Comparative Effectiveness
Research and Children with
Chronic Conditions
James M
M. Perrin
Perrin, M
M.D.
D
Professor of Pediatrics, Harvard Medical School
Director, Center for Child and Adolescent Health Policy
M
MassGeneral
G
l Hospital
H
it l for
f Children
Child
Chronic Conditions in
Childhood
Major growth in past few decades
Increasing evidence supporting both primary
prevention and limiting long-term effects
Commonalities in (some) etiology and
treatment issues
Perccent
Children and Adolescents
with Limitation of Activity
8
7
6
5
4
3
2
1
0
1960
1969-70 1974-75 1979-81 1992-94 1996-98
Newacheck, NHIS Analyses, US data
Chronic Conditions:
Children and Adults
Adult conditions:

small number of common conditions
Child conditions:

large number of (mainly) rare conditions
Most conditions more common in males, especially
before puberty
Most children survive, although developmental,
physical, and psychological outcomes vary
“Typical”
Typical Chronic
Conditions
Cystic fibrosis
Spina
p
bifida
Sickle cell anemia
Hemophilia
p
22,500
60,000
37,500
7,500
,
New Epidemics:
Mainly among school-age children
and youth
y
Obesity
Asthma
ADHD
D
i
Depression
Autism Spectrum
Disorder
*US population estimates, early-mid 2000s
11,250,000*
5 250 000
5,250,000
4,000,000
3 200 000
3,200,000
700,000
Growth in Rates of Chronic
Conditions
1960-1980: Improvements in survival led to
increases in rate of a number of chronic
conditions (>80% survival in 1980; >95%
survival currently)
Marginal impact of newer conditions (eg,
VLBW, in utero toxins, AIDS)
1980-now: New epidemics of common
chronic conditions
Childhood Chronic Conditions
Increasing Prevalence
Parallel developments have led to dramatic
increases in childhood chronic conditions:

Amazing biomedical advances



Children with chronic conditions live longer (eg, CF, leukemia)
More children survive (eg
(eg, NICU
NICU, surgical)
Regressive social changes
Poverty associated with more and more severe
chronic disease – but little evidence of change in
childhood poverty rates over past 40 years
C
C
Changes
in Children’s
Lives
Genetics


Manyy conditions have clear genetic
g
disposition,
p
requiring
q
g
environmental triggers for recognition or manifestation
But, hard to postulate genetic drift
Changing physical (and toxic) environments
and the cleanliness hypothesis
Children’s social environments (and
pp
for p
pediatric counseling)
g)
opportunities
Children’s
Changing Children
s
Environments
Parenting and parent stress
TV and other media
Physical activity
Di t
Diet
Trajectories of Chronic
Conditions
Three cohorts of children ages 2-8 years followed
for six years
1988 1994
1988,
1994, and
d 2000 comparisons
i
40-50% of children with chronic conditions in 1988
do not have them in 1994 (same in 2000)
>60% of those with chronic conditions in 1994 (or
2000)) did not have them six yyears earlier
Increased rates over 12 years (from 12.8% in 1994
to 26.6% in 2006)
VanCleave, Gortmaker, Perrin, JAMA, 2010
Special Problems for CER with
Children and Adolescents
Many rare conditions – small numbers
Development
p
and its effects on condition
manifestations and treatment

Implications for habilitation and rehabilitation
Diversity (sociodemographic) in child and
adolescent population
Dependency (ie, family as unit of observation)
Financing (varied patchwork of support)
Condition-specific
Condition
specific CER and
Rare Disease
Very rare diseases (e.g., SCID, Krabbe’s,
Arginosuccinic
g
Acidemia)) – can it be done?
More common but still rare conditions (e.g.,
CF SCA)
CF,

Mainly thru collaborative networks
Can CER apply to decisions regarding
newborn screening?
IOM CER Priorities:
Conditions
Conditions:








Hearing loss (various surgical interventions)
Adding biologics to inflammatory disease Rx
Surgical line infections
Preventing dental caries
Obesity (school, clinical, social interventions)
Mental health conditions (ADHD
(ADHD, ASD
ASD, MDD
MDD, PTSD)
Asthma (integrated vs episodic care)
Prevention of unintended pregnancy
IOM CER Priorities:
Care Models
Comprehensive vs usual care (VUC) in severe CD
Wraparound and community services in SED
Literacy-sensitive
Literacy
sensitive disease management VUC in CD
Co-location (VUC) in ID and RX of socioemotional and
development disorders, ages 0-3
Models of comprehensive care after NICU stay
Mindfulness-based interventions (VUC) in anxiety,
depression, and CD
Shared decision making (VUC) in children with asthma and
other chronic conditions
Strategies to improve adherence
P di t i Oncology
O
l
Pediatric
Acute lymphocytic leukemia – almost 100%
mortality in early 1960s
S i tifi advances
Scientific
d
in
i understanding
d t di eradication
di ti off
tumor cells, coupled with
Development of research network with active
collaboration in


Common assessment
Implementation of careful research protocols
Current 5 year survival ~95%
C ti Fibrosis
Fib
i F
d ti
Cystic
Foundation
>95% of children with CF in common data registry,
allowing



Understanding of trends (in disease and treatment)
Search for where variations (sociodemographic,
treatment etc.)
etc ) are associated with different clinical
treatment,
outcomes
p
p
Development
of comparative
treatment p
protocols
Similar developments beginning in autism care
P di t i Models
M d l off Care
C
Pediatric
Chronic care model (Dr. Strickland)
What components work?
What are specific issues for children and
adolescents in CCM?
Prevention
 Family
F il ffocus
 Others
