Real-World Evidence – Healthcare Economist

A Retrospective Claims Analysis of the Rate of Complications in Patients Undergoing Treatment for Paroxysmal Nocturnal Hemoglobinuria

2024-11-05Jason Shafrin

That is the title of my recent paper with co-authors Denise Clayton, Glorian Yen, Lincy Geevarghese, Yulin Shi Anem Waheed. The abstract is below. IntroductionParoxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease associated with complications that increase morbidity, such as thrombosis and chronic kidney disease. Limited data exist regarding complications among treated patients outside…

Use of real-world data for measuring treatment effectiveness for target populations

2024-10-25Jason Shafrin

Randomized controlled trials are the gold standard for evaluating treatment efficacy, but effectiveness in the real-world may vary. One reason for this is that clinical trials often have stricter inclusion criteria than is the case for the target treated population. Policymakers, payers, and clinicians may wonder how well the results from the narrower clinical trial…

HARmonized Protocol Template to Enhance Reproducibility (HARPER)

2022-11-16Jason ShafrinLeave a comment

Clinical trials have detailed study protocols and are registered on ClinicalTrials.gov. What level of details are needed for real-world data (RWD) analyses that aim to estimate treatment effects? In Europe, the European Medicines Agency (EMA) requires registration of many study protocols using a template for observational post-authorization safety studies (PASS) conducted by marketing authorization holders.…

What is DARWIN EU?

2022-11-14Jason ShafrinLeave a comment

Real-world data is becoming increasingly important as the amount of data generation increases as does the speed of data processing. In the US, the 21st Century Cures Act was enacted in December 2016 and mandated that the Food and Drug Administration (FDA) create a framework for evaluating the use of real-world data help support the…

Why use real-world evidence for clinical trials?

2019-09-15Jason Shafrin2 Comments

Clinical trials are the hallmark of the drug development process. In most cases, life sciences firms are required to conduct randomized controlled trials (RCTs) to examine their treatment’s safety and efficacy relative to standard of care. While RCTs do a great job at measuring causal inference, there as a number of limitations. First of all,…

FDA sets standards for Real-World Evidence

2018-12-30Jason ShafrinLeave a comment

The 21st Century Cures Act, enacted December 2016, mandated that the FDA consider how to use real-world evidence (RWE) to bring innovative treatments to market faster. To meet this statutory requirement, the FDA has provided guidance outlining its policies for using RWE to support the approval of a new drug indication. What is real-world evidence?…

Advancing the Discussion on Real-World Evidence

2017-11-12Jason Shafrin1 Comment

With the FDA’s introduction of new guidelines surrounding the use of real-world evidence (RWE) in medical device regulatory decisions, FDA Commissioner Scott Gottlieb advances the argument for the utility of RWE. In fact, the FDA is currently considering the role of RWE in evaluating pharmaceutical treatments. Despite much debate over what part RWE should play in…