FDA Drug Approval and the Ethics of Desperation. [PDF]
JAMA Intern Med, 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 ...
Largent EA, Peterson A, Lynch HF.
europepmc +2 more sources
Evolving Landscape of New Drug Approval in Japan and Lags from International Birth Dates: Retrospective Regulatory Analysis. [PDF]
Clin Pharmacol Ther, 2021 The Pharmaceuticals and Medical Devices Agency (PMDA) has approved hundreds of new drugs in recent years. We retrospectively analyzed the new drugs approved in Japan from 2008 to 2019, and identify the first‐in‐world approvals and clarify the current ...
Tanaka M +10 more
europepmc +2 more sources
Are drug targets with genetic support twice as likely to be approved? Revised estimates of the impact of genetic support for drug mechanisms on the probability of drug approval. [PDF]
PLoS Genet, 2019 Despite strong vetting for disease activity, only 10% of candidate new molecular entities in early stage clinical trials are eventually approved. Analyzing historical pipeline data, Nelson et al. 2015 (Nat.
King EA, Davis JW, Degner JF.
europepmc +2 more sources
FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019.
JAMA Intern Med, 2020 This retrospective review assesses the frequency of surrogate measures used for the first time vs subsequent times in a cancer setting and the surrogate’s strength of correlation with patient-centered outcomes.
Chen EY, Haslam A, Prasad V.
europepmc +2 more sources
The use of mechanistic evidence in drug approval. [PDF]
J Eval Clin Pract, 2018 The role of mechanistic evidence tends to be under‐appreciated in current evidence‐based medicine (EBM), which focusses on clinical studies, tending to restrict attention to randomized controlled studies (RCTs) when they are available. The EBM+ programme
Aronson JK +4 more
europepmc +2 more sources
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
core +7 more sources
Patient-Centered Drug Approval: The Role of Patient Advocacy in the Drug Approval Process. [PDF]
J Manag Care Spec Pharm, 2017 Recent approval of eteplirsen for Duchenne muscular dystrophy (DMD), a rare disease with few treatment alternatives, has reignited the debate over the U.S. drug approval process.
Mattingly TJ, Simoni-Wastila L.
europepmc +2 more sources
Adapting Drug Approval Pathways for Bacteriophage-Based Therapeutics. [PDF]
Front Microbiol, 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.
Cooper CJ, Khan Mirzaei M, Nilsson AS.
europepmc +2 more sources
JAMA Oncol, 2019 Importance To date, an empirical evaluation of the quality of control arms in randomized clinical trials (RCTs) leading to anticancer drug approvals by the US Food and Drug Administration (FDA) has not been undertaken.
Hilal T, Sonbol MB, Prasad V.
europepmc +2 more sources
Factors Affecting Drug-Development Strategies in Asian Global Clinical Trials for Drug Approval in Japan. [PDF]
Clin Transl Sci, 2018 We assessed the current status of Asian global clinical trials (GCTs) and factors, such as therapeutic areas, main metabolic enzymes targeted, approval status in the United States or the European Union, development strategies, influencing drug ...
Asano K, Uyama Y, Tohkin M.
europepmc +2 more sources