Nearly 7,000 rare diseases are known to affect roughly 30 million Americans. It is estimated that about 80 percent of rare diseases are genetic and about half of all rare diseases affect children.
Rare Disease Day, which is commemorated internationally on the last day in February, was established to raise awareness about rare diseases, the challenges encountered by those affected, the importance of research to develop diagnostics and treatments, and the impact of these diseases on patients' lives.
The focus of this year’s Rare Disease Day is to join together for better care. Caring for people living with a rare disease is multi-faceted – some patients have access to treatment, while others have no treatment options; some patients are fairly independent, while others require intensive physical assistance and equipment.
This year’s theme is intended to draw attention to these different aspects of rare disease care, and to highlight its importance for rare disease patients and their families.
In honor of Rare Disease Day 2014, the Office of Orphan Products Development (OOPD) will be engaging in the following activities:
- Launching web-based training modules for patients and industry on FDA-related rare disease topics in collaboration with the Center for Drug Evaluation and Research (CDER). The first of these modules will be available on February 28th in recognition of International Rare Disease Day.
- NIH’s Rare Disease Day Conference on February 28th at NIH Masur Auditorium (Building. 10), 8:30 am – 5:00 pm. For More Information: Click Here
- Issuing an FDA Voice Blog for Rare Disease Day to highlight efforts to encourage and accelerate the development of new therapies for pediatric rare diseases.
OOPD’s mission is to promote the development of products for rare diseases. For more information on OOPD programs, Click Here.