The DOI matches its Crossref record.
Trends in clinical success rates and therapeutic focus
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“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]
Citations
Cited by
Sorted by citations
Molecular Docking: Shifting Paradigms in Drug Discovery
Pinzi, L., & Rastelli, G. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20184331 · Cited by 2,537
“Considering the high attrition rates characterizing drug discovery [203,204], the possibilities offered by docking in combination with the approaches outlined here will be important to reduce time and costs in both the development of clinical candidates with better safety profiles (target profiling and ADRs findings)…”
BackgroundWhy 90% of clinical drug development fails and how to improve it?
Sun, D., Gao, W., Hu, H., & Zhou, S. (2022) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2022.02.002 · Cited by 1,763
“The “rule of 5” have been considered in chemical structure design: (1) less than 5 H-bond donors; (2) molecular weight less than 500; (3) cLogP less than 5; (4) less than 10 H-bond acceptors(31).”
BackgroundMethodInfluentialFu, L., Shi, S., Yi, J., Wang, N., He, Y., Wu, Z., Peng, J., Deng, Y., Wang, W., Wu, C., et al. (2024) · Nucleic Acids Research · doi:10.1093/nar/gkae236 · Cited by 1,396
“Addition to them, toxicity, another important evaluation indicator in the drug development process, also accounts for up to 30% of drug development failures ( 2 ,4 ).”
Advancing human genetics research and drug discovery through exome sequencing of the UK Biobank
Szustakowski, J. D., Balasubramanian, S., Kvikstad, E., Khalid, S., Bronson, P. G., Sasson, A., Wong, E., Liu, D., Wade Davis, J., Haefliger, C., et al. (2021) · Nature Genetics · doi:10.1038/s41588-021-00885-0 · Cited by 401
Transfer Learning for Drug Discovery
Cai, C., Wang, S., Xu, Y., Zhang, W., Tang, K., Ouyang, Q., Lai, L., & Pei, J. (2020) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.9b02147 · Cited by 376
Zushin, P.-J. H., Mukherjee, S., & Wu, J. C. (2023) · Journal of Clinical Investigation · doi:10.1172/jci175824 · Cited by 347
“Potential therapeutics from preclinical studies entering phase I trials only had a 10.4% approval rate between 2003 and 2014 (2) and an even lower 6% to 7% rate between 2011 and 2017 (3).”
BackgroundInfluentialZeng, X., Xiang, H., Yu, L., Wang, J., Li, K., Nussinov, R., & Cheng, F. (2022) · Nature Machine Intelligence · doi:10.1038/s42256-022-00557-6 · Cited by 322
Cancer therapy with antibodies
Paul, S., Konig, M. F., Pardoll, D. M., Bettegowda, C., Papadopoulos, N., Wright, K. M., Gabelli, S. B., Ho, M., van Elsas, A., & Zhou, S. (2024) · Nature reviews. Cancer · doi:10.1038/s41568-024-00690-x · Cited by 292
“144,145).”
Knowledge graph-enhanced molecular contrastive learning with functional prompt
Yin, F., Zhang, Q., Zhang, N., Chen, Z., Zhuang, X., Shao, X., Fan, X., & Chen, H. (2023) · Nature Machine Intelligence · doi:10.1038/s42256-023-00654-0 · Cited by 251
“Results Overview of KANO In this paper, we propose KANO, a new KG-enhanced molecular contrastive learning with functional prompt method, which consists of three main components: (1) ElementKG construction and embedding, (2) contrastive-based pre-training and (3) prompt-enhanced fine-tuning.”
BackgroundMethodInfluentialImproving target assessment in biomedical research: the GOT-IT recommendations
Emmerich, C. H., Martinez‐Gamboa, L., Hofmann, M. C. J., Bonin, M., Arbach, O., Schendel, P., Gerlach, B., Hempel, K., Bespalov, A., Dirnagl, U., et al. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0087-3 · Cited by 243
Generative molecular design in low data regimes
Moret, M., Friedrich, L., Grisoni, F., Merk, D., & Schneider, G. (2020) · Nature Machine Intelligence · doi:10.1038/s42256-020-0160-y · Cited by 219
“We compared the effect of this approach on model training in terms of (1) the validity; that is, the percentage of SMILES strings that can be translated back to molecular graphs; (2) the uniqueness, which is the percentage of non-duplicated SMILES strings; and (3) the novelty of the generated molecules calculated as a…”
MethodOpen Targets Platform: facilitating therapeutic hypotheses building in drug discovery
Buniello, A., Süveges, D., Cruz-Castillo, C., Llinares, M. B., Cornu, H., López, I., Tsukanov, K., Roldán‐Romero, J. M., Mehta, C., Fumis, L., et al. (2024) · Nucleic Acids Research · doi:10.1093/nar/gkae1128 · Cited by 251
“For every 10 drugs that enter clinical trials, only one will reach regulatory approval ( 1 ), with 79% of the programs closing due to efficacy or safety concerns ( 2 ).”
Artificial intelligence in drug discovery: applications and techniques
Deng, J., Yang, Z., Ojima, I., Samaras, D., & Wang, F. (2021) · Briefings in Bioinformatics · doi:10.1093/bib/bbab430 · Cited by 244
Machine Learning Toxicity Prediction: Latest Advances by Toxicity End Point
Cavasotto, C. N., & Scardino, V. (2022) · ACS Omega · doi:10.1021/acsomega.2c05693 · Cited by 234
Biased Allosteric Modulators: New Frontiers in GPCR Drug Discovery
Slosky, L. M., Caron, M. G., & Barak, L. S. (2021) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2020.12.005 · Cited by 214
Machine learning in preclinical drug discovery
Catacutan, D. B., Alexander, J., Arnold, A., & Stokes, J. M. (2024) · Nature Chemical Biology · doi:10.1038/s41589-024-01679-1 · Cited by 227
“5,6 .”
Holmes, M. V., Richardson, T. G., Ference, B. A., Davies, N. M., & Davey Smith, G. (2021) · Nature Reviews Cardiology · doi:10.1038/s41569-020-00493-1 · Cited by 184
A disease-associated gene desert directs macrophage inflammation through ETS2
Stankey, C. T., Bourges, C., Haag, L.-M., Turner‐Stokes, T., Piedade, A. P., Palmer-Jones, C., Papa, I., dos Santos, M. S., Zhang, Q., Cameron, A. J., et al. (2024) · Nature · doi:10.1038/s41586-024-07501-1 · Cited by 163
Gioiello, A., Piccinno, A., Lozza, A. M., & Cerra, B. (2020) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.9b01956 · Cited by 153
Tuberculosis Drug Discovery: Challenges and New Horizons
Fernandes, G., Thompson, A. M., Castagnolo, D., Denny, W. A., & dos Santos, J. L. (2022) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.2c00227 · Cited by 153
Predicting the frequencies of drug side effects
Galeano, D., Li, S., Gerstein, M., & Paccanaro, A. (2020) · Nature Communications · doi:10.1038/s41467-020-18305-y · Cited by 147
A critical perspective on 3D liver models for drug metabolism and toxicology studies
Serras, A. S., Rodrigues, J. S., Cipriano, M., Rodrigues, A. V., Oliveira, N. G., & Miranda, J. P. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.626805 · Cited by 138
Role of artificial intelligence in revolutionizing drug discovery
Rehman, A. U., Li, M., Wu, B., Ali, Y., Rasheed, S., Shaheen, S., Liu, X., Luo, R., & Zhang, J. (2024) · Fundamental Research · doi:10.1016/j.fmre.2024.04.021 · Cited by 160
“Advancing a drug candidate to phase-I clinical trial after rigorous optimization at the preclinical stage is considered a significant milestone for both pharmaceutical companies and academic institutions [ 4 ].”
Endothelial Regulation of Drug Transport in a 3D Vascularized Tumor Model
Haase, K., Offeddu, G. S., Gillrie, M. R., & Kamm, R. D. (2020) · Advanced Functional Materials · doi:10.1002/adfm.202002444 · Cited by 126
Wu, C., Lorenzo, G., Hormuth, D. A., Lima, E. A. B. F., Slavkova, K. P., DiCarlo, J. C., Virostko, J. M., Phillips, C. M., Patt, D. A., Chung, C. D., et al. (2022) · Biophysics Reviews · doi:10.1063/5.0086789 · Cited by 147
Wolf, J., Rasmussen, D. K., Sun, Y. J., Vu, J. T., Wang, E., Espinosa, C. A., Bigini, F., Chang, R. T., Montague, A. A., Tang, P. H., et al. (2023) · Cell · doi:10.1016/j.cell.2023.09.012 · Cited by 130
Bacterial therapies at the interface of synthetic biology and nanomedicine
Hahn, J., Ding, S., Im, J., Harimoto, T., Leong, K. W., & Danino, T. (2023) · Nature Reviews Bioengineering · doi:10.1038/s44222-023-00119-4 · Cited by 125
“In general, many drugs fail in clinical trials owing to differences between preclinical models and human patients, which result in inadequate efficacy and excessive toxicity 153 .”
Navigating the complexities of drug development for inflammatory bowel disease
Honap, S., Jairath, V., Danese, S., & Peyrin‐Biroulet, L. (2024) · Nature Reviews Drug Discovery · doi:10.1038/s41573-024-00953-0 · Cited by 123
“Although the highest attrition rates are in early phase clinical trials, failure of phase III IBD assets has occurred 12–15 .”
Yamaguchi, S., Kaneko, M., & Narukawa, M. (2021) · Clinical and Translational Science · doi:10.1111/cts.12980 · Cited by 125
Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discovery
Morimoto, S., Takahashi, S., Ito, D., Daté, Y., Okada, K., Kato, C., Nakamura, S., Ozawa, F., Chai, M., Nishiyama, A., et al. (2023) · Cell stem cell · doi:10.1016/j.stem.2023.04.017 · Cited by 111
Image-based high-content screening in drug discovery
Lin, S., Schorpp, K., Rothenaigner, I., & Hadian, K. (2020) · Drug Discovery Today · doi:10.1016/j.drudis.2020.06.001 · Cited by 118
Meta Learning With Graph Attention Networks for Low-Data Drug Discovery
Lv, Q., Chen, G., Yang, Z., Zhong, W., & Chen, C. Y. (2023) · IEEE Transactions on Neural Networks and Learning Systems · doi:10.1109/tnnls.2023.3250324 · Cited by 112
“D RUG discovery is a high-investment, long-period, and high-risk systems engineering [1].”
BackgroundZhang, J., Li, H., Zhang, Y., Huang, J., Ren, L., Zhang, C., Zou, Q., & Zhang, Y. (2025) · Briefings in Bioinformatics · doi:10.1093/bib/bbaf533 · Cited by 134
Computational drug repurposing: approaches, evaluation of in silico resources and case studies
Tanoli, Z., Fernández‐Torras, A., Özcan, U. O., Kushnir, A., Nader, K. M., Gadiya, Y., Fiorenza, L., Ianevski, A., Vähä-Koskela, M. J. V., Miihkinen, M., et al. (2025) · Nature Reviews Drug Discovery · doi:10.1038/s41573-025-01164-x · Cited by 104
Medicamentos e tratamentos para a Covid-19
Ferreira, L. L. G., & Andricopulo, A. D. (2020) · Estudos Avançados · doi:10.1590/s0103-4014.2020.34100.002 · Cited by 104
Fischer, I., Milton, C., & WALLACE, H. M. (2020) · Toxicology Research · doi:10.1093/toxres/tfaa011 · Cited by 94
Fehlings, M. G., Moghaddamjou, A., Harrop, J. S., et al. (2023) · Journal of Neurotrauma · doi:10.1089/neu.2023.0163 · Cited by 93
“28 In response, advocacy communities have lobbied for revised regulatory guidance on clinical trials for Duchenne muscular dystrophy 29 and ALS, 30 with recommendations such as virtual controls, increased use of secondary outcomes, and potential elimination of the placebo arm.”
BackgroundWei, Y., Li, S., Li, Z., Ziwei, W., & Lin, J. (2022) · Bioinformatics · doi:10.1093/bioinformatics/btac192 · Cited by 94
Replacing Animal Testing with Stem Cell-Organoids : Advantages and Limitations
Park, G., Rim, Y. A., Sohn, Y., Nam, Y., & Ju, J. H. (2024) · Stem Cell Reviews and Reports · doi:10.1007/s12015-024-10723-5 · Cited by 92
“Between 2010 and 2017, clinical trials for drugs had a greater chance of failing phase І, owing to safety and efficacy [ 31].”
BackgroundAnalysis of pharma R&D productivity – a new perspective needed
Schuhmacher, A., Hinder, M., Stein, A. V. S. U., Hartl, D., & Gassmann, O. (2023) · Drug Discovery Today · doi:10.1016/j.drudis.2023.103726 · Cited by 103
Graph Neural Networks in Modern AI-Aided Drug Discovery
Zhang, O., Lin, H., Zhang, X., Wang, X., Wu, Z., Ye, Q., Zhao, W., Wang, J., Ying, K., Kang, Y., et al. (2025) · Chemical Reviews · doi:10.1021/acs.chemrev.5c00461 · Cited by 110
Radio Signals from Live Cells: The Coming of Age of In-Cell Solution NMR
Luchinat, E., Cremonini, M., & Banci, L. (2022) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00790 · Cited by 86
Repurposing drugs for the management of COVID-19
Cusinato, J., Cau, Y., Calvani, A. M., & Mori, M. (2020) · Expert Opinion on Therapeutic Patents · doi:10.1080/13543776.2021.1861248 · Cited by 78
“Since the beginning of the pandemic, remdesivir was one of the most promising candidates, and its repurposing for COVID-19 allowed the development of focused phase III clinical trials in early 2020.”
BackgroundInfluentialAllosteric TYK2 inhibition: redefining autoimmune disease therapy beyond JAK1-3 inhibitors
Jensen, L. T., Attfield, K. E., Feldmann, M., & Fugger, L. H. (2023) · EBioMedicine · doi:10.1016/j.ebiom.2023.104840 · Cited by 88
Use of artificial intelligence to enhance phenotypic drug discovery
Malandraki-Miller, S., & Riley, P. R. (2021) · Drug Discovery Today · doi:10.1016/j.drudis.2021.01.013 · Cited by 84
Translation of AI into oncology clinical practice
Naqa, I. E., Karolak, A. M., Luo, Y., Folio, L. R., Tarhini, A. A., Rollison, D. E. M., & Parodi, K. (2023) · Oncogene · doi:10.1038/s41388-023-02826-z · Cited by 85
“Among various malignancies and proposed treatments, anticancer drugs have less than 10% probability to successfully pass phase I clinical trials, the numbers look more promising for phases II and III [80].”
BackgroundMahalmani, V., Sinha, S., Prakash, A., & Medhi, B. (2022) · Indian Journal of Pharmacology · doi:10.4103/ijp.ijp_860_22 · Cited by 87
“[2,3] The failure rates might be reduced by adopting strict criteria during the preclinical stages of drug development.”
GraphormerDTI: A graph transformer-based approach for drug-target interaction prediction
Gao, M., Zhang, D., Chen, Y., Zhang, Y., Wang, Z., Wang, X., Li, S., Guo, Y., Webb, G. I., Nguyen, A. T. N., et al. (2024) · Computers in Biology and Medicine · doi:10.1016/j.compbiomed.2024.108339 · Cited by 81
Evolution and impact of high content imaging.
Way, G. P., Sailem, H., Shave, S., Kasprowicz, R., & Carragher, N. O. (2023) · SLAS Discovery · doi:10.1016/j.slasd.2023.08.009 · Cited by 67
“The probability of launch statistics are reflected within more recent analyses and as a sub-category, Phase II failure, in particular, has been identified as 79% attributable to safety and efficacy [59].”
Drug Screening in Human Cells by NMR Spectroscopy Allows the Early Assessment of Drug Potency
Luchinat, E., Barbieri, L., Cremonini, M., et al. (2020) · Angewandte Chemie International Edition · doi:10.1002/anie.201913436 · Cited by 70
Machine Learning Empowering Drug Discovery: Applications, Opportunities and Challenges
Qi, X., Zhao, Y., Qi, Z., Hou, S., & Chen, J. (2024) · Molecules · doi:10.3390/molecules29040903 · Cited by 75
“Despite the high investment levels, the approval success rate of launching a small-molecule drug to market from phase I clinical trial is less than 10% [3], highlighting the considerable risk of failure.”
BackgroundHartung, I. V., Huck, B. R., & Crespo, A. (2023) · Nature Reviews Chemistry · doi:10.1038/s41570-022-00451-0 · Cited by 65
Advances in 3D neuronal microphysiological systems: towards a functional nervous system on a chip
Anderson, W. A., Bosak, A. J., Högberg, H. T., Härtung, T., & Moore, M. J. (2021) · In Vitro Cellular & Developmental Biology - Animal · doi:10.1007/s11626-020-00532-8 · Cited by 66
Modulating the Blood–Brain Barrier: A Comprehensive Review
Whelan, R., Hargaden, G. C., & Knox, A. J. S. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13111980 · Cited by 65
“[19], with additional extrapolations made.”
BackgroundInfluentialHou, X., Du, C., Lu, L., Yuan, S., Zhan, M., You, P., & Du, H. (2022) · World Journal of Surgical Oncology · doi:10.1186/s12957-022-02510-8 · Cited by 63
“Meanwhile, when tested in clinical trials, only ~ 5% of the drug candidates can be approved by the governmental drug administration [2, 4].”
BackgroundMazzucato, M., & Li, H. L. (2021) · Journal of Law, Medicine & Ethics · doi:10.1017/jme.2021.8 · Cited by 60
“But neglected diseases are not confined to tropical diseases: Central nervous system disorders, a therapeutic area for which there has been low probability of clinical trial success, have been increasingly marginalised from many companies’ research and development pipelines, leading to an increasing disconnect between…”
BackgroundZhang, S., Yan, Z., Huang, Y., Liu, L., He, D., Wang, W., Fang, X., Zhang, X., Wang, F., Wu, H. R., et al. (2022) · Bioinformatics · doi:10.1093/bioinformatics/btac342 · Cited by 68
“INTRODUCTION The modern drug discovery process is extremely time-consuming and expensive, and yet, only 10-20% of drug candidates can make it to the market (1, 2).”
Background3D human liver spheroids for translational pharmacology and toxicology
Ingelman‐Sundberg, M., & Lauschke, V. M. (2022) · Basic & Clinical Pharmacology & Toxicology · doi:10.1111/bcpt.13587 · Cited by 62
Repurposing metformin to treat age-related neurodegenerative disorders and ischemic stroke
Sharma, S., Nozohouri, S., Vaidya, B., & Abbruscato, T. J. (2021) · Life Sciences · doi:10.1016/j.lfs.2021.119343 · Cited by 62
Recent advances in human respiratory epithelium models for drug discovery
Yaqub, N., Wayne, G. J., Birchall, M., & Song, W. (2021) · Biotechnology Advances · doi:10.1016/j.biotechadv.2021.107832 · Cited by 64
Dendrimers toward Translational Nanotherapeutics: Concise Key Step Analysis
Mignani, S. M., Shi, X., Rodrigues, J., Roy, R., Muñoz‐Fernández, M. Á., Ceña, V., & Majoral, J. (2020) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.0c00395 · Cited by 56
Machine learning and network medicine approaches for drug repositioning for COVID-19
Santos, S. D. S., Torres, M., Galeano, D., Sánchez, M. D. M., Cernuzzi, L., & Paccanaro, A. (2021) · Patterns · doi:10.1016/j.patter.2021.100396 · Cited by 57
“This observation is motivated by the empirical evidence (e.g., Dowden and Munro 25 ) that the probability of success of a candidate drug increases as the candidate drug moves to the next developmental stage in the drug development process.”
InfluentialNear‐Infrared Chemiluminescence Imaging of Chemotherapy‐Induced Peripheral Neuropathy
Liu, J., Huang, J., Wei, X., Cheng, P., & Pu, K. (2023) · Advanced Materials · doi:10.1002/adma.202310605 · Cited by 57
R&D efficiency of leading pharmaceutical companies – A 20-year analysis
Schuhmacher, A., Wilisch, L., Kuss, M., Kandelbauer, A., Hinder, M., & Gassmann, O. (2021) · Drug Discovery Today · doi:10.1016/j.drudis.2021.05.005 · Cited by 59
Jentzsch, V., Osipenko, L., Scannell, J. W., & Hickman, J. A. (2023) · JAMA Network Open · doi:10.1001/jamanetworkopen.2023.24977 · Cited by 53
Proteomics in the pharmaceutical and biotechnology industry: a look to the next decade
Lill, J. R., Mathews, W. R., Rose, C. M., & Schirle, M. (2021) · Expert Review of Proteomics · doi:10.1080/14789450.2021.1962300 · Cited by 57
Advances in targeting RNA modifications for anticancer therapy
Pomaville, M. M., & He, C. (2023) · Trends in cancer · doi:10.1016/j.trecan.2023.04.003 · Cited by 52
Kundu, A., McCoy, L., Azim, N., Nguyen, H. T., Didier, C. M., Ausaf, T., Sharma, A. D., Curley, J. L., Moore, M. J., & Rajaraman, S. (2020) · ACS Biomaterials Science & Engineering · doi:10.1021/acsbiomaterials.0c01184 · Cited by 53
Precision medicine for cardiometabolic disease: a framework for clinical translation
Franks, P. W., Cefalu, W. T., Dennis, J., Florez, J. C., Mathieu, C., Morton, R. W., Ridderstråle, M., Sillesen, H. H., & Stehouwer, C. D. A. (2023) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(23)00165-1 · Cited by 57
Genetic factors associated with reasons for clinical trial stoppage
Razuvayevskaya, O., López, I., Dunham, I., & Ochoa, D. (2024) · Nature Genetics · doi:10.1038/s41588-024-01854-z · Cited by 59
“Recent reports indicate that 79% of clinical studies fail because of a lack of efficacy or safety 2 .”
Added Value of Clinical Sequencing: WGS-Based Profiling of Pharmacogenes
Caspar, S. M., Schneider, T., Meienberg, J., & Mátyás, G. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21072308 · Cited by 51
“Currently, only 10% of all drugs that initiate phase I clinical trials are subsequently approved [2], among other reasons potentially due to neglected patient stratification such as according to metabolizer status in clinical trials.”
BackgroundAppelt‐Menzel, A., Oerter, S., Mathew, S., Haferkamp, U., Hartmann, C., Jung, M., Neuhaus, W., & Pleß, O. (2020) · Current Protocols in Stem Cell Biology · doi:10.1002/cpsc.122 · Cited by 56
How can machine learning and multiscale modeling benefit ocular drug development?
Wang, N., Zhang, Y., Wang, W., Ye, Z., Chen, H., Hu, G., & Ouyang, D. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114772 · Cited by 52
Degraders upgraded: the rise of PROTACs in hematological malignancies
Casan, J. M. L., & Seymour, J. F. (2024) · Blood · doi:10.1182/blood.2023022993 · Cited by 53
Lee, S. Y., & Yoo, S. (2024) · Journal of Cheminformatics · doi:10.1186/s13321-023-00796-8 · Cited by 50
“The results of clinical trials show that approximately 90% of new active substances fail to gain regulatory approval due to poor efficacy and unexpected toxicity [5].”
BackgroundCommon pitfalls in drug target Mendelian randomization and how to avoid them
Gill, D., Dib, M., Cronjé, H. T., Karhunen, V., Woolf, B. A. R., Gagnon, É., Daghlas, I., Nyberg, M., Drakeman, D. L., & Burgess, S. (2024) · BMC Medicine · doi:10.1186/s12916-024-03700-9 · Cited by 51
Bioengineered in vitro skeletal muscles as new tools for muscular dystrophies preclinical studies
Fernández‐Costa, J. M., Fernández‐Garibay, X., Velasco‐Mallorquí, F., & Ramón‐Azcón, J. (2021) · Journal of Tissue Engineering · doi:10.1177/2041731420981339 · Cited by 48
Foley, T. L., Burchett, W., Chen, Q., Flanagan, M. E., Kapinos, B., Li, X.-Y., Montgomery, J. I., Ratnayake, A. S., Zhu, H., & Peakman, M. (2021) · SLAS DISCOVERY · doi:10.1177/2472555220979589 · Cited by 49
Coccia, M. (2023) · Technology in Society · doi:10.1016/j.techsoc.2023.102384 · Cited by 50
Mendelian Randomization Applied to Neurology
Gagnon, É., Daghlas, I., Zagkos, L., Sargurupremraj, M., Georgakis, M. K., Anderson, C. D., Cronjé, H. T., Burgess, S., Arsenault, B. J., & Gill, D. (2024) · Neurology · doi:10.1212/wnl.0000000000209128 · Cited by 50
“14 The MR paradigm is a promising strategy to help accelerate and reduce the cost of the drug development pipeline.”
BackgroundOuédraogo, G., Alépée, N., Tan, B., & Roper, C. S. (2025) · Regulatory Toxicology and Pharmacology · doi:10.1016/j.yrtph.2025.105904 · Cited by 59
Artificial intelligence for small molecule anticancer drug discovery
Duo, L., Liu, Y., Ren, J., Tang, B., & Hirst, J. D. (2024) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2024.2367014 · Cited by 51
Reviving an R&D pipeline: a step change in the Phase II success rate
Wu, S. S., Fernando, K., Allerton, C. M. N., Jansen, K. U., Vincent, M. S., & Dolsten, M. (2020) · Drug Discovery Today · doi:10.1016/j.drudis.2020.10.019 · Cited by 47
Target Validation Using PROTACs: Applying the Four Pillars Framework
Nowak, R. P., & Jones, L. H. (2020) · SLAS DISCOVERY · doi:10.1177/2472555220979584 · Cited by 48
Flourishing tumor organoids: History, emerging technology, and application
Yang, Q., Li, M., Yang, X., Xiao, Z., Tong, X., Tuerdi, A., Li, S., & Lei, L. (2023) · Bioengineering & Translational Medicine · doi:10.1002/btm2.10559 · Cited by 46
“didates from phase I clinical trials to the initiation of late-stage development is below 10%, and the main reason for their failure is inadequate safety or efficacy.(179) Therefore, the establishment of”
BackgroundFernald, K., Förster, P. C., Claassen, E., & van de Burgwal, L. (2024) · Drug Discovery Today · doi:10.1016/j.drudis.2024.104160 · Cited by 56
Peng, Y., Wang, M., Xu, Y., et al. (2020) · Briefings in Bioinformatics · doi:10.1093/bib/bbaa027 · Cited by 43
Microfluidic Liver-on-a-Chip for Preclinical Drug Discovery
Fu, J., Qiu, H., & Tan, C. S. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15041300 · Cited by 42
nach0: multimodal natural and chemical languages foundation model
Livne, M., Miftahutdinov, Z., Tutubalina, E. V., Kuznetsov, M., Polykovskiy, D. A., Brundyn, A., Jhunjhunwala, A., Costa, A. B., Aliper, A., Aspuru‐Guzik, A., et al. (2024) · Chemical Science · doi:10.1039/d4sc00966e · Cited by 61
“…for the biomedical domain, utilizing a variety of model families: for instance, researchers have developed BioBERT 17 , based on BERT with 110 million parameters, and SciFive, based on T5-base and T5-large with 220 and 770 million parameters respectively, using biomedical literature from PubMed.”
MethodSchuhmacher, A., Hinder, M., Brief, E., Gassmann, O., & Hartl, D. (2025) · Drug Discovery Today · doi:10.1016/j.drudis.2025.104291 · Cited by 47
Applications of Artificial Intelligence in Drug Design: Opportunities and Challenges
Thomas, M. C., Boardman, A., García-Ortegón, M., Yang, H., de Graaf, C., & Bender, A. (2021) · Methods in molecular biology · doi:10.1007/978-1-0716-1787-8_1 · Cited by 43
Cohen, B. M., Lehnherr, D., Sezen-Edmonds, M., et al. (2023) · Process Safety and Environmental Protection · doi:10.1016/j.cherd.2023.02.050 · Cited by 42
Creemers, J. H. A., Ankan, A., Roes, K. C. B., Schröder, G., Mehra, N., Figdor, C. G., de Vries, I. J. M., & Textor, J. C. (2023) · Nature Communications · doi:10.1038/s41467-023-37933-8 · Cited by 39
“%) ultimately obtain market approval 2 – 4 .”
Prodrug Approach as a Strategy to Enhance Drug Permeability
de Souza, M. M., Gini, A. L. R., Moura, J. A., Scarim, C. B., Chin, C. M., & dos Santos, J. L. (2025) · Pharmaceuticals · doi:10.3390/ph18030297 · Cited by 47
“Studies indicate that 9 out of 10 approved drugs fail in demonstrating efficacy and safety during various stages of development [2].”
Cell-Type-Specific High Throughput Toxicity Testing in Human Midbrain Organoids
Renner, H., Becker, K., Kagermeier, T., Grabos, M., Eliat, F., Günther, P. S., Schöler, H. R., & Bruder, J. M. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2021.715054 · Cited by 41
The rat as an animal model in chronic wound research: An update
Ghanbari, M., Salkovskiy, Y., & Carlson, M. A. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.122783 · Cited by 52
Yıldırım, Z., Swanson, K., Wu, X., Zou, J., & Wu, J. C. (2024) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-022724-095035 · Cited by 40
Shamul, J. G., Wang, Z., Gong, H., Ou, W., White, A. M., Garcia, D. P. M., Gu, S., Clyne, A. M., Quiñones‐Hinojosa, A., & He, X. (2024) · Nature Biomedical Engineering · doi:10.1038/s41551-024-01250-2 · Cited by 43
“Animal models are currently used widely for preclinical testing of drug transport across the BBB and efficacy in the brain; however, better models are needed since ~92% of drug candidates that show success in animal models fail in clinical trials 98–100 .”
Meta-MolNet: A Cross-Domain Benchmark for Few Examples Drug Discovery
Lv, Q., Chen, G., Yang, Z., Zhong, W., & Chen, C. Y. (2024) · IEEE Transactions on Neural Networks and Learning Systems · doi:10.1109/tnnls.2024.3359657 · Cited by 42
“T HE discovery of new drugs is a system engineering with high investment, long cycle, and high risk [1], [2].”
Clinical trials in-a-dish for cardiovascular medicine
Wu, X., Swanson, K., Yıldırım, Z., Liu, W., Liao, R., & Wu, J. C. (2024) · European Heart Journal · doi:10.1093/eurheartj/ehae519 · Cited by 42
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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review