Real-World Data (RWD) and Evidence (RWE)
In healthcare, the first step of validating a new treatment against an existing one is a randomized controlled trial (or RCT) conducted on a preselected patient population. For the regulators, the data collected from such a trial is still a gold standard for bringing the treatment to the general population. Once a new treatment is approved, healthcare professionals may choose to prescribe it for patients who differ in some ways from participants in the clinical trials. The patients might have other health conditions besides the approved indication or disease and therefore, the outcome of the treatment may be different than the outcome of the RCT. This is where the real-world evidence (or RWE) comes into the picture.
RWE is the evidence collected outside the controlled environment of RCT. Such evidence may be relevant for greater efficacy of the treatment. It helps to understand patient behavior, the disease burden, treatment patterns, and product performance in settings and populations that are representative of everyday clinical practice.
RWE is based on real-world data (or RWD) which is collected into three broad bins:
1) Clinical – Cure rates in real life
2) Economic – Cost associated with medical resource utilization
3) Humanistic – Data on the health-related quality of life of the patient
The RWD can either be collected prospectively (in real time) or retrospectively (by reviewing past data). This data can be administrative data, medical charts or disease registries or healthcare databases, patient chart reviews, electronic health records, physician patient and public surveys, mobile health, wearable technologies, and post marketing observational studies.
Just like clinical trials, RWE has its advantages and limitations.
RWE is based on everyday clinical practice, and this can show how physicians prescribe a product and help patients to use it.
The RWE can be used to look at large and diverse populations, including patients often excluded from clinical trials giving a broader picture.
RWE can evaluate comparisons that may not have been made in a RCT.
RWE allows to collect data typically not gathered in a clinical trial, such as the cost of the disease burden or the impact of treatment on health care costs.
There is a potential for bias in RWE. It is important to ensure that RWE data sources are good representation of the population considering the diversity of the real world.
Data available in the real world may not be complete or of high quality to answer specific questions. Most healthcare information systems are not built to generate computable data that can be used for evidence generation.
Ideally, clinical trials and RWE could be used together to provide a more comprehensive understanding of the treatment or disease. It is an approach that can provide insights into healthcare delivery. But there is a need to have a regulatory framework to generate RWD that can be effectively used to have strong RWE. Such evidence can then be used to help improve the efficiency of clinical research.