Results 61 to 70 of about 32,298 (248)

Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019

Orphanet Journal of Rare Diseases, 2020
Background Pricing and reimbursement decisions for orphan drugs are faced with differences access between European countries depending on each reimbursement policies, evaluation processes and timings.
Xavier Badia, Tania Vico, John Shepherd, Alicia Gil, José Luis Poveda-Andrés, César Hernández   +5 more
doaj   +1 more source

Regulatory harmonization: Evolution, globalization and future directions

British Journal of Clinical Pharmacology, EarlyView.
Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm, Elaine Y. L. Blair, Christine Y. Lu   +2 more
wiley   +1 more source

Precision Medicines’ Impact on Orphan Drug Designation

Clinical and Translational Science, 2019
The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration.
Christine M. Mueller, Gayatri R. Rao, Katherine I. Miller Needleman   +2 more
doaj   +1 more source

External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management

Clinical Pharmacology &Therapeutics, EarlyView.
External control arms (ECAs) have emerged as important tools to provide benchmark evidence on comparator effectiveness and thereby support regulatory decision making in situations where randomized controlled trials are difficult to conduct. However, how key methodological elements for ECAs are documented and implemented in practice and how residual ...
Suguru Okami, Alexandros Sagkriotis
wiley   +1 more source

The Multiple Criteria Qualitative Value‐Based Pricing Framework “MARIE” for Novel Cell and Gene Therapy

Clinical Pharmacology &Therapeutics, EarlyView.
When setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Akina Takami, Ataru Igarashi
wiley   +1 more source

Preisregulierung durch Nutzenbewertung bei seltenen Erkrankungen

Monitor Versorgungsforschung
Ziel der Studie ist eine genaue Untersuchung der populationsnormalisierten Behandlungskosten von Orphan Drugs. Zu diesem Zweck wurde bereits im Jahr 2022 eine Studie zur Betrachtung der Therapiekosten von Orphan Drugs für die Jahre 2005 bis 2021 ...
Lukas Maag MSc, Dr. Ariane Höer, Dr. rer. nat. Gerrit Müller   +2 more
doaj   +1 more source

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries

Frontiers in Pharmacology, 2019
Objective: To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and ...
Ewa Stawowczyk, Krzysztof Piotr Malinowski, Paweł Kawalec, Rafał Bobiński, Jacek Siwiec, Dimitra Panteli, Dimitra Panteli, Dimitra Panteli, Helene Eckhardt, Helene Eckhardt, Helene Eckhardt, Steven Simoens, Antònia Agusti, Antònia Agusti, Marc Dooms, Andrzej Pilc, Andrzej Pilc   +16 more
doaj   +1 more source

Adverse Reactions Identified after Approval of Novel Medicines: Analysis of 339 New Molecular Entities and Therapeutic Biologics

Clinical Pharmacology &Therapeutics, EarlyView.
Postmarketing safety surveillance data identify safety issues not identified in preapproval clinical trials. We describe the frequency of new safety issues overall and by labeling section and the relationship of the most commonly added safety issues with the cumulative number of prescriptions dispensed via retail pharmacies.
Ellen Pinnow, Brendan Day, Esther H. Zhou, Corinne Woods, Nana Dwumfour, Jenni Y. Lee, Ilynn Bulatao, Sanae Cherkaoui, Manish Kalaria, Sonja Brajovic, Nabila Sadiq, Farideh Sistani, Maggie Lee, Grace Chai, Gerald Dal Pan   +14 more
wiley   +1 more source

Exposing Gender Blindness in Business Scholarship on Modern Slavery

Corporate Social Responsibility and Environmental Management, EarlyView.
ABSTRACT The objective of this review is to analyse the treatment of gender within business scholarship on modern slavery and to propose a framework that supports more gender‐sensitive corporate responses. Modern slavery risk is an escalating concern for business, with the United Nations calling for its eradication by 2030.
Kathyayini Kathy Rao, Roger Leonard Burritt, Katherine Leanne Christ   +2 more
wiley   +1 more source

Atomic‐Scale Structurally Locked 1T/2H‐MoS2/ZnS Z‐Scheme Heterojunctions for Prolonged Charge Kinetics and Dual‐Functional Photocatalysis

ENERGY &ENVIRONMENTAL MATERIALS, EarlyView.
Atomic‐scale structurally locked 1T/2H‐MoS2/ZnS direct Z‐scheme heterojunction enabling dual‐functional photocatalysis. The metallic 1T‐MoS2 phase serves as an electronic actuator that bridges charge transfer across the heterointerface, resulting in prolonged charge kinetics, efficient hydrogen evolution, and complete degradation of Malachite Green ...
Jeongjin Lee, Younghwon Kim, Byeong Sub Kwak, Suyeon Lee, Geunhyeong Kim, Buseong Kim, Harim Jeong, Younghwan Im, Sadanand Pandey, Hojun Moon, Misook Kang   +10 more
wiley   +1 more source