Evidence generated in the scenario where patients are taking their medicines in real world is referred to as the real world evidence (RWE). Usually, results of actual human studies in the research environment are considered to be the gold standard when compared to the data obtained from such real world setting, where data from daily clinical practice is available. The human studies in experimental settings often have theories that are tested on a selected group of patients, who randomly receive one of the two or more treatments being studied. This randomisation allows for fair comparison and reduces partiality to the maximum extent possible.
However, the clinical studies come with their own set of limitations. For instance, they have specific selection criteria for patients to make sure that a standardised patient sample is created. However, in the real world, there is no such criteria and no deprived patient can be left out from a certain treatment. Moreover, findings from clinical studies can only be generalised to the type of patients similar to those who were included in those studies. This may make those findings without any use as they may not be applicable to the real patients in the real world setting. However, RWE and studies focusing on RWE can very well supplement the results of a clinical study to possibly bridge the gap between the rigid clinical environment and the harsh real world setting.
On the contrary, there are a few examples that have shown promising results in the clinical study stages, but poor results in the actual daily clinical practice. For example, in the US, a strong disagreement was observed between the results of a real-world, observational study and a large clinical study that compared outcomes of patients using glucose-lowering medications.(1) Similarly, researchers showed poor effect of certain cancer drugs in the real world setting when compared to that observed within the clinical study setting. (2)
About the efficacy of prescribed medicines in the real world setting, it can be debatable. However, in most cases, they can prove to be efficacious because they are mostly studied in the population similar to that they’re being prescribed for. On the other hand, it can’t be argued that all these prescribed medicines (i.e. those available in the market) get to the market after passing through all the stages of a clinical study in the first place. This means that they’re possibly assessed for safety and efficacy in a selected group of patients with some inclusion/exclusion criteria. Therefore, in the real world setting, with no such criteria, the medicine may or may not be effective.
To overcome this dilemma, one can perform large simple studies, where both the clinical as well as real world aspects are bound together to match with each other’s results. Such studies that prove the effectiveness of medications can improve the connection between the clinical and the real world environments within research. However, real world studies also have some limitations. Real world population cannot randomly receive a medication, but a specific one. Patients may not adhere to the doses in the real world setting, thus indirectly questioning the usefulness of the prescribed medicines.
No matter what or how the research is conducted to launch the product into the market, you, as a patient, should share your experience with the medicine you are prescribed. This can be done with a medium called as ‘patient reported outcomes’ (PROs). PROs are provided by patients, so they directly come from the real world setting and thus can prove to be an essential source for the RWE. PROs are increasingly being preferred by the pharmaceutical research companies to take into account the patient insights that can help them greatly improve their research as well as future product strategies. You can say that PROs are like an instant feedback to the research companies from their end consumers, i.e. patients.
Therefore, all prescribed medicines are usually effective in the real world. However, you should always try to be transparent about your treatment with your doctor as your feedback contributes to the RWE, which can ultimately help in a great way with the future scientific research!
- Circulation. 2017; 136:249-59.
- JAMA Intern Med. 2017; 177:276-7.