ASPIRE CLASS 1: Conceiving the Research Idea Katie Derington, PharmD Outcomes Research Fellow in Ambulatory Care Kaiser Permanente Colorado & University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences

Learning Objectives Class 1: Conceiving the Research Idea ASPIRE Class 1: Conceiving the Research Idea Write a research question that meets PICO & FINER criteria Formulate primary and secondary objectives Formulate study hypotheses and select outcome measures for each objective Use a well-written study question to survey primary literature Identify an efficient process for reviewing articles and managing references

Where Do Research Questions Come From?

Where Do Research Questions Come From? Clinical practice Literature Institutional and clinical problems Could be a problem you regularly encounter in practice, or a question that comes up in reading literature, or institutional issues or concerns, such as meeting quality objectives (JCACHO in the hospital, HEDIS in managed care)

Example Research Questions How effective is a clinical pharmacy service in managing patients with diabetes? Why do HMO patients take their prescriptions to outside (out of plan) pharmacies? What are the benefits of Medication Management? Why don’t patients pick up their new bisphosphonate prescriptions?

Components of a good research question PICO Population Intervention Comparison Group Outcome Components of the question

Quality of a good research question FINER Feasibility Interesting Novel Ethical Relevant Quality of the question

Example Question Does a home blood pressure monitoring program improve hypertension control?

Quality/Completeness Check: PICO Does a home blood pressure monitoring program improve hypertension control? Population Intervention Comparison Outcome Prob- Pop- who? Int- pharmacist-managed HBPM program Comparison- pts enrolled vs not enrolled ; pre and post NOT a good comparison here! Outcome- BP control; change in SBP

Quality/Completeness Check: PICO Does a home blood pressure monitoring program improve hypertension control? Population—adult pts with a HTN diagnosis and uncontrolled HTN Intervention—Pharmacist-managed HBPM program Comparison—adult pts meeting inclusion criteria but not enrolled in program Outcome—systolic BP Prob- Pop- who? Int- pharmacist-managed HBPM program Comparison- pts enrolled vs not enrolled ; pre and post NOT a good comparison here! Outcome- BP control; change in SBP

Revised Question Does a pharmacist-managed home blood pressure monitoring program (I) reduce blood pressure to a greater extent (O) in hypertensive patients with uncontrolled BP (P) compared to similar patients receiving usual care (C)?

Quality Check: FINER Feasibility Interesting Novel Ethical Relevant Feas—non-interventional, so Q is getting data– Pt identification? (Administrative) Characteristics? (Administrative) Beliefs? (Survey) Also—enough subjects? Technical expertise? Cost and duration? Scope? Interesting– real patient problem Novel– Confirms or refutes or extends previous findings; Provides new findings Ethical– Relevant– to scientific knowledge; to future research directions—could lead to designing a successful intervention

Elements of a Research Protocol Objectives Background Design Research Question Population Analytical Plan Procedures

Objectives, Hypotheses, and Outcomes What you want to learn Outcome How you plan to measure it Specific Hypothesis What results do you expect?

Study Objectives (Study Aims) What the study will achieve Guides thinking, study design, analysis, and reporting Serves as outline for organizing later sections Methods section should parallel Can have multiple objectives Not too many! Use strong verbs Outlines what the study will achieve Multiple objectives is ok, but make sure one is primary and others are secondary/tertiary Limit number to keep your study focused

Study Outcome(s) This is the specific thing you are going to measure for each objective

Study Outcome(s) Is this specific enough as a study outcome? “The primary outcome of the study was patient satisfaction.” This needs to be much more specific. Include: Satisfaction with what? How is satisfaction being measured? At what time point are we interested in patient satisfaction?

Study Hypothesis Definition: prediction of the relationship between one or more factors and the problem under study More specific than objectives and allow you to statistically evaluate the objective Guides how to conduct the statistics

Study Hypothesis Most appropriate for intervention/evaluative studies Difficult for descriptive/exploratory studies At least 1 hypothesis for each objective can have more than 1 Simple, specific, and stated in advance Stated as null or alternative Statistical analysis based on null hypothesis Not usually possible for descriptive studies Example of more than 1: BP outcomes in general, BP outcomes in specific subgroup

Study Objectives: Example Question: Does a pharmacist-managed home blood pressure monitoring program reduce blood pressure to a greater extent in hypertensive patients with uncontrolled BP compared to similar patients receiving usual care? Primary Objective: Secondary Objective:

Study Objectives: Example Question: Does a pharmacist-managed home blood pressure monitoring program reduce blood pressure to a greater extent in hypertensive patients with uncontrolled BP compared to similar patients receiving usual care? Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes Secondary Objective: Determine patient satisfaction with pharmacist-managed HBPM program

Outcome: Example Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes What would be an appropriate outcome for this objective?

Outcome: Example Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes Systolic blood pressure Diastolic blood pressure Time to blood pressure goal Probably need to be more specific than this: Comparator groups? Time points?

Hypothesis Example Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes Primary Outcome: Systolic blood pressure Null: Alternative:

Hypothesis Example Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes Primary Outcome: Systolic blood pressure Null: There will be no difference in systolic blood pressure in those managed in the pharmacist HBPM program and usual care groups. Alternative: Systolic blood pressure will be lower in those managed using the pharmacist HBPM program compared to usual care. Important to note that there can be multiple ways to word an alternative hypothesis

Blood Pressure Example Summary Question: Does a pharmacist-managed home blood pressure monitoring program increase the proportion of hypertensive patients with uncontrolled BP who achieve their target blood pressure compared to similar patients receiving usual care? Primary Objective: Establish effectiveness of pharmacist-managed HBPM program on blood pressure outcomes Primary Outcome: Change in systolic blood pressure Hypothesis: Systolic blood pressure will be lower in those managed using the pharmacist HBPM program compared to usual care.

Elements of a Research Protocol Background Population Design Objectives Procedures Analytical Plan Research Question

Researching Background: Goals Understand what is known Find the unknown Learn how others have approached this problem Guide your research question, methods, outcomes, etc.

General Search Strategies Tips to insure a high-quality, systematic search: Use at least two databases Check references of studies meeting inclusion criteria Check with content experts to make sure relevant studies are not missed Search for ‘gray’ literature Avoid limiting searches by date Come up with a process for reference management (manual or software assisted) Record search history Start with your PICO question(s) Establish preliminary search terms/themes Establish inclusion and exclusion criteria

Free Resources for Reference Management EndNote Web Version: Register for free access (1 year) Mendeley (https://www.mendeley.com/) Manual management

EndNote Web Resource Pros Cons It’s free(ish) It enables you to maintain a comprehensive electronic reference list It’s widely accessible It’s only free for 1 year The interface is not that intuitive Not all functionality is available work computers

Mendeley Pros Cons May not have access on work computers Free Links to PubMed and other literature sources Plug-in for browsers allow you to add references as you search Plug-in for Microsoft Word makes it easy to use Relatively intuitive/easy Share libraries with other Mendeley users Search function May not have access on work computers !! Caution using pre-populated fields Relies on you to enter information correctly

Manual Reference Management Pros Cons Free Not vulnerable to IT issues It’s like retro, man

During the Literature Search Be organized! Add references to your reference manager Write down things to look up later

Sample Evidence Summary Study N Design Inclusion criteria Intervention Comparator Outcomes Median follow-up Chacon, 2011 675 RCT >18 years KP member Normal Renal function Bloodletting Leeches Syncope 30 days

Once You Have References… Determine which should be included (relevant) Devise a scheme for routinely updating reference list Detail references in your protocol, posters, and manuscript

Assessing Quality of Studies Appropriate study design Application of appropriate statistical testing Identification of bias and conflicts of interest Meaningful synthesis of results Appropriate conclusions drawn Various published quality assessment tools are available (email me if you are interested)

In summary… Start with a good PICO question Think about your objectives, outcomes, and hypotheses before and during your literature search Consult with content experts Be systematic about finding, reviewing, and storing references Use free resources to your advantage

Assignment Begin background literature review for your study Prepare a 5-10 min presentation for your small group session including draft versions of the following: Your research question (use FINER and PICO criteria) Primary and secondary objectives A hypothesis and study outcome(s) for each objective Please bring the above with you to the first class KPCO Central Support Services: 16601 E Centretech Parkway, Aurora CO