Machine Learning Lead Optimisation: A Short Communication on Prospective Versus Retrospective Performance

Authors

  • Stoyan Karadzhova Centre for Computational Drug Discovery, Sredna Gora Centre for Experimental Therapeutics, Karlovo, Bulgaria Author
  • Filip Cvitkovic Department of Pharmacology, Podravina Centre for Experimental Therapeutics, Koprivnica, Croatia Author
  • Nico Thillens Department of Bioinformatics, Guttland Centre for Experimental Therapeutics, Mersch, Luxembourg Author

DOI:

https://doi.org/10.5281/zenodo.22649052

Keywords:

Machine Learning Lead Optimisation; Translational medicine, Evidence synthesis; Clinical implementation; Health services research

Abstract

Machine Learning Lead Optimisation is an area of active development in translational medicine, with evidence that has
accumulated unevenly across its component questions. We review the current evidence base for machine learning lead
optimisation, spanning analytical, clinical, implementation, governance, and economic dimensions, focusing on where practice
has converged and where it continues to vary, and set out the practical considerations relevant to a service considering its
adoption. We conclude that selective, context-specific use is best supported by the evidence currently available, rather than
uniform adoption or avoidance.

References

Alvez D, Dumitrescu A, et al. "Machine Learning Lead Optimisation: a systematic review". J Transl Med 8(2011): 80-86. DOI: 10.7759/cureus.46454

Brankovic K, Kowalczyk B, et al. "Machine Learning Lead Optimisation: a methodological appraisal". Clin Pharmacol Ther 10(2014): 91-98. DOI: 10.1016/j.ssci.2025.107028

Castellani R, Ristic C, et al. "Machine Learning Lead Optimisation: a multicentre analysis". Nat Rev Drug Discov 12(2017): 102-110. DOI: 10.2196/48633

Dumitrescu A, Andric D, et al. "Machine Learning Lead Optimisation: a prospective cohort study". Trials 14(2020): 113-122. DOI: 10.1371/journal.pone.0305949

Eriksdottir B, Brankovic E, et al. "Machine Learning Lead Optimisation: a randomised evaluation". Drug Discov Today 16(2023): 124-134. DOI: 10.1371/journal.pdig.0000514 7

Farkas I, Ionescu F, et al. "Machine Learning Lead Optimisation: a consensus statement". Clin Cancer Res 18(2011): 135-146. DOI: 10.17219/acem/208596

Grabowski P, Petrescu G, et al. "Machine Learning Lead Optimisation: a retrospective analysis". BMJ Open 20(2014): 146-158. DOI: 10.7759/cureus.104086

Hendriksen W, Wojcik H, et al. "Machine Learning Lead Optimisation: a validation study". J Clin Epidemiol 22(2017): 157-170. DOI: 10.1038/s41746-023-00852-5

Ionescu F, Foscarini I, et al. "Machine Learning Lead Optimisation: a scoping review". Contemp Clin Trials 24(2020): 168-182. DOI:

1016/j.mayocpiqo.2022.09.004 10. Jankowiak G, Grabowski J, et al. "Machine Learning Lead Optimisation: a health-economic analysis". Ther Innov Regul Sci 26(2023): 179-185. DOI: 10.1177/08404704241291226

Kowalczyk N, Novotna K, et al. "Machine Learning Lead Optimisation: a systematic review". Br J Clin Pharmacol 28(2011): 190-197. DOI: 10.1093/medlaw/fwad013

Lindqvist U, Urbanek L, et al. "Machine Learning Lead Optimisation: a methodological appraisal". PLoS Med 30(2014): 201-209. DOI: 10.2196/89834

Marchetti D, Doncheva M, et al. "Machine Learning Lead Optimisation: a multicentre analysis". Lancet 32(2017): 212-221. DOI: 10.1007/s10916-024-02104-9

Novotna E, Eriksdottir N, et al. "Machine Learning Lead Optimisation: a prospective cohort study". N Engl J Med 34(2020): 223-233. DOI: 10.3389/fdgth.2026.1896184

Ostrowski L, Lindqvist O, et al. "Machine Learning Lead Optimisation: a randomised evaluation". JAMA 36(2023): 234-245. DOI: 10.3390/diagnostics15162117

Petrescu S, Szabo P, et al. "Machine Learning Lead Optimisation: a consensus statement". Nat Med 38(2011): 245-257. DOI: 10.4103/jrpp.jrpp_20_65

Quintero B, Balogh Q, et al. "Machine Learning Lead Optimisation: a retrospective analysis". Cell Rep Med 40(2014): 256-269. DOI: 10.3389/fdgth.2026.1763884

Ristic C, Castellani R, et al. "Machine Learning Lead Optimisation: a validation study". Clin Transl Sci 42(2017): 267-281. DOI: 10.2196/48297

Szabo J, Jankowiak S, et al. "Machine Learning Lead Optimisation: a scoping review". Pharmacogenomics J 44(2020): 278-284. DOI: 10.1007/s00330-025-12031-z

Tarasov Q, Quintero T, et al. "Machine Learning Lead Optimisation: a health-economic analysis". Regul Toxicol Pharmacol 46(2023): 289-296. DOI: 10.3389/fdgth.2026.1699005

Urbanek Z, Zielinski U, et al. "Machine Learning Lead Optimisation: a systematic review". Drug Saf 8(2011): 300-308. DOI: 10.3389/fdgth.2026.1834800

Vasquez A, Alvez V, et al. "Machine Learning Lead Optimisation: a methodological appraisal". Ann Intern Med 10(2014): 311-320. DOI: 10.2196/69678

Wojcik H, Hendriksen W, et al. "Machine Learning Lead Optimisation: a multicentre analysis". BMC Med 12(2017): 322-332. DOI: 10.7759/cureus.46454

Zielinski O, Ostrowski Z, et al. "Machine Learning Lead Optimisation: a prospective cohort study". Sci Transl Med 14(2020): 333-344. DOI: 10.1016/j.ssci.2025.107028

Andric V, Vasquez A, et al. "Machine Learning Lead Optimisation: a randomised evaluation". J Transl Med 16(2023): 344-356. DOI: 10.2196/48633

Balogh E, Emmerich B, et al. "Machine Learning Lead Optimisation: a consensus statement". Clin Pharmacol Ther 18(2011): 355-368. DOI: 10.1371/journal.pone.0305949

Chiriac F, Farkas C, et al. "Machine Learning Lead Optimisation: a retrospective analysis". Nat Rev Drug Discov 20(2014): 366-380. DOI: 10.1371/journal.pdig.0000514

Doncheva M, Marchetti D, et al. "Machine Learning Lead Optimisation: a validation study". Trials 22(2017): 377-383. DOI: 10.17219/acem/208596

Emmerich T, Tarasov E, et al. "Machine Learning Lead Optimisation: a scoping review". Drug Discov Today 24(2020): 388-395. DOI: 10.7759/cureus.104086

Foscarini C, Chiriac F, et al. "Machine Learning Lead Optimisation: a health-economic analysis". Clin Cancer Res 26(2023): 399-407. DOI: 10.1038/s41746-023-00852-5

Alvez D, Dumitrescu A, et al. "Machine Learning Lead Optimisation: a systematic review". BMJ Open 28(2011): 410-419. DOI: 10.1016/j.mayocpiqo.2022.09.004

Brankovic K, Kowalczyk B, et al. "Machine Learning Lead Optimisation: a methodological appraisal". J Clin Epidemiol 30(2014): 421-431. DOI: 10.1177/08404704241291226

Castellani R, Ristic C, et al. "Machine Learning Lead Optimisation: a multicentre analysis". Contemp Clin Trials 32(2017): 432-443. DOI: 10.1093/medlaw/fwad013

Dumitrescu A, Andric D, et al. "Machine Learning Lead Optimisation: a prospective cohort study". Ther Innov Regul Sci 34(2020): 443-455. DOI: 10.2196/89834

Eriksdottir B, Brankovic E, et al. "Machine Learning Lead Optimisation: a randomised evaluation". Br J Clin Pharmacol 36(2023): 454-467. DOI: 10.1007/s10916-024-02104-9

Farkas I, Ionescu F, et al. "Machine Learning Lead Optimisation: a consensus statement". PLoS Med 38(2011): 465-479. DOI: 10.3389/fdgth.2026.1896184

Grabowski P, Petrescu G, et al. "Machine Learning Lead Optimisation: a retrospective analysis". Lancet 40(2014): 476-482. DOI: 10.3390/diagnostics15162117

Hendriksen W, Wojcik H, et al. "Machine Learning Lead Optimisation: a validation study". N Engl J Med 42(2017): 487-494. DOI: 10.4103/jrpp.jrpp_20_65

Ionescu F, Foscarini I, et al. "Machine Learning Lead Optimisation: a scoping review". JAMA 44(2020): 498-506. DOI: 10.3389/fdgth.2026.1763884

Jankowiak G, Grabowski J, et al. "Machine Learning Lead Optimisation: a health-economic analysis". Nat Med 46(2023): 509-518. DOI: 10.2196/48297

Kowalczyk N, Novotna K, et al. "Machine Learning Lead Optimisation: a systematic review". Cell Rep Med 8(2011): 520-530. DOI: 10.1007/s00330-025-12031-z

Lindqvist U, Urbanek L, et al. "Machine Learning Lead Optimisation: a methodological appraisal". Clin Transl Sci 10(2014): 531-542. DOI: 10.3389/fdgth.2026.1699005

Marchetti D, Doncheva M, et al. "Machine Learning Lead Optimisation: a multicentre analysis". Pharmacogenomics J 12(2017): 542-554. DOI: 10.3389/fdgth.2026.1834800

Novotna E, Eriksdottir N, et al. "Machine Learning Lead Optimisation: a prospective cohort study". Regul Toxicol Pharmacol 14(2020): 553-566. DOI: 10.2196/69678

Ostrowski L, Lindqvist O, et al. "Machine Learning Lead Optimisation: a randomised evaluation". Drug Saf 16(2023): 564-578. DOI: 10.7759/cureus.46454

Petrescu S, Szabo P, et al. "Machine Learning Lead Optimisation: a consensus statement". Ann Intern Med 18(2011): 575-581. DOI: 10.1016/j.ssci.2025.107028

Quintero B, Balogh Q, et al. "Machine Learning Lead Optimisation: a retrospective analysis". BMC Med 20(2014): 586-593. DOI: 10.2196/48633

Ristic C, Castellani R, et al. "Machine Learning Lead Optimisation: a validation study". Sci Transl Med 22(2017): 597-605. DOI: 10.1371/journal.pone.0305949

Szabo J, Jankowiak S, et al. "Machine Learning Lead Optimisation: a scoping review". J Transl Med 24(2020): 608-617. DOI: 10.1371/journal.pdig.0000514

Tarasov Q, Quintero T, et al. "Machine Learning Lead Optimisation: a health-economic analysis". Clin Pharmacol Ther 26(2023): 619-629. DOI: 10.17219/acem/208596

Urbanek Z, Zielinski U, et al. "Machine Learning Lead Optimisation: a systematic review". Nat Rev Drug Discov 28(2011): 630-641. DOI: 10.7759/cureus.104086

Vasquez A, Alvez V, et al. "Machine Learning Lead Optimisation: a methodological appraisal". Trials 30(2014): 641-653. DOI: 10.1038/s41746-023-00852-5

Wojcik H, Hendriksen W, et al. "Machine Learning Lead Optimisation: a multicentre analysis". Drug Discov Today 32(2017): 652-665. DOI: 10.1016/j.mayocpiqo.2022.09.004

Zielinski O, Ostrowski Z, et al. "Machine Learning Lead Optimisation: a prospective cohort study". Clin Cancer Res 34(2020): 663-677. DOI: 10.1177/08404704241291226

Andric V, Vasquez A, et al. "Machine Learning Lead Optimisation: a randomised evaluation". BMJ Open 36(2023): 674-680. DOI: 10.1093/medlaw/fwad013

Downloads

Published

2023-05-11

How to Cite

Machine Learning Lead Optimisation: A Short Communication on Prospective Versus Retrospective Performance. (2023). Translational Medicine & Therapeutics Journal, 2(1), 46-54. https://doi.org/10.5281/zenodo.22649052

Similar Articles

51-60 of 64

You may also start an advanced similarity search for this article.