Real-world evidence — opportunities for big data

Real-world evidence (RWE) has become a trending term in medicine in recent years. However, the term is difficult to correctly grasp and narrow down. We've tried it and explain what real-world evidence means, what role we play in it, and why real-world evidence is the future for evidence-based medicine.

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What exactly is real-world evidence and how is it collected?

RWE generally describes “the presentation of the reality of care through mass data sources.” [1] A cornerstone of this is the collection of meaningful data on drug effectiveness in practice. To date, the generation of RWE has been limited to register data. Register data are data sets that are collected for various purposes and systematically stored in databases. It is usually data sets that are used to bill for drug therapies. These include Master insurance data (gender, age, highest school leaving certificate...), ICD diagnostic data and inpatient stays in hospitals.

Clinical staff collect this data manually and transfer it to the respective registers, i.e. the database of the collecting institution (e.g. health insurance companies). Since this is a more or less voluntary, non-standardized survey, the completeness of this data is relatively low. Another problem is that this data is originally collected for the purpose of billing services and drug monitoring, which is very different from the purpose of research.

Voices from the healthcare sector are obviously getting louder and louder following uniform, standardized data collection. In addition to the main method for generating RWE, the FDA suggests these other methods:

1. Electronic health records
2. Product and disease register
3. Data generated privately by the patient, e.g. Data from other sources that can provide information about health conditions, such as mobile devices

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The role of XO Life in a real supply setting

XO Life is taking a big step forward and providing an innovative communication platform that collects high-quality data from the real care setting on the effects and side effects of drug therapies. The primary goal is to standardize the data sets and make them available to all affected persons as well as medical professionals and research companies in real time.

This creates a further basis of information, through Patient Reported Outcomes (PRO), for drug therapy decisions. Because big data is of little use if it is neither structured nor accessible. The monitoring of the effectiveness of drug therapies is thus intended to be digitized, simplified and more cost-effective. At the same time, due to the large amount of data, the application of precision medicine, which will become increasingly important in the future, is increasing. In this way, treatment strategies and therapies can be individually tailored. Of course, under the strictest data protection regulations and in compliance with all GDPR regulations.

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Real-world evidence as a complement to the clinical “gold standard” of Randomized Controlled Trials (RCT)

For the reasons mentioned above, RWE is ideal for shaping the future of evidence-based medicine in addition to tried and tested research approaches (RCTs). For example, the time and financial costs of RCT studies are not always appropriate.

RCTs are the gold standard for clinical trials. Randomized and controlled treatment groups are used. A randomly selected treatment group receives either the medication or a placebo. In evidence-based medicine, this study design, carried out as an experiment, is particularly suitable for obtaining causality, i.e. a clear answer from a clear question.

But in addition to these advantages, RCTs also have serious disadvantages, which is why they are not appropriate in some cases. For example, RCTs are often expensive and time-consuming. There are also high inclusion criteria, which reduce external validity, i.e. the consistency of the results with the entire population. There is also a tight time limit. This is particularly the case with drugs for the treatment of rare diseases (orphan drugs).

Even at present, the issue is more topical than ever before, since the first vaccines against the corona virus have now been approved. The rapid, conditional approval of vaccines by the EMA often raises questions about the safety of these vaccines. Due to the limited approval, manufacturers of these pharmaceuticals must ensure data collection during use. That is, pharmaceutical manufacturers must continuously collect data from patients taking a drug to monitor potential side effects. Collecting real-world evidence, e.g. through XO Life's ImpactMonitor, promises to be an innovative way to solve the problem. The previously mentioned variety of data, which is collected from a wide variety of groups of people, represents an important advantage of collecting RWE. RWE collected as a complement to RCT studies can significantly summarize the final data situation. Following market approval, it can provide clear evidence as quickly as possible for the benefit-risk profile of drug therapies for young and old, and men and women.

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conclusion

Real-world evidence, which can be generated via the ImpactMonitor, for example, will play a major role in many areas of evidence-based medicine in the future. For example, it helps drug manufacturers learn more quickly about drug effects and side effects. By seamlessly integrating ImpactMonitor into existing processes, we support research institutes and manufacturers in collecting the required data. In addition, real-world evidence can be collected more quickly if RCT studies on new drugs are not appropriate, e.g. due to time limits. In this way, our platform makes a significant contribution to the safety of medicines and targeted individual drug prescriptions to patients.

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sources:

Behrendt, C.A. What is the reality behind the real-world evidence? Vascular Surgery 24, 7—8 (2019). Accessed at: https://link.springer.com/article/10.1007/s00772-018-0474-9; Last accessed 04.01.2021

Good, Leavy, Dreyer. Registries for Evaluating Patient Outcomes: A User's Guide (2020). Accessed at: https://effectivehealthcare.ahrq.gov/sites/default/files/pdf/registries-evaluating-patient-outcomes-4th-edition.pdf; Last accessed 04.01.2021

U.S. Food & Drug Administration. Real-World Evidence (2020). Accessed at: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence; Last accessed 04.01.2021