Real-world evidence – opportunities offered by big data
Real-world evidence (RWE) has become a buzzword in medicine in recent years. Yet the term is difficult to really grasp and define. We have tried to do just that: In this article, we explain what real-world evidence means, the role we play in it and why real-world evidence is the future of evidence-based medicine.
What exactly is real-world evidence and how is it generated?
In general, RWE describes “the representation of the reality of care through mass data sources.”[1] A cornerstone of this is the collection of meaningful data on the effectiveness of drugs in practice. To date, the generation of RWE has been limited to registry data. Registry data are datasets that are collected for various purposes and stored systematically in databases. Most of them are datasets used for billing drug therapies. These include, among others, insured persons' master data (gender, age, highest level of education…), ICD diagnosis data and inpatient hospital stays.
Clinical staff collect these data manually and submit them to the respective registry, i.e., the database of the collecting institution (e.g., health insurers). Since this is a more or less voluntary, non-standardized form of data collection, the completeness of these data is relatively low. Another problem is that these data are originally collected for the purpose of billing services and monitoring drugs, which differs greatly from their intended use in research.
Healthcare stakeholders are increasingly calling for consistent, standardized data collection. In addition to the main method of generating RWE, the FDA proposes the following further methods:
- Electronic health records
- Product and disease registries
- Data generated privately by patients, e.g., data from other sources that can provide insight into their health status, such as from mobile devices
XO Life's role in real-world healthcare
XO Life is taking a major step forward here by providing an innovative communication platform through which high-quality real-world data on the effects and side effects of drug therapies are collected. The primary goal is to standardize the datasets and make them available in real time to all those affected as well as to healthcare professionals and research companies.
In this way, patient-reported outcomes (PROs) create an additional basis of information for decisions on drug therapy. After all, big data is of little use if it is neither structured nor accessible. The aim is to digitalize the monitoring of the effectiveness of drug therapies, simplify it and make it more cost-effective. At the same time, the wealth of data expands the scope for applying precision medicine, which will become increasingly important in the future. This allows treatment strategies and therapies to be tailored to the individual. All of this, of course, under the strictest data protection and in compliance with all provisions of the GDPR.
Real-world evidence as a complement to the clinical “gold standard” of randomized controlled trials (RCTs)
For the reasons outlined above, RWE is ideally suited to shaping the future of evidence-based medicine alongside tried-and-tested research approaches (RCTs). After all, the time and cost involved in RCTs are not always justified.
RCTs are the gold standard for clinical trials. They use randomized and controlled treatment groups. A randomly selected treatment group receives either the drug or a placebo. In evidence-based medicine, this experimental study design is particularly well suited to establishing causality, i.e., obtaining a clear answer to a clearly defined question.
Despite these advantages, however, RCTs also have serious drawbacks, which is why they are not appropriate in some cases. RCTs often involve considerable cost and time. In addition, strict inclusion criteria reduce external validity, i.e., the extent to which the results apply to the general population. There are also tight time constraints. This is particularly the case for drugs used to treat rare diseases (orphan drugs).
The topic is also more relevant than ever right now, as the first vaccines against the coronavirus have been approved. The rapid conditional approval of the vaccines by the EMA often raises questions about their safety. Because of the limited approval, the manufacturers of these pharmaceuticals must ensure data collection that accompanies their use. This means that pharmaceutical manufacturers must continuously collect data from patients taking a drug in order to monitor possible side effects. Collecting real-world evidence, e.g., through XO Life's ImpactMonitor, promises to be an innovative way of solving this problem. The large volume of data mentioned earlier, collected from a wide range of population groups, is a key advantage of generating RWE. RWE collected as a complement to RCTs can significantly strengthen the final body of evidence. Following marketing authorization, it can establish clear evidence of the benefit-risk profile of drug therapies as quickly as possible – for children and adults, young and old, men and women alike.
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. It helps drug manufacturers, for instance, learn about drug effects and side effects more quickly. By seamlessly integrating the ImpactMonitor into existing processes, we support research institutes and manufacturers in collecting the data they need. In addition, real-world evidence can be collected more quickly when RCTs for new drugs are not appropriate, e.g., due to time constraints. In this way, our platform makes a significant contribution to drug safety and to targeted, individualized prescribing for patients.
Sources:
Behrendt, C.A. Was ist die Realität hinter der Real-World-Evidenz?. Gefässchirurgie 24, 7–8 (2019). Accessed at: https://link.springer.com/article/10.1007/s00772-018-0474-9; Last accessed 04.01.2021
Gliklich, 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
