Tomorrow (February 29) is Rare Disease Day, an international day to raise awareness for rare, “orphan” diseases—and a good time to remember why we must protect the Orphan Drug Act and incentivize R&D for new cures.
There are about 7,000 identified “orphan,” or rare, diseases—which, combined, affect more than 300 million people, or as much as 6% of the world’s population.
And 72% are genetic, with 70% starting in childhood.
But they’re rarely covered by the media,says BIO CEO Jim Greenwood, because each disease affects just a small number of patients.
The Orphan Drug Act has helped. Passed by Congress in 1983, the legislation has incentivized development of 400+ innovative treatments for rare diseases, giving hope and a new life to millions of patients around the world suffering from diseases like Duchenne muscular dystrophy, Batten disease, spinal muscular atrophy, and more.
But 95% of rare diseases still have no treatment available—which is why we must protect the legislation’s market exclusivity and tax credit provisions to promote continued investment.
And above all, we can’t enact price controls—because we know they will disincentivize investment in cures across the board, whether for rare diseases or widespread ones, like the coronavirus.
Read more about why the Orphan Drug Act matters:
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