Expert opinion on Real-World Evidence (RWE) in drug development and usage

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Fundings: There were no institutional or private fundings for this article.
Conflict of interests: is the Chief Scientific Officer of EDRA-LSWR Publishing Company and of Inpeco SA Total Lab Automation Company and he is the VP for Regulatory Strategy and Market Access Innovation of VeraSci, USA. In the last year he has provided scientific consultation to AbbVie, USA; BCG, Switzerland; Boehringer-Ingelheim, Germany; Compass Pathways, UK; Johnson & Johnson, USA; Takeda, USA; Vifor, Switzerland.
Authors’ contributions: The authors contributed equally to the realization of the manuscript.
Availability of data and materials: The data underlying this manuscript are available in the article.
Ethical approval: N/A.

Real-World Data (RWD) refers to data on patients’ health status and the provision of health services that are routinely collected from various sources during routine clinical practice. The scientific evidence on the potential benefits and risks of a drug derived from the analysis of RWD, and, therefore, related to the use of drugs in the Real-World setting, is called Real-World Evidence (RWE) (1). Randomized clinical trials (RCTs) are the gold standard of clinical research to assess the efficacy and safety of a drug treatment, but they have a number of important limitations that make their results difficult to generalize to the entire populations of patients treated in everyday clinical practice: this is why RWE is becoming increasingly valuable in clinical and regulatory practice (2). RWD are useful in the pre-marketing phases of drug development, providing information on disease burden and unmet clinical needs, as well as in the post-marketing phases, where scientific evidence can be generated on drug uptake, prescriptive appropriateness, comparative effectiveness research, short- and long-term drug safety and implementation and impact of risk minimisation measures (3). The marketing of new drugs and innovative therapies, such as advanced therapy medicinal products, mutation-driven cancer drugs and digital therapeutics, requires both innovative pricing and reimbursement processes and a post-marketing re-evaluation of the benefit-risk profile of these drugs/therapeutic options through RWE generation (4). On the other hand, the increase in health expenditure linked to the ageing population, together with the limited availability of economic re-sources, requires an integrated assessment of health care costs, especially in the setting of chronicity, for which the development and monitoring of outcome and process indicators through RWD analysis is essential (5). The Italian Society of Pharmacology has developed the present expert opinion document on RWE as a technical-scientific reference framework for institutions (Italian Medicines Agency, Regions and State-Regions Conference), scientific community, health care professionals, and pharmaceutical companies, with the aim of examining the role that RWE can play in five thematic areas: A. Chronicity and Multimorbidity management; B. Regulatory; C. Governance of care processes (overcoming silos budget); D. Population studies; E. Health emergencies.