According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There are more than 7,000 rare diseases that continue to affect more than a million people around the world. Progress in Europe and America has been made thanks to the European Orphan Drug Legislation in 2000 and the American Orphan Drug Act passed in 1983.
Prior to the approved regulations, Europe only had 8 known orphan drugs, and now we have seen more than 100. Unfortunately, prior to 2000 there wasn’t much education and awareness revolving around rare diseases. There were no enticing incentives for biopharmaceutical industries, not to mention the development of orphan drugs is highly costly. Most individuals with rare diseases are children, and sadly most would die before the age of 5 without proper treatment.
Once the US and Europe made regulations, progress started to finally happen. In Europe, regulators incentivized, provided assistance and reduced fees to encourage industries to take time to develop these treatments. A committee was developed to help make the right decisions that favored the patients’ needs, not the industries. The incentives provided were huge for companies like Novartis, and those companies made a huge push for the development of new orphan drugs. The rate increased by 8.2% yearly from the initial approval of the Orphan Drug Regulation to 2015. Not to mention, roughly 130 more drugs hit the marketing circuit in Europe alone, and 605 across many other therapeutic platforms.
While we can now agree that significant change has been made, there is no slowing down. We can be happy with the progress since so many lives have been saved with new drug development, but there is no room for complacency. There are still so many individuals living with debilitating diseases and many dying from them. People should be encouraged to continue the spread of knowledge and awareness. Orphan medication only receives about 4% of all pharmaceutical funding and many predict that will remain the same up to 2020. That is something everyone should be fighting for, these innocent individuals should receive the attention they deserve. They are human like everyone else and deserve an equal opportunity to live.
