Results 101 to 110 of about 88,273 (287)

Through the lens of marketing authorization holders: experience in use of real‐world data and real‐world evidence in drug development and regulatory submissions in EU

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim The aim of this study was to assess the general and product‐specific experiences of MAHs use of RWD/RWE in medicines development and in their regulatory submissions, and to explore organizational aspects of MAHs related to RWD/RWE. Methods An electronic survey was conducted, and information collected directly from MAHs.
Sini M. Eskola, Aleksandar Gigov, Hubertus G. M. Leufkens, Andrew Bate, Marie Louise De Bruin, Helga Gardarsdottir   +5 more
wiley   +1 more source

Ethical hurdles in the prioritization of oncology care [PDF]

, 2016
With finite resources, healthcare payers must make difficult choices regarding spending and the ethical distribution of funds. Here, we describe some of the ethical issues surrounding inequity in healthcare in nine major European countries, using cancer ...
Capri, Stefano, Castanier, Jean-Claude, Cunningham, David, de Groot, Folkert, Flamion, Bruno, Flume, Mathias, Herholz, Harald, Levin, Lars-Åke, Rupprecht, Christoph J., Shalet, Natalie, Solà-Morales, Oriol, Walker, Andrew, Wong, Olivier   +12 more
core   +1 more source

Do drugs approved via expedited approval pathways have therapeutic advantages? A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer, Eliza J. McEwin, Zhicheng Wang, Joel Lexchin, Barbara Mintzes   +4 more
wiley   +1 more source

Book Review: Deforming Tort Reform [PDF]

, 1990
The storms buffeting the tort system over the past two decades have come in three distinct waves. In the late 1960s, steep increases in the insurance costs incurred by health care providers protecting against negligence claims by patients triggered what ...
Page, Joseph A.
core   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?

Orphanet Journal of Rare Diseases, 2012
Since its enactment in 2000, the European Orphan Medicinal Products Regulation has allowed the review and approval of approaching 70 treatments for some 55 different conditions in Europe. Success does not come without a price, however.
Hughes-Wilson Wills, Palma Ana, Schuurman Ad, Simoens Steven   +3 more
doaj   +1 more source

Nanomaterial‐based immune therapeutic strategies in neurodegenerative diseases

BMEMat, EarlyView.
This review highlights the immunomodulatory potential of nanomaterials (NMs) in treating neurodegenerative diseases (NDs). It focuses on their roles in regulating innate and adaptive immune responses to maintain immune homeostasis. By providing insights into these mechanisms, the review lays the groundwork for innovative NMs therapeutic strategies to ...
Xinru Zhou, Jia Liu, Shulin Lai, Suhan Yin, Bingmin Luo, Longquan Shao, Yiyuan Kang   +6 more
wiley   +1 more source

Race, Money and Medicines [PDF]

, 2006
Taking notice of race is both risky and inevitable, in medicine no less than in other endeavors. The literature on race as a classifying tool in clinical research poses this core dilemma: On the one hand, race can be a useful stand-in for unstudied ...
Bloche, Maxwell Gregg
core   +1 more source

Generating health technology assessment evidence for rare diseases [PDF]

, 2014
Objectives: Rare diseases are often heterogeneous in their progression and response to treatment, with only a small population for study. This provides challenges for evidence generation to support HTA, so novel research methods are required.
Facey, Karen, Granados, Alicia, Guyatt, Gordon, Kent, Alastair, Shah, Nilay, van der Wilt, Gert Jan, Wong-Rieger, Durhane   +6 more
core   +1 more source

Design of experiments‐guided membrane capture facilitates integration of continuous virus inactivation using Phi6 as a surrogate virus in an intensified downstream process

Biotechnology Progress, EarlyView.
Abstract The growing demand for cost‐efficient and flexible biomanufacturing has increased interest in process‐intensified downstream platforms. This study evaluates an intensified monoclonal antibody (mAb) purification sequence, where two unit operations traditionally performed in batch mode—Protein A capture and low pH virus inactivation (VI)—are ...
Mario Grünberg, Lisa Lipski, Gabriel Fisicaro, Thomas Duvignau, Karolina Meyer‐Heinrichs, Diana Carmen Mocsy, Thomas‐Josef Filz, Bastian Quaas, Alexandra Stützer   +8 more
wiley   +1 more source