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Abstract

The vast majority of vaccines are prophylactic in nature. As a result, the demonstration of their efficacy paradoxically requires the infectious disease to occur among non-diseased study participants randomized between investigational vaccine and appropriate control groups. The statistics of vaccine efficacy (VE) calculation are nearly entirely and solely based on the number of observed incident disease cases during follow-up. For certain diseases, the sample sizes needed to achieve the required number of observations may reach 5,000 subjects or many more, requiring vaccine mega-trials to be conducted. In this article, the author provides an overview of mega vaccine trials conducted over the last 20 years.

Introduction

Prophylactic vaccines generally are intended for healthy or medically stable populations. Administration to hundreds of thousands or even millions of people in a few yea…

Simplified schematic illustration showing the proposed mechanism of action of FcRn inhibitors. The left side illustrates how IgG molecules enter cells and interact with the neonatal Fc receptors (FcRn), and the right side depicts the impact of FcRn inhibitors on this process. Image by Benff, licensed under [CC BY-SA 4.0], via Wikimedia Commons. Modified from D.D. Patel, J.B. Bussel’s work.

The Fc receptor (FcRn) inhibitor market, led by promising candidates such as efgartigimod, promises to reshape the autoimmune disease treatment landscape. Sales projections for the market top $10 billion, as Driehaus Capital Management estimates. The addressable U.S. patient base spans more than 228,500 individuals across various conditions including myasthenia gravis, warm autoimmune hemolytic anemia (wAIHA) and idiopathic thrombocytopenia purpura (ITP), and more. As the estimated annual treatment cost per patient for targeted…

biopharma research

Vaccine mega-trials: Rare behemoths in the vaccine trial landscape

The multi-billion dollar promise of efgartigimod and the broader FcRn inhibitor market