Ana Mingorance crdn summit 2017

1. Empowering the
impatient patient
revolution
Ana Mingorance PhD
Dracaena Consulting | www.draccon.com
@CNSdrughunter

2. 2
Patients discovering
drugs and how to help
them make that better

3. 3
Patient Centricity

4. Working for patients

5. Working for patients
Passive role

6. Working with patients

7. 7
Why patients need to
be active partners
in drug discovery

8. ~7,000
rare diseases

10. Only 5%
have an
approved
drug

11. That is 1 in 20

12. 10-15
new
approvals
per year

13. 500 years

14. 14
We need more
orphan drugs

15. 15
And we need them
faster

16. 16
Impatient
Revolution

17. 17
My experience
working with patient
organizations (Dravet
syndrome)
1

18. Shape our disease field so that it
very easy for anyone to work on it.
Science Business
Our power as a patient group:

19. #ImpatientRevolution
A book on "company
engagement" for patient
organizations.
http://www.draccon.com/impatient/

20. In most rare diseases the science
is incomplete

21. Strategic research
investments
Disease education
/ awareness
Diagnosis Cells/tools
Animal model
generation
Animal model
validation and
distribution
Patient registries
PROs
Outcomes and
endpoints
Broader trial-market
collaborations
Patient organizations are already leading
many key scientific/medical progresses

22. Decide to make an open-
access mouse model
Scientists “I’ve been wanting to work
on Dravet syndrome, but
the 3 labs that have mouse
models won’t share them.”
Over 350 mice shipped
Dozens of new laboratories
Millions of € into Dravet
Join forces with
other patient groups

23. Who knows how many
patients are taking that
drug?
Drug company? Not sharing that
data.
Neurologists? Most only see 2-8
patients with this disease
National Health System? Don’t know
/ regional level
? ? ?
“… nobody is
really taking
that drug, no
need to include
it in the trial.”
Data from almost 300 families
51% is taking the drug

24. Patient groups are involved in regulatory and
commercial aspects of drug development
Understand and communicate the
business case to pursue an indication
Well-funded organizations
become investors
Involvement in
regulatory approval and
national HTA

25. 25
The ultra-rare
diseases
2

26. 26
Too few patients.
Not worth the
investment for
companies.

27. 27
Going solo

28. Patient 0

29. Four years later, we are
aware of 55 patients
worldwide.
Try supplements based on
that known cause
(repurposing)
Starting companies to identify
suitable
medications

30. Parent-driven
innovation

31. Many of the 7000 rare
diseases are ultra-
rare

32. The majority have a
genetic cause

33. Doctors don’t
know the cause?
Find where to do and interpret
whole exome sequencing
Find genetic
cause
Drug already
exists?
DIY diagnosis and drug development
Repurpose /
off-label
Repurpose /
off-label
Drug already
exists?
Need to develop
new drugs?
Start a
company
Patient-driven
drug discovery
Pharma engagement

34. Patient 2.0

35. ESHG 2015 Conference Press Release:
People want access to their
own genomic data, even
when uninterpretable
03.Jun.2015

37. We need the
23andMe of
DIY drug development

38. Access to technology platforms
and approved compound
libraries

39. Doctors don’t
know the cause?
Find genetic
cause
Drug already
exists?
DIY diagnosis and drug development
Repurpose /
off-label
Repurpose /
off-label
Drug already
exists?
Need to develop
new drugs?
Start a
company
Patient-driven
drug discovery
Find where to do and interpret
whole exome sequencing

40. How do you envision this
one-stop-shop?
 A government-sponsored program at a
centralized location (like a personalized
medicine institute).
 An international collaboration program by the
EC-NIH or IRDiRC involving multiple centers.
 A network of independently-supported service
providers (think Amazon).

41. 41
It is not only
the future…

44. Working for patients

45. Impatient
Revolution
Ana Mingorance PhD
ana@draccon.com
@CNSdrughunter