We call them rare diseases, but there is nothing rare about the suffering they cause. Some 25 million Americans, nearly one in 13, live with rare genetic diseases. … And even though the direct medical costs of rare diseases are estimated at $400 billion a year … less than five percent of them have Food and Drug Administration-approved treatments.
Why so few? Because the economics of drug development work against small patient populations. …
What makes this moment different is that the technology to do something about it finally exists [in] recent advances in mRNA science and CRISPR gene editing. The technology can be reprogrammed for different diseases by inputting a short stretch of genetic code …. Build the system once, and you can redirect it to a new disease by changing that one piece.
Related on the SLP
Radically new science demands radically new thinking about how we regulate, manufacture, pay for and deliver treatments. Ten years from now, if children are still dying of conditions we know how to correct, it will not be because the science wasn’t ready. It will be because we lacked the imagination to build a system worthy of it.
