Rare Disease Product Launch Services

The market for individual rare and orphan disease therapeutics may be small, but the overall market for rare disease drugs is substantial and competitive. There are over 400 million people worldwide in need of highly-specialized rare or orphan drug treatments, including some 30 million (approximately 1 in 10) living in the U.S.. With 7,000 different rare diseases, each affecting fewer than 200,000 people, only 500 have approved treatments. However, the percentage of drugs approved to treat rare or orphan diseases has been rising considerably in the last decade, with more than half (54%) of the FDA’s novel drug approvals in 2022 going to help patients with rare diseases.

That said, launching a rare disease therapy requires overcoming unique challenges in the form of competitive and pricing pressures, stakeholder and regulatory demands, and patient needs and expectations. Because Two Labs has worked on over 200 unique drugs that have the orphan designation assigned to them by the FDA, our experience gives us the ability to provide the biopharma industry with a detailed understanding of what it takes to successfully launch orphan drugs. Unique challenges require non-traditional strategies, and Two Labs is ready to help your company prepare a holistic launch strategy that capitalizes on data and interacts proactively with physicians, patients, advocacy groups, and other stakeholders.