The Orphan Drug Act: What Does “Orphan” Mean?

Douglas Paul, PharmD, PhD discusses the Orphan Drug Act and what the term “Orphan” means in context of the the Rare Disease industry, especially considering that much of the Rare Disease pipeline is for Oncology products.

The Orphan Drug Act was first enacted in the United States in 1983 and was set up to encourage the development of drugs for rare diseases. At that time, drug therapies for these disorders were rarely developed. Thirty four years later, a growing proportion of industry research and development and drug approvals target diseases affecting fewer than 200,000 persons in the United States, the prevalence-based threshold of rare disease under the Act.

Most of the policy response to the orphan drug pricing problem has been to explore new ways to evaluate orphan drug performance following regulatory approval. However, there is little indication that health care payers are successfully pushing back on drug price points.