Rare diseases, including orphan diseases, affect more than 30 million people in the United States. The U.S. Orphan Drug Act defines a rare disease as one that affects fewer than 200,000 people, but despite their low incidence, many of the world’s more than 7,000 rare diseases lack effective treatments and so represent a significant unmet medical need.

Small molecule oral delivery therapies are a crucial part of the treatment landscape for rare diseases, offering patients familiarity and the potential for convenient administration. However, manufacturing these therapies can present unique challenges. If you are responsible for developing one of these therapies, it is important to understand:

• The key challenges in manufacturing small molecule oral delivery therapies for rare diseases
• How Contract Development and Manufacturing Organizations (CDMOs) can provide critical support to bring life-saving treatments to patients