsA mixed-method participatory research approach (the Dialogue Model) was applied, including interviews (n = 19), four focus group discussions (n = 27), a questionnaire (n = 146) and a stakeholder dialogue meeting (n = 30) with patients in the Netherlands.ResultsPatients' physical dis

Overcoming obstacles to repurposing for neurodegenerative disease.

Repurposing Food and Drug Administration (FDA)-approved drugs for a new indication may offer an accelerated pathway for new treatments to patients but is also fraught with significant commercial, regulatory, and reimbursement challenges. The Alzheimer's Drug Discovery Foundation (ADDF) and the Michael J. Fox Foundation for Parkinson's Research (MJFF) convened an advisory panel in October 2013 to understand stakeholder perspectives related to repurposing FDA-approved drugs for neurodegenerative diseases. Here, we present opportunities on how philanthropy, industry, and government can begin to a

Health-related quality of life and associated factors among family caregivers of patients with cancer in oncologic centers of Northwest Ethiopia.

BackgroundProviding care for individuals dealing with long-term illnesses like cancer demands significant amounts of time, energy, and emotional investment, potentially resulting in a challenging and overwhelming quality of life for those providing the care.ObjectiveThe purpose of this study was to assess the level of health-related quality of life (HRQoL) and associated factors among family caregivers of patients with cancer in oncologic centers of Northwest Ethiopia.MethodA cross-sectional study was conducted among 412 family caregivers of patients with cancer who were following treatment at