A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act
LJ Fermaglich, KL Miller - Orphanet Journal of Rare Diseases, 2023 - Springer
Background Rare diseases affect more than 30 million Americans. The passage of the
Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare …
Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare …
Use of US Food and Drug Administration expedited drug development and review programs by orphan and nonorphan novel drugs approved from 2008 to 2021
AN Monge, DW Sigelman, RJ Temple… - JAMA Network …, 2022 - jamanetwork.com
Importance The US Food and Drug Administration (FDA) has 4 programs that can be used
alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough …
alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough …
Investigation of the diagnostic importance and accuracy of CT in the chest compared to the RT-PCR test for suspected COVID-19 patients in Jordan
H Alewaidat, Z Bataineh, M Bani-Ahmad… - …, 2023 - pmc.ncbi.nlm.nih.gov
This article aims to synthesize the existing literature on the implementation of public policies
to incentivize the development of treatments for rare diseases,(diseases with very low …
to incentivize the development of treatments for rare diseases,(diseases with very low …
Cost of exempting sole orphan drugs from Medicare negotiation
Importance The Inflation Reduction Act (IRA) requires Medicare to negotiate prices for some
high-spending drugs but exempts drugs approved solely for the treatment of a single rare …
high-spending drugs but exempts drugs approved solely for the treatment of a single rare …
Assessment of surrogate end point trends in clinical trials to approve oncology drugs from 2001 to 2020 in Japan
Importance A surrogate end point (SEP) is an end point used in clinical trials as an
alternative for measuring the true clinical benefit. The use of SEPs in trials shortens their …
alternative for measuring the true clinical benefit. The use of SEPs in trials shortens their …
[HTML][HTML] Pharmaceutical policy and innovation for rare diseases: A narrative review
This article aims to synthesize the existing literature on the implementation of public policies
to incentivize the development of treatments for rare diseases,(diseases with very low …
to incentivize the development of treatments for rare diseases,(diseases with very low …
Continued cancer drug approvals in Japan and Europe after market withdrawal in the United States: A comparative study of accelerated approvals
Regulatory authorities must balance ensuring evidence of efficacy and safety of new drugs.
Various regulatory pathways, such as the accelerated approval program in the United States …
Various regulatory pathways, such as the accelerated approval program in the United States …
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National …
RM Lomash, O Shchelochkov, RJ Chandler… - Human Gene …, 2023 - liebertpub.com
Orphan drug designation (ODD) is an important program intended to facilitate the
development of orphan drugs in the United States. An orphan drug benefiting pediatric …
development of orphan drugs in the United States. An orphan drug benefiting pediatric …
[HTML][HTML] 'Financial fallout'in the US biopharmaceutical industry: Maximizing shareholder value, regulatory capture, and the consequences for patients
R Whitacre - Social Science & Medicine, 2024 - Elsevier
Patients are suffering the consequences of financialization–as shareholders demand high
returns from pharmaceutical companies, pharmaceuticals squeeze profits out of top-selling …
returns from pharmaceutical companies, pharmaceuticals squeeze profits out of top-selling …
Leveraging real-world data to conduct externally controlled trial for rare diseases with count-type endpoints: utilizing multiple entries–a simulation study
T Sun, E Liao, N Shao, J Luo - Journal of Biopharmaceutical …, 2024 - Taylor & Francis
Conducting randomized controlled trials for medications targeting rare diseases presents
significant challenges, due to the scarcity of participants and ethical considerations. Under …
significant challenges, due to the scarcity of participants and ethical considerations. Under …