Real World Evidence and Data: From Disease management to Evaluating Drug Safety and Effectiveness

Dr. Penelope Ward, Visiting Professor in Pharmaceutical Medicine at Kings College London, David Morgan, Statistical Consultant to the pharmaceutical industry, Dr. Alvaro Quintana, Scientific Expert in Anti-Infectives Hospital BU Emerging Markets in Pfizer, with Dr. Pravin Chopra, Chair, Scientific Advisory Committee, IFAPP Academy, tackled salient concepts and methodology in relation to the generation of Real‐world evidence (RWE) and Real-world Data (RWD), and the essential attributes and pros-and-cons of available modalities.

While the clinical evidence generated through randomized controlled trials (RCTs) is traditionally considered the highest level of evidence to determine efficacy and safety to obtain regulatory approvals for new therapeutic options. These are often inadequate with providing guidance to treating physicians and caregivers to choose optimal treatment for their patients. Increasingly, and across therapeutic areas, implementation and analyses of RWE and RWD are being deployed to answer clinical and pertinent policy and guidelines queries that cannot be comprehensively or directly addressed by data generated from RCTs. In parallel, data from retrospective or prospective observational or "minimal interventional" studies – vis-à-vis pre-defined disease assessments or treatment -- and large-scale registries and pragmatic observational study models provide insights beyond those addressed by traditional RCTs, to inform both clinical development and treatment guidelines.

Notwithstanding this advancement in understanding, several challenges remain and complicate meaningful interpretation and extrapolation of data generated with RWE studies. This includes data-source quality and credibility, elements of study design, compliant clinical data access, ethical concerns around data privacy, the feasibility of relevant and appropriate comparative cohorts, and a potential limitation with generalizability. While data gleaned from clinical health records, disease state assessments, and management registries, and other RWE resources offer valuable perspectives on patient care and treatments, the intrinsic limitations with the obtained data prompt a need for caution and temperance to preempt misleading conclusions and potentially harmful outcomes. The key is successfully balancing appropriate study designs and tools with guidance, even while ensuring operational feasibility.

Additionally, the audience was presented with historical perspectives on RWE and RWD, and the possible implications on healthcare and public policy decision-making, the principles and utility of RWE in understanding effectiveness, safety, and diseases management, the relative value of RWE studies, and pragmatic trials design options vis-à-vis RCTs, in different therapeutic areas and settings, and, importantly, the evolving regulatory environment with RWE/RWD acceptability.