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Trends in clinical success rates and therapeutic focus
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“Human genetics can provide compelling evidence of causation through approaches such as Mendelian randomization (MR).”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
In the citing paper
“Human genetics can provide a tool by which the causal role of an exposure (such as a complex phenotype and/or a drug target) can be characterized 9 .”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
In the citing paper
“Performed well, such studies called Mendelian randomization (MR) analyses, can provide rigorous evidence of causation 9,10 .”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
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“However, this model has demonstrated its limitations, particularly in this era of precision-based medicine, and is manifest to some extent by the high attrition rates in early development of oncology drugs where over 90% of investigational drugs in Phase I never achieve market authorization [2].”
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“In fact, more than 90% of all new active substances that enter clinical phase I fail in subsequent clinical trials [170] and 79% of these failures are due to safety or efficacy issues [170].”
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“Indeed, most drugs fail as a result of low ef fi cacy (45 – 65% of cases) or high toxicity (15 – 35% of cases), and seldom because of strategic, commercial, or operative reasons (Harrison, 2016; Takebe et al., 2018; Dowden and Munro, 2019).”
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“Although many possible treatments for AD are in development [5, 6], drug approval rates are typically low for the nervous system [7] and a recent approval of Aducanumab [8] has proven controversial [9, 10].”
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“%) and poor strategic planning 10% (Dowden and Munro 2019).”
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Drug Discovery Firms and Business Alliances for Sustainable Innovation
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“Approximately 50% of these failures result from drug inef-ficacy, and 30% of trials are halted owing to safety concerns 2,3 , both of which are often attributable to poor target selection during preclinical stages 4 .”
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First-in-Class Small Molecule Degrader of Pregnane X Receptor Enhances Chemotherapy Efficacy
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“Currently, the drug discovery procedure includes 2D cell culture-based tests, then animal model tests, and finally clinical trials of which only 10% are successful [36].”
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GM-Pep: A High Efficiency Strategy to De Novo Design Functional Peptide Sequences
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“Nevertheless, from drug approval to launch of marketing and distribution, it takes about 1.5 years on average (Dowden and Munro, 2019).”
InfluentialNeurologic orphan diseases: Emerging innovations and role for genetic treatments
Kioutchoukova, I. P., Foster, D. T., Thakkar, R. N., Foreman, M. A., Burgess, B. J., Toms, R., Valero, E., & Lucke-Wold, B. P. (2023) · World Journal of Experimental Medicine · doi:10.5493/wjem.v13.i4.59 · Cited by 11
Drug Screening in Human Cells by NMR Spectroscopy Allows the Early Assessment of Drug Potency
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Deng, J., Hou, W., Dong, X., Hajagos, J., Saltz, M. M., Saltz, J. H., & Wang, F. (2021) · Drugs - Real World Outcomes · doi:10.1007/s40801-021-00253-8 · Cited by 7
“In fact, new drug development is well known for its low success rate [28].”
BackgroundArtificial Intelligence in Drug Discovery: Applications and TechniquesPreprint
Deng, J., Yang, Z., Ojima, I., et al. (2021) · arXiv (Cornell University) · doi:10.48550/arxiv.2106.05386 · Cited by 8
Why Clinical Trials Stop: The Role of GeneticsPreprint
Razuvayevskaya, O., López, I., Dunham, I., & Ochoa, D. (2023) · medRxiv · doi:10.1101/2023.02.07.23285407 · Cited by 8
“Recent reports indicate that 79% of clinical studies fail because of the lack of efficacy or safety (Dowden and Munro, 2019).”
BackgroundInfluentialLow-Data Drug Design with Few-Shot Generative Domain Adaptation
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Screening for variable drug responses using human iPSC cohorts
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“Unfortunately, these pipelines often have very high failure rates, with 80% or more drug candidates in major disease areas failing to pass clinical trials [4].”
Computational Strategies Reshaping Modern Drug Discovery
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Metadata
- Authors
- Dowden, H., & Munro, J.
- Corresponding author
- Munro, Jamie
- Venue
- Nature Reviews Drug Discovery
- Published
- 2019-05-08
- Volume
- 18
- Issue
- 7
- Pages
- 495-496
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- OpenAlex
- W2944466104
- Cited by
- 649
- References
- 0
- Citations by year
- 2026: 101 · 2025: 129 · 2024: 140 · 2023: 114 · 2022: 57 · 2021: 57 · 2020: 43 · 2019: 7
- FWCI
- 39.38
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › Science, Research, and Medicine
- Health Sciences › Medicine › Pharmacology (medical) › Historical Medical Research and Treatments
- Health Sciences › Medicine › Internal Medicine › General Medicine and Therapeutics
- Keywords
- clinical success rate, therapeutic focus, drug development, clinical trial results, therapeutic areas
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