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HMS Case Study

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AI in Clinical Trials: Machine Learning for the Analysis of Clinical Data

HMS helped Novartis use machine learning to analyze treatment outcomes and tolerability in patients with advanced breast cancer.

Management Summary

The AI4ANNA study shows which clinical endpoints yield predictive signals with machine learning and where the evaluated models reach their limits. Cross-study validation revealed moderate signals for the objective response rate and progression-free survival, while predictive performance was limited for most safety outcomes. The study therefore provides a solid basis for assessing suitable applications of machine learning and for focusing further analyses on appropriate research questions.

Project summary

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Novartis

Novartis

 

Industry: Pharmaceuticals
Focus: AI & Machine Learning
Use Case: Predictive Machine Learning

Project Objective

Evaluate the predictive potential of clinical trial data

An Overview of the Key Project Aspects

  • Analysis of data from the RIBECCA and RIBANNA studies
  • Comparison of LASSO and XGBoost for clinical predictive models
  • Internal and cross-study validation of the models

Tech Stack

machine learning statistical analysis

The cross-study validation shows for which clinical endpoints machine learning provides predictive signals and where the models reach their limits.

Dorothee Childs

Senior Data Scientist

The Starting Point

Pharmaceutical companies need reliable methods for analyzing clinical trial data. Until now, traditional statistical methods have been the primary tools used for this purpose. While these methods yield important and easily interpretable results, they are sometimes based on linear assumptions.

Machine learning can complement established methods by identifying additional patterns in clinical data. Time-to-event data poses a particular challenge. In such cases, the analysis must account for the fact that events—such as tumor progression—may not occur in all patients during the observation period or may not be fully captured.

The HMS Solution

Machine Learning for Clinical Data

HMS helped Novartis explore the potential of AI in clinical trials.

Investigating Predictive Potential

The AI4ANNA study examined treatment outcomes and tolerability in patients with HR-positive, HER2-negative advanced breast cancer. The study was based on anonymized data from the German RIBECCA and RIBANNA studies.

Comparing and Validating Models

LASSO and XGBoost were used to predict tumor control and safety outcomes. The models were validated within each study through repeated cross-validation and across studies.

Identifying Relevant Baseline Factors

Permutation Feature Importance was used to investigate which clinical baseline features were particularly important for the model predictions. This helped make the model results more interpretable.

The study was presented at the 2022 San Antonio Breast Cancer Symposium. The abstract was published in “Cancer Research”in March 2023 and is documented in the official AACR abstract.

New Findings from Clinical Trial Data

The study showed for which endpoints machine learning could provide additional predictive signals and where the models examined reached their limits.

Customer Benefits

New Insights from Clinical Trial Data

The study showed for which endpoints machine learning could provide additional predictive signals and where the models under investigation reached their limits.

Differentiated by endpoint

Predictive potential assessed by endpoint

Moderate signals were identified for the objective response rate and progression-free survival. For most of the safety outcomes examined, these signals were insufficient.

More interpretable

Relevant baseline factors identified

The analysis revealed which clinical baseline characteristics were particularly relevant to the model's performance.

Validated across studies

Model performance assessed across two datasets

Cross-study validation using RIBECCA and RIBANNA made it possible to assess model performance across two different datasets.

Our Strengths, Your Benefits

Combining Statistical Methods and Machine Learning

HMS helped Novartis explore the potential of machine learning for clinical trial data. In this project, we combined statistical analysis with ML-based predictive models, cross-study validation, and methods for assessing relevant baseline factors.

What You Can Expect from HMS

When analyzing clinical data, model selection, validation, and interpretability must work in tandem. To achieve this, HMS combines established statistical methods with machine learning and expert interpretation.

  • Selection and comparison of suitable models for clinical questions
  • Internal and cross-study validation of predictive performance
  • Transparent analysis of relevant clinical baseline characteristics
Nicolai Henrichs
Nicolai Henrichs
Principal Sales & Solutions Consultant | Partner

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Next Steps and Further Study

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Clinical Data Management and Analytics

HMS provides support for the preparation and analysis of clinical data as a basis for statistical evaluations and advanced machine learning applications.

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