相关论文: What Were They Thinking? Pharmacologic priors impl…

Triple difference designs have become increasingly popular in empirical economics. The advantage of a triple difference design is that, within a treatment group, it allows for another subgroup of the population -- potentially less impacted…

Transitioning from Phase 2 to Phase 3 in drug development, at a rate of $\approx$40%, is the most stringent among phase transitions (Hay et al. (2014)). Yet, success rate at Phase 3 leading to approval is only $\approx$50% (Arrowsmith…

In phase I dose escalation studies for dual-agent combinations, at least one drug often has an established monotherapy dose. Consequently, substantial prior clinical safety data often exist for one or more monotherapies, allowing the study…

Drug discovery is lengthy and expensive, with traditional computer-aided design facing limits. This paper examines integrating quantum computing across the drug development cycle to accelerate and enhance workflows and rigorous…

Most clinical AI systems operate as prediction engines -- producing labels or risk scores -- yet real clinical reasoning is a time-bounded, sequential control problem under uncertainty. Clinicians interleave information gathering with…

The primary objective of Phase I oncology trials is to assess the safety and tolerability of novel therapeutics. Conventional dose escalation methods identify the maximum tolerated dose (MTD) based on dose-limiting toxicity (DLT). However,…

The case-crossover design (Maclure, 1991) is widely used in epidemiology and other fields to study causal effects of transient treatments on acute outcomes. However, its validity and causal interpretation have only been justified under…

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

Novel dose-finding designs, using estimation to assign the best estimated maximum- tolerated-dose (MTD) at each point in the experiment, most commonly via Bayesian techniques, have recently entered large-scale implementation in Phase I…

The keyboard design is a novel phase I dose-finding method that is simple and has good operating characteristics. This paper studies theoretical properties of the keyboard design, including the optimality of its decision rules, coherence in…

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

We consider design issues for toxicology studies when we have a continuous response and the true mean response is only known to be a member of a class of nested models. This class of non-linear models was proposed by toxicologists who were…

Nowadays, more and more clinical trials choose combinational agents as the intervention to achieve better therapeutic responses. However, dose-finding for combinational agents is much more complicated than single agent as the full order of…

The study of experimental design offers tremendous benefits for answering causal questions across a wide range of applications, including agricultural experiments, clinical trials, industrial experiments, social experiments, and digital…

The U.S. Food and Drug Administration (FDA) released a landmark draft guidance in January 2026 on the use of Bayesian methodology to support primary inference in clinical trials of drugs and biological products. For sponsors, the central…

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

Epidemiologists have a growing interest in employing computational approaches to solve analytic problems, with simulation being arguably the most accessible among all approaches. While previous literature discussed the utility of simulation…

Biased-coin designs are used in clinical trials to allocate treatments with some randomness while maintaining approximately equal allocation. More recent rules are compared with Efron's [Biometrika 58 (1971) 403-417] biased-coin rule and…

Most existing evaluations of explainable machine learning (ML) methods rely on simplifying assumptions or proxies that do not reflect real-world use cases; the handful of more robust evaluations on real-world settings have shortcomings in…