ArticleBiometrical journal. Biometrische Zeitschrift2024
Phases of methodological research in biostatistics-Building the evidence base for new methods.
Article in Biometrical journal. Biometrische Zeitschrift, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
37 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A systematic review of simulation studies which compare existing statistical methods to account for non-compliance in randomised controlled trials.BMC medical research methodology · 2023Pooled it
- Variable Selection via Fused Sparse-Group Lasso Penalized Multi-state Models Incorporating Molecular Data.Biometrical journal. Biometrische Zeitschrift · 2025Trial
- A Framework for the Estimation of Quality-Adjusted Life-Years Using Joint Models of Longitudinal and Survival Data.Medical decision making : an international journal of the Society for Medical Decision Making · 2026Article
- Comparison of Methods for Incorporating Related Data When Developing Clinical Prediction Models: A Simulation Study.Biometrical journal. Biometrische Zeitschrift · 2026Article
- A Neutral Comparison of Multivariate Hypothesis Tests for Parallel Group Designs in Rare Disease Settings.Statistics in medicine · 2026Article
- Comparison of Different Methods for the Meta-Analysis of Diagnostic Test Accuracy Studies-A Simulation Study.Biometrical journal. Biometrische Zeitschrift · 2026Article
- Methods for Adjusting for Covariate Measurement Error in Flexible Modeling of Functional Form: Designing a Blinded, Controlled Neutral Comparison Simulation Study.Biometrical journal. Biometrische Zeitschrift · 2026Article
- Novel Distance Regression for Repeated Outcomes With Missing Data: Applications to Longitudinal and Crossover Studies of Microbiome Beta-Diversity.Statistics in medicine · 2026Article
- Tree-based exploratory identification of predictive biomarkers in non-randomized data.BMC medical research methodology · 2026Article
- Design and Analysis of Randomized Clinical Trials With Average Hazard: Practical Guidance and Tools for Implementation.Statistics in medicine · 2026Article
- Agreement between heuristic shrinkage factor and optimal shrinkage factors in logistic regression for risk prediction: a simulation study across different sample sizes and settings.Diagnostic and prognostic research · 2026Article
- Modelling longitudinal and time-to-event data: a phase IV simulation study comparing R package implementations of joint models with time-varying Cox proportional-hazards regression, and the two-stage approach.BMC medical research methodology · 2026Article
- Clustered flexible calibration plots for binary outcomes using random effects modeling.Research synthesis methods · 2026Article
- Defining Utility as a Measure of Preference Under Uncertainty in Phase I-II Oncology Dose Finding Trials.Statistics in medicine · 2026Article
- A New Parametric Accelerated Failure Time Model for Semi-Competing Risks Data.Statistics in medicine · 2026Article
- The statistical software revolution in pharmaceutical development: challenges and opportunities in open source.Drug discovery today · 2026Review
- Multiple Contrast Tests for Count Data: Small Sample Approximations and Their Limitations.Biometrical journal. Biometrische Zeitschrift · 2025Article
- On "Confirmatory" Methodological Research in Statistics and Related Fields.Statistics in medicine · 2025Article
- A Practical Framework to Design Immunization Studies Based on the Beta Distribution.Statistics in medicine · 2025Article
- Rethinking the Handling of Method Failure in Comparison Studies.Statistics in medicine · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
Although new biostatistical methods are published at a very high rate, many of these developments are not trustworthy enough to be adopted by the scientific community. We propose a framework to think about how a piece of methodological work contributes to the evidence base for a method. Similar to the well-known phases of clinical research in drug development, we propose to define four phases of methodological research. These four phases cover (I) proposing a new methodological idea while providing, for example, logical reasoning or proofs, (II) providing empirical evidence, first in a narrow target setting, then (III) in an extended range of settings and for various outcomes, accompanied by appropriate application examples, and (IV) investigations that establish a method as sufficiently well-understood to know when it is preferred over others and when it is not; that is, its pitfalls. We suggest basic definitions of the four phases to provoke thought and discussion rather than devising an unambiguous classification of studies into phases. Too many methodological developments finish before phase III/IV, but we give two examples with references. Our concept rebalances the emphasis to studies in phases III and IV, that is, carefully planned method comparison studies and studies that explore the empirical properties of existing methods in a wider range of problems.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.