- The PCORI Strategic Plan
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- The PCORI Strategic Plan
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- Draft Key Questions: Systematic Review of the Impact of Doula Support During Pregnancy, Childbirth and Beyond (2024)
Past Opportunities to Provide Input
- Patient-Centered Economic Outcomes Landscape (2023-2024)
- Systematic Review of Audio Care for the Management of Mental Health and Chronic Conditions (2023) -- Draft Key Questions
- Proposed New Methodology Standards for Usual Care as a Comparator (2023)
- Stakeholder Views on Components of 'Patient-Centered Value' in Health and Health Care (2023)
- PCORI's Proposed Research Agenda (2021-2022)
- Proposed National Priorities for Health (2021)
- Proposed Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research (2020)
- Proposed New PCORI Methodology Standards (2018)
- Data Access and Data Sharing Policy: Public Comment (2017)
- Proposed New PCORI Methodology Standards (2017)
Comment on the Proposed New and Revised PCORI Methodology Standards (2016)
- 1. Standards for Formulating Research Questions
- 10: Standards for Studies of Diagnostic Tests
- 12. Standards on Research Designs Using Clusters
- 13: General Comments on the Proposed Revisions to the PCORI Methodology Standards
- 2: Standards Associated with Patient-Centeredness
- 3: Standards for Data Integrity and Rigorous Analysis
- 4: Standards for Preventing and Handling Missing Data
- 5: Standards for Heterogeneity of Treatment Effects
- 6: Standards for Data Registries
- 7: Standards for Data Networks as Research-Facilitating Structures
- 8. Standards for Causal Inference Methods
- 9. Standards for Adaptive Trial Designs
- Peer-Review Process Comments (2014)
- Draft Methodology Report Public Comment Period (2012)
Children's Day—celebrated the second Sunday of every June—is a time to mark our commitment to ensuring a healthy future for all the nation’s children. Approximately 74 million Americans, or 24 percent of the population, is under the age of 18, and the complexity of pediatric care is growing rapidly. In the United States, about 20 percent of children have at least one chronic condition, such as obesity, diabetes, asthma, attention-deficit/hyperactivity disorder, or autism spectrum disorder. Comparative clinical effectiveness research (CER) can help provide clinicians, parents, and others who care for children with useful evidence regarding the best health options.
At PCORI, we have funded more than $180 million in CER focused on improving child health and care delivery. Children are also an important focus in the development of PCORI’s PCORnet, the National Patient-Centered Clinical Research Network, which aims to transform clinical research by promoting collaborative partnerships to improve healthcare and advance medical knowledge. PCORnet includes the National Pediatric Learning Health System Clinical Data Research Network (PEDSnet), and five patient-led networks that focus on child health. One of the first demonstrations projects within PCORnet will address long-term effects of antibiotics on children’s growth.
We believe that PCORI’s investment in such studies, coupled with our commitment to engaging patients, caregivers, clinicians, and policy makers in the research projects, will result in better information and improved outcomes. Here we highlight a few of the projects we fund.
Helping Youth with Overweight or Obesity with Bipolar Spectrum Disorders
One of PCORI’s first 10 awards in its Pragmatic Clinical Studies initiative addresses an important question in pediatric mental health. Pragmatic studies are large, diverse, and conducted in routine clinical settings, so they generate information that can quickly integrated into clinical practice.
The recently awarded pediatric pragmatic study plans to enroll 1,800 Cincinnati and New York City area children, ages 8 to 17, who have been diagnosed with bipolar spectrum disorders. The Food and Drug Administration has approved four second-generation antipsychotics—olanzapine, risperidone, aripiprazole, and quetiapine—for such children. However, those drugs can lead to weight gain, which patients and their parents consider the most problematic side effect. To reduce weight gain and improve metabolic health, some clinicians have prescribed metformin, a medication used to treat diabetes. The PCORI-funded study will compare young people treated with or without metformin, along with an intervention that encourages a healthier diet and more physical activity. The research team will collect information on outcomes including weight, metabolic health, treatment adherence and satisfaction, self-esteem, mood, and quality of life.
Using Technology to Better Measure Health Outcomes
A California project seeks to more accurately gauge the impact of health problems on children’s everyday lives. The research team suspected that children, using an age-appropriate survey called the Child Health Rating Inventory Survey, can assess various facets of their health more accurately than traditional measures, such as surveys of parents. The team previously developed an animated, computer-administered survey for children. The team now plans to compare the results of their survey, presented on an iPad, with those of a traditional written questionnaire for parents and children, as well as with health measures in medical records. The team will also add a new facet of the survey that measures surgery-related anxiety immediately before and after the operation. Six hundred children, ages 4 to 10, having tonsillectomies will participate.
Controlling Asthma in Hispanic/Latino Youth
Another California project is benefitting from collaboration between an academic research institute, community clinics, a community-based organization, and a division of the state health department. These groups have teamed up to study the effect of treatments on Hispanic/Latino youth with asthma. While nationwide only 8.3 percent of children have asthma, 20 percent are affected in Imperial County California, the largely Hispanic/Latino, US-Mexican border community where the project is based.
Asthma is affected by factors ranging from air quality to health care, and currently there’s no agreement among researchers, healthcare professionals, or people with asthma on the best treatment methods given these diverse influences. To compare how well a variety of treatments control asthma, the research team will ask parents of 400 patients, ages 6 to 17, about their child’s asthma control and quality of life and also measure lung function. The study will include a community-wide intervention (a health campaign and school program), a clinic intervention (modifying systems and education for physicians and nurses), and a family intervention using community health workers.
Empowering Caregivers to Get Involved in Decision Making
Under any circumstance, medical emergencies create a stressful situation, and parents and guardians of children in need of emergency care may feel especially stressed. An Ohio project is studying whether a computer-based, interactive tool helps caregivers of a child diagnosed with early appendicitis be confident in their decision making and satisfied with care the child receives. The tool informs the patients and caregivers about appendicitis and its treatment options, encourages them be active in guiding the care, and helps them make a treatment decision that is best for the child.
The study will include 200 children, ages 7 to 17, and their parents or caregivers. All will receive a standard surgical consultation, but half will also use the interactive tool. The research team will measure outcomes including participants’ knowledge about appendectomy and antibiotic options and their satisfaction or regret with their treatment decision. The team will also consider the number of days the patient and caregiver spend away from normal activities, any medical complications, and the patient’s quality of life after their chosen treatment.
These are only a sampling of the more than 70 projects that PCORI is supporting to advance children’s health. We currently fund research in each of our five national priorities. We’re excited to see the diversity of topics across our projects and look forward to the study results.
In addition to the funded work, other children’s health questions are currently in PCORI’s topic-prioritization process, used to select questions for targeted funding announcements or high-priority lists. Please visit the lists in our topic prioritization pathway or suggest something new.
We look forward to keeping you informed as our children’s health projects progress toward answering questions important to patients, parents, and others in the healthcare community. You can get learn about other studies by searching our list of funded projects.