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
Personalized or Precision Medicine?
2
Personalized
Precision
Precision Medicine
3
Diagnostics and biomarkers
4
January 15, 2001
Companion Dx Promise for a Better
Future
5
Challenges for Dx
6
Dx Industry Profile vs Others
7
Not Helped by this Situation
8
The Orphan Drugs Act (US, 1983)
9
Impact of the Orphan Drugs Act on Drug
Development
10
Ideal playing field for
small biotechs
Orphan Drug Development
for Companies of All Sizes
11
Key Components of the Orphan
Drugs Act
12
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
Could the Orphan Drug Act be a
template for Orphan Dx Act?
13
Common elements
Need relevant definition
Indication vs target
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.”
Potential Impact of Orphan Dx Act
on Diagnostics Related Projects
15
Equity capital funding
Stronger exclusivity
More likelihood of exit (esp. pre-
commercial)
Help to reduce NNT (if companion Dx)
Next Steps to Make Orphan Dx Act a
Reality
16
The Future?
17
Orphan Drugs Act 1983
Orphan Diagnostics Act 201?
Orphan Drugs and Diagnostics Act?
…sometimes it is easier to amend….
Questions?
18
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