PCORI Methodology Standards:
Academic Curriculum
© 2016 Patient-Centered Outcomes Research Institute. All Rights Reserved.
Module 3: Overview
Category 1: Formulating
Research Questions
Prepared by Zackary Berger, MD, PhD
Eric Bass, MD, MPH
Presented by Zackary Berger, MD, PhD
Why Is Determining Research Questions Essential?
How Does Including the Patient Change Research Questions?
 Answerable questions that matter and are applicable
 Interesting questions that motivate the researcher
 Feasible questions that can be supported
 Albert Wu’s criteria: common, controversial, costly
 Including the patient  research that …
 Matters to patients practically
 The individual, community, public supports
 Patients can participate in
3
Ways to Formulate Questions
Issue
Question
Abnormality
Are these individuals sick or well?
Diagnosis
How accurate is this test for diagnosing disease?
Frequency
How often does this disease occur?
Risk
What factors are associated with an increased risk of this disease?
Prognosis
What are the consequences of having this disease?
Treatment
How does treatment change the course of this disease?
Prevention
Does this intervention keep well people from developing this disease?
Does early detection and treatment improve the course of this disease?
Cause
What conditions lead to the disease?
What are the mechanisms of the disease?
Cost
How much will caring for this illness cost?
Adapted by Jodi Segal from: Fletcher, R., Fletcher, S. W. (2012). Clinical Epidemiology: The Essentials. (Fifth edition). Philadelphia: LWW.
4
Question, Aim, or Hypothesis? Different Approaches to Framing Research
 Research question
 What is the impact on BMI of a multicomponent, nurse-led weight-reduction
intervention including social media, compared to usual care?
 Specific aim
 To assess the relationship between strength of inter-provider social networks and
cancer survivorship care in adult survivors of prostate cancer.
 Research hypothesis
 Telephone interpreting systems versus in-person interpretation are associated with
decreased patient satisfaction.
5
Determining Needs for a New Study
 Which questions are of interest?
 Are there relevant pre-existing reviews?
 Systematic reviews
 Other reviews
 If no reviews, identify existing evidence that addresses the question
 Assess quality
 Identify evidence gaps
 Is evidence conclusive?
• That is, would additional evidence
change the conclusion?
Image: Case, D. (2013). Mind the gap. Via Wikimedia Commons: https://commons.wikimedia.org/wiki/File:Mind_the_gap_Chinglish_sign_in_Shanghai.jpg. Licensed
under Creative Commons Attribution-Share Alike 3.0 Unported (https://creativecommons.org/licenses/by-sa/3.0/deed.en). Accessed July 31, 2015.
6
Consider Using an Analytic Framework
Source: U.S. Preventive Services Task Force. Final Research Plan for Sexually Transmitted Infections: Behavioral Counseling, September 2014. Available at:
http://www.uspreventiveservicestaskforce.org/Page/Document/final-research-plan34/sexually-transmitted-infections-behavioral-counseling1.
Accessed July 31, 2015.
7
Using Directed
Acyclic Graphs to
Identify Variables
That Need to Be
Controlled
for in Estimating
Neighborhood
Health Effects
Source: Fleischer, N. L., & Diez Roux, A. V. (2008).
Using directed acyclic graphs to guide analyses of
neighbourhood health effects: an introduction.
Journal of Epidemiology and Community Health,
62(9), 842–846.
http://doi.org/10.1136/jech.2007.067371
8
PICOTS: Population
 Defining population
 Specific
 Feasible
 Sensitive to patient needs
 Consider whether population is vulnerable
 Good or bad examples?
 “African-Americans with poorly controlled diabetes”
 “Incarcerated men who have successfully completed
a 1-year course of anti-retroviral therapy for
HIV/AIDS”
 “All patients seen in the emergency department with
chest pain”
 “Medical students with depression”
Image: Kmf164, from US Census Bureau data (http://www.census.gov/population/international/data/idb/informationGateway.php). (2014). Population pyramid,
United States, 2014. Via Wikimedia Commons: https://commons.wikimedia.org/wiki/File:Uspop.svg. Public domain. Accessed July 31, 2015.
9
PICOTS: Intervention
 Defining intervention
 Specific
 Feasible to research
 Responsive to patient needs
 Pause here and try writing your own intervention statement
 Strengths/weaknesses of these examples?
 Glucose testing for diabetes
 Primary care for first follow-up visit within a year after discontinuation of
chemotherapy in adult cancer survivors
 Routine health exam
 Intravenous acetaminophen for the treatment of chronic musculoskeletal pain
10
PICOTS: Comparison
 Defining comparison
 Specific
 Detailing difference from intervention
 Responsive to patient needs
 Defining “usual care”
 Pause to write your own comparison
 Good or bad?
 Physical therapy for knee meniscal tear compared to watchful waiting
 Pharmacotherapy for depression compared to standard care
 Metformin and insulin for diabetes compared to dapagliflozin
11
PICOTS: Outcomes
 Defining outcome
 Specific
 Different types of outcomes
• Patient-reported outcomes
• Clinical outcomes
• System outcomes
 Examples of outcomes
 Hemoglobin A1C difference-in-difference at six months
 Patient satisfaction with care
 Patient adherence
 Mortality
 Composite outcome of stroke, myocardial infarction, or other vascular disease
12
Example of Detailed Study Protocol With PICOTS
Research question
What is the effect of a best practice advisory on completion of depression
screening among patients 65 years of age or older in a community practice?
Population
Patients seen in a community practice 65 years of age or older
Intervention
Implementation of a best-practice advisory for MDs in an electronic
health record
Comparison
Usual care
Outcome
Completion of depression screening
Time
Three months
Setting
Outpatient practice
13