Celebrating World Lupus Day: Gaining Ground With Effective Policy

In 2004, national representatives from thirteen countries met in Eaton, United Kingdom, to create the World Lupus Day Proclamation. The Proclamation officially recognized May 10 as World Lupus Day, while acknowledging Lupus as a significant public health concern. Today celebrates a decade-long movement to gain public and private interest to increase financial support, resources and assistance for the chronic, autoimmune disease that affects an estimated 2 million people in the United States alone.

To our team, World Lupus Day highlights a larger movement working to positively change how national health care systems deal with serious unmet medical needs, including orphan diseases. Lupus continues to be defined as an orphan disease due to the lack of treatments available for its patients, grouped together with rarer diseases such as Crohn's Disease, Asperger's Disease and Huntington's Disease, to name a few.

For the better half of the 20th century these orphan diseases were neglected by preventive and medical treatment specialists due to their rarity; ignored by larger research and development institutions due to their lack of financial return.

From 1981 to 1983, California House Representative Henry Waxman and organizations such as the National Organization for Rare Disorders rallied bipartisan support in Congress to push forward H.R. bill 5238 to President Ronald Reagan who signed into law what we know today as the Orphan Drug Act (ODA). Other key markets implemented similar stimulus opportunities shortly after, including Singapore in 1991, Japan in 1993, Australia in 1997 and the European Union in 2000.

In the United States, research and development incentives gained by the ODA include shorter development timelines, greater regulatory success, increased attention to precision medicine and the formation of the NIH Office of Rare Diseases. The ODA has created commercial drivers for research and development institutions, including favorable reimbursements, longer exclusivity, lower marketing costs and faster uptake, among other motivators.

Unified support fostered in the nation's capital ultimately established the ODA and identified the economic and social opportunities for organizations to conduct research and development within the orphan drug market.

Today, the ODA is acknowledged to be a great success by health care professionals and pharmaceutical analysts, to public officials and private investors alike. The 2013 Orphan Drug Report by EvaluatePharma estimated that orphan drug sales will grow to a total $127 billion by 2018 -- double that of the overall prescription drug market. No doubt the orphan drug market has outgrown its name. It has become a very promising time for research and development for orphan diseases such as Lupus.

The Orphan Drug Act's impact on the development of treatments for rare diseases has been impressive. In the decade prior to the ODA, only 10 drugs had been approved in the United States for the treatment of rare diseases. Since the establishment of the ODA by Congress, more than 2,000 compounds have been granted orphan designation and more than 350 drugs have been approved for use against rare diseases. More than 200 orphan diseases are now treatable.

Thanks to the ODA, research and development institutions are better motivated to actively participate in developing and bringing to market treatments for rare diseases. Its success, however, should not undermine the urgency to help more than 30 million Americans who suffer from 7,000 orphan diseases. Most orphan diseases still desperately need new therapeutics, including Lupus. Support for research and development in the field must continue. We should not let budget cuts influence the direction in tackling the burdens associated with these issues. Now more than ever we must back the ODA's purpose and vision, and let effective policy stand.

We celebrate World Lupus Day not only as a moment to bring awareness and resources to Lupus and other orphan diseases, but to highlight what effective policy can do when we work alongside each other, empathetic of each other. The ODA continues to promote collaboration, advance innovation and support the development of initiatives that tackle serious unmet medical needs in the health care industry.

More than 30 years later we celebrate the Orphan Drug Act as we launch our initiative on the 10th anniversary of World Lupus Day. Without the ODA and the momentum it has created for developing treatments for orphan diseases, establishing Mount Tam Biotechnologies -- and initiates with similar goals -- would be a much more arduous and expensive process that would discourage members of the scientific community from working on the changes necessary to ultimately better the health and well-being of millions of people. For this, we are thankful and hopeful that our efforts will play a small part in alleviating the suffering associated with Lupus for people around the world.

Mount Tam Biotechnologies, Inc. is a for-profit company that was formed to further develop, optimize and bring to market leading medical compounds to better the health and well-being of millions of people impacted by autoimmune diseases. The organization's most advanced product focuses on the treatment of Lupus.