Why Almost All Ml Models For Medicine Are Wrong And What We Need For Evidence Based Medical Ai

Why almost all ML models for medicine are wrong-and what we need for evidence-based medical AI

Terms

Concerns

  • many of the models being published are not validated/verified well and do not perform as stated
    • This is based on many assumptions in the design process for these models

Issues with model training:

  • Unreliable ground truths
  • inappropriate decision thresholds
  • assessment metrics are inadequate
  • validation datasets do not represent real world use cases

Ground Truth Concerns

Specifically in medicine, diagnostic categories are not cut and dry categories and there is often blurring/overlap. However ML training assumptions are being made that the labels used are objective and accurate/correct, and then used for predictions. These assumptions on accuracy supremacy lead to noise in the dataset and impact the application of the model.

Threshold optimization

Most ML models optimize global metrics such as AUROC, assuming all decision thresholds are equally important. In medicine this assumption is incorrect:

  • different diseases have different acceptable false positive / false negative tradeoffs
  • decision thresholds depend on clinical context and risk
  • threshold selection therefore has ethical and clinical implications

Assessment metrics

Many commonly reported metrics are unstable or misleading. Problems:

  • accuracy is inflated by class imbalance
  • PPV changes with disease prevalence
  • F1, MCC, sensitivity and specificity depend on the chosen threshold
  • many papers report metrics using an arbitrary threshold of 0.5 Thus recommendations include:
  • report calibration
  • report clinical utility (net benefit)
  • evaluate robustness on heterogeneous and out-of-distribution (OOD) data
  • evaluate performance across clinically relevant thresholds

Calibration

A model should produce probabilities that match real-world outcomes. For example:

  • if the model predicts 80% probability for 100 patients, then roughly 80 should actually have the condition. Calibration should be evaluated around clinically important decision thresholds, not only globally.

Performance uncertainty

Many papers report only point estimates for metrics. The problem is(are):

  • evaluation datasets are often small
  • performance estimates have statistical uncertainty
  • small differences between models may not be meaningful To address these concerns, researchers can use report confidence/credible intervals and use statistical tests when comparing models

External validation

Many studies only evaluate on held-out splits from the same dataset. But this does not show whether the model generalizes

External validation should use:

  • independent hospitals
  • different populations
  • different collection conditions
  • ideally prospective datasets

Temporal validation

Clinical data changes over time:

  • disease prevalence
  • diagnostic criteria
  • laboratory equipment
  • clinical practice
  • patient populations Models should therefore include:
  • temporal validation
  • recalibration
  • post-deployment monitoring
  • MLOps fo continuous evaluation

Recommendations: Real-world data

The paper argues that real-world clinical data is inherently noisy. Rather than assuming perfect labels, ML methods should support:

  • noisy labels
  • soft/probabilistic labels
  • annotator disagreement
  • dataset shift
  • uncertainty estimation
  • OOD detection
  • robust learning