It has been said that the definition of insanity is doing the same thing over and over again, yet expecting different results. How does this relate to real-world data (RWD) and real-world evidence (RWE)? Simply put RWD and RWE represent a new approach to measuring effects of a pharmaceutical and are vital in moving away from repetitive pricing negotiations to talking about value creation and outcomes. Unfortunately, this is where the simplicity ends and the complexity begins.

Before we look forward we need to take a trip back to 1946 and the first randomized controlled trial conducted by Sir Bradford Hill.1 Hill and colleagues set out to study the effects of streptomycin on tuberculosis based on a meticulous set of methods involving admission criteria, randomization, documentation, and analysis, thus forming the foundation of the modern clinical trial design. While innovative and an essential milestone in the study of disease treatment, this approach creates a study environment resembling an aseptic operating suite rather than the messy natural environment of the real world. This is not to say that clinical trials don’t have their place—clearly they do—but we need to balance this controlled environment with that of the actual patient experience.

Key to understanding the usefulness of real-world evidence is an appreciation of its potential for complementing the knowledge gained through traditional clinical trials. With traditional studies there are well-known limitations that make it difficult to generalize findings to larger, more inclusive populations of patients, providers, and health care delivery systems or settings that reflect actual use in practice.2 The recently finalized FDA recommendation on RWE to support regulatory decision-making for medical devices noted that, under the right conditions, data derived from real-world sources can be used to support regulatory decisions. Essentially, RWD and the associated RWE may constitute valid scientific evidence depending on the characteristics of the data.3

Beyond regulatory approvals, insights derived from RWE, when used appropriately, may help to accelerate healthcare innovation and transform patient care.4 Catalyzed by the 21st Century Cures Act, payers and pharmaceutical manufacturers both see opportunities in RWE, but its uptake has been slowed due to incomplete or inconsistent data. The continued evolution of the internet of things (IoT), a system of interconnected devices including wearables, can help solve this problem by increasing not only the quality but the quantity of data that can be used to support RWE generation. For instance, your home digital assistant could be used to perform a Patient Health Questionnaire 9 (PHQ9) to help assess treatment for major depressive disorder. If the assessment shows improvement, then value of that treatment has been validated. In addition to exploring opportunities to utilize IoT, manufacturers should engage payers in consensus-building forums to generate the energy necessary to advance RWE uptake across therapeutic areas.

There still remains much to be determined about RWE and its payoff. Manufacturers may see new opportunities from speed to market, new indication discovery, improved visibility for drug safety, and reduced development costs. Ultimately, RWE will be critical as the healthcare system transitions from cost containment to value generation.


    1. Streptomycin in Tuberculosis Trials Committee. Streptomycin treatment of pulmonary tuberculosis: a medical research council investigation. Br Med J. 1948;2:769-782.