Results 21 to 30 of about 432,821 (259)

Towards a more transparent HTA process in Poland: new Polish HTA methodological guidelines

Journal of Market Access & Health Policy, 2017
Introduction: Health technology assessment (HTA) in Poland supports reimbursement decisions via the Polish HTA Agency (AOTMiT), whose guidelines were updated in 2016.
Krzysztof Lach, Michal Dziwisz, Cécile Rémuzat, Mondher Toumi   +3 more
doaj   +1 more source

RWE Framework: An Interactive Visual Tool to Support a Real-World Evidence Study Design

Drugs - Real World Outcomes, 2019
Objectives Real-world evidence (RWE) is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients.
Andrew D. Xia, Caroline P. Schaefer, Agota Szende, Elke Jahn, Matthew J. Hirst   +4 more
doaj   +1 more source

Association Between Dinutuximab Beta Exposure and Post‐End‐of‐Treatment Survival in Neuroblastoma: A Weighted Patient‐Level Analysis of Three Clinical Studies

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Objectives The association between exposure to dinutuximab beta (DB) and event‐free survival (EFS) or overall survival (OS) of neuroblastoma patients was assessed using data collected during three clinical trials (five cohorts). Methods A systematic review (March 2026) was conducted to identify relevant studies (prospective; registered DB ...
Przemysław Holko, Holger N. Lode, Alberto Garaventa, Carla Manzitti, Roberto Luksch, Dominique Valteau‐Couanet, Marion Gambart, John Anderson, Juliet Gray, Adela Canete, Lucas Moreno, Aleksandra Wieczorek, Cormac Owens, Shifra Ash, Isaac Yaniv, Genevieve Laureys, Hans Loibner, Katarzyna Śladowska, Ruth Ladenstein, Paweł Kawalec   +19 more
wiley   +1 more source

Diversity in randomized clinical trials for peripheral artery disease: a systematic review

International Journal for Equity in Health
Background Significant race and sex disparities exist in the prevalence, diagnosis, and outcomes of peripheral artery disease (PAD). However, clinical trials evaluating treatments for PAD often lack representative patient populations.
Chandler Long, Abimbola O. Williams, Alysha M. McGovern, Caroline M. Jacobsen, Liesl M. Hargens, Sue Duval, Michael R. Jaff   +6 more
doaj   +1 more source

An integrated modelling methodology for estimating global incidence and prevalence of hereditary spastic paraplegia subtypes SPG4, SPG7, SPG11, and SPG15

BMC Neurology, 2022
Background Hereditary spastic paraplegias (HSPs) are progressively debilitating neurodegenerative disorders that follow heterogenous patterns of Mendelian inheritance.
Geert Vander Stichele, Alexandra Durr, Grace Yoon, Rebecca Schüle, Craig Blackstone, Giovanni Esposito, Connor Buffel, Inês Oliveira, Christian Freitag, Stephane van Rooijen, Stéphanie Hoffmann, Leen Thielemans, Belinda S. Cowling   +12 more
doaj   +1 more source

A Model of Access in the Absence of Markets [PDF]

The B.E. Journal of Theoretical Economics, 2016
AbstractEconomists have long known that properly designed markets allocate resources efficiently. However, in many circumstances markets are unfeasible. In this paper, we construct a general model of access which allows us to value different assignments when resources are allocated in the absence of markets. We demonstrate that marginal value schedules
Holzer, Jorge, McConnell, Kenneth
openaire   +2 more sources

A Population‐Based Study on Childhood Aplastic Anemia—Incidence, Outcomes, and Health‐Related Quality of Life

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Childhood aplastic anemia (AA) is a rare disease, and both the disease itself and its treatment cause significant morbidity. We aimed to determine the contemporary incidence of childhood AA in Finland, to compare the clinical characteristics of AA against inherited bone marrow failure syndromes (IBMFS) and refractory cytopenia of ...
Lauri‐Matti Kulmala, Riitta Niinimäki, Tytti Pokka, Anu Huurre, Liisa Järvelä, Sauli Palmu, Tuuli Pöyhönen, Pauliina Utriainen, Henri Aarnivala   +8 more
wiley   +1 more source

Establishing an Apheresis Medicine Program in a Resource‐Constrained Setting: A 5‐Year Experience From Lagos, Nigeria

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Background Establishing a comprehensive apheresis medicine program in a resource‐constrained setting presents significant structural, financial, and logistical challenges. Despite the growing clinical importance of apheresis services globally, published experience from sub‐Saharan Africa remains sparse.
Folasade Adelekan‐Popoola, Shakirat Gold‐Olufadi, Olufunto Olufela Kalejaiye, Abdullateef Ogala, Rasheed A. Balogun   +4 more
wiley   +1 more source

Estimating the broader fiscal consequences of acute hepatic porphyria (AHP) with recurrent attacks in Belgium using a public economic analytic framework

Orphanet Journal of Rare Diseases, 2021
Background Acute hepatic porphyria (AHP) is a rare, debilitating disease characterized by potentially life-threatening attacks often resulting in chronic symptoms that negatively impact daily functioning and quality of life.
Mark P. Connolly, Nikos Kotsopoulos, Sebastian Vermeersch, Julien Patris, David Cassiman   +4 more
doaj   +1 more source

Safety of Daprodustat for the Treatment of Chronic Kidney Disease Anemia: Final Analysis of a Multicenter Postmarketing Surveillance Study in Japan

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction This final analysis of a multicenter, prospective postmarketing surveillance study evaluated the safety of daprodustat in patients with chronic kidney disease anemia in routine clinical practice in Japan. Methods Patients who initiated daprodustat between September 2020 and July 2022 were registered.
Tadao Akizawa, Kyomi Kanaya, Kiyomi Aizawa, Masanori Onoki, Hirofumi Ozeki, Kazuko Suzuki, Ai Hayashi, Tomohiko Asamura   +7 more
wiley   +1 more source