RWD – Healthcare Economist

Treatment Patterns and Healthcare Resource Utilization of Patients With Paroxysmal Nocturnal Hemoglobinuria: A Retrospective Claims Data Analysis

2024-01-04Jason Shafrin

That is the title of my new paper published today in Clinical and Applied Thrombosis/Hemostasis with co-authors Denise Clayton, Glorian Yen, Soyon Lee, Lincy Geevarghese, Yulin Shi, Luyang He, Ying Shen, and Anem Waheed. The abstract is below: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic disorder commonly treated with complement inhibitors such as…

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…

FDA: Why we approved Prograf based on real-world data

2021-08-10Jason ShafrinLeave a comment

Recently, the Food and Drug Administration (FDA) approved Prograf (tacrolimus) in combination with other immunosuppressant drugs for the new indication of preventing organ rejection for patients who have received a lung transplant. While the FDA approving a new indication isn’t typically news, this is a noteworthy event because (i) it marks the first approval of…

Guidance on use of real-world evidence for devices and diagnostics

2021-05-11Jason ShafrinLeave a comment

Real-world evidence is often used to assess the safety of devices and diagnostics. For instance, the FDA’s , Postmarketing Surveillance Program includes the FDA Adverse Event Reporting System (FAERS); FDA may also require some MedTech companies to conduct Post-Approval Studies. However, as new legislation (e.g., 21st Century Cures Act) has passed and new technology has…

FDA’s use of real-world data

2020-10-21Jason ShafrinLeave a comment

The 21st Century Cures Act required the Food and Drug Administration (FDA) establish a program for evaluating the use of real-world data (RWD) to support the approval of new indications for drugs. Real-world data is typically data from either health insurance claims, electronic health records (EHRs), patient registries, or mobile devices. But how has FDA…