Happy 40th - Orphan Drug Act

Rare Diseases have always existed. Prior to modern medicine and technology many more were mistreated, not treated and fatal than they are today. Along with advances in those two disciplines was one major piece of legislation - The Orphan Drug Act of 1983 that has helped change the outcomes and quality of life for many. This year, we in the rare disease world celebrate this fundamental, bipartisan legislation that gave hope and real answers to many. I would like to note that the actual 40th anniversary date of this law being passed was January 4, 1983 - something this big deserves to be celebrated all year long!!

Passed under Ronald Reagan, The Orphan Drug Act, was to provide incentives through taxes credits to pharmaceutical companies to invest in drug development for rare diseases. Think about it, if a rare disease affects less than 200,000 in the U.S. prior to the passage of this law why would a pharmaceutical company invest millions knowing that they would benefit relatively few and therefore make relatively little money (https://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf.

Prior to this legislation there were only 38 drugs that were FDA approved for rare disease treatments and since that number has grown to over 1100 with over 7000 rare diseases officially being recognized. I would like to also mention that this was the same year that the National Organization for Rare Disorders was created. NORD as it is known is one of the leading rare disease advocacy organizations. NORD was created to bring unity to those who were advocating and bringing awareness to the needs of this now recognized rare disease community. Much of the information that I share, I obtain through my experience with NORD, as I the Community Engagement Ambassador for Tennessee's Rare Action Network.

The Orphan Drug Act 1983 and development of organizations such as NORD have paved the way for more advancements. Some of the most widely known are the Human Genome Project in 2003 which started a whole new way of looking at the possibility of medicine using human genetics and https://www.clinicaltials.gov which lists all current medical trials that receive federal funding (https://rarediseases.org/the-orphan-drug-act-turns-40-nord-celebrates-its-impact-on-rare-diseases/).

There is still a lot of work to be done as there are still less than 10% of the 7,000 rare known rare disease with a treatment or cure. There is still a whole network of patients who are undiagnosed with rare or ultra rare diseases. As part of my role within RAN (Rare Action Network) I work to connect with rare patients in Tennessee and those that serve them and then connect them to each other. If you or someone you know and love would like to get connected, please find me through the link provided on my blog or at tnrareaction.org and I will get you connected with people who can help - even if you are not in Tennessee.