We sat down with Raffaele Di Marzo, PV Leadership and Advice at Arithmos.
Graduated in Medicine and Surgery, Raffaele is a Pharmaceutical Physician with more than 30 years of experience.
He has acquired, in national and multinational pharmaceutical companies, a robust knowledge of Pharmaceutical Medicine in its different fields of application (Clinical and Epidemiology, Medical Affairs, Pharmacovigilance) as well as supporting the strategic company functions for the maintenance of the business (Regulatory Affairs, Marketing, Market Access and Commercial).
We asked Raffaele Di Marzo to talk about Real World Data (RWD) and Real World Evidence (RWE).
What are the main differences between RWD and RWE?
There is currently no agreed definition of RWD and RWE and Real World Evidence – Where Are We Now? (N Engl J Med. 2022;386(18):1680-2) emphasises that these terms are being used inconsistently and sometimes interchangeably.
Amongst existing definitions, the Real-World Data for Regulatory Decision Making: Challenges and Possible Solutions for Europe (Clin Pharmacol Ther. 2019;106(1):36-9) defines RWD as “routinely collected data relating to a patient’s health status or the delivery of health care from a variety of sources other than traditional clinical trials”, and RWE as “the information derived from the analysis of RWD”.
RWD is commonly understood as observational data which are most often secondary data from various origins (e.g., electronic healthcare records, claims data, registries) but may also originate from primary data collection (e.g., data collected in an observational study, data collected with digital wearable devices or patients’ or physicians’ surveys) or a combination of both.
The term RWE is commonly used as the result of the analysis of RWD using well-validated and appropriate methods that may combine design elements of observational studies and clinical trials.
A current domain of research is the assessment of whether RWE studies can provide the same results as RCTs performed for the same research question.
Such research does not aim to show that RWE can replace RCTs but it may give confidence in the validity of RWE studies based on healthcare data if they can consistently match the results of published trials and even predict the results of ongoing trials.
RWE is likely to be preferred to RCTs when studying a highly promising treatment for a disease with no other available treatments, where ethical considerations may preclude randomising patients to placebo, particularly if the disease is likely to result in severely compromised quality of life or mortality.
In these cases, RWE could support the evidence and reliability on the safety and effectiveness of the therapy against the typical disease progression observed in the absence of treatment.
RWE might be used to support regulatory decision-making (i.e. drugs approval and price and reimbursement pathway).
The use of RWE in this context depends on several factors and several publications describe components of RWE that determine whether it is relevant and acceptable to focus main elements and recommendations in three domains:
- Data sources
- Study design elements
- Data quality
All the elements cited above represent fundamental principles of pharmacoepidemiology.
While RWD refers to the availability of a large amount of data from different sources, RWE relies on the application of epidemiological methods to analyse such data.
Given the importance taken by RWD and RWE, especially in the context of public health systems, RWD and RWE have become important components of the scientific information supporting medicines evaluation, regulatory decision-making, health technology assessment and public health in general.
How can the Arithmos team support Life Sciences companies?
Arithmos, a consulting company that combines scientific and technology expertise for Life Sciences industry, delivers reliable pharmacovigilance solutions and services in full compliance with regulatory and quality standards, providing added value to accomplish the critical requirements of RWD/RWE.
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