CNN is running a comprehensive package about rare diseases and the crisis patients with rare diseases are facing with by being priced out (or left out) of the drug development market. The CNN story discusses the fact that 6800-7000 rare diseases are still mostly overlooked by pharmaceutical companies because of the small patient population and lack of financial incentives to pharma and biotech companies. With this package, CNN also provides a rare disease resource guide so that people can get informed.
Between the US and EU, there are 60 million (!) people suffering from rare diseases (not exactly a small patient population) of different types. According to the National Institutes of Health, pharmaceutical companies only have treatments for 200 of these rare diseases!
Eurordis and rare disease advocates in Europe are calling for an EU resolution to be passed in June to create a national plan on rare diseases. We need millions of rare disease advocates working together and marching on Washington to help highlight the problem here in the United States.
The entire drug development system in the United States is broken for millions of people and there are no solutions in sight. After weeks of waiting, we don’t even have a Director of NIH named.