News, Events & Blogs
Why February 29th? The History Behind Rare Disease Day
More than 7,000 rare diseases affect an estimated 1 in 10 Americans, yet public awareness remains limited. Rare Disease Day, established in 2008 by EURORDIS, unites over 100 countries each year to promote research, health equity, and global advocacy. Through education, policy initiatives, and patient-driven storytelling, the movement continues to amplify voices that are too often unheard.
Economics and Politics in Rare Disease Treatments: The Rising Cost and Challenges
Orphan drug approvals are rising, offering hope for rare disease patients — but at an average cost of $370,000 per year. With policy shifts, Medicare loopholes, and falling research funding, the future of rare disease treatment hangs in the balance.
Funding challenges in Rare Disease Research: How financial support can transform the Rare Disease Landscape
Rare diseases may each affect only a small number of individuals, but collectively they impact up to 30 million Americans—highlighting a silent crisis in healthcare. Despite scientific progress, rare disease research remains chronically underfunded, resulting in delayed diagnoses, mismanagement, and a lack of effective treatments. This article explores the funding challenges that plague rare disease research and how strategic financial support—from government grants to public-private partnerships—can drive critical advancements. Transforming the rare disease landscape is not just about innovation; it's about giving millions a fair chance at timely diagnosis, effective care, and a better quality of life.
Navigating Clinical Trials: A Guide for Rare Disease Patients
A condition is defined as a rare disease if it affects fewer than 200,000 individuals. Approximately 7,000 conditions meet this definition. In 2016, the United States Food and Drug Administration awarded $23 million dollars towards a four year period that supported research for 21 different rare diseases. However, most rare diseases lack disease-modifying therapies or effective medications for several reasons…