Elsie Melsopp, Ph.D.

Elsie Melsopp serves as the director of the solids formulations group at Alcami in Wilmington, NC. She has a Bachelor of Science in Pharmacy from the University of Puerto Rico and earned her Ph.D. in Pharmaceutical Sciences from the University of Connecticut. She has 25+ years of experience in pharmaceutical product development and manufacturing including oral liquids and solid dosage forms from pre-toxicological development to product validation. She has six scientific publications and is co-inventor of two patents (approval pending).

White Paper: Insight into Orphan Drug Formulations and Regulations

The US Orphan Drug Act of 1983 characterizes an orphan disease as a rare medical condition that affects a population of fewer than 200,000 people. Rare diseases have various causes— most notably, genetics. However, the National Institutes of Health (NIH) states, “many rare diseases, including infections, some rare cancers, and some autoimmune diseases, are not inherited. While researchers are learning more each year, the exact cause of many rare diseases is still unknown” (NIH, 2017).

The term “orphan disease” resulted from pharmaceutical companies not having the resources to design and scale up formulations to treat these diseases, due to the lack of financial support for such highly specialized programs. The Orphan Drug Act (ODA) allows for the necessary financial backing of pharmaceutical companies to develop treatments for rare diseases (NIH, 2017), giving special status to a drug or biological product (drug) upon request of a sponsor. This status is referred to as orphan designation or orphan status.

Case Study: Challenges and Solutions to Formulating an Orphan Drug

According to the Food and Drug Administration and the Orphan Drug Designation program, orphan...