Who is your customer?
What does your customer
consider value?
What are your results
with customers?
Peter Drucker
We hold these truths to be self-evident,
that all men are created equal, that they
are endowed by their Creator with
certain unalienable Rights, that among
these are Life, Liberty and the pursuit of
Happiness.
Clinical Research
ALS-FRS
FVC
Survival
Labs
Treatment
Control
Etc…
15m
12m
9m
6m
3m
Start
Lithium Carbonate & Riluzole
Clinical Evidence
Clinical Evidence
Clinical Care
Date: 2/28/2002
BP: 115/75 (Z01.3), Smoking: no, Weight, 202 lbs
Patient has ALS (G12.2), with loss of function in arms
(M62.8), non ambulatory (R26), FVC, 80% (R06.8)
Med list: 100mg riluzole, baclofen (as needed)
Complaint: insomnia (F51.0), drooling (K11.7)
Rx: ambien, (T1T PO PRN QHS)
Rx: detrol (T3T PO BID)
Rx: Permobil C500 power wheelchair
(Z46.8)
Clinical Life
"It is simply no longer possible to believe
much of the clinical research that is
published, or to rely on the judgment of
trusted physicians or authoritative
medical guidelines. I take no pleasure in
this conclusion, which I reached slowly
and reluctantly over my two decades as
an editor of The New England Journal
of Medicine.”
Marcia Angell, MD
“Given myWhat
status,
is my status?
what is the best
outcome I canWalking
hope
to achieve, and
how
Breathing
do I get there?”
Hands
Speaking
Wellbeing
Productivity
Time
Function
Step 1:
Create/update and
share your health
profile
Step 2:
Find support from
others like you and
compare
experiences
Step 5:
Play an integral
part in your own
health care
Step 3:
Learn from
aggregated
community
Treatment and
Symptom Reports
Step 4:
Take profile to your doctor to have
an improved treatment conversation
Users reported that PatientsLikeMe helped them…
…get engaged to be married
1
PLM, data on file
Social ‘Dose response’ curve
(p<0.001) for differences between “none” and all other categories
Wicks P, et al. Submitted
ALS
Severity of Depression by ALS Functional Rating Scale
36-48 (N=474)
53
24-36 (N=678)
29
39
12-24 (N=522)
40
37
0-12 (N=208)
10%
30%
40%
5
29
50%
60%
2
19
34
20%
2
17
39
28
0%
13
70%
80%
9
90%
100%
100
90
80
70
60
50
Severe
40
Moderate
30
Mild
20
10
0
Today (N=25)
30-90 days (N=23)
90-300 days (N=18)
“Trust, but verify.”
Ronald Reagan
Of Mice and Stephen…….
Of Mice and Stephen…….
30,000+ mice
130+ drugs
Results…
TDI Retest
Published v. TDF
Retest
Difference in the Mean
50
Published
ALS
TDF TDI
40
30
Unfortunately, no
study was repeatable
despite larger number
of animals used . . .
20
10
0
-10
N=417
N=857
Empowered
patient
“A great wind is blowing
and that gives you either
imagination or a headache”
Molecular
medicine
Doctor role
– Catherine the Great
New clinical
data sources
Ongoing
reform
“If you do not know how to
ask the right question, you
discover nothing.”
W. Edwards Deming
Longitudinal RNA
“The future is already here —
it's just not very evenly
distributed”
William Gibson
"All models are wrong, but some are
useful.”
George Edward Pelham Box
“All models are wrong, and increasingly
you can succeed without them.”
Peter Norvig
Who do we work for?
Who is your customer?
What does your customer
consider value?
What are your results
with customers?
Peter Drucker
“Given my status,
what is the best
outcome I can hope
to achieve, and how
do I get there?”
What I have learned…
To shorten the vector (time) it takes to have
quantifiable clinical insight into the effectiveness of
an intervention in a single patient
A grid of meaningful quantifiable phenomic and
molecular markers across human, animal, cellular
and molecular models
Validation, validation, & validation
Scale & commonality of comparison and platforms
across conditions, models, and systems
Patient
Value
Clinical
Research
Clinical
Care
Private
Shared
Subject
Partner
Secure
Accessible
Validated
Learning
Aggregated
Personalized
Closed
Open
An Effective Learning Health System
Truth Standards: Improved definitions of disease, its
measurement, and the variance in symptomatology,
pathology, and functional impact.
Vector Measurement: A shift from static/diagnostic health
concepts to longitudinal vectors with origins and
magnitudes.
Host Health: Improved understanding of the dynamics,
range and incidental variance of normal human health at
the molecular and phenomic level.
Big Data: Understanding the multiplicity and complexity
of individual health will require immense longitudinal real
world real world longitudinal datasets.
For in reason, all government
without the consent of the
governed is the very definition of
slavery.
Jonathan Swift
Thank You