A/B testing is critical for modern technological companies to evaluate the effectiveness of newly developed products against standard baselines. This paper studies optimal designs that aim to maximize the amount of information obtained from online experiments to estimate treatment effects accurately.

Treatment individualization and adaptation over time are crucial for managing chronic diseases.

A targeted learning (TL) framework is developed to estimate the difference in the restricted mean survival time (RMST) for a clinical trial with time-to-event outcomes. The approach starts by defining the target estimand as the RMST difference between investigational and control treatments. Next, an efficient estimation method is introduced: a targeted minimum loss estimator (TMLE) utilizing pseudo-observations. Moreover, a version of the copy reference (CR) approach is developed to perform a sensitivity analysis for right-censoring. The proposed TL framework is demonstrated using a real data application.

He Went to Prison for Gene-Editing Babies. Now He's Planning to Do It Again Chinese scientist He Jiankui wants to end Alzheimer's and thinks Silicon Valley is conducting a "Nazi eugenic experiment." In 2018, a nervous-looking He Jiankui took the stage at a scientific conference in Hong Kong. A hush settled over the packed auditorium as the soft-spoken Chinese scientist adjusted his microphone and confirmed the circulating media reports: He had created the world's first gene-edited babies . Three little girls were born with modifications to their genomes that were intended to protect them against HIV. The changes he'd made to their DNA were permanent and heritable, meaning they could be passed down to future generations.

Why personalized gene editing, genetic resurrections and embryo scoring made our list. Earlier this week, published its annual list of Ten Breakthrough Technologies. As always, it features technologies that made the news last year, and which--for better or worse--stand to make waves in the coming years. They're the technologies you should really be paying attention to. This year's list includes tech that's set to transform the energy industry, artificial intelligence, space travel --and of course biotech and health. Our breakthrough biotechnologies for 2026 involve editing a baby's genes and, separately, resurrecting genes from ancient species.

Our reporters and editors constantly debate which emerging technologies will define the future. Once a year, we take stock and share some educated guesses with our readers. Here are the advances that we think will drive progress or incite the most change--for better or worse--in the years ahead. Rubrik is the exclusive sponsor of the 10 Breakthrough Technologies 2026 and had no editorial influence on this list. Rubrik is a security and AI operations company that aims to secure and accelerate the world's AI transformation.

More accurate and individualized health predictions will allow for preventative factors to be implemented well in advance. In 2026, we will see the beginning of precision medical forecasting. Just as there have been remarkable advances in weather forecasting with the use of large language models, so will there be for determining an individual's risk of the major age-related diseases (cancer, cardiovascular, and neurodegenerative). These diseases share common threads, such as a long incubation phase before any symptoms are manifest, usually two decades or more. They also have the same biologic underpinnings of immunosenescence and inflammaging, terms that characterize an immune system that has lost some of its functionality and protective power, and the accompanying heightened inflammation.

Screening patients for clinical trial eligibility remains a manual, time - consuming, and resource-intensive process. W e present a secure, scalable proof-of - concept system for Artificial Intelligence ( AI)- augmented patient - trial matching that addresses key implementation challenges: integrating heterogeneous electronic health record (EHR) data, facilitating expert review, and maintaining rigorous security standards. Leveraging open-source, reasoning-enabled large language models (LLMs), the system moves beyond binary classification to generate structured eligibility assessments with interpretable reasoning chains that support human-in - the - loop review. This decision support tool represents eligibility as a dynamic state rather than a fixed determination, identifying matches whe n available and offering actionable recommendations that could render a patient eligible in the future . The system aims to reduce coordinator burden, intelligently broaden the set of trials considered for each patient and guarantee comprehensive auditability of all AI - generated outputs. Introduction Applications of artificial intelligence (AI) in healthcare are increasingly focused on improving administrative efficiency and optimizing clinical workflows . Identifying relevant trials and screening them for a particular patient is traditionally manual, time - consuming, and heavily reliant on clinical expertise.

We'll keep following these developments, but this just wasn't their year. If you're a longtime reader, you probably know that our newsroom selects 10 breakthroughs every year that we think will define the future . This group exercise is mostly fun and always engrossing, but at times it can also be quite difficult. We collectively pitch dozens of ideas, and the editors meticulously review and debate the merits of each. We agonize over which ones might make the broadest impact, whether one is too similar to something we've featured in the past, and how confident we are that a recent advance will actually translate into long-term success. There is plenty of lively discussion along the way.

This work presents the development and evaluation of an NLP-enabled probabilistic classifier designed to estimate the probability of technical and regulatory success (pTRS) for clinical trials in the field of neuroscience. While pharmaceutical R&D is plagued by high attrition rates and enormous costs, particularly within neuroscience, where success rates are below 10%, timely identification of promising programs can streamline resource allocation and reduce financial risk. Leveraging data from the ClinicalTrials.gov database and success labels from the recently developed Clinical Trial Outcome dataset, the classifier extracts text-based clinical trial features using statistical NLP techniques. These features were integrated into several non-LLM frameworks (logistic regression, gradient boosting, and random forest) to generate calibrated probability scores. Model performance was assessed on a retrospective dataset of 101,145 completed clinical trials spanning 1976-2024, achieving an overall ROC-AUC of 0.64. An LLM-based predictive model was then built using BioBERT, a domain-specific language representation encoder. The BioBERT-based model achieved an overall ROC-AUC of 0.74 and a Brier Score of 0.185, indicating its predictions had, on average, 40% less squared error than would be observed using industry benchmarks. The BioBERT-based model also made trial outcome predictions that were superior to benchmark values 70% of the time overall. By integrating NLP-driven insights into drug development decision-making, this work aims to enhance strategic planning and optimize investment allocation in neuroscience programs.