THE ORPHAN DRUG MARKET

Mount Tam's most advanced product focuses on the treatment of lupus, an orphan disease that is part of the orphan drug market. 

 

Orphan Disease

The orphan drug market is named after the orphan disease (also referred to as a "rare disease"), which is defined by the FDA as a medical condition that affects 200,000 people or less. The orphan drug is a medical product developed to treat such patients who have serious unmet medical needs. The National Organization for Rare Disorders (NORD) estimates there are currently ~30 million Americans suffering from 7,000 rare, orphan diseases.

 

Lupus

Lupus is part of an expanding orphan drug market that has been consistently growing in patient size, now with more than 1.5 million patients in the United States alone. Although the disease is 7 times larger than the average orphan disease, the FDA continues to define lupus as an orphan disease due to the lack of treatments available for patients—highlighting the need for new targeted medications for the disease.  

 

In the last decade, pharmaceutical companies have been adapting to meet the demand for orphan drugs, as this market enables companies to develop new areas of therapeutics, diagnosis and treatment, monitoring and patient support.

 

The  increased focus on rare diseases started in part because of the 1983 Orphan Drug Act (ODA) in the U.S., and similar stimulus opportunities that later established in Singapore in 1991, Japan in 1993, Australia in 1997, and the European Union in 2000. Now that such incentives are in place around the world, there is new encouragement for the investment of research and development in orphan diseases. This encouragement comes in the form of research and development incentives, including grant programs to fund R&D, waived FDA fees and protocal assistance, shorter development timelines, greater regulatory success, tax credits, and more. Commercial drivers include favorable reimbursements, fewer hurdles for approval, longer exclusivity, lower marketing costs, faster update, and premium pricing.

 

These benefits have transformed the cost and time needed for the research and development of orphan drugs, introduced below from research by EvaluatePharma's Orphan Drug Report 2013. As a result, the last decade has become the most productive decade for orphan drug development.