So Many Ideas, So Little Time
FDA appears ready, even eager, to be pushed on the orphan-drug front. Long viewed as saddled with a bunker mentality, the
agency is undergoing a dramatic course correction at the hands of its two activist public-health officials who are opening
the agency to sunshine ("transparency and accessibility") and its many demanding stakeholders. In February, FDA created a
new position of associate director for rare diseases to keep track of orphan drug NDAs as they move through the approval process—plainly
a response to NORD's long-, often-, and well-articulated critiques.
Whether a new orphan drug review office is on the agenda is a matter of dispute. "We have obtained agreement with high-level
officials at FDA that they will support the creation of a new focused review division for certain rare disease not well covered—if
we can obtain an additional $10 million," he says.
Office of Orphan Product Development head Tim Coté has a different version. "The science itself doesn't support it. Most
orphan diseases do share certain technical problems like the design and recruitment of clinical trails. But as diseases, they
cut across every medical specialty," he says.
Coté, for his part, is hitting the road with workshops for aspiring applicants in Orphan Drug Designation 101. "It's fairly
radical for the agency to go out and speak directly to sponsors and have them generate submissions on site," he says. "We
are trying to demystify the process for researchers who have a fire in the belly about their project but may not even know
what 'IND' stands for." The first workshop, held in February at the Keck Graduate Institute in Claremont, Calif., drew 29
sponsors; four designations have already been granted from the crop of applications. A second workshop is set for August.
Coté is also paying visits to top-10 drugmakers, reminding them that little diseases have large unmet needs. "I really hope
to start seeing results soon," he says. "Getting orphan drugs to market reflects well on the industry and the agency."
Keith Andrus died of cardiac arrest in January. The brown-haired, blue-eyed, much-tattooed patient-activist was 24. In March,
departing PhRMA chief, Billy Tauzin, paid a tearful tribute to Keith during his own farewell speech, screening a short film
of Keith's too-short life movingly narrated by Raychel Bartek. "We always knew Keith would be on the cusp—either the first
generation with a treatment or the last generation without a treatment," Ron Bartek says simply. Is his son's death a moment
to take a break, step away from his nonstop activism? No. "This stopped being just about Keith the day we started," he says