The document summarizes a presentation on opportunities for improving quality, value and population health in pediatric endocrinology through understanding costs and cost-effectiveness. It provides examples of studies analyzing the cost and cost-effectiveness of growth hormone therapy for short stature and different screening strategies for prediabetes and diabetes in children. The results suggest current guidelines may not optimize value and new models of care are needed to improve outcomes and reduce costs.

1. Joyce Lee, MD, MPH
Associate Professor
Pediatric Endocrinology
Child Health Evaluation and Research Unit
University of Michigan
Twitter: @joyclee
Health Outcomes, Quality, and Cost:
Opportunities for Pediatric
Endocrinology
Paul Kaplowitz Endowed Lectureship for
contributions to quality and cost-effective
care in Pediatric Endocrinology

2. Dr. Paul Kaplowitz

3. Disclosures
JAMA Pediatrics
Verily
Business Innovation Factory

4. Disclosures
I work in the laboratory of healthcare
delivery
Pediatric Clinical/Translational
Researcher

5. Clinical
Effectiveness
Knowledge
Clinical
Efficacy
Knowledge
Improved
Quality, Value,
& Population
Health
Basic
Biomedical
Science
T1 T2 T3
Dougherty JAMA 2008
Translational Research
Clinical Efficacy
Research

6. Clinical
Effectiveness
Knowledge
Clinical
Efficacy
Knowledge
Improved
Quality, Value,
& Population
Health
Basic
Biomedical
Science
T1 T2 T3
Dougherty JAMA 2008
Translational Research
Health Services Research
• Access/Quality
• Health Utilization
• Health Outcomes
• Costs/Cost-effectiveness

7. Clinical
Effectiveness
Knowledge
Clinical
Efficacy
Knowledge
Improved
Quality, Value,
& Population
Health
Basic
Biomedical
Science
T1 T2 T3
Dougherty JAMA 2008
Translational Research
Health Services Delivery
• Systems Design
• Quality Improvement Science
• Human-centered Design/Participatory
Design

8. Clinical
Effectiveness
Knowledge
Clinical
Efficacy
Knowledge
Improved
Quality, Value,
& Population
Health
Basic
Biomedical
Science
T1 T2 T3
Dougherty JAMA 2008
Translational Research
“If I publish it, it will be done”

9. 1982
B-blockers post-MI improved mortality
Translating knowledge and therapies
takes too long

10. 2007
25 years later
T Lee NEJM 2007
% of Pts
Receiving
B-Blockers
Post-MI

11. “Intensive therapy effectively delays the onset
and slows the progression of diabetic
retinopathy, nephropathy, and neuropathy in
patients with IDDM”
1983-1993
Diabetes Control and
Complications Trial

12. 23%
26%
21%
0%
20%
40%
60%
80%
100%
<6
6-<13
13-<20
Age
A1c Goals of <7.5% for <18 yrs, <7% for 18+
% Individuals meeting HbA1c targets
(T1D Exchange)
2013
20 years later

13. “If I publish it, it will be done”
There is a translational gap in
Pediatric Endocrinology,
which is why we need the Science
of Health Services Research and
the Science of Health Services
Delivery to achieve the goal of
providing the best possible medical
care for our patients

14. Disclaimer: Measuring costs and cost-
effectiveness are just one aspect of the
science of health services research and
health services delivery
Focus of this talk:
Cost & Cost-effectiveness (CE)

15. Takeaways
Costs/CE affects access to therapies for our
patients
Understanding Costs/CE helps us optimize our
use of health care resources by identifying
which clinical strategies may lead to greater
value for cost
Understanding Costs can help us think about
opportunities for developing new models of care

16. Takeaways
Costs/CE affects access to therapies for our
patients
Understanding Costs/CE helps us optimize our
use of health care resources by identifying
which clinical strategies may lead to greater
value for cost
Understanding Costs can help us think about
opportunities for developing new models of care

18. We built a model based on efficacy results from the
pivotal trial used for the ISS FDA approval &
studies from the literature
Hypothetical cohort of 10 year old boys treated w/
GH compared with an untreated control cohort
5 year duration of tx
GH dosing 0.37 mg/kg/week
5th% weight
30% discontinuation rate in 1st year of tx
Yearly visits with Endo, bone age, TFTs, IGF-1

19. Main Outcome Measures
Incremental cost per child
Incremental growth per child
Incremental cost per inch

20. Results
Incremental
Cost per Child, $
Incremental
Growth per
Child
(inches)
Cost per
Inch, $
99,959 1.9 $52,634

22. Incremental
Cost per
Child, $
Incremental
Growth per
Child, in
Cost
per
Inch, $
Lower Efficacy (1.8 in)
Higher Efficacy (3.9 in)
99,959
99,959
1.2
2.6
81,875
38,783
Age at initiation ages 8-13y
Age at initiation ages 12-16y
81,268
126,123
1.9
1.9
42,792
66,411
Discontinuation rate, 0%
Discontinuation rate, 40%
137,779
87,352
2.6
1.7
53,531
52,174
Treatment Duration, 7y (Ages 8-15y)
Treatment Duration, 10y (Ages 5-15)
122,513
145,550
2.5
3.2
49,396
45,156
Dosing Regimens
Low-dosage GH (0.24 mg/kg per wk)
Standard-dosage GH (0.37 mg/kg per
wk x 2y followed by high-dosage
GH at puberty (0.7 mg/kg per wk) x
3y
Standard-dosage GH (0.37 mg/kg per
wk) x 1y followed by high-dosage
GH at puberty (0.7 mg/kg per wk) x
4y
65,092
155,440
170,866
1.4
3.1
3.4
45,700
49,821
50,384
Sensitivity Analyses

23. Conclusions
Estimate of $52,000 was substantially higher
than a previous cited cost estimate of $35,000
No alternative GH treatment strategies change
the cost-effectiveness of the therapy
The cost of the drug drives CE

24. Takeaways
Cost and CE affects access to therapies for our
patients
Understanding Costs/CE helps us optimize our
use of health care resources by identifying
which clinical strategies may lead to greater
value for cost
Understanding Costs/CE can help us think
about opportunities for developing new models
of care

25. 2010 ADA guidelines
Prediabetes
HbA1c ≥ 5.7% & <6.5%
Diabetes
HbA1c ≥ 6.5%
2010 ADA Guidelines
The guideline was based exclusively on
data from adults. No pediatric data about
test efficacy/effectiveness or cost-
effectiveness.

26. Study Design: Cross-sectional cohort of 254 children
10-17 years with a BMI ≥ 85th%
2-hour OGTT (Gold standard)
Nonfasting HbA1c
Nonfasting 50 gm 1 hour glucose challenge test
Nonfasting Random glucose
Outcome was Dysglycemia (2-hr Glu≥140) as defined 2-
hour OGTT (Prediabetes n=99, Diabetes n=3)
Receiver Operator Characteristic Curves and
Compared Area Under the Curve

27. 0%
25%
50%
75%
100%
0%
25%
50%
75%
100%
Sensitivity(Truepositive)
1-Specificity (False )
Sensitivity(Truepositive)
1-Specificity (False Positive) *p=0.02
1-hr 50 gm
Glucose Challenge
Test (GCT)
Random Glucose
HbA1c
Test performance of nonfasting glucose
tests of glycemia was better than HbA1c
5.7%
110
100
110
120
6.0%
AUC
Random
Glucose
0.68 (0.61-0.76)*
1 hour
GCT
0.70 (0.62-0.78)*
HbA1c
0.55 (0.47-0.64)

28. Strategy
Cutoff
Sensitivity (%)
Specificity
(%)
2 hr OGTT
-
100%
100%
Hemoglobin
A1c
5.7% (ADA)
45%
57%
6.0% (IDF)
32%
74%
6.5%
7%
98%
1-hr Glucose
Challenge
Test (mg/dL)
110
63%
63%
120
44%
81%
Random
Glucose (mg/
dL)
100
55%
67%
110
30%
88%
Test Performance for Detecting
Prediabetes

29. Test Performance for Detecting
Diabetes
Strategy
Cutoff
Sensitivity (%)
Specificity
(%)
Hemoglobin
A1c
5.7% (ADA)
33%
56%
6.0% (IDF)
33%
71%
6.5%
33%
96%

30. Model of a hypothetical cohort of the 2.5
million overweight or obese adolescents 10-19
years of age eligible for screening
Modeled a one-time screening program for
diabetes and dysglycemia from the societal
perspective

31. Screening Strategies Evaluated
2-hour oral glucose tolerance test
Hemoglobin A1c (HbA1c)
Random Glucose
1-hr 50gm Glucose Challenge Test
If positive,
2-hr OGTT

32. Base Case Assumptions
16% prevalence of dysglycemia (n=400,000)
0.02% prevalence of diabetes (n=500)
100% adherence
2-hr OGTT has 100% sensitivity and
specificity
Liese et al, Pediatrics, 2006
Li et al, Diabetes Care, 2009

33. Cost Assumptions
Screening
Strategy
Cost per Screen
($ 2010)*
Patient time
for Testing**
2-hr OGTT
$18.44
135 min
HbA1c
$13.90
15 min
1-hr GCT
$6.80
75 min
Random
Glucose
$5.62
15 min
½ Mean Hourly Wage
(All Occupations)
$10.68/hour
Physician Time
1/5th visit=$20
Direct and Indirect Costs
*Medicare reimbursement rates **Wage data (Bureau of Labor Statistics)

34. Study Outcomes
Proportion of cases (diabetes/dysglycemia)
identified
Total costs (direct & indirect)
Cost per case identified (direct & indirect)

35. Sensitivity Analyses
Alternative estimates of HbA1c test performance
Higher or lower prevalence (±25%)
Differing levels of adherence (75% for nonfasting
and 50% for 2-hr OGTT)
Doubled provider time
Halved HbA1c costs

36. $831,166 (33%)
2 hr OGTT
$312,224 (100%)
HbA1c 5.7%
HbA1c 5.5%
$731,822
(33%)
HbA1c 6.5%
$571,344
(33%)
Cost per Diabetes Case Identified ($)
%ofCasesIdentified
Base Case (100% adherence)
High Effectiveness
Low Cost per case
Low Effectiveness
High Cost Per Case

37. $831,166 (33%)
2 hr OGTT
$312,224 (100%)
HbA1c 5.7%
HbA1c 5.5%
$731,822
(33%)
HbA1c 6.5%
$571,344
(33%)
Cost per Diabetes Case Identified ($)
%ofCasesIdentified
Base Case (100% adherence)

38. $831,166 (33%)
2 hr OGTT
$312,224 (100%)
HbA1c 5.7%
HbA1c 5.5%
$731,822
(33%)
HbA1c 6.5%
$571,344
(33%)
HbA1c 6.5%
$577,843 (32%)
Cost per Diabetes Case Identified ($)
%ofCasesIdentified
Alternative HbA1c Thresholds
HbA1c 5.7% $329,249 (71%)

39. Costs per Dysglycemia Case Identified ($)
%ofCasesIdentified
Alternative HbA1c Thresholds
HbA1c 6.5%
$3370 (7%)
$938 (32%) HbA1c 5.7%
$763 (45%) HbA1c 5.5%
$721 (30%) RPG 110
$709 (44%) 1-hr GCT 120
$571 (63%) 1-hr GCT 110
$498 (55%) RPG 100
2 hr OGTT
$390 (100%)
HbA1c 6.5%
$5754 (4%)
HbA1c 5.7%
$826 (34%)

40. Sensitivity Analyses did not change the
Overall Rankings
Alternative estimates of HbA1c test performance
Higher or lower prevalence (±25%)
Differing levels of adherence (75% for nonfasting
and 50% for 2-hr OGTT)
Doubled provider time
Halved HbA1c costs

41. Conclusions/Implications
HbA1c had lower effectiveness and higher costs
Why would we prioritize a screening test that
performs worse and costs more?
A1c is still useful at diagnosis of diabetes, but
random or 1-hour GCT may be more promising
screening tests
Should the ADA change its policy on HbA1c for
children? What should the AAP recommend?

42. Takeaways
Cost and CE affects access to therapies for our
patients
Understanding Costs/CE helps us optimize our
use of health care resources by identifying
which clinical strategies may lead to greater
value for cost
Understanding Costs/CE can help us think
about opportunities for developing new models
of care

43. Most studies of cost in adults, non-US populations,
privately insured kids, so we studied kids covered
under California Children’s Services
Outcomes: We measured health utilization and
costs for 652 children with presumed T1D enrolled
for the period July 1, 2009, to June 30, 2012.
Aged 0-21 years
Continuously enrolled for at least 365 days
Had an outpt visit for T1D in the year
Were taking insulin

44. $5603
$58
$144
$2930
$1579
0
2000
4000
6000
8000
10000
12000
Overall Population
ED
(0.4%)
(60%) Hospitalizations
(1.3%) Outpatient Clinic
(13%) Monitoring Supplies
Overall Median Annualized Expenditure Rates
(23%) Insulin

45. S/P DKA Episode #3
Flat affect
Lives 2 ½ hours away
3 hour clinic visit
Does not bring meter
HbA1c = 14%

46. Clinical
Effectiveness
Knowledge
Improved
Quality, Value,
& Population
Health
Basic
Biomedical
Science
T1 T2 T3
Dougherty JAMA 2008
Translational Research
Health Services Delivery
• Systems Design
• Quality Improvement Science
• Human-centered Design/
Participatory Design
Health Services Research
• Health Utilization
• Health Outcomes
• Costs/Cost-effectiveness
• Access/Quality
Clinical
Efficacy
Knowledge

47. Design of a Learning Health System
for Type 1 Diabetes

48. Healthcare delivery system as scientific
laboratory
Clinical Care, Research, and Quality
Improvement are no longer separate efforts
but are deliberately designed to be integrated
Research informs practice and practice
informs research
Learning Health System

49. Technology Tools
Focus on Outcomes/Quality Improvement
Collaborative Network
What does a Learning Health System
Consist Of?

50. Patient-reported
Data
Data
Clinician-reported
Data
Clinical Care
Quality
Improvement
Research
A “Data in Once System”

51. Data Capture at Clinical Encounter
through the EMR

52. Research Grade Data is Entered &
Measured Consistently Across All
Centers

53. Collection of Patient Reported Outcomes

54. Technology Tools
Focus on Outcomes/Quality Improvement
Collaborative Network
What does a Learning Health System
Consist Of?

55. Multiple Outcomes are Followed Across
Centers the Network in Real-time

56. Clinical Centers can Measure the Effects
of Improvement Interventions

57. Clinical Centers Can Perform Population
Management

58. Technology Tools
Focus on Outcomes/Quality Improvement
Collaborative Network
What does a Learning Health System
Consist Of?

59. Collaborative Network of Patients &
Caregivers, Clinicians, Researchers
Sharing Virtual/In Person
“Stealing Shamelessly & Sharing
Seamlessly”
Resources, QI Interventions, Innovations
One Patient è Many Patients
One Provider è Many Providers
One Visit è Many Visits

60. Identify her as a high risk patient &
provide clinical support between visits
Depression Screening (QI Intervention)
Diabetes coach (self-management skills)
Peer support (Group Classes)
Diabetes education for millennials
What can a Learning Health System do
for Kayla?

61. $5603
$58
$144
$2930
$1579
0
2000
4000
6000
8000
10000
12000
Overall Population
ED
(0.4%)
(60%) Hospitalizations
(1.3%) Outpatient Clinic
(13%) Monitoring Supplies
(23%) Insulin
“An opportunity”
Overall Median Annualized Expenditure Rates

62. Alternative Payment Models (accountable care
organizations (ACOs), bundled payments, and
advanced primary care medical homes)
Currently at 20% of Medicare
Goal of 30% by 2016
Goals of 50% by 2018

63. “Wilkins demonstrated his organized
approach to patient care. Impressed by the
poor care of children with congenital syphilis
and their lack of follow-up care, he started a
dedicated clinic, organized the care, and
obtained a special social worker to develop a
follow-up system.”

64. Health Outcomes, Cost, Quality,
and Learning Health Systems of Care:
Our opportunity to fulfill
Wilkins’ vision of care
for Pediatric Endocrinology!

65. Valerie Castle, MD
Ram Menon, MD
Gary Freed, MD, MPH
Sarah Clark, MPH
Matthew Davis, MD, MAPP
UM Pediatric Endocrinology
Twitter: @joyclee
Email: joyclee@med.umich.edu
http://www.doctorasdesigner.com/
Acknowledgements
Emily Hirschfeld
Ashley Garrity
Nayla Kazzi
En-Ling Wu
Beth Tarini
Esther Yoon
Jim Gurney
Acham Gebremariam

66. www.diabetesemoticons.com
nightscoutstudy.info