Evidence map›Paper›PMID 39196202›Full record

ReviewNature cardiovascular research2024

The role of early-phase trials and real-world evidence in drug development.

Harriette G C Van Spall, Arnaud Bastien, Bernard Gersh, Barry Greenberg, Reza Mohebi, James Min, Karsten Strauss, Steffen Thirstrup, Faiez Zannad

Abstract readReview
PubMed Publisher
In one paragraph

Review in Nature cardiovascular research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Review
  3. A Fast Path from Innovation to Safe and Effective Medicines.Clinical pharmacology and therapeutics · 2025
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Harriette G C Van SpallDepartment of Medicine, Department of Health Research Methods, Evidence, and Impact; Research Institute of St. Joseph's, McMaster University, Hamilton, Ontario, Canada.ORCID 0000-0002-8370-4569
Arnaud BastienBristol Myers Squibb, Princeton, NJ, USA.
Bernard GershDepartment of Cardiovascular Medicine, Mayo Clinic College of Medicine and Science, Rochester, MN, USA.ORCID 0000-0001-5605-900X
Barry GreenbergDivision of Cardiology, UC San Diego Health, San Diego, CA, USA.
Reza MohebiDivision of Cardiology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
James MinCleerly, Denver, CO, USA.
Karsten StraussOlink, Uppsala, Sweden.
Steffen ThirstrupEuropean Medicines Agency, Amsterdam, the Netherlands.ORCID 0000-0003-0903-682X
Faiez ZannadUniversité de Lorraine, Inserm Clinical Investigation Center at Institut Lorrain du Coeur et des Vaisseaux, University Hospital of Nancy, Nancy, France. f.zannad@chru-nancy.fr.ORCID 0000-0001-7456-1570

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Phase 3 randomized controlled trials (RCTs), while the gold standard for treatment efficacy and safety, are not always feasible, are expensive, can be prolonged and can be limited in generalizability. Other under-recognized sources of evidence can also help advance drug development. Basic science, proof-of-concept studies and early-phase RCTs can provide evidence regarding the potential for clinical benefit. Real-world evidence generated from registries or observational datasets can provide insights into the treatment of rare diseases that often pose a challenge for trial recruitment. Pragmatic trials embedded in healthcare systems can assess the treatment effects in clinical settings among patient populations sometimes excluded from trials. This Perspective discusses potential sources of evidence that may be used to complement explanatory phase 3 RCTs and to speed the development of new cardiovascular medications. Content is derived from the 19th Global Cardiovascular Clinical Trialists meeting (December 2022), involving clinical trialists, patients, clinicians, regulators, funders and industry representatives.

Indexed as

Drug DevelopmentCardiovascular AgentsCardiovascular DiseasesClinical Trials, Phase III as TopicEvidence-Based MedicineHumansObservational Studies as TopicPragmatic Clinical Trials as TopicRandomized Controlled Trials as TopicResearch DesignTreatment OutcomeCardiovascular Agents

Identifiers

PMID39196202

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.