Many existing models for clinical trial outcome prediction are optimized using task-specific loss functions on trial phase-specific data. While this scheme may boost prediction for common diseases and drugs, it can hinder learning of generalizable representations, leading to more false positives/negatives. To address this limitation, we introduce CLaDMoP, a new pre-training approach for clinical trial outcome prediction, alongside the Successful Clinical Trials dataset(SCT), specifically designed for this task. CLaDMoP leverages a Large Language Model-to encode trials' eligibility criteria-linked to a lightweight Drug-Molecule branch through a novel multi-level fusion technique. To efficiently fuse long embeddings across levels, we incorporate a grouping block, drastically reducing computational overhead. CLaDMoP avoids reliance on task-specific objectives by pre-training on a "pair matching" proxy task. Compared to established zero-shot and few-shot baselines, our method significantly improves both PR-AUC and ROC-AUC, especially for phase I and phase II trials. We further evaluate and perform ablation on CLaDMoP after Parameter-Efficient Fine-Tuning, comparing it to state-of-the-art supervised baselines, including MEXA-CTP, on the Trial Outcome Prediction(TOP) benchmark. CLaDMoP achieves up to 10.5% improvement in PR-AUC and 3.6% in ROC-AUC, while attaining comparable F1 score to MEXA-CTP, highlighting its potential for clinical trial outcome prediction. Code and SCT dataset can be downloaded from https://github.com/murai-lab/CLaDMoP.

Clinical trials are the gold standard for assessing the effectiveness and safety of drugs for treating diseases. Given the vast design space of drug molecules, elevated financial cost, and multi-year timeline of these trials, research on clinical trial outcome prediction has gained immense traction. Accurate predictions must leverage data of diverse modes such as drug molecules, target diseases, and eligibility criteria to infer successes and failures. Previous Deep Learning approaches for this task, such as HINT, often require wet lab data from synthesized molecules and/or rely on prior knowledge to encode interactions as part of the model architecture. To address these limitations, we propose a light-weight attention-based model, MEXA-CTP, to integrate readily-available multi-modal data and generate effective representations via specialized modules dubbed "mode experts", while avoiding human biases in model design. We optimize MEXA-CTP with the Cauchy loss to capture relevant interactions across modes. Our experiments on the Trial Outcome Prediction (TOP) benchmark demonstrate that MEXA-CTP improves upon existing approaches by, respectively, up to 11.3% in F1 score, 12.2% in PR-AUC, and 2.5% in ROC-AUC, compared to HINT. Ablation studies are provided to quantify the effectiveness of each component in our proposed method.

Clinical trials are pivotal for developing new medical treatments, yet they typically pose some risks such as patient mortality, adverse events, and enrollment failure that waste immense efforts spanning over a decade. Applying artificial intelligence (AI) to forecast or simulate key events in clinical trials holds great potential for providing insights to guide trial designs. However, complex data collection and question definition requiring medical expertise and a deep understanding of trial designs have hindered the involvement of AI thus far. This paper tackles these challenges by presenting a comprehensive suite of meticulously curated AIready datasets covering multi-modal data (e.g., drug molecule, disease code, text, categorical/numerical features) and 8 crucial prediction challenges in clinical trial design, encompassing prediction of trial duration, patient dropout rate, serious adverse event, mortality rate, trial approval outcome, trial failure reason, drug dose finding, design of eligibility criteria. Furthermore, we provide basic validation methods for each task to ensure the datasets' usability and reliability. We anticipate that the availability of such open-access datasets will catalyze the development of advanced AI approaches for clinical trial design, ultimately advancing clinical trial research and accelerating medical solution development.

The goal of drug discovery and development is to bring new medicines to patients suffering from critical illnesses. Earlier, drug discovery was a tedious process. Bringing a drug to market still takes 10 to 15 years. As a result, there is a lot of interest in finding new approaches to developing drugs using novel technological approaches. Machine learning tools and techniques are proving their importance at every stage of drug discovery and reducing the risk, and lowering the cost and expenditure used in clinical trials. It proves crucial in QSAR analysis, de novo drug design, hit discoveries, target validation, prognostic biomarkers, digital pathology, etc. The discovery of a drug has a lengthy procedure to go through before reaching the market.

Recent infectious disease outbreaks, such as the COVID-19 pandemic and the Zika epidemic in Brazil, have demonstrated both the importance and difficulty of accurately forecasting novel infectious diseases. When new diseases first emerge, we have little knowledge of the transmission process, the level and duration of immunity to reinfection, or other parameters required to build realistic epidemiological models. Time series forecasts and machine learning, while less reliant on assumptions about the disease, require large amounts of data that are also not available in early stages of an outbreak. In this study, we examine how knowledge of related diseases can help make predictions of new diseases in data-scarce environments using transfer learning. We implement both an empirical and a theoretical approach. Using empirical data from Brazil, we compare how well different machine learning models transfer knowledge between two different disease pairs: (i) dengue and Zika, and (ii) influenza and COVID-19. In the theoretical analysis, we generate data using different transmission and recovery rates with an SIR compartmental model, and then compare the effectiveness of different transfer learning methods. We find that transfer learning offers the potential to improve predictions, even beyond a model based on data from the target disease, though the appropriate source disease must be chosen carefully. While imperfect, these models offer an additional input for decision makers during pandemic response.

Human doctors with well-structured medical knowledge can diagnose a disease merely via a few conversations with patients about symptoms. In contrast, existing knowledge-grounded dialogue systems often require a large number of dialogue instances to learn as they fail to capture the correlations between different diseases and neglect the diagnostic experience shared among them. To address this issue, we propose a more natural and practical paradigm, i.e., low-resource medical dialogue generation, which can transfer the diagnostic experience from source diseases to target ones with a handful of data for adaptation. It is capitalized on a commonsense knowledge graph to characterize the prior disease-symptom relations. Besides, we develop a Graph-Evolving Meta-Learning (GEML) framework that learns to evolve the commonsense graph for reasoning disease-symptom correlations in a new disease, which effectively alleviates the needs of a large number of dialogues. More importantly, by dynamically evolving disease-symptom graphs, GEML also well addresses the real-world challenges that the disease-symptom correlations of each disease may vary or evolve along with more diagnostic cases. Extensive experiment results on the CMDD dataset and our newly-collected Chunyu dataset testify the superiority of our approach over state-of-the-art approaches. Besides, our GEML can generate an enriched dialogue-sensitive knowledge graph in an online manner, which could benefit other tasks grounded on knowledge graph.

Search engine logs have a great potential in tracking and predicting outbreaks of infectious disease. More precisely, one can use the search volume of some search terms to predict the infection rate of an infectious disease in nearly real-time. However, conducting accurate and stable prediction of outbreaks using search engine logs is a challenging task due to the following two-way instability characteristics of the search logs. First, the search volume of a search term may change irregularly in the short-term, for example, due to environmental factors such as the amount of media or news. Second, the search volume may also change in the long-term due to the demographic change of the search engine. That is to say, if a model is trained with such search logs with ignoring such characteristic, the resulting prediction would contain serious mispredictions when these changes occur. In this work, we proposed a novel feature selection method to overcome this instability problem. In particular, we employ a seasonal-adjustment method that decomposes each time series into three components: seasonal, trend and irregular component and build prediction models for each component individually. We also carefully design a feature selection method to select proper search terms to predict each component. We conducted comprehensive experiments on ten different kinds of infectious diseases. The experimental results show that the proposed method outperforms all comparative methods in prediction accuracy for seven of ten diseases, in both now-casting and forecasting setting. Also, the proposed method is more successful in selecting search terms that are semantically related to target diseases.

The graph structure of biomedical data differs from those in typical knowledge graph benchmark tasks. A particular property of biomedical data is the presence of long-range dependencies, which can be captured by patterns described as logical rules. We propose a novel method that combines these rules with a neural multi-hop reasoning approach that uses reinforcement learning. We conduct an empirical study based on the real-world task of drug repurposing by formulating this task as a link prediction problem. We apply our method to the biomedical knowledge graph Hetionet and show that our approach outperforms several baseline methods.