“The Orphans of Medical Research: Finding the Cure for Rare Diseases”
By Rachel Dokko, Operations Executive
An orphan disease is any rare and often fatal disease that affects a small percentage of the population. Most of us live our entire lives unaware that there are about 7,000 rare diseases that affect 350 million people worldwide. These individuals and families fight every day to find some hope for the future.
Unfortunately, many researchers are unwilling to invest time and money into treatments for rare diseases that will only benefit a handful of individuals. The big money is in conditions like heart disease that affect more people and therefore bring in more revenue. Drug development for orphan diseases is simply not cost effective.
In 1983, Congress passed the Orphan Drug Act to encourage pharmaceutical companies and independent researchers to develop these orphan drugs and expedite the development process. The law gives tax credits and aid to companies working on rare diseases and also allows the company the exclusive right to sell the drug for seven years. During the 20-year-period after the Orphan Drug Act was passed, the FDA approved 231 orphan disease treatments. Since 2002, 263 new drugs have been approved and placed on the market.
Although much progress has been made, the fight is far from over. Only 494 drugs exist for 7,000 rare diseases, which means that 95 percent of them have no treatment. Families look to researchers and dream about the day a drug will be developed for their loved one. It is up to all of us to raise awareness and educate the community about orphan diseases so that no affected individual and family are without hope.
Citation: picture copied from: https://globalgenes.org/the-rare-diseases-list-you-must-see-it-to-believe-it/
Siblings with a Mission is an international organization established to serve and support siblings of individuals with special needs. All images are found on Google images and are solely used for education purposes. The stories and advice provided by Siblings with a Mission are not to be replaced by professional advice and counseling but to be considered as an additional source of support.
By Rachel Dokko, Operations Executive
An orphan disease is any rare and often fatal disease that affects a small percentage of the population. Most of us live our entire lives unaware that there are about 7,000 rare diseases that affect 350 million people worldwide. These individuals and families fight every day to find some hope for the future.
Unfortunately, many researchers are unwilling to invest time and money into treatments for rare diseases that will only benefit a handful of individuals. The big money is in conditions like heart disease that affect more people and therefore bring in more revenue. Drug development for orphan diseases is simply not cost effective.
In 1983, Congress passed the Orphan Drug Act to encourage pharmaceutical companies and independent researchers to develop these orphan drugs and expedite the development process. The law gives tax credits and aid to companies working on rare diseases and also allows the company the exclusive right to sell the drug for seven years. During the 20-year-period after the Orphan Drug Act was passed, the FDA approved 231 orphan disease treatments. Since 2002, 263 new drugs have been approved and placed on the market.
Although much progress has been made, the fight is far from over. Only 494 drugs exist for 7,000 rare diseases, which means that 95 percent of them have no treatment. Families look to researchers and dream about the day a drug will be developed for their loved one. It is up to all of us to raise awareness and educate the community about orphan diseases so that no affected individual and family are without hope.
Citation: picture copied from: https://globalgenes.org/the-rare-diseases-list-you-must-see-it-to-believe-it/
Siblings with a Mission is an international organization established to serve and support siblings of individuals with special needs. All images are found on Google images and are solely used for education purposes. The stories and advice provided by Siblings with a Mission are not to be replaced by professional advice and counseling but to be considered as an additional source of support.