RWD can be categorised in four data types:

1. Patient claims data - including hospital, provider and prescription claims.
2. Patient/Medical registries - therapy-related information collected from prospective observational cohort studies of patients who have a particular disease and/or are receiving a particular treatment or intervention.
3. Electronic health records (EHR) / Electronic medical records (EMR) - a patient-level electronic record of health information collected from a single provider practice (EMR) or from multiple healthcare practices (EHR) .
4. Web/social data - relates to patient interaction on diseases, treatment experiences and side effects. Some sites and social networks allow patients to form communities, groups and discuss experiences (e.g. Medhelp, PatientsLikeMe).

Applications of RWD / RWE in Healthcare

The development of sophisticated new analytical capabilities, such as predictive modelling and machine learning, now allows companies to analyse and understand these healthcare data at scale.

The results of RWD analyses provide RWE that can be used to:

• Inform the recruitment action plan - RWD and site-specific data about existing and available patient populations can accelerate recruitment and engagement by enabling predictability in site enrollment and avoiding over-promising and under-delivering patients to the study.
• Inform clinical programme decisions - by providing insights into treatment effectiveness, risks and safety, as well as predicting success, time and cost.
• Set appropriate pricing - RWE is central to healthcare technology assessments (HTA) by which buyers judge if a new drug or intervention is cost-effective in their healthcare system.
• Compare alternative treatment - regimens, drugs and dosage effectiveness can be compared to determine the best treatment option for particular diseases for particular patient profiles.
• Evaluate product cost-efficiency - patient outcomes in real world settings can be analysed to generate, understand and demonstrate value of pharmaceutical and medical device innovations in a real-world environment. To gauge its impact on improving the quality of healthcare, RCTs need to be supplemented or followed up with RWE.
• Support and complement RCT data - by providing a more reliable picture as it helps understand diseases/conditions and their patterns, and by learning about drug/device effectiveness in the real world, as opposed to controlled clinical conditions (RCT focuses on demonstrating efficacy).
• Identify new markets - RWE can be used to identify patient populations that are underserved.
• Increase healthcare understanding - of real-life treatment pathways, treatment sequences, length of required treatment and the resources required plus specific disease processes.
• Facilitate product development - by using RWE in early drug discovery and development programmes to identify diseases which represent a significant burden in populations.

RWD/RWE Challenges

RWD/RWE aren’t necessarily a panacea, as there are issues related to consistency and validation in particular. To summarise some of the key challenges:

• There is no widely accepted scope for RWD across the pharmaceutical industry. In fact, there are many different types of research that fit under the banner of RW data studies and these rely on different study designs and different data sources, which can lead to inconsistencies in communication and interpretation.
• Data collected in a routine healthcare setting must be stringently curated, validated and standardized to enable the generation of robust RWE. Also, there are some methodological challenges that should be considered, such as a lack of randomisation, bias and issues around data quality.
• Validating data generated outside the healthcare environment can be difficult, as in the case of structured patient-generated data from PatientsLikeMe research networks.
• Considering the mechanism of data collection and experimental design, RWE studies generally cannot yield definitive causal inference because of the many other factors and variants that are involved.

RWE is therefore not yet seen as a substitute for RCT, unless there is a clear reason why the use of RCT is not feasible. In fact, in the UK RWE is only seen as supplementary to phase 2 and phase 3 data, or in cases of ongoing evidence collection such as situations where regulators are willing to use available evidence to make initial decisions but require more evidence as time progresses.

Future of RWD/RWE

The healthcare community is increasingly turning to RWE to inform their decisions, alongside evidence from RCTs. Recently, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have stated their ambition for greater use of RWE to support applications for new indications. The FDA recently released its draft guidance document entitled “Use of real-world evidence to support regulatory decision-making for medical devices.”, while the EMA published a paper on the “Use of real world data in development programmes”.