RES 101 – Intro to Research for the Non-Researcher, Part II

MGN • Improving Access to Genetic Services

IBEMC • Understanding “Natural” History of IBEM

IBEMCSN • Supporting patient centered research

MGN – Midwest Genetics NetworkIBEMC – Inborn Errors of Metabolism CollaborativeIBEMCSN – IBEMC Stakeholder NetworkHRSA – Health Resources and Services AdministrationNIH – National Institutes of HealthPOCRI – Patient Centered Outcomes Research Institute

($$ - HRSA)

($$ - HRSA & NIH)

($$ - PCORI)

Achieving Victory over the Short Term

Objective: Understand some basics of clinical researchOUTLINE FOR NEXT TWO WEBINARS

Working Definition of Clinical Research

Working Definition of Peer Review

Statistics, a Friend and a Foe

Patient Reported Outcomes

Patient Reported Outcomes Continued

Clinical research funded by PCORI

RECALL THAT THE GOAL IS NOT TO MAKE SCIENTISTS OUT OF ALL OF YOU!

Outcome has a Specific Meaning in ResearchOutcome: How something turns out

In Research: an end result often assessed by a lab test or some other observation by a researcher or clinician• Phe levels at goal•Developmental milestone reached

Patient Reported Outcomes

A patient-reported outcome is an end result assessed by the patient based on how they feel or what they have experienced•Quality of life

•Current pain level

•Satisfaction with carehttp://www.govint.org/our-services/outcomes/

Measuring Patient Reported Outcomes -It’s Not Complicated, I PROMIS

PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health in adults and children. It can be used with the general population and with individuals living with chronic conditions.

Demonstration of PROMIS ToolsNIH PROMIS Webpage

Why are Patient Reported Outcomes Important to IBEMCSN

Patient-reported outcomes • often measure things patients and their

caregivers care about

• are the best way to measure things only a patient or their caregiver would know (for example how you feel, if you have pain, are you satisfied with care)

• are preferred in PCORI research studies

• may provide valuable insight to your clinicians that allows them to improve care

Questions About Patient Reported Outcomes

Comparative Effectiveness Research (CER)

Definition of Comparative Effectiveness Research (CER)

Research that comparesoutcomes to determine what options for health care work best for whom.

Three Key Parts to a CER Question

http://www.pcori.org/get-involved/suggest-patient-centered-research-question/how-write-practical-useful-research-question

CER Questions are Organized Around Research Frameworks

Research Frameworks are tools that help ask and answer research questions in a standardized way

Three general types of frameworks• Theoretical - for asking questions based on unproven evidence

• Empirical – for asking questions based on evidence from your own experience or that of others

• Contextual – for asking questions that are specifically intended to change practice or policy

PCORI Prefers Research Projects that Use a Contextual Framework

“Research without a framework will provide results, but without an underlying framework as context, the results may not be that helpful in guiding practice, education, or research.” University of Southern California Library

Resource, original author unidentified

PICOTS is a Widely Used Framework for CER and other Clinical ResearchPopulation: Population impacted by the research questions, often based on a specific health problem or care delivery system

Intervention: The main treatment, test, or approach for the health problem/care delivery system under investigation

Comparator: Alternative treatment, test, or approach to the main intervention (may be multiple comparators)

Outcome: Measurable effect of the intervention/comparator (lab value, test result, quality of life measure, etc)

Time Frame: Time frame needed to implement the intervention and assess outcomes

Setting: Setting of interest

PICOTS PracticePopulation

Intervention

Comparator(s)

Outcomes

Timing

Setting