期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (6)
In the statistical literature, treatment effects in clinical trials are frequently described as either ITT or per-protocol effects. The estimand given......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (3)
Data on the Likert scale are ubiquitous in medical research, including randomized trials. Statistical analysis of such data may be conducted using the......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (4)
Historically early phase oncology drug development programmes have been based on the belief that more is better. Furthermore, rule-based study designs......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (6)
The role and value of statistical contributions in drug development up to the point of health authority approval are well understood. But health autho......
期刊： PHARMACEUTICAL STATISTICS, 2023; ()
Matching reduces confounding bias in comparing the outcomes of nonrandomized patient populations by removing systematic differences between them. Unde......
期刊： PHARMACEUTICAL STATISTICS, 2023; ()
Cross-over designs are commonly used in randomized clinical trials to estimate efficacy of a new treatment. They have received a lot of attention, par......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (3)
This short communication supports that rule-based study designs such as the '3 + 3' study design are still being used in early phase oncology developm......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (6)
Pre-specification of the primary analysis model is a pre-requisite to control the family-wise type-I-error rate (T1E) at the intended level in confirm......
期刊： PHARMACEUTICAL STATISTICS, 2023; ()
Non-inferiority trials compare new experimental therapies to standard ones (active control). In these experiments, historical information on the contr......
期刊： PHARMACEUTICAL STATISTICS, 2023; ()
We compare the performance of nonparametric estimators for the mean number of recurrent events and provide a systematic overview for different recurre......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (3)
Basket trials evaluate a single drug targeting a single genetic variant in multiple cancer cohorts. Empirical findings suggest that treatment efficacy......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (1)
Matching and stratification based on confounding factors or propensity scores (PS) are powerful approaches for reducing confounding bias in indirect t......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (2)
Dose-finding designs for phase-I trials aim to determine the recommended phase-II dose (RP2D) for further phase-II drug development. If the trial incl......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (5)
Large pharmaceutical companies maintain a portfolio of assets, some of which are projects under development while others are on the market and generat......
期刊： PHARMACEUTICAL STATISTICS, 2023; 22 (5)
In the context of vaccine efficacy trial where the incidence rate is very low and a very large sample size is usually expected, incorporating historic......
