Are Orphan Drugs On The Rise?

If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of the market. Orphan drugs accounted for less than 10% of spending in 2017. Yet the number of approvals for Orphan drugs was at an all-time high. In order to understand this the National Organization for Rare Diseases (NORD) commissioned a study on the topic of Orphan Drugs. Keep reading to learn more, or follow the original story here for more information.

What are Orphan Drugs?

In the simplest terms, an orphan drug is a medication designed for the treatment of a rare condition– generally, in the US, this means a condition that affects fewer than 200,000 people in the country. While each condition itself is rare, the “rare” umbrella contains over 7,000 conditions and diseases– which means about 1 in 10 people in the US have a rare disease. Because the conditions are rare, there often isn’t a large enough market to support pharmaceutical companies developing the treatment. To mitigate this, the government provide support and advantages to companies researching rare diseases. The company can be awarded an orphan drug designation by the FDA, which provides incentives for rare research. This designation, and the benefits that come with it, allow for the research and development of treatments which would otherwise be ignored.

Finding Out

Describing 2017, NORD’s president speaks of a “year of innovation and advancement.” He is speaking specifically about advances in rare disease medicine. During 2017, the Food and Drug Administration (FDA) approved more treatments for rare diseases than during any previous year. In 2017, the FDA granted approval for 80 orphan drug statuses. Within the first eight months of 2018, the FDA already approved almost 60. NORD’s study points to the Orphan Drug Act as a major motivator that enables research to move forward.

Another factor being considered about the Orphan Drug Act is the way it affects treatment cost. According to NORD’s study, the Orphan Drug Act is not affecting the cost of medications in a significant way. Among the most used rare therapies in the US, the median cost for treatment was under $10,000 per year.

Overall, these statistics help to paint a better picture of how orphan drugs fit into the medical landscape. Nearly 56% of the US’s drug-spending in 2017 went to treatments for non-orphan diseases. Orphan drugs make up just under 10% of the annual spending– an almost 2% increase from the previous year. Orphan drug research continues to grow.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email