ReviewOrphanet journal of rare diseases2020
A roadmap to using historical controls in clinical trials - by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG).
Review in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06163924 (To Investigate the Effect of Multidisciplinary Care Approach on Cardiovascular Risk Modification in Prostate Cancer Patients Receiving Androgen Deprivation Therapy), which is not on this map. Cited by 70 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.
To Investigate the Effect of Multidisciplinary Care Approach on Cardiovascular Risk Modification in Prostate Cancer Patients Receiving Androgen Deprivation Therapy
Who cites it
70 citing papers in PubMed.
- Plitidepsin in combination with dexamethasone (ADMYRE trial) versus an external control arm of pomalidomide plus dexamethasone in patients with relapsed/refractory multiple myeloma.Annals of hematology · 2026Trial
- Wearable Device-Based Intervention for Promoting Patient Physical Activity After Lung Cancer Surgery: A Nonrandomized Clinical Trial.JAMA network open · 2024Trial
- Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls.Annals of clinical and translational neurology · 2023Trial
- Comparing g-computation, propensity score-based weighting, and targeted maximum likelihood estimation for analyzing externally controlled trials with both measured and unmeasured confounders: a simulation study.BMC medical research methodology · 2023Trial
- Multi-Frequency RF Combined with Intense Pulsed Light Improves Signs and Symptoms of Dry Eye Disease Due to Meibomian Gland Dysfunction.Clinical ophthalmology (Auckland, N.Z.) · 2023Trial
- Toward precision neuromodulation in ADHD: rTMS-evoked excitability as a biomarker and ccPAS as an alternative protocol.NeuroImage. Clinical · 2026Article
- External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.Molecular therapy. Advances · 2026Article
- AI in Drug Discovery: Clinical Failures, Regulatory Reality, and the Validation Crisis Behind the Hype.Pharmaceuticals (Basel, Switzerland) · 2026Review
- The design of a small Bayesian hybrid-controlled trial utilizing time-to-event data from six published sources that used best alternative care to treat liver-predominant metastasis from primary ocular melanoma.Contemporary clinical trials communications · 2026Article
- Borrowing using historical-bias power prior with empirical Bayes.Journal of biopharmaceutical statistics · 2026Article
- Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial.Clinical and translational science · 2026Review
- Artificial intelligence-generated synthetic data for cancer research and clinical trials.Nature reviews. Cancer · 2026Review
- Dynamic Borrowing From Historical Controls via the Synthetic Prior With Covariates in Randomized Clinical Trials.Statistics in medicine · 2026Article
- The Placebo Effect in Rare Disease Clinical Trials: Measurement, Impact, and Statistical Approaches for Patient-as-Own-Control Designs.Clinical and translational science · 2026Review
- Evaluating the Feasibility of Using Historical Placebo Control in Osteoarthritis Trials.Epidemiologia (Basel, Switzerland) · 2026Article
- Tailored interventions to prevent functional decline after a Sentinel Fall presenting to the Emergency Department (iSeFallED) - protocol for a pragmatic mixed-methods implementation study.BMC geriatrics · 2026Article
- A call for principle-based acceptance of synthetic patients: establishing the AI-driven foundation for regulatory submissions.Frontiers in digital health · 2026Review
- Optimising clinical trial methods for complex regional pain syndrome: a methodological framework (OptiMeth-CRPS).Pain reports · 2025Article
- Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).Therapeutic innovation & regulatory science · 2025Review
- Hope is Not a Strategy: Using Robust Real-World Evidence to Make Better Clinical Development Decisions.Therapeutic innovation & regulatory science · 2025Article
10 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Historical controls (HCs) can be used for model parameter estimation at the study design phase, adaptation within a study, or supplementation or replacement of a control arm. Currently on the latter, there is no practical roadmap from design to analysis of a clinical trial to address selection and inclusion of HCs, while maintaining scientific validity. This paper provides a comprehensive roadmap for planning, conducting, analyzing and reporting of studies using HCs, mainly when a randomized clinical trial is not possible. We review recent applications of HC in clinical trials, in which either predominantly a large treatment effect overcame concerns about bias, or the trial targeted a life-threatening disease with no treatment options. In contrast, we address how the evidentiary standard of a trial can be strengthened with optimized study designs and analysis strategies, emphasizing rare and pediatric indications. We highlight the importance of simulation and sensitivity analyses for estimating the range of uncertainties in the estimation of treatment effect when traditional randomization is not possible. Overall, the paper provides a roadmap for using HCs.
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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.