By Dr Bina Rawal, Board Director at Vann
Image by Gerd Altmann from Pixabay
In the world of cancer research and drug development, there is a lot of focus on real world data. So what exactly do we mean by this?
Real world data (RWD) in medicine is data that is generated from a heterogeneous or mixed patient population in a real-world setting, as opposed to data derived from randomised controlled clinical trials. RWD can be derived directly from electronic health records, health insurance claims and also patient surveys, such as those offered on the Vann platform.
To understand why RWD is relevant, it’s important to first understand randomised, double-blind, controlled clinical trials (RCTs). When a new drug is being developed by a pharmaceutical or biotechnology company, they have to satisfy rigid requirements to prove that the drug is safe, effective and manufactured to a high quality standard before a regulator will ‘approve’ or give a licence to the company to market the drug. During this drug development process, the most sure way to demonstrate safety and efficacy is through RCTs. Additionally, RCTs can be carried out by researchers when the drug is being tested against different comparators or to evaluate different dosage regimens.
The term randomised means that when a patient agrees to join a trial, they are assigned to a treatment or control group completely randomly or by chance – this avoids any bias that would arise if the investigator could choose which groups to allocate specific patients to. Double-blind means that neither the patient nor the investigator or clinicians looking after the patient know which treatment or control group the patient has been allocated to – this avoids patients or clinicians knowing or thinking that they know what to expect and thereby influencing the results. Controlled means that the new drug is being compared to something else, either an identical dummy version of the drug (placebo) or another accepted treatment (active comparator) – this allows the investigator to see the size of the effect that the new drug has in relation to no active treatment (placebo) or in relation to the currently accepted gold standard treatment.
In order to get clear read-outs from RCTs, these studies often have very stringent inclusion and exclusion criteria which means that the patient population accepted into an RCT is often more homogeneous (similar to each other) than the pool of patients in the real world with that disease. For example, patients enrolled into an RCT may be limited to a certain age range, severity of disease, types of concurrent conditions/medications etc.
This is why RWD is so important – it is a way to look at the outcomes in a wider population of patients who have a given condition and receive a given treatment - without major inclusion/exclusion criteria being applied. This allows the researchers, manufacturers and regulators to see how well a wider pool of patients fare on a treatment once the drug has passed regulatory hurdles and it can be generally prescribed to patients in the normal way.
As well as seeing how well the treatment works, RWD studies can allow investigators to get more information on the side effects of treatments and how they affect patients’ quality of life more broadly. This more qualitative information is vital to improving the patient’s experience and it also feeds back into the drug development process to improve future treatments. Vann works with cancer researchers to provide patients access to surveys that will collect RWD to address important questions about cancer treatments and outcomes. The aim is for this collaboration between cancer patients, researchers and Vann to lead to tangible improvements in the quality of care.