Results 21 to 30 of about 88,754 (289)

Expert Consensus on the Application of Multiple criteria Decision Analysis in Comprehensive Clinical Evaluation of Orphan Drugs(2022)

罕见病研究, 2022
Comprehensive clinical drug evaluation is an important technical tool for decision-making on drug supply. Traditional evaluation on health technology provides methodological references for comprehensive clinical evalu-ation of drugs.
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, China Alliance for Rare Diseases   +1 more
doaj   +1 more source

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study

Frontiers in Pharmacology, 2022
Background: Orphan drugs are used for the diagnosis, prevention and treatment of rare diseases that, in the European Union, are defined as disorders affecting no more than 5 persons in 10,000.
Francesca Gorini, Michele Santoro, Anna Pierini, Lorena Mezzasalma, Silvia Baldacci, Elena Bargagli, Alessandra Boncristiano, Maurizia Rossana Brunetto, Paolo Cameli, Francesco Cappelli, Giancarlo Castaman, Barbara Coco, Maria Alice Donati, Renzo Guerrini, Silvia Linari, Vittoria Murro, Iacopo Olivotto, Paola Parronchi, Francesca Pochiero, Oliviero Rossi, Barbara Scappini, Andrea Sodi, Alessandro Maria Vannucchi, Alessio Coi   +23 more
doaj   +1 more source

Analysis of patient access to orphan drugs in Turkey

Orphanet Journal of Rare Diseases, 2021
Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases.
Güvenç Koçkaya, Sibel Atalay, Gülpembe Oğuzhan, Mustafa Kurnaz, Selin Ökçün, Çiğdem Sar Gedik, Mete Şaylan, Nazlı Şencan   +7 more
doaj   +1 more source

Expert Consensus on the Application of Multi-criteria Decision Analysis in Clinical Comprehensive Evaluation of Orphan Drugs(2022)

Xiehe Yixue Zazhi, 2022
Comprehensive drug evaluation is an important technical tool for decision-making on drug supply. Traditional health technology of evaluation provides methodological references for comprehensive clinical evalu-ation of drugs.
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, National Rare Diseases Committee
doaj   +1 more source

Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union

Saudi Pharmaceutical Journal, 2023
Background: Rare diseases are chronic, serious, and life-threatening conditions that have not received sufficient attention from drug developers due to their rarity.
Bander Balkhi, Asma Almuaither, Saeed Alqahtani   +2 more
doaj   +1 more source

Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022

Frontiers in Pharmacology, 2023
Introduction: Over 400 million patients worldwide suffer from rare diseases. Access to orphan drugs is, therefore, crucial for this population. China has been actively working on improving orphan drug accessibility in the past decades, especially since ...
Jia Liu, Yue Yu, Yue Yu, Mingkang Zhong, Mingkang Zhong, Chunlai Ma, Chunlai Ma, Rong Shao   +7 more
doaj   +1 more source

Orphan drug: Development trends and strategies

Journal of Pharmacy and Bioallied Sciences, 2010
The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in
Aarti Sharma, Abraham Jacob, Manas Tandon, Dushyant Kumar   +3 more
doaj   +1 more source

Pricing and reimbursement of orphan drugs: the need for more transparency

Orphanet Journal of Rare Diseases, 2011
Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients.
Simoens Steven
doaj   +1 more source

Are payers treating orphan drugs differently?

Journal of Market Access & Health Policy, 2014
Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years.
Joshua P. Cohen, Abigail Felix
doaj   +1 more source

Rare diseases and orphan drugs

Annali dell'Istituto Superiore di Sanità, 2011
According to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council, rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10 000 persons in the European Community.
Domenica Taruscio, Fiorentino Capozzoli, Claudio Frank   +2 more
doaj   +1 more source