Orphan drug backers seek rule changes

Emil Kakkis is a man on a mission to speed the way drugs get to people with rare diseases.
The former chief medical officer at BioMarin Pharmaceutical Inc. — one of the trailblazing companies of the so-called “orphan drug” industry — is leading efforts in Washington, D.C., to rewrite how those drugs are judged by the Food and Drug Administration. Why use a statistics-intensive process set up for diseases with millions of patients, Kakkis’ thinking goes, to divine the safety and effectiveness of the rarest of the rare diseases?
Changing the process to rely on markers in mid-stage trials rather than historical data, he said, will translate into less risk, more investment, more jobs and more treatments to patients faster.