Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2019 Jan 29;6(1):1565804.
doi: 10.1080/20018525.2019.1565804. eCollection 2019.

Effectiveness trials: critical data to help understand how respiratory medicines really work?

Andreas Heddini  1 , Josefin Sundh  2 , Magnus Ekström  3 , Christer Janson  4
Affiliations
Review

Effectiveness trials: critical data to help understand how respiratory medicines really work?

Andreas Heddini et al. Eur Clin Respir J. .

Abstract

Most of the information about the benefits, safety aspects, and cost effectiveness of pharmacological treatment in the respiratory field has been obtained from traditional efficacy studies, such as randomised controlled trials (RCT). The highly controlled environment of an RCT does not always reflect everyday practice. The collection, analysis, and application of effectiveness data to generate Real World Evidence (RWE) through pragmatic trials or observational studies therefore has the potential to improve decision making by regulators, payers, and clinicians. Despite calls for more RWE, effectiveness data are not widely used in decision making in the respiratory field. Recent advances in data capture, curation, and storage combined with new analytical tools have now made it feasible for effectiveness data to become routine sources of evidence to supplement traditional efficacy data. In this paper, we will examine some of the current data gaps, diverse types of effectiveness data, look at proposed frameworks for the positioning of effectiveness data, as well as provide examples from therapeutic areas. We will give examples of both previous effectiveness studies and studies that are ongoing within the respiratory field. Effectiveness data hold the potential to address several evidentiary gaps related to the effectiveness, safety, and value of treatments in patients with respiratory diseases.

Keywords: COPD; Effectiveness; asthma; co-morbidity; efficacy RCT; evidence-base; levels of evidence; pragmatic trials; real-world evidence; respiratory disease.

PubMed Disclaimer

Figures

Figure 1.
Figure 1.
Conceptual framework for therapeutic research. Adapted from Roche et al. [14] (Reproduced with permission from the publisher).
Figure 2.
Figure 2.
Positioning and role of effectiveness studies in the evidence generation chain.
See this image and copyright information in PMC

References

    1. Treweek S, Zwarenstein M.. Making trials matter: pragmatic and explanatory trials and the problem of applicability. Trials. 2009June3;10:37 PubMed PMID:19493350; PubMed Central PMCID:PMC2700087. - PMC - PubMed
    1. Godwin M, Ruhland L, Casson I, et al. Pragmatic controlled clinical trials in primary care: the struggle between external and internal validity. BMC Med Res Methodol. 2003December22;3:28 PubMed PMID:14690550; PubMed Central PMCID:PMC317298. - PMC - PubMed
    1. ABPI The vision for real world data – harnessing the opportunities in the UK. 2011[cited 2018 September] Available from: http://abpi.org.uk/media/1378/vision-for-real-world-data.pdf
    1. Roland M, Torgerson DJ. What are pragmatic trials? Bmj. 1998January24;316(7127):285 PubMed PMID:9472515; PubMed Central PMCID:PMC2665488. - PMC - PubMed
    1. Holgate S, Bisgaard H, Bjermer L, et al. The Brussels declaration: the need for change in asthma management. Eur Respir J. 2008December;32(6):1433–7. PubMed PMID:19043008. - PubMed

LinkOut - more resources