This piece was originally published on WBUR’s Cognoscenti.
I want to live in a world where children have never heard of hemophilia or cystic fibrosis, because we have all but eradicated those diseases.
This isn’t a dream. The biotech industry is beginning to explore how we might impact genetic and metabolic diseases, by decisively intervening at birth with genetic therapies capable of lifelong transformation.
The Orphan Drug Act, enacted in 1983, gives incentives for biotech and pharmaceutical companies to develop drugs for rare diseases, sometimes known as “orphan” diseases. The incentives have done wonders, spurring the development and approval of hundreds of new therapeutics. But the vast majority of those drugs are for adults.
Prior to 2000, fewer than 10 percent of Orphan Drug Act approvals were for pediatric therapies. In the last two decades, that percentage has gone up, but even so, fewer than 25 percent of approved orphan drugs are designated for use in children, and just a fraction of those are approved for use in infants.
Read the full piece on WBUR’s Cognoscenti.