Estimation of clinical trial success rates and related parameters
Previous estimates of drug development success rates rely on relatively small samples from databases curated by the pharmaceutical industry and are subject to potential selection biases. Using a sample of 406 038 entries of clinical trial data for over 21 143 compounds from January 1, 2000 to Octobe...
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| Format: | Article |
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Oxford University Press (OUP)
2020
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| Online Access: | https://hdl.handle.net/1721.1/128480 |
| _version_ | 1811089059341139968 |
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| author | Wong, Chi Heem Siah, Kien Wei Lo, Andrew W |
| author2 | Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory |
| author_facet | Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory Wong, Chi Heem Siah, Kien Wei Lo, Andrew W |
| author_sort | Wong, Chi Heem |
| collection | MIT |
| description | Previous estimates of drug development success rates rely on relatively small samples from databases curated by the pharmaceutical industry and are subject to potential selection biases. Using a sample of 406 038 entries of clinical trial data for over 21 143 compounds from January 1, 2000 to October 31, 2015, we estimate aggregate clinical trial success rates and durations. We also compute disaggregated estimates across several trial features including disease type, clinical phase, industry or academic sponsor, biomarker presence, lead indication status, and time. In several cases, our results differ significantly in detail from widely cited statistics. For example, oncology has a 3.4% success rate in our sample vs. 5.1% in prior studies. However, after declining to 1.7% in 2012, this rate has improved to 2.5% and 8.3% in 2014 and 2015, respectively. In addition, trials that use biomarkers in patient-selection have higher overall success probabilities than trials without biomarkers. |
| first_indexed | 2024-09-23T14:13:07Z |
| format | Article |
| id | mit-1721.1/128480 |
| institution | Massachusetts Institute of Technology |
| last_indexed | 2024-09-23T14:13:07Z |
| publishDate | 2020 |
| publisher | Oxford University Press (OUP) |
| record_format | dspace |
| spelling | mit-1721.1/1284802022-10-01T19:51:02Z Estimation of clinical trial success rates and related parameters Wong, Chi Heem Siah, Kien Wei Lo, Andrew W Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory Massachusetts Institute of Technology. Department of Electrical Engineering and Computer Science Sloan School of Management Previous estimates of drug development success rates rely on relatively small samples from databases curated by the pharmaceutical industry and are subject to potential selection biases. Using a sample of 406 038 entries of clinical trial data for over 21 143 compounds from January 1, 2000 to October 31, 2015, we estimate aggregate clinical trial success rates and durations. We also compute disaggregated estimates across several trial features including disease type, clinical phase, industry or academic sponsor, biomarker presence, lead indication status, and time. In several cases, our results differ significantly in detail from widely cited statistics. For example, oncology has a 3.4% success rate in our sample vs. 5.1% in prior studies. However, after declining to 1.7% in 2012, this rate has improved to 2.5% and 8.3% in 2014 and 2015, respectively. In addition, trials that use biomarkers in patient-selection have higher overall success probabilities than trials without biomarkers. 2020-11-13T21:39:39Z 2020-11-13T21:39:39Z 2018-01 2017-11 2019-02-22T16:30:21Z Article http://purl.org/eprint/type/JournalArticle 1465-4644 1468-4357 https://hdl.handle.net/1721.1/128480 Wong, Chi Heem et al. “Estimation of Clinical Trial Success Rates and Related Parameters.” Biostatistics 20, 2 (January 2018): 273–286 © 2018 Oxford University Press http://dx.doi.org/10.1093/BIOSTATISTICS/KXX069 Biostatistics Creative Commons Attribution 4.0 International license https://creativecommons.org/licenses/by/4.0/ application/pdf Oxford University Press (OUP) Oxford University Press |
| spellingShingle | Wong, Chi Heem Siah, Kien Wei Lo, Andrew W Estimation of clinical trial success rates and related parameters |
| title | Estimation of clinical trial success rates and related parameters |
| title_full | Estimation of clinical trial success rates and related parameters |
| title_fullStr | Estimation of clinical trial success rates and related parameters |
| title_full_unstemmed | Estimation of clinical trial success rates and related parameters |
| title_short | Estimation of clinical trial success rates and related parameters |
| title_sort | estimation of clinical trial success rates and related parameters |
| url | https://hdl.handle.net/1721.1/128480 |
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