Health Outcomes, Quality, and Cost: Opportunities for Pediatric Endocrinology

Joyce Lee, MD, MPHAssociate ProfessorPediatric EndocrinologyChild Health Evaluation and Research UnitUniversity of MichiganTwitter: @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

Dr. Paul Kaplowitz

DisclosuresJAMA PediatricsVerilyBusiness Innovation Factory

DisclosuresI work in the laboratory of healthcare deliveryPediatric Clinical/Translational Researcher

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


Clinical Efficacy

Knowledge


Health

Basic Biomedical

Science

T1 T2 T3

Dougherty JAMA 2008


Health Services Research •  Access/Quality •  Health Utilization •  Health Outcomes •  Costs/Cost-effectiveness


Clinical Efficacy

Knowledge


Health

Basic Biomedical

Science

T1 T2 T3

Dougherty JAMA 2008


Health Services Delivery •  Systems Design •  Quality Improvement Science •  Human-centered Design/Participatory

Design


Clinical Efficacy

Knowledge


Health

Basic Biomedical

Science

T1 T2 T3

Dougherty JAMA 2008


“If I publish it, it will be done”

1982

B-blockers post-MI improved mortality

Translating knowledge and therapies takes too long

200725 years later

T Lee NEJM 2007

% of Pts Receiving

B-Blockers Post-MI

“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

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

“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

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)

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

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

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

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

Results

IncrementalCost per Child, $

Incremental Growth per

Child(inches)

Cost per Inch, $

99,959 1.9 $52,634

IncrementalCost per Child, $

Incremental Growth per

Child, in

Cost per

Inch, $Lower Efficacy (1.8 in)Higher Efficacy (3.9 in)

99,95999,959

1.22.6

81,87538,783

Age at initiation ages 8-13yAge at initiation ages 12-16y

81,268126,123

1.91.9

42,79266,411

Discontinuation rate, 0%Discontinuation rate, 40%

137,77987,352

2.61.7

53,53152,174

Treatment Duration, 7y (Ages 8-15y)Treatment Duration, 10y (Ages 5-15)

122,513145,550

2.53.2

49,39645,156

Dosing RegimensLow-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

65,092155,440

170,866

1.43.1

3.4

45,70049,821

50,384

Sensitivity Analyses

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

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

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.

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

0%

25%

50%

75%

100%

0% 25% 50% 75% 100% Sens

itivi

ty (T

rue

posi

tive)

1-Specificity (False )

Sens

itivi

ty (T

rue

posi

tive)

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)

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

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%

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

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

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

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)

Study Outcomes

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

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

$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 ($)

% o

f Cas

es Id

entifi

ed

Base Case (100% adherence)

High Effectiveness Low Cost per case

Low Effectiveness High Cost Per Case

$831,166 (33%)

2 hr OGTT $312,224 (100%)

HbA1c 5.7%

HbA1c 5.5% $731,822

(33%) HbA1c 6.5% $571,344

(33%)


% o

f Cas

es Id

entifi

ed

Base Case (100% adherence)

$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%)


% o

f Cas

es Id

entifi

ed

Alternative HbA1c Thresholds

HbA1c 5.7% $329,249 (71%)

Costs per Dysglycemia Case Identified ($)

% o

f Cas

es Id

entifi

ed

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%)

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

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?

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

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

$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

“Patients, caregivers, clinicians and researchers work together to choose care based on best evidence; together they drive discovery as natural outgrowth of patient care; and ensure innovation, quality, safety and value, all in real-time.”

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



Health

Basic Biomedical

Science

T1 T2 T3

Dougherty JAMA 2008


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

Design of a Learning Health System for Type 1 Diabetes

Healthcare delivery system as scientific laboratoryClinical Care, Research, and Quality Improvement are no longer separate efforts but are deliberately designed to be integratedResearch informs practice and practice informs research

Learning Health System

Technology Tools Focus on Outcomes/Quality Improvement Collaborative Network

What does a Learning Health System Consist Of?

Patient-reported Data

Data

Clinician-reported Data

Clinical Care Quality Improvement Research

A “Data in Once System”

Data Capture at Clinical Encounterthrough the EMR

Research Grade Data is Entered & Measured Consistently Across All Centers

Collection of Patient Reported Outcomes



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

Clinical Centers can Measure the Effects of Improvement Interventions

Clinical Centers Can Perform Population Management



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 ProvidersOne Visit è Many Visits

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?

$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

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

“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.”

Health Outcomes, Cost, Quality, and Learning Health Systems of Care:

Our opportunity to fulfill Wilkins’ vision of care

for Pediatric Endocrinology!

Valerie Castle, MD Ram Menon, MD Gary Freed, MD, MPH Sarah Clark, MPH Matthew Davis, MD, MAPP UM Pediatric Endocrinology

Twitter: @joyclee Email: [email protected]

http://www.doctorasdesigner.com/

Acknowledgements

Emily Hirschfeld Ashley Garrity Nayla Kazzi En-Ling Wu Beth Tarini Esther Yoon Jim Gurney Acham Gebremariam

www.diabetesemoticons.com nightscoutstudy.info

Health & Medicine

Health Outcomes, Quality, and Cost: Opportunities for Pediatric Endocrinology