PCT Definitions

Clinical Trial

Effectiveness Study

Clinical studies investigating health interventions whose design does not follow the design of a randomised controlled clinical trial and aims to reflect health intervention effectiveness in routine clinical practice.

Effectiveness studies do not typically include randomisation of trial subjects, but there are exceptions (e.g. pragmatic clinical trials). For the purposes of GetReal, effectiveness studies include, but are not limited to, the following: pragmatic clinical trials, non-interventional/ observational studies, drug utilisation studies, post-authorisation efficacy/safety studies. (See also: “real-world studies”, “drug utilisation study”, “pragmatic clinical trial” and “non-interventional/ observational study”)

Large Simple Trials

Large simple trials are pragmatic, randomised clinical trials with minimal data collection protocols that are narrowly focused on clearly defined outcomes that are important to patients as well as clinicians. Their large sample size provides the adequate statistical power to detect a small difference in effects between treatments in a situation where a moderate difference in an important outcome may be important. Additionally, LST’s include follow-up that mimics normal clinical practice. LST’s are by definition pragmatic clinical trials. (See also “pragmatic clinical trials”)

[Source:imi GetReal Glossary – 25 Oct 2016– (Adapted from Stroup 2011, Peto 1995)]*

* Stroup, T.S., What can large simple trials do for psychiatry? Am J Psychiatry, 2011. 168(2): p. 117-9.; Peto, R., R. Collins, and R. Gray, Largescale randomized evidence: large, simple trials and overviews of trials. J Clin Epidemiol, 1995. 48(1): p. 23-40; Clinical Trials Transformation Initiative. Large Simple Trials: Facilitating the Use of Large

Low-Intervention Clinical Trial (LICT)

‘Low-intervention clinical trial’ means a clinical trial which fulfils all of the following conditions:

(a) the investigational medicinal products, excluding placebos, are authorised;

(b)  according to the protocol of the clinical trial,

(i)  the investigational medicinal products are used in accordance with the terms of the marketing authorisation; or

(ii)  the use of the investigational medicinal products is evidence-based and supported by published scientific evidence on the safety and efficacy of those investigational medicinal products in any of the Member States concerned; and

(c) the additional diagnostic or monitoring procedures do not pose more than minimal additional risk or burden to the safety of the subjects compared to normal clinical practice in any Member State concerned;

[as per Article 2.2.3 of Regulation EU/2014/536]

Normal Clinical Practice

 ‘Normal clinical practice’ means the treatment regime typically followed to treat, prevent, or diagnose a disease or a disorder.

[as per Article 2.2.6 of Regulation EU/2014/536]

Pragmatic Clinical Trial (PCT)

 A study comparing health interventions among a randomised, diverse population representing clinical practice, and measuring a broad range of health outcomes. To ensure generalizability, pragmatic trials should represent the intended patients to whom the treatment will be applied as best as possible. For instance, inclusion criteria would be broad (e.g. allowing co-morbidity, co-medication, wider age range, etc.), the follow-up would not be (or not much) interventional and allowing for treatment switching etc. (See also “large simple trials” and “real-world studies”)

[Source:imi GetReal Glossary – 25 Oct 2016– Adapted from Schwartz, 1967, Tunis, 2003 & Roland, 1998]*

* Schwartz, D., & Lellouch, J. (1967). Explanatory and pragmatic attitudes in therapeutical trials. Journal of chronic diseases, 20(8), 637-648.; Tunis, S. R., Stryer, D. B., & Clancy, C. M. (2003). Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. Jama, 290(12), 1624-1632.; Roland, M. and D.J. Torgerson, What are pragmatic trials? BMJ, 1998. 316(7127): p. 285.

Real World Data

 An umbrella term for data regarding the effects of health interventions (e.g. safety, effectiveness, resource use, etc) that are not collected in the context of highly-controlled RCT’s. Instead, RWD can either be primary research data collected in a manner which reflects how interventions would be used in routine clinical practice or secondary research data derived from routinely collected data. Data collected include, but are not limited to, clinical and economic outcomes, patient-reported outcomes (PRO) and health-related quality of life (HRQoL). RWD can be obtained from many sources including patient registries, electronic medical records, and claims databases. (See also “randomised controlled clinical trial”, “real-world evidence” and “real-world study”)(Adapted from Garrisson, 2007 and GetReal, 2016)

[Source: imi GetReal Glossary – 25 Oct 2016– Adapted from Garrisson, 2007]*

* Garrison et al. (2007). Using Real-World Data for Coverage and Payment Decisions: The ISPOR Real-World Data Task Force Report. Value in Health 10:5, 2007

Real World Evidence

Real World Evidence (RWE) is the evidence derived from the analysis and/or synthesis of real-world data (RWD). (See also “real-world data”, “real-world study”)

[Source:imi GetReal Glossary – 25 Oct 2016]

Real World Studies

Studies investigating health interventions whose design does not follow the design of a highly-controlled RCT and aims to reflect health intervention effectiveness in routine clinical practice. Real world studies do not typically include randomisation of trial subjects, but there are exceptions (e.g. pragmatic clinical trials). For the purposes of GetReal, real-world studies include, but are not limited to, the following: pragmatic clinical trials, non-interventional/ observational studies, drug utilization studies, post-authorisation efficacy/safety studies. RWS, by definition, generate RWD, which can subsequently be analysed and/or synthesized to produce RWE. (See also: “real-world data”, “real-world evidence”, “effectiveness study”, “pragmatic clinical trial”)

[Source:imi GetReal Glossary – 25 Oct 2016]

Standard of Care

Care delivered by a healthcare provider for a specific patient which should correspond to the care that an averagely competent physician in the same field would provide under similar circumstances.

[Source:imi GetReal Glossary – 25 Oct 2016– Adapted from Strauss, 2009]*

* Strauss D.C., Thomas J.M. (2009). What Does the Medical Profession Mean By “Standard of Care?” JCO Nov 10, 2009: e192-193; published online on September 21, 2009.