Transcript Timothy Coté

ICORD 2009:
Facilitating Cooperation in Regulatory Efforts
Timothy Cote, MD MPH
Director, FDA Office of Orphan Products
Development, Food and Drug Administration,
Rockville, MD, USA
Monday, Feb 23, 2009
International Collaborative Regulatory
Actions
 Designations
The joint form
Annual reports
Exchange
 Grants
 Neglected tropical diseases
 The Science of Small Clinical Trails
 Search for Forgotten Orphan Drugs of Special
Promise---We must be accountable in holding
the People’s data.
The search for forgotten orphan drugs of
promise
 25 years! 1,892 orphan designations as of
August 2008 (1,960+ now) and 334 marketing
approvals---what happened to the rest?
 Some were junk.
 Some were toxic.
 Some were forgotten.
 Drug development is not a strictly Darwinian
process where the fit (i.e. safe/effective) always
win.
Definition: Forgotten Orphan Drugs?
 Some IND* data was submitted to FDA
 IND was begun before Jan 1 2006
 No record of recent activity on the IND.
 Hasn’t been approved for
its designated orphan indication,
another orphan indication
or a common disease
(i.e. not on the market).
*Investigational New Drug, a file of incoming data
submitted to FDA.
Forgotten Orphans Waiting at FDA
1892
Designations
1983 through 8/31/2008
327
Approved
Orphan
Indication
137
179
Approved
For another
Orphan
Indication
Approved
For a Common
Disease
Indication
361
Recent INDs
2006 through
8/31/2008
316
No IND
Available
572
Older Designations,
Not-Approved with an IND
306
Active
INDs
88
CBER
Biologics
59
WithdrawnI
NDs
26
Terminated
INDs
266
Forgotten Orphans
70
Inactive
INDs
23
Other
INDs Status
Plan: Re-examine Forgotten Orphans
Study design
Study enrollment
Study results for Efficacy
Study safety signals
Then…if there are forgotten orphans of
great promise; reinvigorate them!
Summary: Rare Diseases Erase National
Boundaries
International body of science
International pharmaceutical industry
International network of patients
To be effective proponents of developing
drugs for rare diseases, regulators must
work internationally.