Evidence map›Paper›PMID 27774032›Full record

ArticlePragmatic and observational research2015

Efficacy and effectiveness trials have different goals, use different tools, and generate different messages.

Franz Porzsolt, Natália Galito Rocha, Alessandra C Toledo-Arruda, Tania G Thomaz, Cristiane Moraes, Thais R Bessa-Guerra, Mauricio Leão, Arn Migowski, André R Araujo da Silva, Christel Weiss

Erratum issuedAbstract read
In one paragraph

Article in Pragmatic and observational research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing 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

26 citing papers in PubMed.

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  10. Review
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  12. [Covid-19: Twin Method for Verifying Real-World Effectiveness Under Everyday Conditions].Gesundheitswesen (Bundesverband der Arzte des Offentlichen Gesundheitsdienstes (Germany)) · 2023
    Article
  13. Article
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  15. Article
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  19. Canadian family physician Medecin de famille canadien · 2019
    Article
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Franz PorzsoltHealth Care Research, Department of General and Visceral Surgery, University Hospital Ulm, Ulm, Germany; Institute of Clinical Economics (ICE) eV, Ulm, Germany.
Natália Galito RochaDepartment of Physiology and Pharmacology, Biomedical Institute, Universidade Federal Fluminense, Niterói.
Alessandra C Toledo-ArrudaDepartment of Physiology and Pharmacology, Biomedical Institute, Universidade Federal Fluminense, Niterói.
Tania G ThomazDepartment of Physiology and Pharmacology, Biomedical Institute, Universidade Federal Fluminense, Niterói; Cardiovascular Sciences, Universidade Federal Fluminense, Niterói.
Cristiane MoraesCardiovascular Sciences, Universidade Federal Fluminense, Niterói.
Thais R Bessa-GuerraCardiovascular Sciences, Universidade Federal Fluminense, Niterói.
Mauricio LeãoInstitute of Nuclear Medicine, University Hospital Antonio Pedro, Niterói.
Arn MigowskiNational Cancer Institute (INCA), Rio de Janeiro, Brazil; National Institute of Cardiology (INC), Rio de Janeiro, Brazil.
André R Araujo da SilvaDepartment of Mother and Child, Faculty of Medicine, Universidade Federal Fluminense, Niterói, Rio de Janeiro, Brazil.
Christel WeissInstitute of Clinical Economics (ICE) eV, Ulm, Germany; Department of Medical Statistics, Medical Faculty Mannheim, University of Heidelberg, Mannheim, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The discussion about the optimal design of clinical trials reflects the perspectives of theory-based scientists and practice-based clinicians. Scientists compare the theory with published results. They observe a continuum from explanatory to pragmatic trials. Clinicians compare the problem they want to solve by completing a clinical trial with the results they can read in the literature. They observe a mixture of what they want and what they get. None of them can solve the problem without the support of the other. Here, we summarize the results of discussions with scientists and clinicians. All participants were interested to understand and analyze the arguments of the other side. As a result of this process, we conclude that scientists tell what they see, a continuum from clear explanatory to clear pragmatic trials. Clinicians tell what they want to see, a clear explanatory trial to describe the expected effects under ideal study conditions and a clear pragmatic trial to describe the observed effects under real-world conditions. Following this discussion, the solution was not too difficult. When we accept what we see, we will not get what we want. If we discuss a necessary change of management, we will end up with the conclusion that two types of studies are necessary to demonstrate efficacy and effectiveness. Efficacy can be demonstrated in an explanatory, ie, a randomized controlled trial (RCT) completed under ideal study conditions. Effectiveness can be demonstrated in an observational, ie, a pragmatic controlled trial (PCT) completed under real-world conditions. It is impossible to design a trial which can detect efficacy and effectiveness simultaneously. The RCTs describe what we may expect in health care, while the PCTs describe what we really observe.

Indexed as

explanatory trialideal study conditionspragmatic controlled trialpragmatic trialrandomized controlled trialreal-world conditions

Identifiers

PMID27774032
PMCPMC5045025

What OpenQuestion holds

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Registered trials

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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.