Who is your customer? What does your customer consider value? What are your results with customers? Peter Drucker

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1 Who is your customer? What does your customer consider value? What are your results with customers? Peter Drucker

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3 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.

4 Start 3m 6m 9m 12m 15m Clinical Research Lithium Carbonate & Riluzole Treatment Control ALS-FRS FVC Survival Labs Etc

5 Clinical Evidence

6 Clinical Evidence

7 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)

8 Clinical Life

9 "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

10 Given my status, what is the best What is my status? outcome I can Walking hope to achieve, and how Breathing do I get there? Hands Speaking Wellbeing Productivity

11 Function Time

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13 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

14 Users reported that PatientsLikeMe helped them get engaged to be married 1 PLM, data on file

15 Social Dose response curve (p<0.001) for differences between none and all other categories Wicks P, et al. Submitted

16 ALS

17 Severity of Depression by ALS Functional Rating Scale (N=474) (N=678) (N=522) (N=208) % 10% 20% 30% 40% 50% 60% 70% 80% 90% 100%

18 Severe Moderate Mild Today (N=25) days (N=23) days (N=18)

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20 Trust, but verify. Ronald Reagan

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25 Of Mice and Stephen.

26 Of Mice and Stephen. 30,000+ mice 130+ drugs

27 Difference in the Mean Results Published v. TDF TDI Retest Published ALS TDF TDI Unfortunately, no study was repeatable despite larger number of animals used N=417 N=857

28 Empowered patient A great wind is blowing and that gives you either imagination or a headache Catherine the Great Molecular medicine Doctor role New clinical data sources Ongoing reform

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30 If you do not know how to ask the right question, you discover nothing. W. Edwards Deming

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34 Longitudinal RNA

35 The future is already here it's just not very evenly distributed William Gibson

36 "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

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38 Who do we work for? Who is your customer? What does your customer consider value? What are your results with customers? Peter Drucker

39 Given my status, what is the best outcome I can hope to achieve, and how do I get there?

40 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

41 Patient Value Clinical Research Clinical Care

42 Private Subject Shared Partner Secure Accessible Validated Learning Aggregated Personalized Closed Open

43 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.

44 For in reason, all government without the consent of the governed is the very definition of slavery. Jonathan Swift

45 Thank You