Successful Use of Existing Opportunities for Orphan Drug...
Transcript of Successful Use of Existing Opportunities for Orphan Drug...
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Successful Use of
Existing Opportunities for
Orphan Drug Approval
Catarina Edfjäll, Ph.D.
VP, Head of EU and International Regulatory Affairs,
Shire HGT, Switzerland
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To be as brave as the people we help
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Orphan Drug Approval
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~7000 Rare Diseases
~1% covered by treatment in EU
> 25 mio Patients still waiting…
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OMP Marketing Authorisations in the EU
≈ 7/year
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Orphan Drug Approval
Median review time*
235 days (FDA)
381 days (EMA)
* NEJM (2012): Downing et al.
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We do have some tools – let’s use them!
• Conditional MA
• Accelerated Review
• Protocol Assistance
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• Protocol Assistance
• Innovation Task Force
• Business Pipeline meetings
• EMA/HTA consultations
• Parallel Sci. Advise with FDA
• Pre-submission meetings
• EMA SME office
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Accelerating Orphan Drug Development: The
VPRIV® Story
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Gaucher Disease
Collapsed
vertebrae
Pathologic
fracture
Erlenmeyer flask
deformity
Osteonecrosis/
Osteopenia
Bone crisis
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To be as brave as the people we help
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Searching the globe for Gaucher patients …
• USA: Priority Review by the FDA (6 m)
• EU: Accelerated Review by the CHMP (8 m)
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To be as brave as the people we help
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VPRIV approved in 39 countries
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Can we reduce the
number of hurdles
for OMP Approval?
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Flexibility
Predictability
Continuity
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~60% of ODs
developed for
paediatric
indications
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Let’s learn from each other!
Global Harmonisation of regulatory requirements
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You don’t know if it works until
you try
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Orphan Drug Approval
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If we can be as brave as the
people we help,
if we can be as brave as the
specialists we serve,
then we will be very brave indeed
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Thank you for listening!