Finding the most promising indications for novel treatments in oncology

Maren Eckhoff1, Stefan Klingelschmitt2, Lisbeth Van Ruijssevelt3

  • 1McKinsey & Company, London, UK.

NPJ Precision Oncology
|March 26, 2026
PubMed

Insights

This study introduces INSPIRE, a machine learning model that uses real-world patient data to predict effective cancer therapies. The approach successfully identified 70% of later drug approvals, potentially speeding up treatment selection.

Area of Science:

  • Oncology
  • Computational Biology
  • Machine Learning

Background:

  • Developing effective cancer therapies involves significant costs and complexities.
  • Identifying optimal treatment indications for specific drugs, like anti-PD-1, remains a challenge.
  • Real-world data holds potential for improving treatment selection.

Purpose of the Study:

  • To develop a machine learning model that predicts effective cancer therapy indications.
  • To leverage representation learning on real-world patient data for treatment discovery.
  • To accelerate the identification of drugs like anti-PD-1 for specific cancer types.

Main Methods:

  • Trained a machine learning model (INSPIRE) to rank cancer indications based on anti-PD-1 treatment relevance.
  • Utilized histology-based indications and patient medical journey data for feature generation.
  • Employed an embedding approach for biological relevance and reference indications.

Main Results:

  • The INSPIRE model prioritized 70% of subsequent anti-PD-1 inhibitor approvals when trained on pre-clinical data.
  • The approach demonstrated the ability to predict the biological relevance of treatments for specific indications.
  • Successfully identified potential effective treatments by analyzing patient medical journey events.

Conclusions:

  • INSPIRE can accelerate the selection and testing of effective cancer drugs.
  • This approach has the potential to reduce healthcare costs and improve patient care.
  • Representation learning on real-world data offers a promising avenue for precision oncology.

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