ArticleClinical and translational science2019
Characteristics of Single Pivotal Trials Supporting Regulatory Approvals of Novel Non-orphan, Non-oncology Drugs in the European Union and United States from 2012-2016.
Article in Clinical and translational science, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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
10 citing papers in PubMed.
- Critical analysis of withdrawn drug applications in marketing authorization procedures of the European Medicines Agency (EMA) 2006-2024.Naunyn-Schmiedeberg's archives of pharmacology · 2026Article
- Epistemological Tensions in the EU HTA Joint Clinical Assessment: The Illusion of Judgment-Free Evaluation.Journal of market access & health policy · 2026Article
- Scoping review of the role of pharmacometrics in model-informed drug development.Journal of pharmacokinetics and pharmacodynamics · 2025Article
- Protocol Development for Investigator-Sponsored Clinical Studies.Clinical and translational science · 2025Article
- Real-world evidence to support regulatory submissions: A landscape review and assessment of use cases.Clinical and translational science · 2024Review
- Sodium Phenylbutyrate and Tauroursodeoxycholic Acid: A Story of Hope Turned to Disappointment in Amyotrophic Lateral Sclerosis Treatment.Clinical drug investigation · 2024Review
- Navigating the challenges of initiating pediatric device trials - a case study.Journal of clinical and translational science · 2024Article
- Regulators, Pivotal Clinical Trials, and Drug Regulation in the Age of COVID-19.International journal of health services : planning, administration, evaluation · 2021Article
- Aducanumab: evidence from clinical trial data and controversies.Drugs in context · 2021Article
- Local Vibrational Therapy for Essential Tremor Reduction: A Clinical Study.Medicina (Kaunas, Lithuania) · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
For regulatory approval of a new medicine, the gold standard for demonstration of efficacy has traditionally been a minimum of two positive, adequate, and well-controlled clinical trials. Nevertheless, drugs to treat cancer and rare diseases are usually approved based on a single and often uncontrolled pivotal trial. In contrast, little is known about single pivotal trial approvals for non-orphan, non-oncology drugs. Between 2012 and 2016, 23 novel therapeutic drugs were approved by the US Food and Drug Administration (FDA) and/or the European Medicines Agency (EMA) for 27 non-orphan, non-oncology indications each based on a single pivotal trial. Although there was considerable variation in the nature and strength of the efficacy evidence supporting these drug approvals, the majority (85%) of the pivotal trials were randomized and controlled. For all superiority trials, the primary outcome was met with a statistical significance of P ≤ 0.005. Most approvals were supported by additional efficacy data from nonpivotal studies.
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.