CTO Drug Approval Prediction Baseline

A pre-trial LightGBM classifier trained on the CTO dataset to predict clinical trial success outcomes from structured trial metadata alone.

What's honest about this model

  • Uses ONLY features known at trial registration time (phase, enrollment, sponsor type, DMC presence, title keywords)
  • Temporal split: trained on trials registered before 2020, tested on 2020+
  • No leakage from completion status or post-trial results
  • No LLM reasoning β€” pure structured features + gradient boosting

Performance (temporal holdout)

Metric Value
ROC-AUC 0.869
PR-AUC 0.640
Brier 0.141

Test set: 4,359 trials registered 2020+, 24.6% success rate.

Feature importance (top 10)

  1. title_length β€” proxy for trial complexity
  2. enrollment_log β€” trial size
  3. study_year β€” temporal drift in success rates
  4. num_arms β€” design complexity
  5. phase_num β€” later phases have higher success rates
  6. is_fda_regulated β€” regulated trials differ
  7. has_dmc β€” data monitoring committee presence
  8. is_industry β€” industry vs academic sponsor
  9. cond_cancer β€” oncology trials
  10. cond_alzheimer β€” neurodegenerative indication

Caveats

  • Labels are weakly supervised (derived from trial linkage + status, not direct FDA approval tracking)
  • Title-based condition classification is crude
  • No molecular features, no competitive landscape, no regulatory designations
  • This is a research baseline, not investment advice

Citation

Gao et al. "Automatically Labeling Clinical Trial Outcomes: A Large-Scale Benchmark" (arXiv:2406.10292)

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Paper for mottledMantis/cto-drug-approval-baseline