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.
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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.
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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
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Disease modification in osteoarthritis; pathways to drug approval [PDF]
Osteoarthritis and Cartilage Open, 2020 Summary: Objective: To summarize proceedings of a workshop convened to discuss advances in disease modifying osteoarthritis (OA) drugs and regulatory challenges in bringing these drugs to market. Design: Summary of a one day workshop held in Washington,
Jeffrey N. Katz +7 more
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Analysis of safety specifications in risk management plan at the time of drug approval and addition of clinically significant adverse reactions in the package insert post‐approval in Japan [PDF]
Pharmacology Research & Perspectives, 2023 Recently, post‐marketing safety measures have been considered critical in Japan due to the globalization of drug development and the introduction of new drug approval systems.
Rieko Saito, Seiko Miyazaki
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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.
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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.
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ChemAP: predicting drug approval with chemical structures before clinical trial phase by leveraging multi-modal embedding space and knowledge distillation [PDF]
Scientific ReportsRecent studies showed that the likelihood of drug approval can be predicted with clinical data and structure information of drug using computational approaches. Predicting the likelihood of drug approval can be innovative and of high impact.
Changyun Cho +4 more
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Patient-Centered Drug Approval: The Role of Patient Advocacy in the Drug Approval Process. [PDF]
J Manag Care Spec Pharm, 2017 Mattingly TJ, Simoni-Wastila L.
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The European journal of health economics : HEPAC : health economics in prevention and care, 2023 Background Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan ...
D. Michaeli +4 more
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