Challenges and Opportunities to Bolster the Effectiveness of Childhood Screening

Page created by Anne Warner
 
CONTINUE READING
Challenges and Opportunities to
                                        Bolster the Effectiveness of
                                        Childhood Screening
                                         Rosalind B. King, PhD,a Elizabeth Neilson, PhD, MPH, MSN,b Tracy M. King, MD, MPHa

Collectively, through the articles in                               The term “screening” is used                                     with the condition.1 Accessible
this supplement, the authors identify                               differently across various disciplines                           follow-up thus becomes an important
numerous challenges to advancing                                    and research contexts. In the context                            issue to consider if the benefits of
the science of childhood screening                                  of prevention, screening is defined as                           screening, diagnosis, and treatment
but also note exciting opportunities                                the systematic testing of                                        are to be quantified at a population
for future research, including                                      asymptomatic individuals to identify                             level.
potential advances in terminology,                                  people at an increased risk (or early
theoretical frameworks, and                                         stage) of a disease or condition, with                           “Surveillance” is another term that
methodologic approaches. The                                        the aim of providing information and                             has different connotations in
authors’ contributions are grounded                                 other services that reduce the risk of                           different contexts. In the public
in the existing standards of pediatric                              complications.1–3 Although this                                  health context, surveillance is
research but enhanced through                                       definition is relatively                                         generally not considered to be an
contributions from developmental                                    straightforward, quantifying the                                 intervention but, rather, an ongoing
science, econometrics, data science,                                benefits of such screening in clinical                           system for collecting, analyzing, and
and public health scholarship.                                      practice can be a challenge because                              using information to assess disease
Implementation of the novel and                                     of factors such as lack of consistent                            risk and prevalence in a population.
rigorous approaches described                                       follow-up diagnostic evaluation and                              Within the United States, surveillance
herein (and the development of new,                                 treatment, even in clinical trials. One                          testing at the population level
yet unimagined innovations) may                                     proposition to address this issue is to                          conducted to monitor for disease
hold promise for identifying                                        expand the definition of screening                               prevalence may stimulate prevention
approaches to surveillance and                                      from mere receipt of a screening test                            and control efforts2 but may not
screening that will be the most                                     to the entire process from invitation                            explicitly be linked to follow-up and
effective in improving children’s                                   to screening among asymptomatic                                  treatment. In pediatric clinical
health, development, and                                            people through completion of the                                 settings, however, the term
well-being.                                                         treatment of the people identified                               “surveillance” has also been used to

a
    Eunice Kennedy Shriver National Institute of Child Health and Human Development and bOffice of Disease Prevention, National Institutes of Health, Bethesda, Maryland

Drs King, Neilson, and King drafted and approved the final manuscript as submitted and agree to be accountable for all aspects of the work.
DOI: https://doi.org/10.1542/peds.2021-050693K
Accepted for publication Mar 31, 2021
Address correspondence to Rosalind B. King, PhD, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, 6710B Rockledge Dr, Room
2305, Bethesda, MD 20892-7002. E-mail: rosalind.king@nih.gov
PEDIATRICS (ISSN Numbers: Print, 0031-4005; Online, 1098-4275).
Copyright © 2021 by the American Academy of Pediatrics
FINANCIAL DISCLOSURE: The authors have indicated they have no financial relationships relevant to this article to disclose.
FUNDING: No external funding.
POTENTIAL CONFLICT OF INTEREST: The authors have indicated they have no potential conflicts of interest to disclose.

                                    Downloaded from www.aappublications.org/news by guest on July 26, 2021
PEDIATRICS Volume 148, number s1, July 2021:e2021050693K                                                                                                  SUPPLEMENT ARTICLE
refer to active monitoring at the            children on pathways to different               underrepresented groups are
individual level, which can trigger          outcomes. These selective forces,               included in studies, they may
referrals or interventions.4                 often shaped by experiences of                  contribute proportionally less data,
Innovative methods for assessing             socioeconomic disadvantage, can                 particularly data that can link the
the impact of surveillance and               lead to disparities in access to the            delivery of screening to subsequent
screening, in all their various forms,       preventive care visits in which                 health outcomes.
are needed to understand and                 important screenings occur. These
improve their contributions to               selective forces can also directly              One solution noted by Wallis9 is to
improved child health outcomes.              influence health outcomes,                      consider additional systems with
                                             regardless of whether certain                   regular contact with children. Her
Gardner et al5 illustrate one such           screenings are delivered. Another               proposal to draw on child care
approach for assessing the impact of         consequence of selection is that the            settings dovetails with the socio-
screening using an empirical                 same behavior may be labeled as                 ecological model. Teachers and
demonstration of a simulation                within the normative range for                  other early education professionals
model. They outline the qualitative          members of one group and                        have daily interactions with young
dimensions of the screening event            pathologized for members of                     children and their families; they also
and subsequent treatment process                                                             regularly observe young children’s
                                             another. Taken together, these
that must hold for interventions to                                                          interpersonal skills with other
                                             factors may lead to either
be effective: acceptability,                                                                 children and caregivers. This
                                             overestimates or underestimates of
accessibility, and fidelity. Their goal                                                      microsystem is, therefore, not only a
                                             the impact of screening on health
is to show impact at the level of                                                            context through which to
                                             outcomes of interest. Better
population health. Their results                                                             understand children’s behavioral
                                             strategies for understanding and
reveal that even the best of                                                                 health but also a source of data that
                                             accounting for these socio-ecological
screening interventions are                                                                  can contribute to the evidence base
                                             forces are needed to improve the
vulnerable to failures at each step in                                                       for a variety of screening
                                             implementation of childhood
the health care delivery system.                                                             recommendations.
                                             screening and translate screening to
They call for greater integration
                                             improved health outcomes.                       In their article, Graif et al8 also call
both within sectors of health care
and between health care providers                                                            for community partnerships and
                                             In her article, Wallis9 describes how
and community contexts, such as                                                              data exchanges. Wu et al10 expand
                                             these disparities play out in the
schools. Schools and primary care                                                            on this idea of “Moving Beyond the
                                             specific example of autism
providers complement each other;                                                             Clinic to the Community.” They call
                                             screening. Diagnostic algorithms for
the former setting is accessible to                                                          for a mesosystems approach of
                                             autism screening were developed
children whose parents lack health                                                           linking across the contexts of
                                             largely on the basis of evidence
insurance or other ways to pay for                                                           families, schools, neighborhoods,
                                             generated in homogenous samples
medical visits, whereas the latter                                                           and parents’ workplaces through a
                                             of children. As a result, they may              combination of individual surveys,
setting can reach teenagers who
                                             imperfectly predict the risk for                dyadic data, and administrative
have reduced engagement with or
                                             children from underrepresented                  records. Community- and systems-
have dropped out of formal
education.                                   groups. The potential impact of this            level longitudinal linkages create the
                                             lack of representation in                       potential for modeling shared risks
Building on a tradition of bringing          foundational studies is magnified               in different communities on the
Bronfenbrenner’s ecological systems          among low-income children who                   basis of webs of connectedness.
theory into health research,6,7 Graif        experience discontinuities in                   Social media and genomic data
et al8 outline a 5-stage socio-              primary care. In these cases,                   present further sources of currently
ecological model for pediatric               electronic health record evidence of            untapped potential. Such blending of
screening. Their 5 stages situate            screening and the management of                 multiple data sources can be done to
individual children within                   screening results may be missing,               provide a more holistic and
broadening contexts from the                 whether because of a lack of                    meaningful portrait of key health,
microsystems of families, social             preventive care, the dispersion of              developmental, and functional
networks, and health care providers          data among multiple providers, or               outcomes.
to the macrosystems of populations           the use of low-resourced providers
and public policy. They highlight the        who are unable to maintain                      Kemper et al11 focus on another
selection that can occur within each         electronic health records. Thus, even           aspect of the ecological model, the
of these levels, often placing               when children from                              passage of time.6 They acknowledge

                                Downloaded from www.aappublications.org/news by guest on July 26, 2021
S2                                                                                                                           KING et al
that considering each screening                 recommended developmental                       not reveal impacts for many years.
event in isolation, without                     screenings for infants and toddlers             In their work in econometrics,
consideration of the child’s history            than commonly used adult health                 Heckman et al16 also provide
or context, may be easiest to                   measures do and, thus, may have                 examples of methodologic
implement in busy clinical settings             greater utility for revealing the               techniques that may be applied to
and analyze for research purposes.              effects of screening-associated                 pediatric prevention. For example,
However, this oversimplified                    interventions in randomized clinical            they combine bootstrapping and
approach fails to consider critical             trials. Moving this area forward may            kernel matching imputation to
factors, such as the complexities of            require translational collaborations            estimate the societal rate-of-return
human development and                           between developmental                           and benefit-to-cost ratios of a
relationships between coexisting                psychologists conducting basic                  preschool intervention program
health conditions, that can influence           behavioral science research and                 targeted at young children from
screening results but also suggest              applied work by physician-scientists            families experiencing socioeconomic
the most appropriate intervention               and public health researchers.                  disadvantage. These methods allow
for a particular individual and                                                                 projections to populations beyond
setting. Innovative approaches to               Brown and Kemper13 take this call               those directly observed in the data
                                                for rethinking outcomes one step                and across decades of life after the
generating and analyzing
                                                further, by challenging the field to            intervention occurs.
longitudinal data and for linking
                                                consider more broadly who may
such data to key outcomes are
                                                benefit from screening, rather than
needed accelerate the science of                                                                CONCLUSIONS
                                                simply what is being measured.
clinical preventive service delivery.                                                           Although there are many
                                                They question the core screening
Their arguments further explicate                                                               methodologic challenges to creating
                                                principle of limiting consideration of
the calls from Wallis9 and Wu et al10                                                           the evidence base for pediatric
                                                benefit to that which is direct to the
for creative approaches to                                                                      screening, the opportunities cited
                                                patient. Benefits may also accrue to
identifying data sources and analytic                                                           above are relevant to multiple
                                                other individuals across the
strategies.                                                                                     stakeholders: funders (eg, National
                                                ecological contexts in which children
                                                are embedded: family members,                   Institutes of Health [NIH]),
Another component of a                                                                          researchers, practitioners, health
                                                peers (such as classmates), and
developmentally informed approach                                                               care systems, other systems that
                                                future coworkers and neighbors in
for new models is to consider novel                                                             serve children (such as schools),
                                                the broader community. Economist
framing of outcomes. Thus,                                                                      public health officials, and
                                                James Heckman14 has written for
Silverstein et al12 lay out the case                                                            policymakers. One effort in which
                                                decades about the measurable
for assessing positively framed                                                                 these groups could collaborate is
                                                benefit to society of early
outcomes, such as subjective well-                                                              data sharing to create larger data
                                                investment in children’s cognitive
being, in addition to traditional                                                               systems. The NIH has invested
                                                and noncognitive skills. By an
negatively framed clinical outcomes,                                                            deeply in data sharing policies for
                                                extension of this reasoning,
such as the absence of a disease or                                                             funding recipients17 and sharing the
                                                screening for lead in one child that
symptoms. This perspective may tap                                                              agency’s own resources18 as well as
                                                results in the prevention of
into measures that hold greater                 exposure to lead for a sibling yields           supporting efforts in the related
meaning for children and families.              a benefit to society by preventing              areas of data science19 and open
To children in elementary school,               decline in the other child’s cognitive          science.20 The 5-stage socio-
the ability to hear frequencies                 and noncognitive skills. Connecting             ecological model can provide
across the full spectrum of human               screening to a public health                    theoretical grounding for the
vocalization is arguably far less               framework also incorporates the                 development of such systems
important than their ability to hear            consideration of justice, whereby it            through its attention to the multiple
well enough to make friends and                 is ethical to screen a child who will           contextual dimensions of screening.
follow instructions in the classroom.           not receive a direct benefit if the
For their parents, an outcome of                harms to that child are minimal and             In these articles, the authors also
interest might be hearing well                  benefits to others are maximized.               highlight the scientific potential in
enough to make the required                                                                     innovative methodologic techniques
academic progress for on-time                   Finally, Grosse et al15 describe the            and study designs. The development
promotion to the next grade. These              complexities of conducting cost-                and use of subjective well-being
school-aged outcomes occur in much              effectiveness analyses of screening             measures that are validated for
closer time proximity to the                    interventions in childhood that may             children, adolescents, and families

PEDIATRICS Volume 148, number s1, July 2021
                                  Downloaded from www.aappublications.org/news by guest on July 26, 2021                           S3
hold promise for moving the                 generation, improvement, and                        recommendations. Am J Prev Med.
evidence base forward beyond                implementation of the evidence base                 2018;54(1, suppl 1):S63–S69
viewing health as merely the                and resulting recommendations                    2. Farid-Kapadia M, Askie L, Hartling L, et al.
absence of disease.21 Innovative            holds considerable potential for                    Do systematic reviews on pediatric topics
study designs and analytic methods          generating knowledge that can lead                  need special methodological
(such as accelerated longitudinal           to better health outcomes during                     considerations? BMC Pediatr. 2017;
designs,22 synthetic cohorts,23 or          childhood and across the life course.               17(1):57
regression discontinuity designs24)                                                          3. Melnyk BM, Grossman DC, Chou R, et
can be used to reduce the need to           ACKNOWLEDGMENTS                                     al; US Preventive Services Task Force.
rely on randomized controlled trials                                                            USPSTF perspective on evidence-based
                                            The authors acknowledge the sup-
that require extended durations of                                                              preventive recommendations for chil-
                                            port of Dr David Murray, Director of
follow-up before results about the                                                              dren. Pediatrics. 2012;130(2). Available
                                            the NIH Office of Disease Preven-                   at: www.pediatrics.org/cgi/content/
long-term impact of certain                 tion, and Dr Diana Bianchi, Director                full/130/2/e399
exposures or interventions are              of the Eunice Kennedy Shriver Na-
reported. Econometric techniques                                                             4. Lipkin PH, Macias MM; Council on Chil-
                                            tional Institute of Child Health and
applied through approaches such as                                                              dren With Disabilities, Section on Devel-
                                            Human Development. They are also                    opmental and Behavioral Pediatrics.
cost-benefit and cost-effectiveness         grateful to the other members of                    Promoting optimal development: identify-
analyses may address issues of the          the workshop planning group: Dr                     ing infants and young children with de-
lack of data to study causal                Rachel Ballard (NIH Office of Dis-                  velopmental disorders through
inference over long periods of time         ease Prevention), Dr Rao Divi (NIH                  developmental surveillance and screen-
by incorporating simulation                 National Cancer Institute), Dr Dena                 ing. Pediatrics. 2020;145(1):e20193449
modeling, big data and data                 Fischer (NIH National Institute of               5. Gardner W, Bevans K, Keller KJ. The
analytics, and natural experiments.         Dental and Craniofacial Research),                  potential for improving the population
In addition, simulation models can          Dr Dionne Godette-Greer (NIH Office                 health effectiveness of screening: a
be used to help policy makers               of Disease Prevention), Dr Ralph                    simulation study. Pediatrics. 2021;148(1):
evaluate whether screening should           Hingson (NIH National Institute on                  e2021050693C
be provided more broadly and in             Alcohol Abuse and Alcoholism), Dr                6. Eriksson M, Ghazinour M,
alternative settings when resources         Alex Kemper (Nationwide Children’s                  Hammarstr€om A. Different uses of
for conducting pilot and                    Hospital), Dr Iris Mabry-Hernandez                  Bronfenbrenner’s ecological theory in
implementation studies are limited.         (Agency for Healthcare Research                     public mental health research:
                                            and Quality), Dr Suril Mehta (NIH                   what is their value for guiding
A final important use of the socio-         National Institute of Environmental                 public mental health policy
ecological model is to bring in the         Health Sciences), Dr Charlotte Pratt                and practice? Soc Theory Health.
social determinants of health,              (NIH National Heart, Lung, and                      2018;16(4):414–433
including structural racism. The NIH        Blood Institute), Dr Eve Reider (NIH             7. Grzywacz JG, Fuqua J. The social ecology
offers research resources for the           National Institute of Mental Health),               of health: leverage points and linkages.
inclusion of the social determinants        and Dr Karen Sirocco (NIH National                  Behav Med. 2000;26(3):101–115
of health, such as data collection          Institute on Drug Abuse). The views              8. Graif C, Meurer J, Fonatana M. An ecolog-
protocols.25 Along with sister              expressed by the authors are their                  ical model to frame the delivery of
agencies in the US Department of            own and do not necessarily repre-                   pediatric preventive care. Pediatrics.
Health and Human Services, the NIH          sent those of NICHD, ODP, NIH, the                  2021;148(1):e2021050693D
is also soliciting input from the           Department of Health and Human                   9. Wallis KE. The roadmap to early and equi-
scientific community about                  Services, or the federal government.                table autism identification. Pediatrics.
ameliorating structural racism in the                                                           2021;148(1):e2021050693E
conduct of scientific research.26 For
                                                                                            10. Wu AC, Graif C, Mitchell SG, Meurer J,
example, a recent request for                                                                   Mandl KD. Creative approaches for
information issued by the Agency              ABBREVIATION
                                                                                                assessing long-term outcomes in
for Healthcare Research and Quality           NIH: National Institutes of Health                children. Pediatrics. 2021;148(1):
called for specific attention to the                                                            e2021050693F
introduction of racial and/or ethnic                                                        11. Kemper AR, Letostak T, Grossman DC. In-
bias by the clinical algorithms that        REFERENCES                                          corporating longitudinal surveillance into
shape medical decision-making and            1. Kemper AR, Krist AH, Tseng CW, et al.           the delivery of pediatric screening serv-
the provision of care.27 This ongoing           Challenges in developing U.S. Preventive        ices. Pediatrics. 2021;148(1):
scientific enterprise dedicated to the          Services Task Force child health                e2021050693G

                               Downloaded from www.aappublications.org/news by guest on July 26, 2021
S4                                                                                                                                KING et al
12. Silverstein M, Kemper AR, Henderson JT,     18. Trans-NIH BioMedical Informatics                    can be used to enhance prediction of
    Mabry-Hernandez I. Importance of                Coordinating Committee (BMIC). Data                 early childhood psychopathology. Behav
    assessing well-being for United States          sharing resources. Available at: https://           Res Ther. 2019;123:103484
    Preventive Services Task Force                  www.nlm.nih.gov/NIHbmic/index.html.              24. Venkataramani AS, Bor J, Jena AB. Re-
    Recommendations. Pediatrics. 2021;              Accessed March 26, 2021                              gression discontinuity designs in health-
    148(1):e2021050693H                         19. National Institutes of Health. Data science          care research. BMJ. 2016;352:i1216
13. Brown MJ, Kemper AR. Considering                themes. Available at: https://datascience.
                                                                                                     25. National Institutes of Health. PhenX Social
    screening when there may be minimal             nih.gov/. Accessed March 26, 2021
                                                                                                         Determinants of Health (SDOH) Assess-
    direct benefit to the child. Pediatrics.    20. National Institutes of Health. Open                  ments Collection. Available at: https://
    2021;148(1):e2021050693I                        Science Prize announces epidemic                     www.nimhd.nih.gov/programs/collab/
14. Heckman JJ. Skill formation and the eco-         tracking tool as a grand prize winner.              phenx/index.html. Accessed March 26,
    nomics of investing in disadvantaged            Available at: https://www.nih.gov/                   2021
                                                    news-events/news-releases/open-
    children. Science. 2006;312(5782):                                                               26. National Institutes of Health. NIH stands
                                                    science-prize-announces-epidemic-
    1900–1902                                                                                            against structural racism in biomedical
                                                    tracking-tool-grand-prize-winner.
15. Grosse SD, Kemper AR, Prosser LA. Data                                                               research. Available at: https://www.nih.
                                                    Accessed March 26, 2021
    needs for economic evaluations of                                                                    gov/about-nih/who-we-are/nih-director/
                                                21. National Institutes of Health. Your Healthiest       statements/nih-stands-against-structural-
    screening in pediatric primary care: a
                                                    Self: wellness toolkits. Available at:               racism-biomedical-research. Accessed
    research framework. Pediatrics.                 https://www.nih.gov/health-information/              March 26, 2021
    2021;148(1):e2021050693J                        your-healthiest-self-wellness-toolkits.
                                                                                                     27. Agency for Healthcare Research and
16. Heckman JJ, Moon SH, Pinto R, Savelyev          Accessed March 26, 2021
                                                                                                         Quality. Request for information on the
    PA, Yavitz A. The rate of return to the     22. Galbraith S, Bowden J, Mander A. Accel-              use of clinical algorithms that have the
    High/Scope Perry Preschool Program. J           erated longitudinal designs: an overview             potential to introduce racial/ethnic bias
    Public Econ. 2010;94(1–2):114–128               of modelling, power, costs and handling              into healthcare delivery. Available at:
17. Wolinetz C. NIH releases new policy for         missing data. Stat Methods Med Res.                  https://www.federalregister.gov/
    data management and sharing. Available          2017;26(1):374–398                                   documents/2021/03/05/2021-04509/
    at: https://nexus.od.nih.gov/10/29/         23. Luby J, Allen N, Estabrook R, et al. Map-            request-for-information-on-the-
    nih-releases-new-policy-fordata-                ping infant neurodevelopmental precur-               use-of-clinical-algorithms-that-have-
    management-and-sharing/. Accessed               sors of mental disorders: how synthetic              the-potential-to-introduce. Accessed
    March 26, 2021                                  cohorts & computational approaches                   March 26, 2021

PEDIATRICS Volume 148, number s1, July 2021
                                  Downloaded from www.aappublications.org/news by guest on July 26, 2021                                         S5
Challenges and Opportunities to Bolster the Effectiveness of Childhood
                                  Screening
            Rosalind B. King, Elizabeth Neilson and Tracy M. King
                           Pediatrics 2021;148;s51
                      DOI: 10.1542/peds.2021-050693K

Updated Information &         including high resolution figures, can be found at:
Services                      http://pediatrics.aappublications.org/content/148/Supplement_1/s51
References                    This article cites 18 articles, 3 of which you can access for free at:
                              http://pediatrics.aappublications.org/content/148/Supplement_1/s51#
                              BIBL
Permissions & Licensing       Information about reproducing this article in parts (figures, tables) or
                              in its entirety can be found online at:
                              http://www.aappublications.org/site/misc/Permissions.xhtml
Reprints                      Information about ordering reprints can be found online:
                              http://www.aappublications.org/site/misc/reprints.xhtml

                 Downloaded from www.aappublications.org/news by guest on July 26, 2021
Challenges and Opportunities to Bolster the Effectiveness of Childhood
                                  Screening
            Rosalind B. King, Elizabeth Neilson and Tracy M. King
                           Pediatrics 2021;148;s51
                      DOI: 10.1542/peds.2021-050693K

The online version of this article, along with updated information and services, is
                       located on the World Wide Web at:
      http://pediatrics.aappublications.org/content/148/Supplement_1/s51

Pediatrics is the official journal of the American Academy of Pediatrics. A monthly publication, it
has been published continuously since 1948. Pediatrics is owned, published, and trademarked by
the American Academy of Pediatrics, 345 Park Avenue, Itasca, Illinois, 60143. Copyright © 2021
by the American Academy of Pediatrics. All rights reserved. Print ISSN: 1073-0397.

                   Downloaded from www.aappublications.org/news by guest on July 26, 2021
You can also read