Rare Disease Day is What KGI’s All About
This year Rare Disease Day at KGI is dedicated to raising awareness of orphan drugs for rare diseases-something which is greatly needed according to Dr. Ian Phillips, director of KGI's Center for Rare Disease Therapies.
"Rare Disease Day brings attention to the millions of people who suffer from a rare disease. Most of these diseases are genetic, chronic and debilitating. Some are fatal," Phillips said. "But, because they are rare, patients and their families are often left to fight the battle alone. Rare Disease Day at KGI, which emphasizes the Institute's relationship with companies producing new medicines and therapies, is an important opportunity to help raise awareness of the problem."
The February 28th event to be held on KGI's Claremont campus includes an internationally respected panel of biopharmaceutical industry leaders, including Dr. Surkirti Bagal, direct of medical affairs and clinical, Pfizer; James Banting, head of business development, Sigma Tau; Robert Baffi, executive vice president of technical operations, BioMarin; Chris Garabedian, chief executive officer, Sarepta, and Vinzenz Ploerer, president and CEO, Brace, among others. Dr. Tim Coté, KGI faculty member, principal of the Maryland-based Coté Orphan Consulting, and former director of the FDA's Office of Orphan Product Development will give a lunchtime talk, "Small is Beautiful: Orphan Drugs as Garage Science." In the afternoon, there will be screening of the documentary film "Fight to Live." Directed by two-time academy award winner Barbara Kopple, the film explores the complexities of the FDA's drug approval process and follows the lives of people suffering from debilitating, incurable diseases, including their fights to gain access to drugs that may prolong their lives.
Did you know?
- There are approximately 7,000 different types of rare diseases and disorders, with more being discovered each day
- 30 million people in the United States are living with rare diseases. This equates to 1 in 10 Americans or 10 percent of the U.S. population.
- It is estimated that 350 million people worldwide suffer from rare diseases.
- If all of the people with rare diseases lived in one country, it would be the world's 3rd most populous country.
- Approximately 50 percent of the people affected by rare diseases are children
- 30 percent of children with rare diseases will not live to see their 5th birthday
- According to the Kakkis EveryLife Foundation, 95 percent of rare diseases have not one single FDA-approved drug treatment
- During the first 25 years of the Orphan Drug Act (passed in 1983), only 326 new drugs were approved by the FDA and brought to market for all rare disease patients combined
- Approximately 50 percent of rare diseases do not have a disease specific foundation supporting or researching their rare disease
*Statistics courtesy of the Global Genes Project.