9 Things You Need To Know About Orphan Drugs

This week in Washington, there’s a new congress in town, but not the one you’re probably used to hearing about. Instead of replicating the partisan politics at play on Capitol Hill, the annual World Orphan Drug Congress (WODC) convenes passionate stakeholders seeking to advance development of a class of medicines that often work to fill unmet needs.

But unless you’re a part of the health care industry, there’s a good chance that you have never heard the words “orphan drug” before. So, in honor of this 6th annual WODC, here are 9 things you need to know to understand orphan drugs:

  1. Orphan drugs defined.  At its simplest, an orphan drug is a class of medicine that has not been developed for large numbers of patients. One reason orphan drugs are so important is because they treat rare diseases.

  2. So what’s a rare disease?  In the United States, we consider a disease rare when it affects fewer than 200,000 people (and in Europe it’s fewer than 1 in 2,000 people.) It’s important to note that a rare disease isn’t just one disease with one treatment. In fact, there are over 7,000 different rare diseases.

  3. Cancers can be rare diseases, too. Some of the most challenging rare diseases include cancers that affect very small populations. Cancers like soft tissue sarcoma, bladder cancer and hepatocellular carcinoma each affect under 200,000 people per year, therefore qualifying them as rare.

  4. Most rare diseases are genetic. When it comes to most rare diseases, you can’t pick them up off the street or from a subway station. According to the National Institutes of Health, more than 80% of rare diseases are genetic in origin.

  5. Rare diseases affect nearly 1 in 10 Americans.  Although the word “rare” may trick us into thinking these diseases don’t have a big impact, the reality is stark. As many as 30 million Americans may be affected by rare diseases.

  6. The fight is on. Researchers have been working to find new treatments for people with rare diseases for many years, and the pipeline shows it. The latest numbers indicate there are more than 450 orphan drugs currently in development to help treat rare diseases.  

  7. There are policies in place to help. Beyond the WODC, the US Congress has shown that it is willing to help. For example, they adopted the First Orphan Drug Act in 1983, which helps support companies in their pursuit to make life better for people with rare diseases through the development of orphan drugs.

  8. The policies are making a difference. In the past 3 decades, the FDA has approved more than 500 orphan drugs to help Americans living with rare diseases. In fact, nearly 1/3 of all FDA orphan drug approvals in the last 5 years were for rare diseases.

  9. But more work needs to be done. We are only breaking the surface on finding treatments for the known rare diseases. As it stands, we can only treat 5% of rare diseases

Want to learn more? Here are 10 online rare disease resources that can help!