An analytics approach to designing clinical trials for cancer

Abstract

Since chemotherapy began as a treatment for cancer in the 1940s, cancer drug development has become a multi-billion dollar industry. Combination chemotherapy remains the leading treatment for advanced cancers, and cancer drug research and clinical trials are enormous expenses for pharmaceutical companies and the government. We propose an analytics approach for the analysis and design of clinical trials that can discover drug combinations with significant improvements in survival and toxicity. We first build a comprehensive database of clinical trials. We then use this database to develop statistical models from earlier trials that are capable of predicting the survival and toxicity of new combinations of drugs. Then, using these statistical models, we develop optimization models that select novel treatment regimens that could be tested in clinical trials, based on the totality of data available on existing combinations. We present evidence for advanced gastric and gastroesophageal cancers that the proposed analytics approach a) leads to accurate predictions of survival and toxicity outcomes of clinical trials as long as the drugs used have been seen before in different combinations, b) suggests novel treatment regimens that balance survival and toxicity and take into account the uncertainty in our predictions, and c) outperforms the trials run by the average oncologist to give survival improvements of several months. Ultimately, our analytics approach offers promise for improving life expectancy and quality of life for cancer patients at low cost.