Results 111 to 120 of about 88,273 (287)

Extending life for people with a terminal illness: a moral right and an expensive death? Exploring societal perspectives [PDF]

, 2015
BACKGROUND: Many publicly-funded health systems apply cost-benefit frameworks in response to the moral dilemma of how best to allocate scarce healthcare resources.
Baker, Rachel M., Collins, Marissa, Deogaonkar, Rohan, Donaldson, Cam, Mason, Helen, McHugh, Neil, van Exel, Job, Williamson, Laura   +7 more
core   +4 more sources

More Than a Question of Correlation: Characterization of the Evidentiary Basis for Biomarker Surrogates Used in European Marketing Authorizations

Clinical Pharmacology &Therapeutics, EarlyView.
Traditionally, clinical outcomes measuring how a patient feels, functions, or survives are preferred endpoints in clinical trials; however, some may take a long time to manifest in slowly developing diseases. Biomarkers, if properly validated, can serve as surrogate endpoints, acting as substitutes for clinical outcomes.
Renske Johanna Grupstra, Viktoriia Starokozhko, Elisabeth Bakker, Anna Maria Gerdina Pasmooij, Peter Gerardus Maria Mol   +4 more
wiley   +1 more source

Incentives for orphan drug research and development in the United States

Orphanet Journal of Rare Diseases, 2008
Background The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions.
Rodriguez-Monguio Rosa, Seoane-Vazquez Enrique, Szeinbach Sheryl L, Visaria Jay   +3 more
doaj   +1 more source

A Comparison of Regional Decisions for Doses and Administrations of New Drugs: Concordance, Discordance, and Dependencies

Clinical Pharmacology &Therapeutics, EarlyView.
Regional differences in approved drug dosages and administration are shaped by complex factors, including clinical data, prior regulatory decisions, and region‐specific considerations. This study investigated how such factors are associated with variations in approved doses across the United States Food and Drug Administration (FDA), European Medicines
Sachiko Mita, Shunsuke Ono
wiley   +1 more source

Unveiling Hepatic Protein Alterations in Neonatal and Infant Biliary Atresia

Clinical Pharmacology &Therapeutics, EarlyView.
Pediatric populations differ from adults in drug elimination capacity. While current scaling methods account for enzyme and transporter maturation, they overlook comorbidities, such as biliary atresia (BA), a liver disease appearing within the first 2–8 weeks of life that can progress to cirrhosis.
Zubida M. Al‐Majdoub, Martyn Howard, Brahim Achour, Jill Barber, Naved Alizai, Amin Rostami‐Hodjegan   +5 more
wiley   +1 more source

Determinants of orphan drug health technology assessment in South Korea: an empirical analysis

Frontiers in Pharmacology
BackgroundOrphan drug pricing in South Korea, including its workings and determinants, is a complicated aspect of the country’s healthcare system. Therefore, this study identified the factors associated with the pricing of orphan drugs in South Korea ...
Se Hee Lee, Sung Kyung Lee, Jong Hyuk Lee, Joon Seok Bang   +3 more
doaj   +1 more source

The hope for neglected diseases: R&D incentives [PDF]

Neglected diseases are neglected because they cannot generate enough return on R&D to pharmaceutical firms. This paper analyzes and compares existing proposals for public intervention in R&D for neglected diseases.
Brigitte Granville, Eshref Trushin
core  

Promoting Academic Entrepreneurship in Europe and the United States: Creating an Intellectual Property Regime to Facilitate the Efficient Transfer of Knowledge from the Lab to the Patient [PDF]

, 2015
In 2014, the European Commission announced the launch of a study of knowledge transfer by public research organizations and other institutes of higher learning “to determine which additional measures might be needed to ensure an optimal flow of knowledge
Bagley, Constance E., Tvarnø, Christina D.   +1 more
core   +1 more source

Exercise limitations in amyloid cardiomyopathy assessed by cardiopulmonary exercise testing—A multicentre study

ESC Heart Failure, Volume 12, Issue 2, Page 1326-1335, April 2025.
Abstract Aims Amyloid cardiomyopathy is caused by the deposition of light chain (AL) or transthyretin amyloid (ATTR) fibrils, that leads to a restrictive cardiomyopathy, often resulting in heart failure (HF) with preserved or reduced ejection fraction.
Robin Willixhofer, Mauro Contini, Michele Emdin, Damiano Magrì, Alice Bonomi, Elisabetta Salvioni, Fabrizio Celeste, Alberico Del Torto, Claudio Passino, Christophe D.J. Capelle, Chiara Arzilli, Emiliano Fiori, Nicolò Capra, Christina Kronberger, Nikita Ermolaev, Andreas Kammerlander, Beatrice Musumeci, Giuseppe Vergaro, Vincenzo Castiglione, René Rettl, Giacomo Tini, Andrea Baggiano, Iacopo Fabiani, Susanna Sciomer, Roza Badr Eslam, Piergiuseppe Agostoni   +25 more
wiley   +1 more source

Policy Networks and Policy Entrepreneurship in the EU: Explaining Structural Policy Change in Pharmaceutical Innovation Incentives and Health Technology Assessment

European Policy Analysis, EarlyView.
ABSTRACT Policy process research has excelled in explaining structural policy change within national settings, but extensions and applications to the EU level have long proven challenging for scholars. Given that the EU is currently experiencing its longest period of Treaty stability since the 1980s—having evolved into a sui generis political system ...
Vassilis Karokis‐Mavrikos
wiley   +1 more source