Results Summary

PCORI funded the Pilot Projects to explore how to conduct and use patient-centered outcomes research in ways that can better serve patients and the healthcare community. Learn more.

Background

Healthcare researchers need to know what outcomes are most important to patients so that they can study the outcomes that matter to them. In many cases, researchers do not know patients’ priorities when they are doing research on treatments for different conditions. This is especially true of studies that examine conditions that affect children because the common view is that children cannot tell us what matters to them. Without information on what health outcomes are important to patients, researchers may identify “best” treatments that are not what children and their parents would truly prefer.

Project Purpose

The goal of this project was to create and test a way to allow children and their parents to prioritize health outcomes that matter most to them.

Methods

The researchers reviewed two well-known lists of health outcomes and looked at outcome measures used to study six childhood conditions.

The researchers then interviewed 63 youth ages 12 to 17 and 147 parents of children ages 5 to 17. The children and youth were attending clinics at Children’s Hospital of Philadelphia. The researchers asked the children, youth, and parents to describe health outcomes that mattered most to them.

Using the outcome measures from the lists of health outcomes, measures used in research, and information gathered from the interviews, the researchers created a list of health outcome names and definitions that children and their parents could choose from when indicating what outcomes are most important to them.

The researchers tested the list of outcome names and definitions with 21 high school students ages 14 to 17 and 22 parents of children ages 8 to 17. The researchers tested whether the names and definitions were easy for youth and parents to understand. The research team revised and retested outcome names and definitions until most of the youth and parents understood them.

The researchers then created an online tool using the tested names and definitions of healthcare outcomes. The tool lets children and parents rank the outcomes in the order of those that matter most to them.  

The researchers tested how well the online tool could work for young people with autism spectrum disorder (ASD). The team asked 32 youth and young adults with ASD ages 12 to 30 and 109 parents of youth and young adults with ASD to use the tool to choose the outcomes that mattered most to them. Then the researchers compared what young people with ASD and their parents chose with what researchers use when they study ASD.

Findings

The researchers created 68 health outcome names and definitions that youth and parents understood. Almost half of the outcomes that youth and parents said were most important to them were not commonly used in research studies on ASD.

Limitations

Because the researchers used studies of just six childhood conditions to create their list of outcomes, the list may be missing some health outcomes that are important for other conditions.

Importance of Findings

This project showed that researchers may not be studying many health outcomes that children, youth, and their parents think are the most important. The study identified a method that researchers can use to find out what outcomes are most important to children, youth, and their parents.

Sharing the Results

The researchers presented their work at several national and international conferences. They are preparing articles to send to journals. They plan to make the online tool publicly available.

More to Explore...

Dissemination Activities

Through limited competition, PCORI awarded 25 of the 50 Pilot Projects up to $50,000 to support dissemination and implementation of their activities and findings through the PCORI Pilot Project Learning Network (PPPLN) funding. The deliverables listed below are a result of convenings and conferences supported by this funding, whose efforts align with the PCORI strategic goal of disseminating information and encouraging adoption of PCORI-funded research results.

Period: September 2015 to April 2016
Budget: $49,420

2016 Webinar Series

Introduction to Human Subjects Protection Challenges in PCOR
Clarifying Definitions: Who is a human subject? Is it research?
Preparing Patients and Other Stakeholders to Uphold Ethical Research Principles

Presentations

"Navigating IRB Issues for PCOR: A Framework for Engagement and Protection"
2015 Annual AcademyHealth Research Meeting

"Investigating Human Subjects Issues in Patient-Centered Outcomes Research"
2014 Public Responsibility in Medicine and Research Advancing Ethical Research Conference

Project Information

Katherine B. Bevans, PhD
Children's Hospital of Philadelphia (CHOP)
$668,127
Development of Methods for Identifying Child and Parent Health Outcomes

Key Dates

June 2012
June 2015
2012
2015

Study Registration Information

Tags

Has Results
Award Type
Funding Opportunity Type
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: March 4, 2022