Results 41 to 50 of about 109,656 (184)

Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Orphanet Journal of Rare Diseases, 2019
Background High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool.
Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S. Hoch, Paul Grootendorst   +5 more
doaj   +1 more source

Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non‐orphan drugs approved by the FDA

Clinical and Translational Science, 2022
Clinical pharmacology is an integral discipline supporting the development, regulatory evaluation, and clinical use of drugs for the treatment of both common and rare diseases.
Julie Hsieh, Martina Sahre, Xinning Yang, Rajanikanth Madabushi, Anuradha Ramamoorthy   +4 more
doaj   +1 more source

Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development

Medicine in Drug Discovery, 2023
Only 5% of orphan diseases have approved drugs, leading to a high demand for new treatment. As a result, pharmaceutical companies have shifted their focus of drug development to orphan diseases.
Ryo Okuyama
doaj   +1 more source

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

PLoS ONE, 2016
BackgroundConcerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.MethodsWe conducted a retrospective, propensity score matched study of publicly-
Dyfrig A Hughes, Jannine Poletti-Hughes
doaj   +1 more source

Impacts of adjustment of National Reimbursement Drug List on orphan drugs volume and spending in China: an interrupted time series analysis

BMJ Open, 2023
Objective To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China.Design We used an interrupted time series design covering the period from 2016 to 2018 ...
Yu Xie, Hua Xing, Zihao Wang, Hongbin Yi, Fenghao Shi, Liping Kuai, Dongyan Xu   +6 more
doaj   +1 more source

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children.
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source

Update on the Research of an Emulgel for the Effective Treatment of Atopic Dermatitis: Clinical Investigation in Children

Gels
Atopic dermatitis (AD) is a chronic inflammatory skin condition that affects up to 25% of children and impairs both skin barrier function and quality of life. This study examined the effectiveness of an emulgel containing hyaluronic acid, glycerol, grape
Almudena Gómez-Farto, Ana Leticia Jiménez-Escobar, Noelia Pérez-González, Amy Lozano-White, Jésica Expósito-Herrera, Trinidad Montero-Vílchez, Beatriz Clares, Salvador Arias-Santiago   +7 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

Developing Methodology for the Creation of Clinical Practice Guidelines for Rare Diseases : A Report from RARE-Bestpractices [PDF]

, 2015
Date of Acceptance: 29/05/2015 The research leading to these results has received funding from the (FP7/2007–2013), under grant agreement n 305690 (RARE-Bestpractices project). The opinions, presented here reflect only the authors’ views.
RARE-Bestpractices Consortium
core   +1 more source

Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?

Voices in Bioethics, 2019
The mainstream media and general public have expressed frustration in recent months with the confluence of capitalism and the pharmaceutical research and development enterprise.
Michael Menconi
doaj   +1 more source