Characterizing attributes of innovation of technologies for healthcare: a systematic review
Objectives Characterizing and evaluating the holistic value of innovative healthcare
technologies (eg treatments, services) constitutes a crucial goal to maximize limited …
technologies (eg treatments, services) constitutes a crucial goal to maximize limited …
Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare …
KL Miller, LJ Fermaglich, J Maynard - Orphanet journal of rare diseases, 2021 - Springer
Background Orphan drug designations are a useful proxy to investigate trends in rare
disease drug development. Drug developers must receive a designation before they are …
disease drug development. Drug developers must receive a designation before they are …
Molecular imaging in drug discovery and development
JR Lindner, J Link - Circulation: Cardiovascular Imaging, 2018 - Am Heart Assoc
Noninvasive imaging has played an increasing role in the process of cardiovascular drug
development. This review focuses specifically on the use of molecular imaging, which has …
development. This review focuses specifically on the use of molecular imaging, which has …
Investigating the landscape of US orphan product approvals
KL Miller, M Lanthier - Orphanet journal of rare diseases, 2018 - Springer
Abstract Background The Orphan Drug Act was enacted in 1983 to encourage the
development of drugs for rare diseases. Previous research has attempted to examine the …
development of drugs for rare diseases. Previous research has attempted to examine the …
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 …
Orphan drug expenditures in the United States: a historical and prospective analysis, 2007–18
V Divino, M DeKoven, M Kleinrock, RL Wade… - Health Affairs, 2016 - healthaffairs.org
The Orphan Drug Act of 1983 established incentives for the development of drugs that treat
rare, or orphan, diseases. We used the IMS Health MIDAS database of audited …
rare, or orphan, diseases. We used the IMS Health MIDAS database of audited …
Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies
KL Miller, S Kraft, A Ipe, L Fermaglich - Expert opinion on orphan …, 2021 - Taylor & Francis
ABSTRACT Background The Orphan Drug Act was created to stimulate the development of
drugs and biologics for rare diseases. Investigating products that have received orphan drug …
drugs and biologics for rare diseases. Investigating products that have received orphan drug …
The Orphan Drug Act at 35: observations and an outlook for the twenty-first century
On the thirty-fifth anniversary of the adoption of the Orphan Drug Act (ODA), we describe the
enormous changes in the markets for therapies for rare diseases that have emerged over …
enormous changes in the markets for therapies for rare diseases that have emerged over …
Do investors value the FDA orphan drug designation?
KL Miller - Orphanet Journal of Rare Diseases, 2017 - Springer
Abstract Background The Orphan Drug Act is an important piece of legislation that uses
financial incentives to encourage the development of drugs that treat rare diseases. This …
financial incentives to encourage the development of drugs that treat rare diseases. This …
The potential benefit of expedited development and approval programs in precision medicine
Background: Increased understanding of the molecular causes of disease has begun to
fulfill the promise of precision medicine with the development of targeted drugs, particularly …
fulfill the promise of precision medicine with the development of targeted drugs, particularly …