Integrating real-life studies in the global therapeutic research framework

Roche, N.; Reddel, Helen K.; Agustí, Àlvar; Bateman, Eric D.; Krishnan, Jerry A.; Martin, Richard J.; Papi, Alberto; Postma, Dirkje S.; Thomas, Michael; Brusselle, Guy; Israel, Elliot; Rand, Cynthia M.; Chisholm, Alison; Price, David

doi:10.1016/s2213-2600(13)70199-1

Cited by 113 publications

(120 citation statements)

References 10 publications

Supporting

Mentioning

115

Contrasting

Unclassified

Order By: Relevance

“…The progressive increase of real life studies published in the last years and their recognized scientific value [8,9] underline that their role is becoming constructive in respiratory medicine, attracting research interest and debate [10,11]. The second part of the review will consider some of the advantages and disadvantages, strengths and weaknesses of different types of trials and comment on the complementarity between RCTs and real life studies.…”

Section: Introductionmentioning

confidence: 99%

Randomized controlled trials and real life studies. Approaches and methodologies: a clinical point of view.

Saturni¹,

Bellini²,

Braido³

et al. 2014

Pulmonary Pharmacology & Therapeutics

202

155

View full text Add to dashboard Cite

a b s t r a c tRandomized Controlled Trials (RCTs) are the "gold standard" for evaluating treatment outcomes providing information on treatments "efficacy". They are designed to test a therapeutic hypothesis under optimal setting in the absence of confounding factors. For this reason they have high internal validity. The strict and controlled conditions in which they are conducted, leads to low generalizability because they are performed in conditions very different from real life usual care. Conversely, real life studies inform on the "effectiveness" of a treatment, that is, the measure of the extent to which an intervention does what is intended to do in routine circumstances. At variance to RCTs, real life trials have high generalizability, but low internal validity. Recently the number of real life studies has been rapidly growing in different areas of respiratory medicine, particularly in asthma and COPD. The role of such studies is becoming a hot topic in respiratory medicine, attracting research interest and debate.In the first part of this review we discuss some of the advantages and disadvantages of different types of RCTs and analyze the strengths and weaknesses of real life trials, considering the recent examples of some studies conducted in COPD. We then discuss methodological approaches and options to overcome some of the limitations of real life studies.Comparing the conclusions of effectiveness and efficacy trials can provide important pieces of information. Indeed, these approaches can result complementary, and they can guide the interpretation of each other results.

show abstract

Section: Introductionmentioning

confidence: 99%

Randomized controlled trials and real life studies. Approaches and methodologies: a clinical point of view.

Saturni¹,

Bellini²,

Braido³

et al. 2014

Pulmonary Pharmacology & Therapeutics

202

155

View full text Add to dashboard Cite

show abstract

“…For people with asthma, quality management in primary care is a real, viable and accessible solution to asthma care. This is reflected in guidelines [2,5, 6], in research trends (as we see pragmatic research sourcing participants in primary care) [7,8] and it is evidenced in a multitude of primary healthcare initiatives established to support primary care physicians to optimally manage asthma in the community.Optimal asthma management: are we achieving it? Or is it an enigma?…”

mentioning

confidence: 99%

Asthma management in primary care: caring, sharing and working together

Bosnic‐Anticevich

2016

Eur Respir J

View full text Add to dashboard Cite

@ERSpublicationsWhy aren't primary care physicians and pharmacists collaborating more to manage and monitor asthma medication issues? http://ow.ly/YB2p6While historically chronic illnesses have been "stewarded" by different practitioners in different countries [1] with the increasing number of people with chronic illnesses and the ever rising costs of healthcare, the primary care setting is viewed as the long-term custodian of chronic illness management [2][3][4]. For people with asthma, quality management in primary care is a real, viable and accessible solution to asthma care. This is reflected in guidelines [2,5, 6], in research trends (as we see pragmatic research sourcing participants in primary care) [7,8] and it is evidenced in a multitude of primary healthcare initiatives established to support primary care physicians to optimally manage asthma in the community.Optimal asthma management: are we achieving it? Or is it an enigma? These are questions that primary care researchers and healthcare practitioners have been burdened with for a long time. Why? Because evidence, from large-scale and population-based studies confirms that, despite our ever-increasing knowledge and the abundance of research in the practice/clinical field, asthma remains a major health and economic burden. It is estimated that ∼300 million people live with asthma world-wide [2] and a high proportion of people living with asthma are classified as having poorly controlled disease [4,[9][10][11][12][13]. Asthma has significant economic impact both on individuals and on the healthcare system. A recent report determined that the net annual value of the burden of disease of asthma in Australia (based on disability-adjusted life years) was AUD 24.7 billion, with projected government costs for 2016-2019 being AUD 4.0 billion [14].For primary care practitioners, the current status of asthma in the community is at the very least disappointing, if not frightening; especially given the efforts put into improving asthma care at all levels (health system, research, global reports, practice initiatives, patient support and public awareness). The reality is that the multifaceted initiatives are falling short of addressing the multifactorial issues associated with sub-optimal management in real life [2,10,11,[15][16][17][18]: issues spanning across patient, healthcare practitioners and healthcare-system domains [12,19].One of the major issues in practice is asthma medication use. Medication management is a well-recognised, documented and investigated aspect of asthma management. Consistently research and practice indicate that many of the issues associated with sub-optimal management of asthma are related to the way in which people use their medicines, i.e. a high proportion of patients are nonadherent and a majority do not master or maintain correct inhaler technique [9,10]. Effective solutions to inappropriate and incorrect asthma medication use have been identified through high quality research; however, if these strategies are being implemented in...

show abstract

“…While randomized clinical trials examining COPD treatment provide essential outcome data with high internal validity, the study population typically includes highly selected patients which limits generalizability to the real-life clinical practice COPD population [21]. Alternatively, analyses of real-world clinical practice databases that include the broad population of patients receiving treatment, such as in the current study, address the generalizability of treatment outcomes in real-life clinical management of diverse patients [22]. Strengths of the Patients were matched on the basis of gender, moderate and severe COPD exacerbations in baseline (categorized), ICS use in baseline (yes/no), LABA use in baseline (yes/no), LAMA use in baseline (yes/no), and age (±6 years).…”

Section: Discussionmentioning

confidence: 99%