What defines a rare disease?

A rare disease is a condition affecting a small percentage of the population. In the U.S., it impacts fewer than 200,000 people, while in Europe, it's defined as affecting fewer than 1 in 2,000 individuals. Criteria vary slightly by region but focus on prevalence.

How many rare diseases exist, and how many people are affected globally?

There are over 7,000 known rare diseases, with new ones continually being identified. Globally, an estimated 300-400 million people live with a rare disease, making it a significant public health concern despite individual conditions being uncommon.

What are the biggest challenges in diagnosing and treating rare diseases?

Challenges include delayed or misdiagnosis due to lack of awareness among healthcare professionals, difficulty in finding specialized experts, limited research funding, and small patient populations making clinical trials complex and drug development costly.

What is an 'orphan drug'?

An orphan drug is a pharmaceutical product developed to treat rare diseases. Governments often provide incentives like tax credits, reduced fees, and market exclusivity to encourage pharmaceutical companies to develop these drugs, as they might not otherwise be profitable.