1. Clinical Studies Continuum
What features are special with regard to Comparative Effectiveness and Patient Centered Outcomes Research

2. Definition and Purpose of CER
CER is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical condition or to improve the delivery of care.
The purpose of CER is to assist consumers, clinicians, purchasers and policy makers to make informed decisions that will improve health care at both the individual and population levels.

3. Distinguishing Characteristics of CER
Informing specific clinical or policy decision
Comparing at least two approaches or interventions
Describing results at the subgroup level, where possible
Measuring benefits in real-world populations in real-world clinical situations
Applying appropriate methods and data sources

4. Poll Everywhere Question
What is a reason why the US needs of comparative effectiveness research?
Current biomedical studies often have restricted patient populations.
Current biomedical studies often have very tightly supervised protocols that are hard to duplicate in practice.
The questions asked in current studies do not answer the real decision-making needs of clinicians and patients.
All of the above.

5. Definition and Purpose of PCOR
From PCORI:
Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options.

6. This research answers patient-centered questions, such as:
“Given my personal characteristics, conditions, and preferences, what should I expect will happen to me?”
“What are my options, and what are the potential benefits and harms of those options?”
“What can I do to improve the outcomes that are most important to me?”
“How can clinicians and the care delivery systems they work in help me make the best decisions about my health and health care?”

7. Poll Everywhere Question
All comparative effectiveness research is also patient-centered outcomes research.
True
False
Not all CER is PCOR

8. Once you have an articulated question
The study design will:
Connect the specific aims, research question, and hypotheses through the approach by identifying the study population, collecting appropriate data, and using the best tools to analyze the data.

9. Major steps in final design/approach
Research Methods/Study Design
Study Population
Measures
Data Collection/Procedures
Data Management
Statistical Analyses
Statistical Power

10. Threats to validity
Classic texts by Campbell and Stanley (1963 and 1966)
These threats may apply to both experimental and quasi- experimental designs
You must account for these in the design of your study and in the analysis plan

11. Threats to validity: 8 identified
History
Maturation
Testing
Instrumentation
Statistical regression
Selection
Experimental mortality
Diffusion of treatments

12. Threats to validity: 8 identified
History
Events, other than the experimental treatments, influence results.

13. Threats to validity: 8 identified
Maturation
During the study, changes occur within subjects, such as psychological changes

14. Threats to validity: 8 identified
Testing
Exposure to a pretest or intervening assessment influences performance on a posttest.

15. Threats to validity: 8 identified
Instrumentation
Testing instruments or conditions are inconsistent; or pretest and posttest are not equivalent, creating an illusory change in performance.

16. Threats to validity: 8 identified
Statistical regression
Scores of subjects that are very high or very low tend to regress towards the mean during retesting.

17. Threats to validity: 8 identified
Selection
Systematic differences exist in subjects’ characteristics between treatment groups.

18. Threats to validity: 8 identified
Experimental mortality
Subject attrition may bias the results.

19. Threats to validity: 8 identified
Diffusion of treatments
Implementation of one condition influences subjects in another condition.

20. A continuum of clinical studies (not exhaustive of all designs)
Case study (clinical) or case series
Observational study – no control
Observational study – with control
“Natural” experiments, e.g., Oregon-Medicaid
Pre-post designs
Experimental designs
Individually randomized controlled trial
Cluster or group randomized controlled trial
Stepped wedge design

21. Valid Scientific Evidence
21 CFR (Device Regulations)
Includes
Well-controlled investigations
Partially controlled studies
Studies and objective trials without matched controls
Well-documented case histories
Reports of significant human experience with a marketed device

22. Differences Between Efficacy and Effectiveness Studies
Efficacy study
Effectiveness study
Question
Does the intervention work under ideal circumstance?
Does the intervention work in real-world practice?
Setting
Resource-intensive ‘ideal setting’
Real-world everyday clinical setting
Study population
Highly selected, homogenous population Several exclusion criteria
Heterogeneous population Few to no exclusion criteria
Providers
Highly experienced and trained
Representative usual providers
Intervention
Strictly enforced and standardized; No concurrent interventions
Applied with flexibility; Concurrent interventions and cross-over permitted

23. What’s Unique about CER
Direct head-to-head comparisons of competing treatments, prevention modes, or health system interventions (or variations)
Topics oriented to clinical decision-making
Tests, treatment, stategiess for prevention, care, delivery and monitoring.
Study design and endpoints relevant to a wide group of stakeholders:
Patients, clinicians, purchasers, policy makers
Study populations should be representative of clinical practice.

24. What’s Unique about PCOR
The stakeholder, especially patient voice, is heard throughout the study – from conception, to design, through conduct, and dissemination and implementation
The outcomes are centered around what is important to patients.
Note: patient-centered outcomes come in a variety of measures and are not synonomous with patient reported outcomes.

25. What to Consider in Study Design related to CER and PCOR
Consider “real-world” populations and “real- world” clinical situations
Ensure the comparison is between legitimate alternatives
Ensure the data will answer the question with a focus on the clinical or health care system decision to be made and that the results are translatable to your organization
Ensure, where possible, all stakeholders are represented throughout the study process