This site contains promotional information intended only for healthcare professionals resident in Great Britain.
Why Real-World Data?
Key characteristics of RCTs and real-world analyses
Strengths and limitations of RCTs and RWE data sources
Professor Axel Finckh covers important points such as the definitions of both Randomised Controlled Trials (RCTs) and Real World Evidence (RWE), the key differences in the patient populations, the data sources and how they are interpreted.
LTE=long-term extension; PY=patient-years; RA=rheumatoid arthritis; RCT=randomised controlled trial; RWE=real-world evidence
|RCTs1–3||RWE data sources1–3|
|• Interventional design
• Always prospective
• Data from prespeciﬁed assessment of relatively homogeneous patient population selected using well-deﬁned eligibility criteria
• Patient treatment based on randomised assignment to treatment or control/comparator groups
• Bias is controlled by random treatment assignment
|• Observational design
• Often retrospective
• Data from routine clinical practice and relevant point-of-care assessments of relatively heterogeneous patient population
• Patient treatment based on prescriber decision (not randomised)
• A variety of statistical approaches are used to minimise bias
Findings from real-world analyses should not be directly compared with those from RCTs1–3
RCT=randomised controlled trial; RWE=real-world evidence
|RCTs - Key Strengths1||RCTs - Key limitations1|
|• Adequately powered and scientifically robust
• Internal validity due to unbiased methods, e.g. narrowly defined study population, randomisation, blinding, inclusion of control groups
• Provide substantial information regarding the efficacy and safety of interventional products
• Prospective design
• Prespecified, well-defined endpoints
|• Can be constrained by ethical and practical considerations
• May lack external validity and generalisability to different settings; not reflective of real-life clinical settings
• Lack evidence required for medical decision-making or guiding patient‑centred care
• High investment in finances, resources and time
• Often conducted over a shorter period of time than required to fully assess the clinical and economic impact of a medical intervention
• Volunteer bias
• ‘Placebo’ response
|Additional characteristics of real-world data sources1,3*|
• Can assess risk factors that may be considered unethical in RCTs
• Reflect real-world outcomes, treatment patterns and clinical decision-making
• Less likely to be protocol-driven
• Analyses performed at low cost and over a short time frame
• Registries may have a longer time frame than RCTs (variable, detailed, longitudinal information)
• Large size of databases may identify outcomes of patients with rare events
• More diverse population of patients
• Generalisability of data about treatment impacts and use, costs for health services
• Lack of standardisation and randomisation; patient groups may not be comparable
• Susceptible to sample bias (if specific to one geographic location), observational bias and recall bias
• Risk factors/outcomes may change during follow-up
• Increased cost of data collection with larger number of patients and clinical settings
• Potential for reduced data quality (missing data, data entry/coding errors)
• Lack of statistical power to detect events other than specified key endpoints
• May lack a control or comparator group
• Difficult to distinguish cause and effect
*This is an overview of general strengths and limitations of real-world data sources, which may vary with each specific real-world data source type.
RCT=randomised controlled trial; RWE, real-world evidence
In this episode, Dr Ai Lyn Tan and Dr Sarah Mackie highlight the recent real-world evidence on shared decision making, treatment persistence and second-line treatment strategies in the context of Rheumatoid Arthritis (RA) with Janus Kinase inhibitors.
Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search
for MHRA Yellow Card in Google Play or Apple App Store
Adverse events should also be reported to Pfizer Medical Information on 01304 616161
To access further materials, resources and receive communication about medicines and vaccines promoted by Pfizer.
This site is intended only for healthcare professionals resident in the United Kingdom. If you are a member of the public wishing to access information on a specific medicine, please visit www.medicines.org.uk/emc
This website is brought to you by Pfizer Limited, a company registered in England
and Wales under No. 526209 with its registered office at Ramsgate Road, Sandwich, Kent, CT13 9NJ
Copyright © 2021 Pfizer Limited. All rights reserved.
VAT registration number GB201048427
These pages are not intended for patients or for members of the general public. The healthcare professional web pages contain promotional content.
I confirm that I am a healthcare professional* resident in the United Kingdom.
If you select 'No', you will be redirected to Pfizer.co.uk where you will be able to access reference information on Pfizer's prescription medicines.
*The ABPI Code definition for healthcare professional is members of the medical, dental, pharmacy and nursing professionals and any other persons who in the course of their professional activities may administer, prescribe, purchase, recommend or supply a medicine.
PP-PFE-GBR-3863. November 2021
OK, We will need you to sign in before we can determine if you are aligned with a Pﬁzer promotional colleague.
If you have already registered with pfizerpro.co.uk and select ‘yes’, you will be directed to the sign-in page where you will be required to enter your username and password.
Would you like to register or sign in now?