Most people are familiar with the old adage, “The squeaky wheel gets the grease.” My grandmother used to go a bit further by pointing out, “But the greased wheel that continues to squeak gets replaced.” I suppose she was trying to teach me a lesson about not asking for too much or being appreciative of what I had already. However, the first part of that phrase is all that most people know or remember. They know it, I’m sure, because it makes sense. The powers-that-be cannot fix a problem if they don’t know anything about it.
That is why the announcement from China’s Ministry of Health was so encouraging earlier this year. They are publishing a list of rare diseases, such as hemophilia, Fabry disease, and tuberous sclerosis complex, in order to justify funding and legislation for these diseases.
With a recognized list of rare diseases including Fabry, some officials and people living with these diseases hope that the government will enact legislation similar to the United States’ Orphan Drug Act. The U.S. law encourages pharmaceutical companies to develop “orphan” drugs.
Currently, people living with these rare diseases have few options for treatment. They must choose among three options: take medicine off-label, illegally purchase the medicine on the black market, or go untreated.
As reported by Business Insider, authors of the list hope that having documentation for rare diseases will encourage the governments to establish guidelines for tax breaks, preferential policies for approval, and regulation of the marketplace. All of these are effects the Orphan Drug Act had in the United States. Since it was enacted more than 30 years ago, over 600 drugs for rare diseases have been approved by the U.S. FDA. That averages approximately one-and-a-half approvals per month.