There is a lot of talk about real-world evidence (RWE) and its growing importance, but as this commonly-used term has no official definition, what's all the fuss about? How is RWE used now and what will its role be in the future?
The term real-world evidence is commonly used to refer to non-randomised clinical trial (RCT) data generated in a healthcare setting. This is sometimes extended to cover patient socio-economic data and environmental data. But however you chose to define it, RWE is seen to offer significant advantages over randomised controlled trials (RCTs), which despite being recognised as the gold standard for evidence-based medicine, have well documented limitations. In the age of value-driven healthcare resource management, RCTs, designed for regulatory approval, cannot deliver all the evidence needed to support payer coverage decisions.
So how can RWE help?
RWE complements RCT data by providing
- Measures of effectiveness instead of efficacy
- Larger and more representative sample sizes over longer periods of time
- Recording of long-term patient behaviours like adherence.
Importantly, in today's resource-limited healthcare systems, it also allows us to measure real-world costs through the analysis of care delivery and resource utilisation.
The rise of RWE
But RWE is not new. From a regulatory perspective it is already in routine use in the EU, particularly for products already approved where it is primarily used for safety monitoring and drug utilisation. The tipping point right now is that RWE is increasingly being seen as a viable data source, now that the information capabilities of healthcare systems mean that this information can be captured as part of routine monitoring. Electronic Health Records (EHR), national repositories of curated claims data, clinical registries, wearable tech and apps are all facilitating the ability to collect real-world data.
As our ability to capture this data becomes easier there is also increasing interest in the use of RWE by payers and decision-makers. The rise of health technology assessment (HTA) globally puts significant emphasis on health economic and outcome research (HEOR) analysis and RWE provides critical inputs into this. In addition, methods employed to establish value-based healthcare benefit design will demand more RWE. Payers will have to evaluate RWE outcomes and establish pricing and reimbursement criteria. Identifying the more efficient disease management pathways will help to alleviate the resource constraint to healthcare systems worldwide. In the US, manufacturers and payers are increasingly partnered together in value-based contracts and outcomes based on RWE are often the centrepiece in these types of arrangements.
Making the most of RWE
However, as data capture becomes easier the challenge remains of how to take full advantage of RWE. RWE covers many different data sources, many of which are growing at an extremely fast rate and this level of data generation needs technical expertise to be collated, analysed and interpreted. Data quality and data integrations can be extremely demanding and shouldn't be underestimated.
But as more and more data is being generated, it is in effect 'owned' by different sources. Therefore, at the heart of this is the need for collaboration. In the future, policymakers will need to have a compliance framework that encourages cross-functional collaboration between multiple stakeholders (including researchers, payers, patients, manufacturers and providers) to help to improve both the quality and quantity of RWE utilisation in healthcare.
There are already collaborative initiatives to support the development and utilisation of RWEs in the US, including the US meaningful use of EHR requirements and the CMS open Medicare data but uptake outside the US has been slower. A recent publication in BMC Heal Serv Res, 'Is real-world evidence influencing practice? A systematic review of CPRD research in NICE guidance', concluded that despite a slow uptake of RWE in clinical and therapeutic guidelines, there is an increasing trend in the use of healthcare system data to inform clinical practice. To meet this demand organisations need to work together to enable or improve data access, undertake translational and relevant research and establish sources of reliable evidence.
The increasing availability of RWE, combined with a growing desire from regulatory and HTA bodies, is leading to RWE being incorporated more often into the necessary data packages for reimbursement. However, obstacles such as the quality of data and the integration of disparate sources of data will provide a challenge in the near future. Ultimately, sporadic examples of RWE making the difference for reimbursement will become the norm.
Case studies: examples of where RWE allowed reimbursement
- NICE assessment of total hip replacement and resurfacing arthroplasty for end-stage arthritis (TA304) is based on data collected through UK National Joint Registry, an RWE data source.
- RWE data presented at ECCO this year showed that switching to the biosimilar Remsima (infliximab) from the originator had no negative effect on safety and efficacy in 10 real-world studies.
- Zaltrap re-submission to the Scottish Medicines Consortium (SMC) included RWE (pooled data from two open-label, single-arm studies) to demonstrate prolonged safety and QoL benefit.
Vicky O'Connor is managing director and Janek Hendrich is an associate management consultant at WG.