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Predictors of orphan drug approval [PDF]
Orphanet Journal of Rare Diseases, 2010 Results More orphan drugs were developed in the US during the first ten years of the US Orphan Drug Act (1983-1992, N=73) and during the first ten years of the EU Regulation on Orphan Medicinal products (2000-2009, N=112) than in the EU (2000-2009, N=59). Orphan drug approval was strongly associated with previous experiences of the sponsor in obtaining
Heemstra Harald
doaj +7 more sources
Adapting drug approval pathways for bacteriophage-based therapeutics
Frontiers in Microbiology, 2016 The global rise of multi-drug resistant bacteria has resulted in the notion that an antibiotic apocalypse is fast approaching. This has led to a number of well publicized calls for global funding initiatives to develop new antibacterial agents. The long
Callum Cooper +3 more
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Clinical and Translational Science, 2021 The current success rate of a drug candidate, from the beginning of the clinical trial to receiving marketing approval, is about 10%–20%, and it has not changed during the past few decades. Therefore, pharmaceutical companies are under pressure to select
Shingo Yamaguchi +2 more
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Drugs, Devices, and the FDA: Part 1 [PDF]
JACC: Basic to Translational Science, 2016 Over the last 150 years, the U.S. Food and Drug Administration (FDA) has evolved from a small division of the U.S. Patent Office to 1 of the largest consumer protection agencies in the world.
Gail A. Van Norman, MD
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Milbank Quarterly, 2017 Policy Points: Randomized trials-the gold standard of evaluating effectiveness-constitute a small minority of existing evidence on agents given accelerated approval. One-third of randomized trials are in therapeutic areas outside of FDA approval and less
Huseyin Naci +2 more
exaly +2 more sources
Predictors of orphan drug approval in the European Union [PDF]
, 2008 Objective: To encourage the development of drugs for rare diseases, orphan drug legislation has been introduced in the USA (1983) and in the EU (2000).
A Rinaldi +17 more
core +6 more sources
An open science pathway for drug marketing authorization-Registered drug approval.
PLoS Medicine, 2021 Florian Naudet and co-authors propose a pathway involving registered criteria for evaluation and approval of new drugs.
Florian Naudet +4 more
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FDA Drug Approval and the Ethics of Desperation.
JAMA Internal Medicine, 2021 In justifying the accelerated approval of aducanumab (Aduhelm; Biogen), US Food and Drug Administration (FDA) officials emphasized that many patients with Alzheimer disease and their families “made it clear that they are willing to accept the trade-off ...
E. Largent, Andrew Peterson, H. F. Lynch
semanticscholar +1 more source
Rheumatology and Therapy, 2023 Introduction This drug utilization study evaluated the impact of 2019 label changes on real-world febuxostat utilization among patients with gout. We describe the numbers and proportions of patients initiating febuxostat as new users (allopurinol-naïve ...
Alexandra Z. Sosinsky +4 more
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The impact of external innovation on new drug approvals: A retrospective analysis [PDF]
International Journal of Pharmaceutics, Volume 563, Pages 273-281,
2019, 2021 Pharmaceutical companies are relying more often on external sources of innovation to boost their discovery research productivity. However, more in-depth knowledge about how external innovation may translate to successful product launches is still required in order to better understand how to best leverage the innovation ecosystem.
arxiv +1 more source