Real-world evidence (RWE) has been used extensively for satisfying post-launch regulatory requirements, mainly about drug safety. Of late, it has been realized that RWE has a wide range of applications across various phases of a product lifecycle, starting from the pre- to post-launch phases. These applications aim to address the particular needs of different healthcare stakeholders, including payers and healthcare professionals (HCPs). (1)

RWE has now found widespread acceptance among different healthcare stakeholders, including regulators, Health Technology Assessment (HTA) bodies, payers, and HCPs. The usefulness of real-world data (RWD) and the analytical approaches to gather RWE have resulted in the appreciation of the value RWE brings in addressing the needs of these stakeholders. This expansion is accompanied by the digitization of healthcare and innovation in technology and analytics. (1)

Recent digital and analytical advancements are allowing the application of RWE in new ways. For instance, with RWE, we can predict disease progression, patient outcomes and responses, or the risk of adverse events while increasing the efficiency of R&D investments and accelerating market access.(2) RWE can also facilitate the interpretation of the impact of patient characteristics and behaviors on health outcomes.

Section 3022 of the 21st Century Cures Act by the USFDA provides guidance on evaluating the applications of RWD and RWE in the approval of new indications or post-approval study requirements.(3) As a result, the USFDA has the strategic framework to enhance the use of RWD/ RWE in regulatory decision-making.(4) In a gist, this guidance suggests including the data, studies, or analyses of RWD, which adhere to the competent and reliable scientific evidence (CARSE) standards, in the promotional communication.(3, 4)

Many trends, such as rising costs and competition, scientific improvements, tech-savvy stakeholders, advanced regulatory shifts, and interoperability of data and technologies, are driving healthcare stakeholders to emphasize value and patient outcomes. Payers are progressively shifting to outcomes-based contracts; providers are aiming to gain privileged status with them; and patients want more ownership of their outcomes. In this changing environment, RWE insights are becoming critical for the right treatment to the right patient at the right time. They’re also crucial for quantifying outcomes and representing the value of interventions. Given the substantial interruption to RCTs and the need to quickly understand the patient burden and potential therapies for COVID-19, RWE is gaining more attention than ever.(2)

Early adopters are looking to invest in building advanced RWE analytical competencies. Communicating the value of RWE to the stakeholders is essential and it can be done by focusing on eight main elements, i.e. strategy and vision (by defining the products and value-chain factors that would benefit from advanced RWE analytics); value positioning (by targeting an urgent business question and demonstrating the RWE value in addressing it), organizational models and processes (by setting up models to oversee RWE initiative strategies, skillsets, and governance); skills (by building expertise with those who understand how RWE operates and delivers value); culture (by considering RWE crucial in strategic discussions and development programs and adopting novel approaches, and accepting  uncertainty in trying new things); partnerships (by collaborating with analytics companies, academic associations, and innovative start-ups); data (by collaborating with data providers helps to gain access to data and develop data to solve business-critical queries); and tools and environments (by creating platforms for conducting basic experiments with cases and delivery models).(2) Research shows that companies need not excel at all eight, but should aim to build a leading position in at least a handful.

Pharmaceutical companies are using RWE to inform strategic and operational decisions, such as clinical trial design, patient enrollment, financial estimations, or commercial resource allocation.(1) A recent example of using RWE in promotional materials is Boehringer Ingelheim’s GioTag research study.(5) GioTag was a real-world retrospective, observational, non-blinded study assessing the impact of treatment with afatinib followed by osimertinib in EGFR-mutated non-small cell lung cancer (NSCLC) patients with T790 mutations. Patients with NSCLC having non-resistant EGFR mutations, as confirmed on an FDA-approved test, are prescribed afatinib as the first-line treatment. Study researchers used electronic health record (EHR) data to show that overall survival among patients who received this therapy sequence was almost four years, a finding not reported in the clinical study. These findings have been widely publicized globally and circulated through various abstracts and peer-reviewed manuscripts. For instance, the results were depicted on the display boards at the European Society for Medical Oncology 2019 conference in Barcelona, Spain. This could be one of the first instances of the promotional use of RWE at a medical conference, and it could translate into a trend as the frequency of RWE-based studies increases.(6)

Whether or not RWE meets the standards for “substantial evidence” of effectiveness is still debatable. Researchers are developing methods to overcome certain limitations, such as confounding, data quality, and bias. However, pharmaceutical companies can speed up their shift from product-focused to patient-focused organizations by optimizing the RWE with advanced analytics and work on communicating the value of RWE analytics for all involved stakeholders. Now is the time to set the sights on the next possibility of evidence generation.(5, 6)

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  1. Gores M, Patel M. RWE: From “nice to have” to “must have”. IQVIA 2018. Available at:
  2. Champagne D, et al. Creating value from next-generation real-world evidence. McKinsey & Company. July 2020. Available at:
  3. 21st Century Cures Act. Public Law 114–255—DEC. 13, 2016. Available at:
  4. Framework for FDA’s Real-World Evidence Program. December 2018. Available at:
  5. Hochmair MJ, Morabito A, Hao D, et al. Sequential afatinib and osimertinib in patients with EGFR mutation-positive non-small-cell lung cancer: final analysis of the GioTag study. Future Oncol 2020; 16(34):2799-2808.
  6. Kish J. Effectively communicating real-world data and real-world evidence: Different stakes for different stakeholders. 2020. Available at:

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