11/14/2011 1 TIM COTE MD MPH TIM COTE, MD MPH CMO: NATIONAL ORGNAIZATION FOR RARE DISORDERS (NORD) PROFESSOR OF REGULATORY PRACTICE: KECK GRADUATE INSTITUTE (KGI) NORD and KGI National Organization for Organization for Rare Diseases Keck Graduate Institute
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NORD and KGI - PACT GROUPpactgroup.net/system/files/catdworkshop_cote.pdf · Big Pharma Cultural Impediments Unlearn Th tit ’tb dThat it can’t be done for such little money That
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11/14/2011
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TIM COTE MD MPHTIM COTE, MD MPHCMO: NATIONAL ORGNAIZATION FOR RARE DISORDERS (NORD)
PROFESSOR OF REGULATORY PRACTICE: KECK GRADUATE INSTITUTE (KGI)
NORD and KGI
National Organization forOrganization for Rare Diseases
Keck Graduate Institute
11/14/2011
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On a personal note…
I will never do that again!again!
Israel stole my heart….
Economic Realities: 1982
Rare diseases = Few pill buyers
1973-1982: 10 new drugs for rare diseases
≈7,000 rare diseases; 25 million people
Congressmen/Senators besieged by parents/caregivers.parents/caregivers.
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Abbey Myer:Grassroots Political Mover
Rare diseases: Individually infrequentIndividually infrequent but collectively common.
“They are like orphans in that they yrequire special care.”---Henry Waxman
The New Deal of the ODA
Get a drug designated with“orphan drug status”drug status Show (with data) that it’s“promising” For treating <200,000 person in the US
When?---When you’ve got “promise”; Animal When? When you ve got promise ; Animal model data is fine
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Summary
Orphans drug development is passionate stuffstuff.
Orphans are very different from non orphans.
Gene therapies and stem cell therapies can be “orphan drugs”, offer perhaps the best hope.
With 7,000 diseases, there is an enormous amount of need, new models beckon.