ReviewClinical pharmacology and therapeutics2020
Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation.
Review in Clinical pharmacology and therapeutics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 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
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 68 citations in OpenAlex.
- Extended dual antiplatelet therapy with ticagrelor 60 mg in patients with prior myocardial infarction: The design of ALETHEIA, a multi-country observational study.Clinical cardiology · 2021Pooled it
- Investigational Use of Real-World Data as a Hybrid Control in Pancreatic Ductal Adenocarcinoma From the Randomized Phase Ib/II MORPHEUS Trial.Clinical pharmacology and therapeutics · 2025Trial
- Bridging evidence worlds: policy and practice pathways for integrating clinical trials and real-world data in drug development and HTA.Frontiers in pharmacology · 2026Article
- Challenges and Criteria for Single-Arm Trials Leading to an Added Benefit in German Health Technology Assessments.PharmacoEconomics · 2025Article
- Emulating randomized trials by observational database studies: the RCT-DUPLICATE initiative in COPD and asthma.American journal of epidemiology · 2025Article
- AI in optimized cancer treatment: laying the groundwork for interdisciplinary progress.Oxford open immunology · 2025Article
- Predicting Individual Treatment Effects: Challenges and Opportunities for Machine Learning and Artificial Intelligence.Kunstliche intelligenz · 2025Article
- Examining the Effect of Missing Data and Unmeasured Confounding on External Comparator Studies: Case Studies and Simulations.Drug safety · 2024Article
- Machine Learning Methods to Estimate Individualized Treatment Effects for Use in Health Technology Assessment.Medical decision making : an international journal of the Society for Medical Decision Making · 2024Review
- Interpretable artificial intelligence to optimise use of imatinib after resection in patients with localised gastrointestinal stromal tumours: an observational cohort study.The Lancet. Oncology · 2024Observational
- Challenges to Using Big Data in Cancer.Cancer research · 2023Review
- Real-world data: a brief review of the methods, applications, challenges and opportunities.BMC medical research methodology · 2022Review
- The Randomised Controlled Trial at the Intersection of Research Ethics and Innovation.Pharmaceutical medicine · 2022Article
- Rationale, Strengths, and Limitations of Real-World Evidence in Oncology: A Canadian Review and Perspective.The oncologist · 2022Article
- A Review of Causal Inference for External Comparator Arm Studies.Drug safety · 2022Review
- Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program.Molecular autism · 2022Article
- Analysis of a Real-World Progression Variable and Related Endpoints for Patients with Five Different Cancer Types.Advances in therapy · 2022Article
- Favorable safety profile of moderate hypofractionated over normofractionated radiotherapy in breast cancer patients: a multicentric prospective real-life data farming analysis.Radiation oncology (London, England) · 2022Article
- Commentary: Constipation caused by anti-calcitonin gene-related peptide migraine therapeutics explained by antagonism of calcitonin gene-related peptide's motor-stimulating and prosecretory function in the intestine.Frontiers in physiology · 2022Article
- Real World Data in Health Technology Assessment of Complex Health Technologies.Frontiers in pharmacology · 2022Article
Corrections and comments
- Commented on by
- Commented on by
Authors and funding
10 authors at 3 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Real-world data and patient-level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods acceptable for regulators and other decision makers will require their testing and validation in broadly the same way one would evaluate a new drug: prospectively, well-controlled, and according to a pre-agreed plan. From a European regulators' perspective, the established methods qualification advice procedure with active participation of patient groups and other decision makers is an efficient and transparent platform for the development and validation of novel study designs.
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.