Development and validation of a machine learning-supported strategy of patient selection for osteoarthritis clinical trials: the IMI-APPROACH study.

To efficiently assess the disease-modifying potential of new osteoarthritis treatments, clinical trials need progression-enriched patient populations. To assess whether the application of machine learning results in patient selection enrichment, we developed a machine learning recruitment strategy targeting progressive patients and validated it in the IMI-APPROACH knee osteoarthritis prospective study. We designed a two-stage recruitment process supported by machine learning models trained to rank candidates by the likelihood of progression. First stage models used data from pre-existing cohorts to select patients for a screening visit. The second stage model used screening data to inform the final inclusion. The effectiveness of this process was evaluated using the actual 24-month progression. From 3500 candidate patients, 433 with knee osteoarthritis were screened, 297 were enrolled, and 247 completed the 2-year follow-up visit. We observed progression related to pain ( The machine learning-supported recruitment resulted in enriched selection of progressive patients. Further research is needed to improve structural progression prediction and assess this strategy in an interventional trial.

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© 2023 The Authors.

Déclaration de conflit d'intérêts

A. Bay-Jensen is a full-time employee and shareholder of Nordic Bioscience. C. Ladel was an employee of Merck KGaA at the project start. A. Lalande is employed by Institut de Recherches Internationales Servier. J. Larkin is employed by, and shareholder in GlaxoSmithKline. I.K. Haugen consults for Novartis and has received funding from Pfizer. M. Kloppenburg receives consulting fees from Abbvie, Pfizer, Levicept, GlaxoSmithKline, Merck-Serono, Kiniksa, Flexion, Galapagos, Jansen, CHDR, Novartis, and UCB. F. Berenbaum reports personal fees from AstraZeneca, Boehringer, Bone Therapeutics, CellProthera, Expanscience, Galapagos, Gilead, Grunenthal, GSK, Eli Lilly, Merck Sereno, MSD, Nordic, Nordic Bioscience, Novartis, Pfizer, Roche, Sandoz, Sanofi, Servier, UCB, Peptinov, 4 ​P Pharma, 4 Moving Biotech and grants from TRB Chemedica, outside the submitted work. F.J. Blanco has received consulting fees or other remuneration from AbbVie, Pfizer, UCB, Bristol-Myers Squibb, Roche, Servier, Bioiberica, Sanofi, Grünenthal, GlaxoSmithKline, Lilly, Janssen, Regeneron, Amgen, and TRB Chemedica, outside the submitted work. A. Mobasheri receives fees/funding from Merck KGaA, Kolon TissueGene, Pfizer, Galapagos-Servier, Image Analysis Group (IAG), Artialis, Aché Laboratórios Farmacêuticos, AbbVie, Guidepoint Global, Alphasights, Science Branding Communications, GSK, Flexion Therapeutics, Pacira Biosciences, Sterifarma, Bioiberica, SANOFI, Genacol, Kolon Life Science, BRASIT/BRASOS, GEOS, MCI Group, Alcimed, Abbot, Laboratoires Expansciences, SPRIM Communications, Frontiers Media, and University Health Network (UHN) Toronto. No other disclosures were reported.