ArticleClinical pharmacology and therapeutics2014
A proposal for integrated efficacy-to-effectiveness (E2E) clinical trials.
Article in Clinical pharmacology and therapeutics, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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
16 citing papers in PubMed.
- Thrombolytic removal of intraventricular haemorrhage in treatment of severe stroke: results of the randomised, multicentre, multiregion, placebo-controlled CLEAR III trial.Lancet (London, England) · 2017Trial
- Managing Sjögren's Disease in Pediatric Patients.Paediatric drugs · 2026Review
- Real World Data in Health Technology Assessment of Complex Health Technologies.Frontiers in pharmacology · 2022Article
- Use of Real-World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design.Clinical pharmacology and therapeutics · 2022Review
- Real-World Evidence Should Be Used in Regulatory Decisions About New Pharmaceutical and Medical Device Products for Diabetes.Journal of diabetes science and technology · 2019Article
- Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice.Clinical pharmacology and therapeutics · 2019Article
- Recognizing that Evidence is Made, not Born.Clinical pharmacology and therapeutics · 2019Article
- A collaborative, academic approach to optimizing the national clinical research infrastructure: The first year of the Trial Innovation Network.Journal of clinical and translational science · 2018Article
- EFFICACY-TO-EFFECTIVENESS CLINICAL TRIALS.Transactions of the American Clinical and Climatological Association · 2018Article
- Maintenance efficacy designs in psychiatry: Randomized discontinuation trials - enriched but not better.Journal of clinical and translational science · 2017Review
- Adaptation through Collaboration: Developing Novel Platforms to Advance the Delivery of Advanced Therapies to Patients.Frontiers in medicine · 2017Review
- PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms.Clinical pharmacology and therapeutics · 2016Article
- Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need.Clinical pharmacology and therapeutics · 2016Article
- From adaptive licensing to adaptive pathways: delivering a flexible life-span approach to bring new drugs to patients.Clinical pharmacology and therapeutics · 2015Article
- 7(th) Annual Symposium on Self-Monitoring of Blood Glucose (SMBG), May 8-10, 2014, Helsinki, Finland.Diabetes technology & therapeutics · 2014Article
- Getting real in clinical trials.Nature reviews. Drug discovery · 2014Article
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
We propose an "efficacy-to-effectiveness" (E2E) clinical trial design, in which an effectiveness trial would commence seamlessly upon completion of the efficacy trial. Efficacy trials use inclusion/exclusion criteria to produce relatively homogeneous samples of participants with the target condition, conducted in settings that foster adherence to rigorous clinical protocols. Effectiveness trials use inclusion/exclusion criteria that generate heterogeneous samples that are more similar to the general patient spectrum, conducted in more varied settings, with protocols that approximate typical clinical care. In E2E trials, results from the efficacy trial component would be used to design the effectiveness trial component, to confirm and/or discern associations between clinical characteristics and treatment effects in typical care, and potentially to test new hypotheses. An E2E approach may improve the evidentiary basis for selecting treatments, expand understanding of the effectiveness of treatments in subgroups with particular clinical features, and foster incorporation of effectiveness information into regulatory processes.
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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.