If you need a patient registry software application, please contact us at info@zlynger.com
Real-world evidence (RWE) studies are very important because they allow healthcare sector stakeholders to make decisions with greater certainty and accuracy by revealing the reality of approved treatments and diseases more effectively.
How do physicians know whether a new, recently authorized drug is equally effective for patients with different characteristics? How can the toxicity of a new drug be assessed in the long term? How do commercialized drugs work in a larger population under the conditions of standard clinical practice?
These are some questions that the pharmaceutical industry is trying to answer through RWE studies.These questions cannot always be satisfactorily answered when conducting clinical trials before drug approval.
The aim of this article is to describe in detail why generating and processing real-world evidence —or real-world data (RWD)— is becoming essential across the healthcare system and the importance of using a suitable software application to collect and manage data in these studies.
In this post, RWE studies will be defined first and, then, frequently asked questions will be answered: Why are RWE studies important? What type of RWE studies are there? What is the difference between a clinical trial and an RWE study? How is data collected in RWE studies?
What is a Real-World Evidence (RWE) Study?
In the late 17th century, medical officers of the British navy observed in routine clinical practice how scurvy was spreading among their crew.
From these observations and their analysis, they deduced that it came from a lack of vitamin C.
In other words, the knowledge of scurvy, a rare disease at the time, and its treatment with citrus fruits corresponds to the collection of clinical data from sailors, soldiers and prisoners of the British Crown in that century [1].
Therefore, an RWE study can be defined as the result of analyzing global information gathered from patients during routine clinical practice.
The U.S. Food and Drug Administration (FDA) defines Real-World Evidence as: “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of Real-World Data (RWD)” [2].
Thanks to technology, the health sector has become aware of the great value of RWD and RWE across healthcare. In 2018, the FDA officially announced a new program to promote the agency’s use of real-world evidence, which was mandated by the 21st Century Cures Act.
This law was designed to help accelerate medical product development and bring new innovations and advances to patients who need them faster and more efficiently [3].
Why Are RWE Studies Important?
RWE studies are important because they provide new insights into several areas of healthcare.
By using real-world evidence, stakeholders speed up the evolution of new therapies, improve diagnosis, can make decisions about the best treatments for patients with rare diseases, and they are able to find out more about the efficacy and safety of the latest drugs [4].
How can RWE studies offer these advantages? RWE studies provide a comprehensive scope of what actually happens in real life, from the general population, not from a sample of the population (like in clinical trials). The difference between these two types of clinical studies (RWE vs clinical trials) will be noted below.
Before specifying which areas of healthcare benefit from RWE studies, these are the 3 steps of how it works:
- Real-world data (RWD) is collected and transformed into RWE through robust analysis.
- Real-world evidence (RWE) provides meaningful information on what happens in daily practice and why.
- Stakeholders in the healthcare ecosystem use these new insights to improve outcomes and, consequently, patient’s health [5].
RWE studies are predominantly useful for the stakeholders listed below, in order to meet their commitment to healthcare and improve the effectiveness of a product across its lifecycle:
Drug Developers
In the framework of pharmaceutical research, RWE helps clinical investigators to determine the efficacy of a new treatment by providing additional information to the traditional clinical trial results on the use of a medicine in routine clinical care.
Physicians
The role of healthcare professionals in generating RWE is crucial. They enter clinical data into clinical registries. In return, physicians can leverage this information to make accurate treatment decisions and understand a disease better.
Regulators
And of course, these treatments need to be safe and effective.That is the job of regulators, who monitor safety and adverse reactions before and after a pharmaceutical product is marketed. In this way, regulators assist regulatory agencies (like the FDA) in decision-making.
Therefore, the use of real-world testing has the potential to influence drug approval and speed up the pharmaceutical development process.
Payers
Within the healthcare ecosystem, payers also have to come to a conclusion based on the question that answers what matters most to them: Does a medical product provide enough benefits to patients in the short or long-term? And is the price justified?
The spectrum of value that payers perceive from real-world evidence includes informed safety monitoring, utilization management and cost/value analysis [6].
What Types of RWE Studies Are There?
RWE can be generated by different study designs or analyses, including observational studies, which can be prospective and/or retrospective [7].
Prospective and retrospective methods are used for many research studies in the health sector. The main difference between the two is the timing of the research and the generation of the source data and the outcome.
Prospective Studies
Prospective studies involve following individuals over a period of time in a prospective manner and collecting information about them as their characteristics or circumstances change. They are patients who are observed as they suffer a disease or get a specific treatment, and they are included in the study before or while these circumstances occur [8].
There are various large prospective studies in progress, for example, The Nurses Health Studies, established in 1976, which investigates risk factors for chronic diseases in women. This study has followed 280,000 male and female nurses to date [9].
Retrospective Studies
Retrospective studies collect a sample of individuals who already had a disease, condition, or treatment, and information is gathered about their past without prospective follow-up.
This can be done through interviews, in which participants are asked to recall important events, or by identifying relevant administrative data to complete information about past events and circumstances [10].
A common way of conducting retrospective RWE studies is to collect detailed clinical data already registered in patient records and charts, at clinical sites. Then, this data is entered in a database for analysis.
A significant example of a retrospective study is the death of 43,000 adult males in India. This study found that smoking, which increases the incidence of clinical tuberculosis, is a cause of half the male tuberculosis deaths in India [11].
What Is the Difference between an RWE Study and a Clinical Trial?
To date —and this will continue to be the case in the future—, clinical trials have been the traditional research format —the Gold Standard— in the approval of new therapies. However, clinical trials are not enough, according to the experts.
The main difference between these two research formats is that an RWE study is observational while a clinical trial (for example a randomized controlled trial; RCT) is interventional.
In clinical trials, a small segment of the population suffering from the disease in question is selected and a new therapy is tested in a controlled environment for a certain period of time. This segment somewhat represents the whole population.
Whereas an RWE study may collect data from a broader population from different sources and with different time conditions. These data may include subgroups that would not have been considered in clinical trials, such as pregnant women, diabetics, obese people, etc:
“Researchers can study how new therapies work among patients with comorbidities, certain age groups, or specific socio-demographic groups. Real-world evidence can also help researchers understand what happens to a patient over a lifetime, not just throughout the RCT period.” [12]
Most health professionals agree with the fact that RWE studies are an important complement to RCTs, and that RCTs need RWE.
Both types of studies should be used in a mutually productive, non-competitive way to improve patient care, quality, outcomes, and safety and, what’s more, to avoid mistakes in new therapies.
AstraZeneca concludes: “RWE complements the evidence generated by RCTs and provides healthcare decision-makers with the confidence to choose the right treatment options for patients.” [13]
What is the Source from which RWE Data is Collected?
As previously stated, RWD is gathered from a variety of sources. In fact, the main source is the information provided by patients in the medical practice.
But there are additional important sources according to the type of data obtained [14]:
- Clinical data: collection of data available in a patient’s medical records. This includes diagnoses, medications, tests, allergies, vaccinations, treatment plans, patient demographics, family history, comorbidities, procedure and treatment history, as well as outcomes.
- Patient-generated data: data from patient-reported outcome (PRO) surveys. The information collected from this surveillance is valuable to researchers as it comes directly from the patient, revealing the patient’s situation outside the medical context, in hospital stays and procedures.
- Cost and utilization data: information based on claims and public databases, which provides insight into health service utilization, population coverage and prescription patterns.
- Public health data: information derived from government data, a key source for stakeholders in order to improve the needs of their populations.
How is Data Collected in RWE Studies?
This article has highlighted the key role of RWE studies and data collection in improving health care services and clinical research.
In order to leverage data collected from patients in observational RWE studies, this data needs to be clear, accurate and structured for analysis.
Therefore, in RWE studies data is gathered through web-based patient registries. For example, hospitals enter information into a database, called an electronic data capture (EDC) system, or electronic case report form (eCRF); a software application that records patient information for clinical research.
What Software Solution Does Zlynger Provide for RWE Studies?
Zlynger provides a customized web-based patient registry tool for RWE studies. Physicians and clinical research teams can use our registry tool to enter and store clinical data through customized forms specifically designed to collect and analyze the information they need.
If you need a patient registry software application, please contact us at info@zlynger.com
References:
[1] Comment by Palacios, M. (2019). The quality of Research with Real-World Evidence. Revista Colombia Médica, vol. 50, nº 3. Cited from an article by Harrison, M. (May 2013) Journal for Maritime Research.
[2] Comment by Gil, A. and Granjo, P. (2021). Introduction to Real-World Data & Evidence. Drug Development RWD-RWE Blog. Available from: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence [Accessed March 2021]
[3] Klonoff, D. C. (2019). The New FDA Real-World Evidence Program to Support Development of Drugs and Biologics. Journal of Diabetes Science and Technology. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7196856/. [Accessed September 2021]
[4] Ibid [2] (Suggested citation: https://worldcdg.org/drug-development-rwd-rwe/introduction-real-world-data-evidence.)
[5] Echeazu, Benjamin. 9 Ways Real World Evidence is Changing Healthcare. Real-World Evidence Blog. https://www.arbormetrix.com/blog/. Published August 2021. [Accessed September 2021]
[6] Real-World Evidence. Trending Topics. https://veradigm.com/real-world-evidence. [Accessed September 2021]
[7] Real-World Evidence. U.S. Food and Drug Administration.
https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Published 2020. [Accessed September 2021]
[8] Prospective vs retrospective studies. 2.4. Introduction to Longitudinal Studies. The Learning Hub at UCL Institute of Education. Learning Hub | Prospective vs retrospective studies (closer.ac.uk). [Accessed September 2021]
[9] Glen, Stephanie. Prospective Study: Definition, Examples. From StatisticsHowTo.com. Elementary Statistics for the rest of us! https://www.statisticshowto.com/prospective-study/. Published 2016. [Accessed September 2021]
[10] Ibid [8]
[11] Dr. Gajalakshmi, Vendhan PhD, Prof. Peto, Richard FRS. Smoking and mortality from tuberculosis and other diseases in India: retrospective study. The Lancet. Volume 362, Issue 9383,16 August 2003, Pages 507-515. From: Smoking and mortality from tuberculosis and other diseases in India: retrospective study of 43 000 adult male deaths and 35 000 controls – ScienceDirect. [Accessed September 2021]
[12] Ibid [5]
[13] Sajan, Khosla, White, Robert, Medina, Jesús. Real world evidence (RWE) – a disruptive innovation or the quiet evolution of medical evidence generation? (nih.gov). Published online 2018 Aug. 29. [Accessed September 2021]
[14] Ibid [5]
