Funding challenges in Rare Disease Research: How financial support can transform the Rare Disease Landscape

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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.

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Navigating Clinical Trials: A Guide for Rare Disease Patients

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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…

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Sometimes It’s a Zebra

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Sometimes It’s a Zebra

In medical school many doctors are taught the old saying “when you hear hoof beats, think horses, not zebras”, which means that doctors should generally consider the most likely possibility first when thinking of a diagnosis.

But what do you do when it is a zebra?

What makes a disease rare? In the United States, the Orphan Drug Act defines a disease as rare if it affects fewer than 200,000 Americans or less than one in 2,000 people. A disease is considered ultra-rare if it affects less than one in 50,000 people.

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