The term real-world evidence (RWE) commonly applies to data generated in non-randomized clinical trials (RCTs) in a healthcare setting, which sometimes also covers patient socio-economic data and environmental data. Irrespective of the definition, RWE offers significant advantages over RCTs that have well documented limitations, despite being recognized as the gold standard for evidence-based medicine. Today, in the era of value-driven healthcare resource management, RCTs designed for regulatory approval cannot deliver all the evidence needed to support payer coverage decisions. Here, RWE plays a role of complementing RCT data in a way of measuring effectiveness, providing larger and more representative sample sizes over longer periods of time, and recording long-term patient behaviors like adherence. (1)

Nonetheless, RWE has been in routine use for a while in the EU, particularly for products already approved for safety monitoring and drug utilization. Electronic health records (EHR)/ medical records (EMR), national repositories of curated claims data, clinical registries, wearable technologies and apps- all facilitate the ability to collect real-world data. Easier data capture contributes to the increasing interest in the use of RWE by payers and decision-makers. Increase in health technology assessment (HTA) applications worldwide significantly highlights the health economic and outcome research (HEOR) analysis, with critical inputs from RWE. Moreover, methods for establishing value-based healthcare benefit design will demand more RWE. Also, payers need to evaluate RWE outcomes in order to establish pricing and reimbursement criteria. (1)

The fundamental aspect of healthcare involves the core ability to access data, describing the current standard of care, along with gaps and deficiencies in the care model, and social or patient-reported data, i.e. patient reported outcomes (PRO). This is where real-world data (RWD) originates – countless sources linked together to provide a view of a patient’s health history that can be acted upon using insights from advanced analytics. (2) However, as numerous data sources are growing at an extremely fast rate, it’s a challenge to collate, analyze and interpret this level of data generation. Data quality and data integrations can be extremely demanding and shouldn’t be underestimated.

Collaborative initiatives are in place to sustain the development and utilization of RWEs in the West. Although, the uptake of RWE in clinical and therapeutic guidelines is slow, there is an increasing trend in the use of healthcare system data for informed clinical practice. To meet this demand, companies need to work together to enable or improve data access, undertake translational and relevant research and establish sources of reliable evidence. Furthermore, RWE is now becoming the foundation for new pricing strategies that can more explicitly provide therapeutic value to patients and health outcomes benefits to health systems and risk-bearers. RWE also provides a new basis for establishing business-to-business relationships or for having leadership conversations between large risk-bearing health systems and increasingly analytic-centric health insurers and payers. (1,2)

It’s believed that, RCT data streamlining can be easier if RWE is appropriately adopted by sponsors of new drugs and devices and regulatory agencies. RWE generates new forms of evidence that many decision makers are thinking to consider alongside traditional RCT evidence. Certainly, prospective trials analyzing RWD together with classic RCT data are complementing to RCTs. (2)

As sources of RWE become available to a greater extent, RWE is being incorporated more often into the necessary data packages for reimbursement, along with a growing desire from regulatory and HTA bodies. However, hindrances in terms of data quality and the integration of disparate sources of data will provide a challenge in the near future. Furthermore, pharmaceutical companies must keep up with the pace of advances in RWE through their own investment and partnerships in RWD sources and respective analytics services. They should be reshaping their necessary skill sets to best address the value, market, and financial opportunities that RWE will offer them.

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  1. The reality of real-world evidence. PMLive. January 2017.
  2. Elton J. The reality behind real-world data and real-world evidence. August 2015.

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