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. Author manuscript; available in PMC: 2023 Jan 5.
Published in final edited form as: Curr Probl Pediatr Adolesc Health Care. 2022 Jan 5;51(12):101126. doi: 10.1016/j.cppeds.2021.101126

Table 2.

Examples of study designs and data elements well-suited to answer selected research questions.

Example Research Questions Example Interventions Patient-Centered Outcomes Types of Data Required Possible Study Designs
Is trazodone an effective medication for sleep problems in children with NI? Trazodone therapy

  • Sleep quality

  • Participation in school/therapies

  • Caregiver mental health

  • Patient/parent-reported outcomes

  • Caregiver data

  • Pharmacy data

 Randomized-controlled trial, either placebo controlled or with active control (melatonin)
How do we best prepare children with medical complexity for major, high-risk surgeries? Are their post-operative outcomes better if we prepare them before surgery? Pre-surgical optimization of nutrition, respiratory condition, and pain management

  • Length of stay

  • ICU utilization

  • Adequate pain control

  • Return to baseline health and functioning

  • Improvement of symptoms

  • Return to school/outpatient therapies

  • Administrative/billing

  • Patient/parent-reported outcomes

  • Clinical and lab data

 Randomized-controlled trial, cluster randomized at hospital level
Does access to family navigator services to address needs related to social determinants of health improve health outcomes? Routine provision of family navigation services within complex care programs

  • Connected to services

  • Caregiver mental health

  • Fewer emergency visits and hospitalizations

  • Administrative/billing

  • Patient/parent-reported outcomes

  • Caregiver data

 Stepped-wedge cluster-randomized trial
Are bisphosphonates effective for treating osteopenia in children with severe NI? Bisphosphonate therapy

  • Improvement in bone mineral density

  • Reduction in fractures

  • Administrative/billing

  • Clinical and lab data

  • Pharmacy data

 Comparison of pre-intervention and post-intervention measurements for the intervention group to an untreated control group
Would a seizure action plan that goes beyond just giving rectal diazepam make a difference in epilepsy hospitalizations? Statewide implementation of a stepwise seizure action plan

  • Reduction in epilepsy-related emergency visits and hospitalizations

  • Administrative/billing

  • Pharmacy data

 Interrupted time series analysis