Taken together, rare diseases affect one in ten Americans
Biopharmaceutical research is entering an exciting new era with a growing understanding of the human genome. Scientific advances have given researchers new tools to explore rare diseases, which are often more complex than common diseases. According to the
Although a rare disease is defined as one that affects fewer than 200,000 people in the U.S., there are approximately 7,000 rare diseases and collectively they affect nearly 30 million people, or one in 10 Americans.
“Biopharmaceutical scientists are better equipped than ever before to bring new treatments – and hope – to patients battling one of the nearly 7,000 rare diseases. In order to capitalize on this scientific promise, we need to ensure that we have a policy and regulatory environment that helps foster this vital work,” said PhRMA President and CEO
The 452 medicines and vaccines in development for rare diseases – all in human clinical trials or under review by the
Rare diseases provide opportunities to study human physiology and biomedical science from unique perspectives, leading to insight into more common disorders. Advances in science and technology, such as personalized medicine, are creating new opportunities to improve and expand both research and development into rare diseases.