For those looking to learn more about real-world evidence (RWE), there is a wealth of available resources, but it can be difficult to know where to start. Here, we’ve rounded up some key readings from around the web to help anyone, regardless of scientific background, become well-versed in RWE. We start with causality, the foundational underlying concept and goal of running real-world data analyses, move through the impact RWE has had to date, on to the FDA’s demonstrated interest in its use for regulators and its potential for the future.
The Book of Why: The New Science of Cause and Effect
Judea Pearl & Dana Mackenzie
As an axiom, "correlation is not causation" has evolved into a decades-long taboo in science and academia of nearly any talk of causation. In The Book of Why: The New Science of Cause and Effect, Judea Pearl and Dana Mackenzie usher in a new era for science in which advanced modeling tools enable data analyses that answer causal questions.
Real-world evidence: From activity to impact in healthcare decision making
McKinsey & Company
As health care continues to focus on outcomes and value, real-world evidence can facilitate better decision-making by providers, biopharma, and payers. In this report, McKinsey explores the benefits, risks, challenges, and potential applications of RWE to fit this role, concluding with a goal to create organizational cultures in which RWE drives innovation in health care.
Second Annual Real-World Evidence Benchmarking Survey:
Mission Critical: Biopharma companies are accelerating real-world evidence adoption, investment and application
Deloitte’s Second Annual RWE Benchmarking Survey indicates a continued and increased adoption of real-world evidence by global life sciences companies. Ninety percent of respondents report investing in RWE, and using it to support the development of value-based contracting agreements, regulatory submissions, and improved trial design.
How Real-World Evidence Is Playing Out In The Real World
Informa Pharma Intelligence
Real-world evidence is gaining traction throughout the health care sphere, but how effective has it been at improving outcomes and lowering costs? In this recorded webinar, Informa editors and biopharma leaders discuss case studies and applications of RWE to evaluate its utility for biopharma.
Framework for FDA’s Real-World Evidence Program
U.S. Food and Drug Administration
The Framework for FDA’s Real-World Evidence Program presents the agency’s three-year strategy to evaluate how real-world data can be used in regulatory decision-making. The document marks a new phase in fulfilling the requirements of the 21st Century Cures Act to accelerate medical product development and innovation, giving patients faster access to new treatments.
FDA Issues Real-World Evidence Framework for Drugs and Biologics
National Law Review
FDA’s Framework for Real-World Evidence Program provides guidelines for the agency’s use of RWE in drug approvals to date, and in the future. In this article, the National Law Review walks through the significance of the announcement and shines light on why FDA Commissioner Scott Gottlieb calls RWE a “top strategic priority.”
When and How can Real World Data Analyses Substitute for Randomized Controlled Trials
Clinical Pharmacology & Therapeutics
Real-world evidence is known as a faster and cheaper alternative to randomized controlled trials, but many remain unsure of the validity of RWE-driven insights for regulatory decision-making. This study, co-authored by Jessica Franklin, Ph.D., and Sebastian Schneeweiss, M.D., Sc.D., explores when and how to use real-world data for regulatory-grade analyses.
Precision medicine: Opening the aperture
McKinsey & Company
Thanks to the health care advances brought about by real-world data and analytics, personalized medicine now allows health care professionals to deliver the right medicines to the right patients at the right times. Here, McKinsey & Company reports on the expanding capabilities of precision medicine.
Real-world evidence: Driving a new drug-development paradigm in oncology
McKinsey & Company
Cancer is soon to become the leading cause of death in the United States, and despite the development of innovative therapies, costs and other impediments slow the progress of scientific breakthroughs. Here, McKinsey offers case studies and examples of how real-world evidence is likely to lead to improved treatment for people with cancer.
Pfizer uses real-world data to score Ibrance breast cancer nod in males
For a variety of reasons, it’s difficult to conduct clinical trials for rare disease therapies. In the case of male breast cancer, which affects less than 3,000 men each year, Pfizer turned to real-world data analyses to expand the indication of breast cancer treatment Ibrance to male patients. The FDA approved the expansion, strengthening the case for using real-world evidence in regulatory decision-making.
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