ArticleFrontiers in pharmacology2021
Emulated Clinical Trials from Longitudinal Real-World Data Efficiently Identify Candidates for Neurological Disease Modification: Examples from Parkinson's Disease.
Article in Frontiers in pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 21 citations in OpenAlex.
- Systematically exploring repurposing effects of antihypertensives.Pharmacoepidemiology and drug safety · 2022Pooled it
- When evidence meets artificial intelligence.Lancet regional health. Americas · 2026Review
- From past to future: Digital approaches to success of clinical drug trials for Parkinson's disease.Journal of Parkinson's disease · 2026Review
- Review
- From In Silico Hypothesis to Validation: The Role of Real-World Evidence in the Preliminary Verification of AI-Generated Drug-Repositioning Candidates: A Comprehensive Review.Journal of clinical medicine · 2026Review
- Design and Implementation of Observational Studies Emulating a Target Trial.JAMA network open · 2026Article
- Artificial Intelligence to Guide Repurposing of Drugs.Annual review of medicine · 2026Review
- Network-based prediction and real-world patient data observation identify doxycycline as a repurposable drug in Alzheimer's disease.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Article
- Association of Medication Use and 8-Year Mortality Risk in Patients With Parkinson Disease: Drug-Wide Trial Emulation.Neurology · 2025Observational
- Use of β-adrenoreceptor drugs and Parkinson's disease incidence in women from the French E3N cohort study.Journal of Parkinson's disease · 2025Article
- Valsartan: An Angiotensin Receptor Blocker Modulates BDNF Expression and Provides Neuroprotection Against Cerebral Ischemic Reperfusion Injury.Molecular neurobiology · 2024Article
- Foundational model aided automatic high-throughput drug screening using self-controlled cohort study.medRxiv : the preprint server for health sciences · 2024Article
- Artificial intelligence and open science in discovery of disease-modifying medicines for Alzheimer's disease.Cell reports. Medicine · 2024Review
- Disease modification in inflammatory skin disorders: opportunities and challenges.Nature reviews. Drug discovery · 2023Review
- GABA-A Alpha 2/3 but Not Alpha 1 Receptor Subunit Ligand Inhibits Harmaline and Pimozide-Induced Tremor in Rats.Biomolecules · 2023Article
- Identifying Protective Drugs for Parkinson's Disease in Health-Care Databases Using Machine Learning.Movement disorders : official journal of the Movement Disorder Society · 2022Article
- The use of real-world data in drug repurposing.Translational and clinical pharmacology · 2021Review
- Observational
- Framework for identifying drug repurposing candidates from observational healthcare data.JAMIA open · 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
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Real-world healthcare data hold the potential to identify therapeutic solutions for progressive diseases by efficiently pinpointing safe and efficacious repurposing drug candidates. This approach circumvents key early clinical development challenges, particularly relevant for neurological diseases, concordant with the vision of the 21st Century Cures Act. However, to-date, these data have been utilized mainly for confirmatory purposes rather than as drug discovery engines. Here, we demonstrate the usefulness of real-world data in identifying drug repurposing candidates for disease-modifying effects, specifically candidate marketed drugs that exhibit beneficial effects on Parkinson's disease (PD) progression. We performed an observational study in cohorts of ascertained PD patients extracted from two large medical databases, Explorys SuperMart (N = 88,867) and IBM MarketScan Research Databases (N = 106,395); and applied two conceptually different, well-established causal inference methods to estimate the effect of hundreds of drugs on delaying dementia onset as a proxy for slowing PD progression. Using this approach, we identified two drugs that manifested significant beneficial effects on PD progression in both datasets: rasagiline, narrowly indicated for PD motor symptoms; and zolpidem, a psycholeptic. Each confers its effects through distinct mechanisms, which we explored via a comparison of estimated effects within the drug classification ontology. We conclude that analysis of observational healthcare data, emulating otherwise costly, large, and lengthy clinical trials, can highlight promising repurposing candidates, to be validated in prospective registration trials, beneficial against common, late-onset progressive diseases for which disease-modifying therapeutic solutions are scarce.
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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.