It is generally supported that outcomes of patients, personnel or organisations are improved through evidence-based clinical practice. Over the years, the process for developing best practice has moved from being primarily expert knowledge-based to being primarily evidence-based, however, there is still concern regarding the sources of evidence.
But there is still heavy reliance on randomised controlled trials (RCTs) for generating evidence for clinical guidelines as, according to many hierarchies of evidence, they are considered to be the ‘gold standard’. However, there are several disadvantages which make evidence from RCTs appear less practical in terms of application to patient care, a key one being the fact that RCTs are generally conducted under controlled conditions on a small number of patients over a fairly short period of time. Even if treatment proves effective in a trial, this does not mean the same effect will translate into the general population as patients in the ‘real world’ can often be more diverse in terms of age, ethnicity and gender, and tend to have more co-morbidities which may have an impact on the efficacy of a treatment. Therefore there is a limit to the type of evidence that can be generated from RCTs to address key clinical questions that clinicians face on a daily basis. Additionally, the cost of running clinical trials and the increased interest in obtaining return on investment in healthcare make matters more complicated. An emerging solution is the use of routinely collected data or clinical databases, research outputs of which are often collectively called real-world evidence (RWE).
Such data presents several advantages: it helps to strengthen current understanding of healthcare delivery and the outcomes of patients; it greatly increases the potential of generating new knowledge as researchers can work to answer important clinical questions (which may have not been possible otherwise), and it can support the development of evidence-based personalised medicines through the linking of electronic medical records (EMRs) to genomic data sets. EMRs may also enable patients to take a more empowered role in their care through accessing their records.
Real-world evidence could help by improving the quantity and quality of evidence used in guidelines
When considering the use of RWE as sources of evidence towards best practice though, we need to be mindful that RCTs remain the gold standard. Entirely disrupting the status quo of the process of developing guidelines will still take a few decades, especially in developing countries, but with such unmet need and unequal access to care, we should not allow (what is thought to be) ‘best’ to become an enemy of ‘good enough’. RWE could indeed help with the dissemination of key information by bridging the knowledge gap for clinicians and by improving the quantity and quality of evidence used in guidelines. This translational process is already happening. In a recent systematic review we noted that RWE from a UK electronic medical record database has been used inconsistently, but increasingly, in the last decade to inform guidelines published in the country. The increased uptake in recent years shows that this area of R&D in healthcare is changing and we are now working during a phase in this transition.
To fully capitalise on the potential value of using RWE, researchers need to ensure they undertake research of translational value to the healthcare community. Organisations that develop guidelines should also work to identify RWE sources that will give a more realistic view of how an intervention works in actual healthcare settings. Safe havens conducting data linkage should expand linkages to various data sources that provide an immense opportunity to not only get a fuller picture of a person’s history, but also investigate the interactions and associations between different treatments/diseases in different settings and possibly develop predictors of health outcomes.
It will take a structured and collaborative multidisciplinary approach to look to the future and overcome the barriers to making best use of RWE, namely: capacity and cost, skills and resource, and technology and access, all while maintaining public trust. Cross-sector collaboration will be the catalyst to enable global synergies and the positive shift in the reality versus expectations scale.
