Unbiased Analysis of Today's Healthcare Issues
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…
How do you evaluate treatment efficacy and safety outside of the clinical trial setting? This is not just a question of academic interest. In last week’s JAMA, Rubin 2021 writes about some of the challenges of evaluating COVID-19 vaccines outside of a clinical trial setting. In an interesting, two-day workshop last week, the Duke Margolis…
Is your observational research study following best practices? Is your methodology transparent? To help answer these questions, the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) Network created the STROBE guidelines. The STROBE guidelines–an acronym for The Strengthening the Reporting of Observational Studies in Epidemiology–aim to improve the transparency of the methods behind observational…
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…
Is treatment A better than treatment B? This is a standard question in health economics. Typically, the answer is determined by a clinical trial. Treatment efficacy (as measured in a trial), however, may not match treatment effectiveness (as measured in the real world). For instance, patient adherence to treatment may be worse, if the treatment…
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,…
That is the title of my latest article in Pharmaceutical Market Europe. An excerpt is below. Randomised controlled trials (RCTs) are regarded as the gold standard of scientific evidence, and for good reason. By randomizsing a treatment across study arms, RCTs eliminate patient-treamtent selection bias, resulting in reliable causal inference. In contrast, in the real…