Orphan Diagnostics Act: Pipe Dream or Reality (ODRD-USA-2016, Boston, MA)
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Transcript of Orphan Diagnostics Act: Pipe Dream or Reality (ODRD-USA-2016, Boston, MA)
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Orphan Diagnostics Act:
Pipe Dream or Reality* M. (Ken) Kengatharan, PhD., MBA.
President, Armetheon; Chairman, Renexxion; General Partner, Atheneos Capital
*my own views and not the views of the organizations I am affiliated with 1
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Personalized or Precision Medicine?
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Personalized
Precision
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Precision Medicine
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Diagnostics and biomarkers
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January 15, 2001
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Companion Dx Promise for a Better
Future
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Challenges for Dx
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Dx Industry Profile vs Others
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Not Helped by this Situation
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The Orphan Drugs Act (US, 1983)
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Impact of the Orphan Drugs Act on Drug
Development
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Ideal playing field for
small biotechs
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Orphan Drug Development
for Companies of All Sizes
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Key Components of the Orphan
Drugs Act
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Prevalence
7-year period of exclusive marketing after
approval
Assistance in clinical study designs
Funding for clinical testing
Tax credits
Waiver of Prescription Drug User Fee Act
(PDUFA) filing fees
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Could the Orphan Drug Act be a
template for Orphan Dx Act?
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Common elements
Need relevant definition
Indication vs target
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From the EU ca. 2008
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“Question 9: Should the EU have an orphan regulation on
medical devices and diagnostics?”
“Orphan medical devices and orphan diagnostics: At first
sight, investigating the possibility of introducing similar
incentives as for orphan drugs might seem like a good idea,
but implementation and identifying possible products
(medical devices and diagnostics) which are exclusively for
rare diseases will or may prove difficult or impossible in
practice.”
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Potential Impact of Orphan Dx Act
on Diagnostics Related Projects
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Equity capital funding
Stronger exclusivity
More likelihood of exit (esp. pre-
commercial)
Help to reduce NNT (if companion Dx)
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Next Steps to Make Orphan Dx Act a
Reality
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The Future?
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Orphan Drugs Act 1983
Orphan Diagnostics Act 201?
Orphan Drugs and Diagnostics Act?
…sometimes it is easier to amend….
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Questions?
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