In pharmaceutical R&D, predicting the efficacy of a pharmaceutical in treating a particular disease prior to clinical testing or any real-world use has been challenging. In this paper, we propose a flexible and modular machine learning-based approach for predicting the efficacy of an untested pharmaceutical for treating a disease. We train a machine learning model using sets of pharmaceutical-pathway weight impact scores and patient data, which can include patient characteristics and observed clinical outcomes. The resulting model then analyses weighted impact scores of an untested pharmaceutical across human biological molecule-protein pathways to generate a predicted efficacy value. We demonstrate how the method works on a real-world dataset with patient treatments and outcomes, with two different weight impact score algorithms We include methods for evaluating the generalisation performance on unseen treatments, and to characterise conditions under which the approach can be expected to be most predictive. We discuss specific ways in which our approach can be iterated on, making it an initial framework to support future work on predicting the effect of untested drugs, leveraging RWD clinical data and drug embeddings.

DeepMind, a Google-owned artificial intelligence (AI) company based in the United Kingdom, made scientific history when it announced last November that it had a solution to a 50-year-old grand challenge in biology--protein folding. This AI machine learning breakthrough may help accelerate the discovery of new medications and novel treatments for diseases. On July 15, 2021 DeepMind revealed details on how its AI works in a new peer-reviewed paper published in Nature, and made its revolutionary AlphaFold version 2.0 model available as open-source on GitHub. The three-dimensional (3D) shape and function of proteins are determined by the sequence of its amino acids. AlphaFold predicts three-dimensional (3D) models of protein structures.

As Ginny Rometti, the current CEO of IBM and also the first woman to head the company is quoted as saying: "Some people call this artificial intelligence, but the reality is this technology will enhance us. So instead of artificial intelligence, I think we'll augment our intelligence." It is true that AI can give us more efficient method but it is not likely to be able to tell us meaning. We need leadership in healthcare to address legitimate challenges and to give direction to our innovations. As a species, we hope technology will help us work smarter and not harder, but we also want it help us work more meaningfully.