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

14

“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